January | February | March | April | May | June | July | August | September | October | November | December |
| Company | Product | Description | Indication | Phase II status | Date | Therapeutic area |
| AB Science SA, of Paris | Masitinib | Oral tyrosine kinase inhibitor targeting mast cells and macrophages | Metastatic castrate-resistant prostate cancer | FDA cleared the IND for a phase III study in patients eligible for chemotherapy; top-line data expected by end of 2020 | 1/9/20 | Cancer |
| Abbvie Inc., of North Chicago | Venclyxto (venetoclax) | Bcl-2 inhibitor | Chronic lymphocytic leukemia | EMA’s Committee for Medicinal Products for Human Use granted a positive opinion for use in combination with obinutuzumab in previously untreated patients | 1/31/20 | Cancer |
| Acepodia Inc., of San Francisco and Taipei, Taiwan | ACE-1702 | Natural killer cell therapy | HER2-expressing solid tumors | FDA cleared the IND for a phase I study | 1/9/20 | Cancer |
| Advaxis Inc., of Princeton, N.J. | ADXS-504 | Listeria monocytogenes secreting antigen-adjuvant fusion proteins | Prostate cancer | FDA cleared IND for the phase I study | 1/7/20 | Cancer |
| AI Therapeutics Inc., of Guilford, Conn. | LAM-002 | PIKfyve kinase inhibitor that activates transcription factor EB | Follicular lymphoma | Conducted type C meeting with FDA and reached concurrence on design of a pivotal trial that could support accelerated approval | 1/16/20 | Cancer |
| Aprea Therapeutics Inc., of Boston | APR-246 | Small molecule targeting p53 | Myelodysplastic syndromes | FDA granted breakthrough therapy for use in combination with azacitidine in patients whose disease as susceptible TP53 mutation | 1/30/20 | Cancer |
| Aravive Inc., of Houston | AVB-500 | Therapeutic recombinant fusion protein shown to neutralize GAS6 activity by binding to GAS6 with very high affinity | Clear cell renal cell carcinoma | FDA cleared the company's IND; phase Ib will evaluate escalating doses in combination with cabozantinib, while phase II will evaluate the recommended dose with a primary endpoint of progression-free survival | 1/13/20 | Cancer |
| Astellas Pharma Canada Inc., of Markham, Ontario, part of Astellas Pharma Inc. | Xospata (gilteritinib) | FLT3 inhibitor | Relapsed/refractory acute myeloid leukemia | Health Canada approved for use in adults whose disease had FLT3 mutation | 1/14/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | Hepatocellular carcinoma | FDA granted orphan drug designation | 1/20/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Tremelimumab | Monoclonal antibody targeting CTLA4 | Hepatocellular carcinoma | FDA granted orphan drug designation | 1/20/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Advanced ovarian cancer | FDA granted priority review in combination with bevacizumab for maintenance treatment of patients who are in complete or partial response for first-line platinum-based chemotherapy with bevacizumab; agency also accepted sNDA for filing; PDUFA date is set for the second quarter | 1/13/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | FDA accepted the supplemental NDA seeking approval for use in patients with a homologous recombination repair gene mutation after progression on a new hormonal agent; granted a priority review, with PDUFA date in the second quarter of 2020 | 1/20/20 | Cancer |
| Bayer AG, of Leverkusen, Germany | Nubeqa (darolutamide) | Nonsteroidal androgen receptor inhibitor | Nonmetastatic castration-resistant prostate cancer | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in combination with androgen deprivation therapy | 1/31/20 | Cancer |
| Biolinerx Ltd., of Tel Aviv, Israel | Motixafortide (BL-8040) | Functions as CXCR4 antagonist | Pancreatic cancer | European Commission granted orphan designation | 1/14/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | Kinase inhibitor | Gastrointestinal stromal tumor | FDA approved for use in treating adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations | 1/10/20 | Cancer |
| Bridge Biotherapeutics Inc., of Seongnam, South Korea | BBT-176 | EGFR tyrosine kinase inhibitor | Advanced non-small-cell lung cancer | FDA cleared the IND for a phase I/II study | 1/19/20 | Cancer |
| Bridgebio Pharma Inc., of San Francisco | Infigratinib | FGFR1-3 selective tyrosine kinase inhibitor | Cholangiocarcinoma | FDA granted fast track designation for first-line treatment of adults with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations and orphan drug designation for the treatment of cholangiocarcinoma | 1/6/20 | Cancer |
| Bristol-Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) and Yervoy (ipilimumab) | PD-1-inhibiting antibody and CTLA4-targeting antibody | Metastatic or recurrent non-small-cell lung cancer | FDA accepted supplemental BLA seeking approval of the combination for first-line treatment of patients whose disease harbors no EGFR or ALK genomic tumor aberrations | 1/15/20 | Cancer |
| Cellectar Biosciences Inc., of Florham Park, N.J. | CLR-131 | Phospholipid-drug conjugate | Lymphoplasmacytic lymphoma | FDA granted orphan drug designation | 1/6/20 | Cancer |
| Celularity Inc., of Warren, N.J. | CYNK-001 | Placental hematopoietic stem cell-derived allogeneic NK cell therapy | Glioblastoma multiforme | FDA cleared IND application | 1/22/20 | Cancer |
| Checkpoint Therapeutics Inc., of New York | Cosibelimab (formerly CK-301) | Monoclonal antibody of IgG1 subtype that directly binds to PD-L1 | Metastatic cutaneous squamous cell carcinoma | Company confirmed plan with FDA to submit a BLA in 2021 based on data from the ongoing phase I trial in which a third of the enrollment is completed | 1/13/20 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | Rubraca (rucaparib) | PARP inhibitor | Metastatic castrate-resistant prostate cancer | FDA accepted the supplemental NDA seeking approval for use as a monotherapy in patients with BRCA1/2-mutant recurrent disease; agency granted priority review, with PDUFA date of May 15, 2020 | 1/15/20 | Cancer |
| Cytodyn Inc., of Vancouver, Wash. | PRO-140 (leronlimab) | A CCR5 antagonist | Metastatic triple-negative breast cancer | Company filed for breakthrough therapy designation with the FDA | 1/13/20 | Cancer |
| Eli Lilly and Co., of Indianapolis | Selpercatinib (LOXO-292) | RET inhibitor | RET fusion-positive non-small-cell lung cancer, RET-mutant medullary thyroid cancer or RET fusion-positive thyroid cancer | FDA accepted the NDA; PDUFA action date in third quarter of 2020 | 1/29/20 | Cancer |
| Epizyme Inc., of Cambridge, Mass. | Tazverik (tazemetostat) | EZH2 inhibitor | Epithelial sarcoma | Approved by FDA | 1/23/20 | Cancer |
| Exelixis Inc., of Alameda, Calif., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Cabometyx (cabozantinib) | Tyrosine kinase inhibitor | Unresectable hepatocellular carcinoma after progression on systemic therapy | Applied to the Japanese Ministry of Health, Labor and Welfare for Manufacturing and Marketing Approval | 1/29/20 | Cancer |
| Fate Therapeutics Inc., of San Diego | FT-516 | Off-the-shelf natural killer cell cancer immunotherapy | Solid tumors | FDA approved IND application | 1/13/20 | Cancer |
| F-star Therapeutics Ltd., of Cambridge, U.K. | FS-120 | Tetravalent bispecific antibody targeting CD137 and OX40 | Advanced malignancies | FDA accepted IND for phase I study | 1/30/20 | Cancer |
| Genentech, unit of Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | PD-L1 inhibitor | Hepatocellular carcinoma | sBLA submitted to FDA for study drug in combination with Avastin (bevacizumab, Roche) to treat unresectable disease in those with no prior systemic therapy; agency reviewing under Real-Time Oncology Review pilot program | 1/27/20 | Cancer |
| Genmab A/S, of Copenhagen, and Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex (daratumumab) | Monoclonal antibody targeting CD38 | Newly diagnosed multiple myeloma | European Commission granted marketing authorization for the drug in combination with bortezomib, thalidomide and dexamethasone, for use in patients who are eligible for autologous stem cell transplant | 1/20/20 | Cancer |
| Genprex Inc., of Austin, Texas | Oncoprex | TUSC2 gene stimulator | Non-small-cell lung cancer | FDA granted fast track designation for the immunogene therapy in combination with osimertinib (Tagrisso, Astrazeneca plc) | 1/21/20 | Cancer |
| Glaxosmithkline plc, of London | Belantamab mafodotin (GSK-2857916) | Immunoconjugate comprising a humanized anti-BCMA monoclonal antibody conjugated to auristatin F via noncleavable linker | Relapsed or refractory multiple myeloma | FDA granted priority review for the BLA seeking approval for treating patients whose prior therapy included an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody | 1/21/20 | Cancer |
| Hillstream Biopharma Inc., of Chester, N.J. | HSB-1216 | Cancer stem cell inhibitor salinomycin encapsulated in the Quatramer delivery system | Small-cell lung cancer | FDA granted orphan drug designation | 1/6/20 | Cancer |
| Incyte Corp., of Wilmington, Del. | Pemigatinib | Inhibitor of FGFR isoforms 1, 2 and 3 | Locally advanced or metastatic cholangiocarcinoma | EMA validated the MAA seeking approval for use in disease with FGFR2 fusion or rearrangement that is relapsed or refractory after at least 1 line of systemic therapy | 1/7/20 | Cancer |
| Inhibitor Therapeutics Inc., of Tampa, Fla. | Suba-itraconazole | Lanosterol-14 demethylase inhibitor | Prostate cancer | FDA cleared IND application for phase IIb combination study with docetaxel and prednisone in metastatic castration-resistant disease, with radiographic progression-free survival as primary endpoint | 1/22/20 | Cancer |
| Innate Pharma SA, of Marseille, France | Lumoxiti (moxetumomab pasudotox-tdfk) | CD22-directed immunotoxin | Relapsed or refractory hairy cell leukemia | EMA accepted MAA seeking approval for use in adults who have received at least 2 prior systemic therapies, including treatment with a purine nucleoside analogue | 1/2/20 | Cancer |
| Innate Pharma SA, of Marseille, France | Lacutamab (IPH-4102) | Anti-KIR3DL2 humanized cytotoxicity-inducing antibody | Advanced T-cell lymphomas | Decided to suspend enrollment of new patients in Tellomak phase II trial following discussions with regulatory authorities regarding GMP deficiencies at a manufacturing subcontractor site that manages fill and finish operations of clinical vials; FDA has placed trial on partial hold, while U.K.’s MHRA agreed for trial to continue as planned with current available supply; awaiting feedback from French regulators | 1/9/20 | Cancer |
| Innate Pharma SA, of Marseille, France | Lacutamab (IPH-4102) | Anti-KIR3DL2 humanized cytotoxicity-inducing antibody | Relapsed/refractory Sezary syndrome and mycosis fungoides | French regulators agreed the Tellomak phase II trial can resume recruitment of new patients who have received at least 2 prior lines of systemic therapy; no new peripheral T-cell lymphoma patients can enroll into the study until a new GMP-certified batch of drug is available though currently enrolled PTCL patients can continue treatment | 1/13/20 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Erleada (apalutamide) | Androgen receptor inhibitor | Metastatic hormone-sensitive prostate cancer | European Commission granted extended marketing authorization for the drug in combination with androgen deprivation therapy | 1/29/20 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson and Johnson | Imbruvica (ibrutinib) | Bruton's tyrosine kinase inhibitor | First-line chronic lymphocytic leukemia | Submitted a type II variation application to the EMA to expand label to include the drug plus rituximab | 1/20/20 | Cancer |
| Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. | KTE-X19 | CAR T targeting CD19 | Relapsed or refractory mantle cell lymphoma | EMA validated the MAA | 1/28/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1-inhibiting antibody | Bacillus Calmette-Guerin BCG-unresponsive, high-risk, non-muscle invasive bladder cancer | FDA approved for use as monotherapy for patients with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy | 1/9/20 | Cancer |
| Neoimmunetech Inc., of Rockville, Md. | Hyleukin-7 (NT-I7) | IL-7 receptor agonist | Solid tumors | FDA cleared IND application for phase Ib/IIa basket study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) to treat relapsed/refractory advanced disease | 1/27/20 | Cancer |
| OBI Pharma Inc., of Taipei, Taiwan | OBI-999 | Antibody-drug conjugate targeting glycolipid antigen Globo H | Gastric cancer | FDA granted orphan designation | 1/21/20 | Cancer |
| Orion Corp., of Espoo, Finland | Nubeqa (darolutamide) | Androgen receptor inhibitor | Non-metastatic castration-resistant prostate cancer | Japan’s Ministry of Health, Labor and Welfare granted marketing authorization | 1/23/20 | Cancer |
| Pfizer Inc., of New York | Ruxience (rituximab) | Biosimilar to Mabthera; anti-CD20 monoclonal antibody | Non-Hodgkin lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and pemphigus vulgaris | EMA’s Committee for Medicinal Products for Human Use recommended marketing approval | 1/31/20 | Cancer |
| Pharmacyte Biotech Inc., of New York | Cell-in-a-Box | CYP2B1 gene modulator | Pancreatic cancer | Product that will be used in upcoming phase IIb trial in locally advanced, inoperable disease passed each of FDA's required release tests for safety and functionality | 1/27/20 | Cancer |
| Precigen Inc., of Germantown, Md. | PRGN-3006 | Nonviral CAR T expressing a chimeric antigen receptor, membrane-bound interleukin?15 and a kill switch | Relapsed or refractory acute myeloid leukemia | FDA granted orphan drug designation | 1/6/20 | Cancer |
| Precision Biosciences Inc., of Durham, N.C. | PBCAR-269A | Allogeneic CAR T-cell therapy | Multiple myeloma | FDA accepted the IND and granted orphan drug designation | 1/13/20 | Cancer |
| Rhovac AB, of Stockholm | RV-001 | Antigen-based cancer therapy | Prostate cancer | Received approval from Belgian and German regulators for phase IIb RhoVac-002 study | 1/10/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Polivy (polatuzumab vedotin) | Antibody-drug conjugate targeting CD79b | Relapsed or refractory diffuse large B-cell lymphoma | European Commission granted conditional marketing authorization for the drug in combination with bendamustine plus Mabthera (rituximab) | 1/21/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Tucatinib | Small-molecule tyrosine kinase inhibitor selective for HER2 | Locally advanced unresectable or metastatic HER2-positive breast cancer | EMA validated the marketing authorization and begins formal review process; MAA seeking approval for use in combination with trastuzumab and capecitabine in adults, including those with brain metastases, who have received at least 2 prior anti-HER2 treatment regimens | 1/31/20 | Cancer |
| Surface Oncology Inc., of Cambridge, Mass. | SRF-617 | Anti-CD39 antibody | Advanced solid tumors | FDA cleared IND | 1/27/20 | Cancer |
| Surface Oncology Inc., of Cambridge, Mass. | SRF-388 | Anti-IL-27 antibody | Hepatocellular and renal cell carcinoma | FDA cleared IND | 1/27/20 | Cancer |
| TG Therapeutics Inc., of New York | Umbralisib | Oral, once-daily, dual inhibitor of PI3K-delta and CK1-epsilon | Previously treated marginal zone lymphoma and follicular lymphoma | Initiated rolling NDA submission to FDA; expects to complete submission in first half of 2020 | 1/16/20 | Cancer |
| Alucent Biomedical Inc., of Salt Lake City | Natural vascular scaffolding technology | Standard angioplasty with linking of structural proteins in wall of blood vessel | Peripheral artery disease | FDA cleared firm to start phase I trial in patients with PAD of the lower extremities | 1/9/20 | Cardiovascular |
| Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Eicosapentaenoic acid | Cardiovascular disease | Approved by Health Canada for use in reducing risk of cardiovascular events in statin-treated patients with elevated triglycerides who are at high risk of CV events due to established CV disease, or diabetes, at have at least 1 other CV risk factor | 1/2/20 | Cardiovascular |
| Arena Pharmaceuticals Inc., of San Diego | APD-418 | ?3-adrenergic receptor antagonist and cardiac myotrope | Decompensated heart failure | FDA granted fast track status | 1/16/20 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | Sodium-glucose co-transporter 2 inhibitor | Heart failure with reduced ejection fraction | FDA accepted the supplemental NDA and granted a priority review; FDA action date is in the second quarter of 2020 | 1/6/20 | Cardiovascular |
| Cynata Therapeutics Ltd., of Melbourne, Australia | CYP-002 | Cymerus mesenchymal stem product | Critical limb ischemia | Received approval from the U.K.’s MHRA to proceed with phase II study | 1/14/20 | Cardiovascular |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Ozempic (once-weekly semaglutide) | GLP-1 analogue | Reducing risk of major adverse cardiovascular events in adults with type 2 diabetes and established CV disease | FDA approved a label expansion based on supplemental NDA | 1/16/20 | Cardiovascular |
| Plx Pharma Inc., of Sparta, N.J. | Vazalore | Liquid-filled aspirin capsule | Atherosclerotic cardiovascular disease and diabetes | FDA requested additional data to bridge the new formulation to the original approved formulation for the 325-mg strength; company and agency in alignment on data required for approval for the 81-mg dose; plans to submit both in the second quarter of 2020 | 1/6/20 | Cardiovascular |
| Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., of Tokyo | Sofpironium bromide | Anticholinergic | Primary axillary hyperhidrosis | Submitted an NDA for approval of manufacturing and marketing in Japan based on phase III data. | 1/10/20 | Dermatologic |
| Cabaletta Bio Inc., of Philadelphia | DSG3-CAART | Chimeric autoantibody receptor T cells targeting DSG3 | Pemphigus vulgaris | FDA granted orphan drug designation | 1/29/20 | Dermatologic |
| Follicum AB, of Lund, Sweden | FOL-005 | Modified, short version of endogenous protein osteopontin | Alopecia | German Medicines Agency and German Ethics Committee cleared start of phase IIa trial of topical formulation of drug | 1/30/20 | Dermatologic |
| Janssen Pharmaceutical Cos., of Beerse, Belgium, part of Johnson & Johnson | Stelara (ustekinumab) | Targets IL-23/IL-12 pathway | Moderate to severe plaque psoriasis | European Commission approved expanded use for pediatric patients, ages 6-11 | 1/23/20 | Dermatologic |
| Merakris Therapeutics LLC, of Research Triangle Park, N.C. | Dermacyte liquid | Amniotic wound care liquid for subcutaneous infection | Nonhealing venous stasis ulcers | Filed a pre-IND meeting request with FDA | 1/30/20 | Dermatologic |
| Pharming Group NV, of Leiden, the Netherlands | Ruconest (conestat alfa) | C1 esterase inhibitor | Hereditary angioedema | EMA approved a type II variation for a new production facility | 1/21/20 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Monoclonal antibody targeting interleukin-4 and interleukin-13 | Moderate to severe atopic dermatitis | FDA accepted the supplemental BLA for children ages 6 to 11; PDUFA target action date is May 26, 2020 | 1/28/20 | Dermatologic |
| Aeglea Biotherapeutics Inc., of Austin, Texas | ACN-00177 | Engineered human enzyme therapy | Homocystinuria | Filed CTA with the U.K.'s Medicines and Healthcare Products Regulatory Agency; company expects to begin a phase I/II trial in the second quarter | 1/13/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | Subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | Began a rolling submission of its NDA, with completion expected early this year; Lumasiran was granted pediatric rare disease designation by the FDA | 1/10/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Givosiran | RNAi therapeutic targeting aminolevulinic acid synthase 1 | Acute hepatic porphyria | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in adults and adolescents 12 and older; if approved, drug will be commercialized under brand Givlaari | 1/31/20 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | Lokelma (sodium zirconium cyclosilicate) | Non-absorbed potassium binder | Hyperkalemia | National Medical Products Administration in China approved the drug | 1/6/20 | Endocrine/Metabolic |
| Avrobio Inc., of Cambridge, Mass. | AVR-RD-02 | Gene therapy | Gaucher disease | FDA cleared the IND to expand ongoing phase I/II study to U.S. | 1/14/20 | Endocrine/Metabolic |
| Chiasma Inc., of Needham, Mass. | Mycapssa (octreotide) | Binds to somatostatin receptors | Acromegaly | Disclosed NDA resubmitted to FDA seeking use for maintenance treatment in adults; company expects 6-month review | 1/10/20 | Endocrine/Metabolic |
| Cyprium Therapeutics Inc., of New York, a Fortress Biotech Inc. company | CUTX-101 | Copper histidinate | Menkes disease | FDA granted rare pediatric disease designation | 1/16/20 | Endocrine/Metabolic |
| Daiichi Sankyo Europe GmbH, of Munich, Germany, part of Daiichi Sankyo Co. Ltd. | Bempedoic acid | Oral, once-daily ATP citrate lyase inhibitor | Reduction of low-density lipoprotein cholesterol | EMA’s Committee for Medicinal Products for Human Use recommended approval to treat primary hypercholesterolemia (heterozygous familial and nonfamilial) or mixed dyslipidemia as an adjunct to diet; in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with maximum tolerated statin dose; and alone or in combinate with other lipid-lowering therapies in patients who are statin-intolerant or for whom a statin is contraindicated | 1/31/20 | Endocrine/Metabolic |
| Daiichi Sankyo Europe GmbH, of Munich, Germany, part of Daiichi Sankyo Co. Ltd. | Bempedoic acid/ezetimibe fixed-dose combination | Oral, once-daily ATP citrate lyase inhibitor/cholesterol absorption inhibitor | Reduction of low-density lipoprotein cholesterol | EMA’s Committee for Medicinal Products for Human Use recommended approval for adults with primary hypercholesterolemia (heterozygous familial and nonfamilial) or mixed dyslipidemia as an adjunct to diet: in combination with a statin in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin in addition to ezetimibe; alone in patients who are either statin-intolerant or for whom a statin is contraindicated and are unable to reach LDL-C goals with ezetimibe alone; and in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets with or without a statin | 1/31/20 | Endocrine/Metabolic |
| Denali Therapeutics Inc., of South San Francisco | DNL-301 | Recombinant form of iduronate 2-sulfatase enzyme engineered using Enzyme Transport Vehicle technology | Hunter syndrome | Submitted an IND to the FDA | 1/14/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | LY-900014 | Fast-acting insulin lispro | Type 1 and type 2 diabetes | EMA’s Committee for Medicinal Products for Human Use issued positive opinion for use as part of a multiple daily injection regimen or delivered by insulin pump | 1/31/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim Pharmaceuticals Inc., of Ridgefield, Conn. | Trijardy XR (empagliflozin/linagliptin/metformin hydrochloride extended-release tablets) | SGLT2 inhibitor, DPP-4 inhibitor and mitochondrial respiratory chain inhibitor | Type 2 diabetes | FDA approved the drug | 1/27/20 | Endocrine/Metabolic |
| Janssen Pharmaceutical Co., of Toronto, a unit of New Brunswick, N.J.-based Johnson & Johnson | Invokana (canagliflozin) | Sodium-glucose co-transporter 2 inhibitor | Type 2 diabetes mellitus and diabetic nephropathy with albuminuria | Health Canada expanded indication for drug to include reduction in the risk of end-stage kidney disease, doubling of serum creatinine and cardiovascular death | 1/28/20 | Endocrine/Metabolic |
| Logicbio Therapeutics Inc., of Cambridge, Mass. | LB-001 | Recombinant adeno-associated viral vector with human methylmalonyl-COA mutase gene | Methylmalonic acidemia | Submitted IND to FDA for a phase I/II trial | 1/10/20 | Endocrine/Metabolic |
| Minoryx Therapeutics SA, of Barcelona, Spain | Leriglitazone (MIN-102) | Brain-penetrant, orally bioavailable and selective PPAR? agonist | X-linked adrenoleukodystrophy | FDA granted fast track designation for all forms of X-ALD, including adrenomyeloneuropathy and childhood cerebral ALD | 1/9/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Fiasp | Insulin | Diabetes | FDA approved the drug as a new mealtime insulin option for children | 1/6/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Rybelsus (semaglutide tablets 7 mg or 14 mg) | GLP-1 analogue | Type 2 diabetes patients with established cardiovascular disease | Prescribing information updated to include analysis from primary endpoint of Pioneer 6 CVOT study showing hazard ratio for time to first 3-component major adverse cardiovascular event (HR 0.79 [95% CI: 0.57, 1.11]) | 1/16/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Rybelsus (oral semaglutide) | GLP-1 analogue | Type 2 diabetes | EMA’s Committee for Medicinal Products for Human Use recommended approval for treating adults with insufficiently controlled diabetes to improve glycemic control as an adjunct to diet and exercise | 1/31/20 | Endocrine/Metabolic |
| Polaryx Therapeutics Inc., of Paramus, N.J. | PLX-200 | Binds to the retinoid X receptor-alpha | Late infantile neuronal ceroid lipofuscinosis | FDA approved the IND | 1/20/20 | Endocrine/Metabolic |
| Recordati SpA, of Milan, Italy | Isturisa (osilodrostat) | Cortisol synthesis inhibitor | Cushing’s syndrome | European Commission granted marketing authorization for treating endogenous Cushing’s syndrome in adults | 1/15/20 | Endocrine/Metabolic |
| Abivax SA, of Paris | ABX-464 | REV protein modulator | Ulcerative colitis | FDA approved IND application to treat individuals with moderate to severe disease; first U.S. patients expected to be enrolled in second quarter into ongoing phase IIb trial | 1/21/20 | Gastrointestinal |
| Albireo Pharma Inc., of Boston | Odevixibat | Bile acid modulator | Biliary atresia | FDA cleared the IND for a global pivotal study | 1/29/20 | Gastrointestinal |
| Anokion SA, of Cambridge, Mass. | KAN-101 | Antigen-mediated immune tolerance | Celiac disease | FDA accepted an IND for a phase I study | 1/21/20 | Gastrointestinal |
| Evoke Pharma Inc., of Solana Beach, Calif. | Gimoti (metoclopramide) | Works via antagonist activity at D2 receptors | Acute and recurrent diabetic gastroparesis | FDA accepted resubmission of the 505(b)(2) NDA; PDUFA date of June 19, 2020 | 1/21/20 | Gastrointestinal |
| Jubilant Pharma Ltd., of Yardley, Pa. | Drax Exametazime | Leukocyte labeling diagnostic | Intra-abdominal infection and inflammatory bowel disease | Health Canada approved the diagnostic | 1/27/20 | Gastrointestinal |
| Ferring Pharmaceuticals SA, of Saint-Prex, Switzerland | Propess (dinoprostone) | Prostaglandin E2 | Cervical ripening in labor | Japan’s Ministry of Health, Labor and Welfare approved use for patients at term (from 37 completed weeks of gestation) | 1/23/20 | Genitourinary/Sexual Function |
| Aruvant Sciences Holdings Ltd., of Basel, Switzerland | ARU-1801 | HBG gene stimulator | Sickle cell disease | FDA granted orphan drug designation in the indication | 1/22/20 | Hematologic |
| Aruvant Sciences, of New York, part of Roivant Sciences Ltd. | ARU-1801 | Modified fetal hemoglobin gene therapy | Sickle cell disease | FDA granted rare pediatric disease designation | 1/8/20 | Hematologic |
| Astellas Pharma Inc., of Tokyo, and Fibrogen Inc., of San Francisco | Evrenzo (roxadustat) | Oral inhibitor of hypoxia-inducible factor prolyl¬ hydroxylase | Anemia associated with chronic kidney disease | Submitted supplemental NDA to Japan’s Ministry of Health, Labour and Welfare for use in nondialysis-dependent patients | 1/30/20 | Hematologic |
| Imago Biosciences Inc., of San Francisco | IMG-7289 (bomedemstat) | Small molecule that inhibits lysine-specific demethylase 1 | Essential thrombocythemia | FDA granted fast track designation | 1/13/20 | Hematologic |
| Orchard Therapeutics plc, of London | OTL-102 | Ex vivo autologous hematopoietic stem cell gene therapy | X-linked chronic granulomatous disease | FDA granted orphan drug designation | 1/29/20 | Hematologic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Moderately to severely active rheumatoid arthritis | Health Canada approved use of the drug in patients who have had an inadequate response or intolerance to methotrexate | 1/7/20 | Immune |
| Aimmune Therapeutics Inc., of Brisbane, Calif. | Palforzia (peanut [Arachis hypogaea] allergen powder-dnfp, formerly AR-101) | Characterized oral desensitization immunotherapy | Peanut allergy | FDA approved oral therapy to treat individuals 4 through 17 years of age with confirmed peanut allergy, with boxed warning; REMS addresses initial dose escalation, up-dosing and pharmacy procedures | 1/31/20 | Immune |
| Elsalys Biotech SA, of Lyon, France | Leukotac (inolimomab) | Immunotherapy antibody targeting IL-2 | Graft-vs.-hosts disease | Available again in France after regulators granted temporary authorization of use cohort for treating GVHD, corticosteroid-resistant or corticosteroid-dependent, with grade II-IV | 1/9/20 | Immune |
| Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif. | GLPG-1690 | Autotaxin inhibitor | Systemic sclerosis | FDA and the European Commission granted orphan drug designation | 1/29/20 | Immune |
| Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Bioresorbable polymeric matrices | Chronic rhinosinusitis | FDA cleared IND to include U.S. sites in the ongoing phase II Lantern study | 1/6/20 | Immune |
| Mesoblast Ltd., of Melbourne, Australia | Remestemcel-L | Therapy comprising culture-expanded mesenchymal stem cells derived from bone marrow of an unrelated donor | Steroid-refractory acute graft-vs.-host disease | FDA confirmed receipt of rolling BLA seeking approval for use in children; final module of BLA will be filed in January, and company will request expedited FDA review | 1/2/20 | Immune |
| Novartis AG, of Basel, Switzerland | Mayzent (siponimod) | Sphingosine 1-phosphate receptor modulator | Secondary progressive multiple sclerosis with active disease | European Commission approved the drug | 1/20/20 | Immune |
| Sorrento Therapeutics Inc., of San Diego, and Mabpharm Ltd., of Taizhou, China | Infliximab biosimilar | Monoclonal antibody targeting tumor necrosis factor | Rheumatoid arthritis, psoriatic arthritis, Crohn's disease, ulcerative colitis, ankylosing spondylitis and plaque psoriasis | Filed an NDA with the National Medical Products Administration in China | 1/6/20 | Immune |
| Vitalis LLC, New York | VTS-72 | Fumarate plus VTS-Aspirin | Relapsing-remitting multiple sclerosis | Based on the meeting minutes from a type C meeting with the FDA, company plans to start a phase III study in 2020 with potential to submit an NDA in the first half of 2021, seeking approval for use in patients who experience the fumarate flush | 1/7/20 | Immune |
| Abbvie Inc., of North Chicago | Maviret (glecaprevir/pibrentasvir) | Once-daily, single-tablet combination of NS3/4A inhibitor and NS5A inhibitor | Hepatitis C virus | EMA’s Committee for Medicinal Products for Human Use recommended change to marketing authorization to shorten treatment duration from 12 to 8 weeks in treatment-naïve, compensated cirrhotic, chronic HCV patients with genotype 3 infection | 1/31/20 | Infection |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-22 | Subcutaneously administered PD-L1 antibody | Hepatitis B | China’s NMPA clears IND for clinical studies in chronic hepatitis B patients | 1/22/20 | Infection |
| Astellas Pharma Inc., of Tokyo | Mycamine (micafungin for injection) | Antifungal | Invasive candidiasis | FDA approved supplemental NDA for use in treating candidemia, acute disseminated candidiasis, Candida peritonitis and abscesses without meningoencephalitis and/or ocular dissemination in pediatric patients younger than 4 months | 1/8/20 | Infection |
| Blueberry Therapeutics Ltd., of Macclesfield, U.K. | BB-2603 | Nanoformulated, topical form of terbinafine | Onychomycosis | FDA cleared IND for a phase IIb study | 1/28/20 | Infection |
| Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. | Ryanodex (dantrolene sodium for injectable suspension) | Ryanodine receptor 1 antagonist | External heat stroke | Resubmitted NDA to FDA seeking approval for use in addition to body cooling; a PDUFA date of 6 months is expected; company received a complete response letter in July 2017 | 1/9/20 | Infection |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | CHIKV-VLP | Virus-like particle vaccine | Chikungunya virus | Came to an agreement with the EMA to pursue a plan to conduct a safety and immunogenicity phase III trial using Serum Neutralizing Antibodies as an immune correlate of protection to predict clinical benefit | 1/13/20 | Infection |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | Vaxchora (cholera vaccine, live, oral) | Single-dose vaccine | Cholera | EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for use as active immunization against disease caused by Vibrio cholerae serogroup O1 in adults and children 6 and older | 1/31/20 | Infection |
| Meissa Vaccines Inc., of South San Francisco | MV-012-968 | Live attenuated vaccine | Prevention of respiratory syncytial virus infection | FDA granted fast track status | 1/10/20 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Dificid (fidaxomicin) | Cytochrome P450 reductase inhibitor; RNA polymerase inhibitor | Clostridioides difficile infection | FDA approved NDA for oral suspension and sNDA for tablet formulation to treat C. diff-associated diarrhea in children 6 months and older | 1/27/20 | Infection |
| Nabriva Therapeutics plc, of Dublin | Contepo (fosfomycin) | Antibiotic | Complicated urinary tract infections | FDA acknowledged receipt of NDA resubmission | 1/8/20 | Infection |
| Novavax Inc., of Gaithersburg, Md. | Nanoflu | Recombinant quadrivalent seasonal influenza vaccine | Influenza | FDA granted fast track designation for use in adults, 65 and older | 1/15/20 | Infection |
| Pulmatrix Inc., of Lexington, Mass. | PUR-1900 | Inhaled itraconazole antifungal candidate | Allergic bronchopulmonary aspergillosis in asthma patients | FDA granted fast track designation | 1/30/20 | Infection |
| Synthetic Biologics Inc., Rockville, Md. | SYN-004 (ribaxamase) | Degrades certain I.V. beta-lactam antibiotics | Allogeneic hematopoietic cell transplant recipients receiving I.V. beta-lactam antibiotics in response to fever | Based on the final meeting minutes from a type C meeting with the FDA, company plans to run a placebo-controlled phase Ib/IIa study in up to 36 patients with 3 sequential cohorts administered a different study-assigned intravenous beta-lactam antibiotic | 1/7/20 | Infection |
| Tennor Therapeutics Ltd., of Suzhou, China | TNP-2092 | Targets RNA polymerase, DNA gyrase and topoisomerase IV | Prosthetic joint infections | FDA accepted an IND for a phase I study | 1/20/20 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1553 | Monovalent, single-dose, live-attenuated vaccine candidate | Chikungunya | End-of-phase II meeting scheduled with FDA for Feb. 24, 2020, during which company will present its plan for phase III studies and potential licensure | 1/7/20 | Infection |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Verdiperstat | Myeloperoxidase inhibitor | Amyotrophic lateral sclerosis | FDA notified collaborators at Sean M. Healey & AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital they may proceed with clinical investigation | 1/22/20 | Musculoskeletal |
| Carmell Therapeutics Inc., of Pittsburgh | BHA | Bone healing accelerant | Long bone fractures | FDA cleared the IND for enrollment of patients in a pivotal study to support a BLA | 1/7/20 | Musculoskeletal |
| Clene Nanomedicine Inc., of Salt Lake City | CNM-Au8 | Concentrated, aqueous suspension of clean-surfaced faceted nanocrystalline gold | Amyotrophic lateral sclerosis | Received formal acceptance from FDA to proceed with CNM-Au8 as 1 of first 3 drug regimens in Healey ALS Platform Trial | 1/24/20 | Musculoskeletal |
| Denali Therapeutics Inc., of South San Francisco | DNL-343 | EIF2B activator | Amyotrophic lateral sclerosis and other neurodegenerative disorders | CTA cleared for phase I trial in healthy volunteers | 1/14/20 | Musculoskeletal |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | p38alpha/beta mitogen activated protein kinase inhibitor | Facioscapulohumeral muscular dystrophy | FDA granted orphan drug designation | 1/29/20 | Musculoskeletal |
| Ra Pharmaceuticals Inc., of Cambridge, Mass. | Zilucoplan | Amyotrophic lateral sclerosis | Complement C5 factor inhibitor | FDA cleared IND for Healey ALS platform trial, with zilucoplan selected as an initial candidate | 1/22/20 | Musculoskeletal |
| Ultragenyx Pharmaceuticals Inc., of Novato, Calif., and Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab-twza) | Recombinant fully human monoclonal IgG1 antibody against FGF23 | Tumor-induced osteomalacia | Submitted an sBLA to the FDA for tumors that cannot be curatively resected or localized | 1/13/20 | Musculoskeletal |
| Affiris AG, of Vienna | Affitope PD-01 | Synuclein alpha inhibitor | Parkinson's disease | FDA provided feedback on pre-IND submission for planned phase II trial in early stage disease; based on agency's guidance, trial initiation in U.S. and Europe expected in second half of 2020 | 1/27/20 | Neurology/Psychiatric |
| Alkermes plc, of Dublin | ALKS-3831 (olanzapine/samidorphan) | Atypical antipsychotic | Schizophrenia or bipolar I disorder | FDA accepted the NDA; PDUFA target action date is Nov. 15, 2020 | 1/28/20 | Neurology/Psychiatric |
| Baudax Bio Inc., of Malvern, Pa. | Meloxicam (intravenous) | Cyclooxygenase 2 inhibitor | Pain | FDA set Feb. 20, 2020, PDUFA date for NDA to treat moderate to severe pain | 1/22/20 | Neurology/Psychiatric |
| Cadent Therapeutics Inc., of Cambridge, Mass. | CAD-1883 | Positive allosteric modulator of SK channels | Spinocerebellar ataxia | FDA accepted IND for a phase II trial | 1/23/20 | Neurology/Psychiatric |
| Corium International Inc., of Menlo Park, Calif. | Adlarity (donepezil transdermal patch) | Acetylcholinesterase inhibitor | Alzheimer's disease | FDA accepted NDA filing for treatment of individuals with mild, moderate and severe disease, setting PDUFA date of July 30, 2020 | 1/27/20 | Neurology/Psychiatric |
| Durect Corp., of Cupertino, Calif. | Posimir (bupivacaine extended-release solution) | Binds to intracellular portion of voltage-gated sodium channels and blocks sodium influx into nerve cells | Postoperative pain | FDA’s Anesthetic and Analgesic Drug Products Advisory Committee voted 6-6 on the question of whether the efficacy, safety and overall risk-benefit profile of Posimir support approval of the NDA | 1/16/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Fycompa (perampanel) | Anti-epileptic drug | Partial-onset seizures | Supplemental NDAs approved in Japan for use as monotherapy of partial-onset seizures and for partial-onset seizures in pediatric patients, ages 4 and older; regulators also approved a new fine granule formulation | 1/23/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Dayvigo (lemborexant) | Binds to orexin receptors OX1R and OX2R | Insomnia | Approved in Japan | 1/22/20 | Neurology/Psychiatric |
| Genetex Biotherapeutics LLC, of Sarasota, Fla., and Ultragenyx Pharmaceutical Inc., of Novato, Calif. | GTX-102 | Antisense oligonucleotide | Angelman syndrome | IND filed with FDA is active; enrollment in phase I/II study expected to start in first half of 2020 | 1/15/20 | Neurology/Psychiatric |
| Intelgenx Corp., of Saint Laurent, Quebec | Montelukast Versafilm | CysLT1 antagonist | Mild to moderate Alzheimer’s disease | Health Canada issued no objection letter in response amended clinical trial application for ongoing phase IIa Buena study | 1/15/20 | Neurology/Psychiatric |
| Intellipharmaceutics International Inc., of Toronto | Aximris XR (oxycodone extended-release tablets) | Long-acting, abuse-deterrent formulation of opioid drug | Pain | FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee voted to not support approval for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate | 1/15/20 | Neurology/Psychiatric |
| Ipsen SA, of Paris | Dysport (clostridium botulinum type A toxin-haemagglutinin complex) | Neurotoxin | Focal spasticity of upper limbs in pediatric cerebral palsy patients, 2 and older | U.K.’s Medicines and Healthcare Products Regulatory Agency granted a license update | 1/6/20 | Neurology/Psychiatric |
| Janssen Pharmaceutical Cos., of Johnson & Johnson, of New Brunswick, N.J. | Spravato (esketamine) nasal spray | S-enantiomer of racemic ketamine | Major depressive disorder | Submitted type II variation application to the EMA seeking to expand use as acute short-term treatment, co-administered with oral antidepressant therapy, for rapid reduction of depressive symptoms in adults with a moderate to severe depressive episode who have current suicidal ideation with intent | 1/15/20 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | Sunosi (solriamfetol) | Dopamine and norepinephrine reuptake inhibitor | Narcolepsy (with or without cataplexy) or obstructive sleep apnea | European Commission approved the drug to improve wakefulness and reduce excessive daytime sleepiness | 1/20/20 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | JZP-258 (sodium oxybate + potassium oxybate + calcium oxybate + magnesium oxybate) | GABA B receptor agonist | Cataplexy; narcolepsy | NDA submitted to FDA to treat cataplexy and excessive daytime sleepiness in individuals 7 and older with narcolepsy; with submission, company redeemed rare pediatric disease priority review voucher, acquired in 2018 from Spark Therapeutics Inc. | 1/22/20 | Neurology/Psychiatric |
| Kyowa Kirin Co. Ltd., of Tokyo | Istradefylline (KW-6002) | Adenosine A2A receptor antagonist | Parkinson's disease | MAA validated by the EMA | 1/6/20 | Neurology/Psychiatric |
| Nektar Therapeutics Inc., of San Francisco | Oxycodegol (NKTR-181) | Selective mu-opioid receptor agonist | Chronic pain | FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee unanimously voted 27-0 against recommending approval of drug for use as second-line management of chronic low back pain in adults with pain severe enough to require daily, around-the-clock, long-term opioid treatment; as a result of the vote, the company decided to withdraw its NDA and make no further investment in the program | 1/14/20 | Neurology/Psychiatric |
| Neurelis Inc., of San Diego | Valtoco (diazepam) | Nasal spray; formulation of diazepam | Epilepsy | FDA approved Valtoco as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity distinct from a patient's usual seizure pattern; first rescue treatment for people with epilepsy aged 6 and older; drug has orphan drug exclusivity | 1/13/20 | Neurology/Psychiatric |
| Newron Pharmaceuticals SpA, of Milan, Italy | Evenamide | Targets voltage-gated sodium channels for the treatment of schizophrenia | Positive symptoms of schizophrenia | Reached agreement with FDA on design and conduct of explanatory studies required to address potential safety issues raised by the agency | 1/9/20 | Neurology/Psychiatric |
| OWP Pharmaceuticals Inc., of Naperville, Ill. | Quetiapine fumarate | Antipsychotic | Schizophrenia and bipolar disorder | Received pre-IND acknowledgement from FDA for liquid formulation at aimed providing alternative delivery for drug | 1/8/20 | Neurology/Psychiatric |
| Paion AG, of Aachen, Germany | Anerem (remimazolam) | Ultra-short-acting intravenous benzodiazepine sedative/anesthetic | General anesthesia | Licensee Mundipharma GmbH received approval by Japan’s Ministry of Health, Labor and Welfare | 1/23/20 | Neurology/Psychiatric |
| Renetx Bio Inc., of New Haven, Conn. | AXER-204 | Human fusion protein | Chronic spinal cord injury | FDA granted fast track designation | 1/8/20 | Neurology/Psychiatric |
| Seelos Therapeutics Inc., of New York | SLS-002 | Intranasal racemic ketamine | Acute suicidal ideation and behavior in patients with major depressive disorder | Requested a type C meeting with FDA for guidance on an adaptive phase III study | 1/6/20 | Neurology/Psychiatric |
| Supernus Pharmaceuticals Inc., of Rockville, Md. | SPN-812 (viloxazine hydrochloride) | Serotonin norepinephrine modulating agent | Attention deficit hyperactivity disorder | FDA accepted NDA, assigning PDUFA date of Nov. 8, 2020 | 1/21/20 | Neurology/Psychiatric |
| Teva Pharmaceuticals USA Inc., a unit of Jerusalem-based Teva Pharmaceuticals Co. Ltd. | Ajovy (fremanezumab-vfrm) | Monoclonal antibody targeting calcitonin gene-related peptide | Migraine prevention | FDA approved an autoinjector for the drug | 1/28/20 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | AV-101 | Oral NMDAR glycine site antagonist | Dyskinesia in Parkinson’s disease | FDA cleared the IND for a phase II study | 1/30/20 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Roclanda (netarsudil and latanoprost ophthalmic solution) | Combination of Rho kinase inhibitor with prostaglandin analogue | Elevated intraocular pressure in open-angle glaucoma or ocular hypertension | EMA accepted for review MAA; opinion from agency’s Committee for Medicinal Products for Human Use expected in late 2020 | 1/2/20 | Ocular |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Rocklatan (netarsudil and latanoprost) | Noradrenaline transporter inhibitor; PGF2 alpha agonist; RHO-associated protein kinase inhibitor | Open-angle glaucoma; ocular hypertension | FDA approved company's sterile fill production facility in Athlone, Ireland, to produce drug product | 1/21/20 | Ocular |
| Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab-trbw) | Fully human monoclonal antibody IGF-1R inhibitor | Thyroid eye disease | Approved by FDA | 1/21/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France, and Bausch + Lomb, of Rochester, N.Y. | Vyzulta (latanoprostene bunod ophthalmic solution) 0.024% | Prostaglandin F2 alpha analogue | Reduction of intraocular pressure in open-angle glaucoma or ocular hypertension | Bausch + Lomb received approval in Hong Kong and Argentina | 1/16/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France, and Bausch and Lomb | Vyzulta | Latanoprostene bunod ophthalmic solution, 0.024% | Open-angle glaucoma and ocular hypertension | Received approval in Mexico to reduce intraocular pressure | 1/13/20 | Ocular |
| Proqr Therapeutics NV, of Leiden, the Netherlands | QR-421a | RNA-based oligonucleotide | Retinitis pigmentosa | FDA granted rare pediatric disease designation for RP caused by mutations in exon 13 of the USH2A gene | 1/30/20 | Ocular |
| Biostage Inc., of Holliston, Mass. | Cellspan Esophageal Implant | Biocompatible scaffold with patient's own cells | Esophageal implant | Received formal FDA response to IND application, detailing specific questions and clarifications; company is finalizing responses | 1/2/20 | Other/Miscellaneous |
| Arrevus Inc., of Raleigh, N.C. | ARV-1801 | Sodium fusidate tablet | Cystic fibrosis pulmonary exacerbations | FDA granted qualified infectious disease product designation | 1/28/20 | Respiratory |
| Proteostasis Therapeutics Inc., of Boston | Dirocaftor, posenacaftor and nesolicaftor | A CFTR potentiator, corrector and amplifier | Cystic fibrosis | U.K.'s Medicines and Healthcare Products Regulatory Agency outlined a path forward for a phase III program, with 2 trials beginning concurrently this year; the Choices trial seeks to translate responses from an ex vivo organoids study of PTI modulators in rare CF mutations, while the More trial will seek to confirm efficacy and tolerability results from a recently completed phase II study | 1/13/20 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 | Genetically targeted agent | Alcohol use disorder | Received favorable opinions for Finnish Medicines Agency and National Committee on Medical Research Ethics to start phase III study | 1/9/20 | Toxicity and Intoxication |
| Alx Oncology Inc., of Burlingame, Calif. | ALX-148 | Next-generation CD47 myeloid checkpoint inhibitor | Head and neck squamous cell carcinoma | FDA granted fast track status | 2/18/20 | Cancer |
| Alx Oncology Inc., of Burlingame, Calif. | ALX-148 | Next-generation CD47 myeloid checkpoint inhibitor | HER2-positive gastric of gastroesophageal junction carcinoma | FDA granted fast track status for second-line treatment | 2/18/20 | Cancer |
| Aptorum Group Ltd., of Hong Kong | SACT-1 | Repurposed drug; small molecule | Neuroblastoma | Plans to leverage 505(b)(2) pathway and submit IND to FDA in second half of 2020 | 2/10/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-1387 | Inhibitor of apoptosis proteins inhibitor | Advanced pancreatic cancer | Received approval from the Center of Drug Evaluation, China NMPA, to start a phase Ib/II study testing the drug in combination with chemotherapy (nab-paclitaxel plus gemcitabine) | 2/24/20 | Cancer |
| Astex Pharmaceuticals Inc., of Pleasanton, Calif., a subsidiary of Otsuka Pharmaceutical Co. Ltd. | Oral C-DEC (cedazuridine and decitabine) | Fixed-dose combination of cytidine deaminase inhibitor and hypomethylating agent | Myelodysplastic syndromes | FDA accepted for priority review the NDA seeking approval for use in previously untreated patients with intermediate- and high-risk disease, including chronic myelomonocytic leukemia | 2/11/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | KIT kinase inhibitor | Fourth-line gastrointestinal stromal tumor | FDA extended PDUFA action date to May 14, 2020, to review data from the ongoing phase III Voyager study | 2/6/20 | Cancer |
| Bold Therapeutics Inc., OF Vancouver, British Columbia | BOLD-100 | Cancer resistance pathway inhibitor | Gastrointestinal cancer | Health Canada cleared initiation of phase Ib combination trial with Folfox in gastric, pancreatic, colorectal and bile duct cancers | 2/10/20 | Cancer |
| Bristol-Myers Squibb Co., of New York | Lisocabtagene maraleucel | Autologous anti-CD19 CAR T-cell immunotherapy | Large B-cell lymphoma | FDA accepted BLA to treat adults with relapsed/refractory disease after at least 2 prior therapies, granting priority review and setting PDUFA date of Aug. 17, 2020 | 2/13/20 | Cancer |
| Bristol-Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | Anti-PD-1 antibody | Esophageal cancer | Japan’s Ministry of Health, Labor and Welfare approved for use in patients with unresectable advanced or recurrent disease that has progressed following chemotherapy | 2/21/20 | Cancer |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | Adenosine A3 receptor agonist | Hepatocellular carcinoma | Submitted protocol design and registration plan for pivotal phase III trial to EMA's CHMP, seeking to evaluate efficacy of drug in individuals with advanced disease with underlying Child Pugh B7 cirrhosis whose cancer progressed on first-line therapy; study design of single phase III trial, already endorsed by FDA, would enable concurrent approval in U.S. and Europe | 2/13/20 | Cancer |
| Coimmune Inc., of Durham, N.C. | CMN-001 (formerly AGS-003) | Dendritic cell-based immunotherapy | Advanced metastatic renal cell carcinoma | FDA cleared IND for phase IIb trial | 2/19/20 | Cancer |
| Debiopharm Group, of Lausanne, Switzerland | Debio-1143 | Antagonist of inhibitor of apoptosis proteins | Squamous cell carcinoma of the head and neck | FDA granted breakthrough therapy designation | 2/27/20 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Ripretinib | Dual KIT/PDGFR-alpha inhibitor | Gastrointestinal stromal tumor | FDA accepted NDA under Oncology Center of Excellence Real-Time Oncology Review pilot program, granting priority review and setting Aug. 13, 2020, PDUFA date | 2/12/20 | Cancer |
| Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. | Pemfexy (pemetrexed for injection; branded alternative to Alimta) | Chemotherapy | Non-small-cell lung cancer and mesothelioma | FDA granted final approval; indications include locally advanced or metastatic nonsquamous NSCLC in combination with cisplatin; locally advanced or metastatic nonsquamous NSCLC patients whose disease has not progressed after 4 cycles of platinum-based first-line chemotherapy, as maintenance treatment; locally advanced or metastatic nonsquamous NSCLC after prior chemotherapy as a single agent; and malignant pleural mesothelioma patients whose disease is unresectable or who are otherwise not candidates for curative surgery in combination with cisplatin | 2/10/20 | Cancer |
| Eli Lilly and Co., of Indianapolis | Cyramza (ramucirumab) | VEGF receptor 2 antagonist | Non-small-cell lung cancer | FDA’s Oncology Drugs Advisory Committee voted 6-5 that the drug plus erlotinib demonstrated a favorable benefit/risk profile for patients with untreated metastatic EGFR-positive disease based on results of the phase III Relay study | 2/26/20 | Cancer |
| Epizyme Inc., of Cambridge, Mass. | Tazverik (tazemetostat) | Methyltransferase inhibitor | Relapsed or refractory follicular lymphoma | FDA accepted NDA seeking approval for use in patients who have received at least 2 prior lines of systemic therapy; agency granted priority review, with a PDUFA date of June 18, 2020 | 2/14/20 | Cancer |
| Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. | Pedmark | Sodium thiosulfate | Prevention of cisplatin-induced hearing loss (ototoxicity) in children with solid tumors | Completed rolling submission of an NDA to the FDA for intravenous use; also submitted an MAA to the EMA | 2/11/20 | Cancer |
| Genentech, of South San Francisco, a member of the Roche Group, and Halozyme Therapeutics Inc., of San Diego | Fixed-dose combination of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase | HER2-targeted antibodies with hyaluronidase administered by subcutaneous injection | HER2-positive breast cancer | FDA accepted the BLA seeking approval for use in combination with intravenous chemotherapy | 2/25/20 | Cancer |
| Genentech, of South San Francisco, a unit of Roche AG | Tecentriq (atezolizumab) | PD-L1-targeting antibody | Advanced nonsquamous and squamous non-small-cell lung cancer | FDA accepted supplemental BLA and granted priority review; application seeks approval for patients without EGFR or ALK mutations with high PD-L1 expression as determined by biomarker testing; PDUFA date is June 19, 2020 | 2/19/20 | Cancer |
| Glaxosmithkline plc, of London | Zejula (niraparib) | PARP inhibitor | First-line maintenance treatment for platinum-responsive advanced ovarian cancer | FDA accepted the sNDA under the real-time oncology review pilot program | 2/24/20 | Cancer |
| Glaxosmithkline plc, of London | Zejula (niraparib) | PARP inhibitor | Advanced ovarian cancer | EMA validated type II variation for use of drug as maintenance treatment in the first-line setting for women who responded to platinum-based chemotherapy regardless of biomarker status; formal review process for EMA’s CHMP begins | 2/27/20 | Cancer |
| Glaxosmithkline plc, of London | Belantamab mafodotin | BCMA monoclonal antibody conjugated to auristatin F | Multiple myeloma | EMA accepted MAA to treat relapsed/refractory disease in individuals whose prior therapy included immunomodulatory agent, proteasome inhibitor and anti-CD38 antibody; accelerated assessment granted by CHMP | 2/3/20 | Cancer |
| Glycomimetics Inc., of Rockville, Md. | GMI-1359 | Inhibits adhesion molecules E-selectin and CXCR4 | Osteosarcoma | FDA granted orphan and rare pediatric disease designations | 2/4/20 | Cancer |
| Harbour Biomed Therapeutics Ltd., of Cambridge, Mass. | HBM-9167 | Humanized IgG1 monoclonal antibody targeting PD-L1 | Nasopharyngeal cancer | FDA cleared the IND for a phase II study; agency also granted orphan designation | 2/27/20 | Cancer |
| Incyte Corp., of Wilmington, Del. | Capmatinib | Selective MET inhibitor | Non-small-cell lung cancer | FDA accepted the NDA and granted priority review; seeking approval for use in locally advanced or metastatic MET exon 14-skipping mutated disease | 2/11/20 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex | Daratumumab | Relapsed/refractory multiple myeloma | Submitted an sBLA to the FDA for Darzalex in combination with Kyprolis (carfilzomib) and dexamethasone | 2/11/20 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | Oral selective inhibitor of nuclear transport | Relapsed or refractory diffuse large B-cell lymphoma | FDA accepted sNDA, granted priority review and assigned PDUFA action date of June 23, 2020 | 2/19/20 | Cancer |
| Kiadis Pharma NV, of Amsterdam | Off-the-shelf natural killer cells from universal donors | Off-the-shelf natural killer cells from universal donors | Acute myeloid leukemia | FDA cleared clinical study by the Ohio State University in relapsed/refractory AML | 2/26/20 | Cancer |
| Kite, of Santa Monica, Calif., a Gilead Co. | KTE-X19 | CAR T-cell therapy | Relapsed or refractory mantle cell lymphoma | FDA accepted the BLA and granted priority review designation | 2/10/20 | Cancer |
| Kleo Pharmaceuticals Inc., of New Haven, Conn. | KP-1237 | CD38-targeting antibody recruiting molecule | Multiple myeloma post-transplant | FDA cleared IND for study testing the drug in combination with patients' natural killer cells that will begin the first half of 2020 | 2/6/20 | Cancer |
| Marker Therapeutics Inc., of Houston | MultiTAA | MAPP multi-antigen targeted T-cell therapy | Post-transplant acute myeloid leukemia | FDA lifted the clinical hold on planned trial investigating safety and efficacy | 2/11/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1-targeting antibody | Multiple cancer indications | FDA issued complete response letter regarding supplemental BLAs seeking to update dosing frequency to include a 400-mg dose infused over 30 minutes every six weeks | 2/19/20 | Cancer |
| Monopar Therapeutics Inc., of Chicago | Camsirubicin | Doxorubicin analogue | Soft-tissue sarcoma | European Commission granted orphan designation | 2/18/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Designed to destroy tumors through physical cell death when activated by radiotherapy | Head and neck squamous cell cancer | FDA granted fast track designation for use, with or without cetuximab, in patients with locally advanced disease who are not eligible for platinum-based chemotherapy | 2/10/20 | Cancer |
| Novartis AG, of Basel, Switzerland | Capmatinib (INC-280) | MET inhibitor | METex14 mutated advanced non-small-cell lung cancer | FDA granted priority review | 2/11/20 | Cancer |
| Noxopharm Ltd., of Sydney | Veyonda (idronoxil) | S1P inhibitor | Soft tissue sarcomas | FDA cleared IND to test combination with doxorubicin | 2/25/20 | Cancer |
| Pharmamar SA, of Madrid, Spain, and Jazz Pharmaceuticals plc, of Dublin | Lurbinectedin | Inhibits oncogenic transcription in tumor-associated macrophages | Small-cell lung cancer | FDA accepted NDA for filing with priority review, seeking accelerated approval for use in patients who have progressed after prior platinum-containing therapy; PDUFA date of Aug. 16, 2020 | 2/17/20 | Cancer |
| Puma Biotechnology Inc., of Los Angeles | Nerlynx (neratinib) | HER2-targeting therapy | Breast cancer | FDA approved a supplemental NDA for use in combination with capecitabine for treatment of adults with advanced or metastatic HER2-positive disease who have received 2 or more prior anti-HER2-based regimens in the metastatic setting | 2/26/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | Anti-PD-L1 antibody | Small-cell lung cancer | China’s NMPA approved for use in combination with carboplatin and etoposide for first-line treatment of patients with extensive-stage disease | 2/14/20 | Cancer |
| Scancell Holdings plc, of Nottingham, U.K. | SCIB-1 | Immunoglobulin Fc receptor modulator | Melanoma | FDA approved IND for U.S. arm of phase II study in 25 individuals with metastatic disease also receiving pembrolizumab (Keytruda, Merck & Co. Inc.), using electroporation delivery device; study will assess whether addition of study drug to checkpoint inhibitor results in improved tumor response rate, progression-free survival and overall survival | 2/3/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Tucatinib | Erbb2 tyrosine kinase receptor inhibitor | Breast cancer | FDA accepted NDA for study drug, in combination with trastuzumab and capecitabine, to treat locally advanced unresectable or metastatic HER2-positive disease, including brain metastases, in individuals who received at least 3 prior HER2-directed agents; agency granted priority review, setting Aug. 20, 2020, PDUFA date | 2/13/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash., and Astellas Pharma Inc., of Tokyo | Padcev (enfortumab vedotin-ejfv) | Antibody-drug conjugate directed against Nectin-4 | Urothelial cancer | FDA granted breakthrough therapy designation for use in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with unresectable locally advanced or metastatic disease who are unable to receive cisplatin-based chemotherapy in the first-line setting | 2/19/20 | Cancer |
| Sellas Life Sciences Group, of New York | Nelipepimut-S | Targets the Wilms tumor 1 protein | Triple-negative breast cancer | Based on feedback from type C meeting with FDA, company finalized design for phase III registration-enabling study in TNBC patients in adjuvant setting after standard treatment; if successful, study may be considered basis of BLA filing | 2/14/20 | Cancer |
| Sirnaomics Inc., of Gaithersburg, Md. | STP-705 | siRNA therapeutic | Hepatocellular carcinoma | Granted orphan drug designation by the FDA | 2/11/20 | Cancer |
| Steba Biotech SA, of Luxembourg | Tookad (padeliporfin dipotassium) | Vascular-targeted photodynamic therapy | Prostate cancer | FDA’s Oncologic Drugs Advisory Committee voted 13-2 against recommending approval of the NDA seeking use in men with localized early stage disease | 2/26/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Alunbrig (brigatinib) | Anaplastic lymphoma kinase tyrosine kinase inhibitor | First-line ALK-positive metastatic non-small-cell lung cancer | FDA granted a priority review for the sNDA; PDUFA action date of June 23, 2020 | 2/24/20 | Cancer |
| Tessa Therapeutics Ltd., of Singapore | CD30 CAR T | CD30-directed autologous CAR T-cell therapy | Relapsed/refractory CD30-positive classical Hodgkin lymphoma | FDA granted regenerative medicine advanced therapy designation | 2/27/20 | Cancer |
| Tetra Bio-Pharma Inc., of Ottawa | Qixleef | Dried flower botanical cannabinoid | Advanced cancer patients with uncontrolled pain | In a letter of advice and type B meeting with the FDA, the agency validated the company's nonclinical program; the agency also validated the company's approach to ensuring the quality and lot-to-lot variation across the nonclinical and clinical programs | 2/24/20 | Cancer |
| Ultragenyx Pharmaceutical Inc., of Novato, Calif., and Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Recombinant fully human monoclonal IgG1 antibody | FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia) | FDA accepted for review supplemental BLA seeking use in patients whose disease cannot be curatively resected or localized; agency assigned priority review, with PDUFA date of June 18, 2020 | 2/27/20 | Cancer |
| United Therapeutics Corp., of Research Triangle Park, N.C., and Deka Research & Development Corp., of Manchester, N.H. | Remunity pump | Subcutaneous delivery system for Remodulin (treprostinil) | Pulmonary arterial hypertension | FDA granted additional 510(k) clearance for drug reservoirs that have been prefilled by specialty pharmacies | 2/24/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Omburtamab | Monoclonal antibody targeting B7-H3 | CNS/leptomeningeal metastases from neuroblastoma | Completed type B pre-BLA meeting with FDA and reached alignment on accelerated approval pathway, along with rolling BLA; company expects to complete rolling BLA within 10 weeks | 2/27/20 | Cancer |
| Esperion Inc., of Ann Arbor, Mich. | Nexletol (Bempedoic acid) | ATP citrate lyase inhibitor | Heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein cholesterol (LDL-C) | FDA approval for bempedoic acid as an adjunct to diet and maximally tolerated statin therapy | 2/21/20 | Cardiovascular |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | BET inhibitor | Major adverse cardiac events | FDA granted breakthrough therapy designation, in combination with standard of care to include high-intensity statins, for secondary prevention of MACE in individuals with type 2 diabetes and recent acute coronary syndrome | 2/3/20 | Cardiovascular |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Berotralstat | Plasma kallikrein inhibitor | Hereditary angioedema | Submitted an NDA to the Japanese Pharmaceuticals and Medical Devices Agency | 2/3/20 | Dermatologic |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Berotralstat (BCX-7353) | Oral kallikrein inhibitor | Prevention of hereditary angioedema attacks | FDA accepted the NDA and set PDUFA date of Dec. 3, 2020 | 2/18/20 | Dermatologic |
| Novan Inc., of Morrisville, N.C. | SB-206 | Nitric oxide-releasing topical gel | Molluscum contagiosum | FDA granted a type C meeting scheduled for April 1, 2020 | 2/6/20 | Dermatologic |
| Revance Therapeutics Inc., of Newark, Calif. | Daxi | DaxibotulinumtoxinA | Moderate to severe glabellar lines | FDA accepted BLA; PDUFA action date of Nov. 25, 2020; agency is not currently planning to hold an advisory committee meeting | 2/6/20 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Onpattro (patisiran) | RNAi therapeutic | Hereditary transthyretin-mediated amyloidosis | Approved by Brazilian Health Regulatory Agency (ANVISA) | 2/26/20 | Endocrine/Metabolic |
| Diurnal Group plc, of Cardiff, U.K. | Alkindi Sprinkle (hydrocortisone) | Glucocorticoid receptor agonist | Adrenal gland hypofunction | FDA accepted NDA for review as replacement therapy for adrenal insufficiency in infants, children and adolescents, setting PDUFA date of Sept. 29, 2020 | 2/13/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Trulicity (dulaglutide) | GLP-1 receptor agonist | Reduction of major adverse cardiovascular events in adults with type 2 diabetes | FDA approved the expanded indication for patients who have established cardiovascular disease or multiple cardiovascular risk factors | 2/21/20 | Endocrine/Metabolic |
| Horizon Therapeutics plc, of Dublin | Procysbi (cysteamine bitartrate) delayed-release oral granules | Cystine-depleting agent | Nephropathic cystinosis | FDA approved oral granule dosage for use in adults and children, 1 and older | 2/18/20 | Endocrine/Metabolic |
| Logicbio Therapeutics Inc., of Cambridge, Mass. | LB-001 | Recombinant adeno-associated vector with human methylmalonyl-COA mutase gene | Methylmalonic acidemia | The FDA placed a clinical hold on the IND submission | 2/11/20 | Endocrine/Metabolic |
| Lysogene SA, of Paris | LYS-SAF302 | Gene therapy designed to deliver functional copy of the SGSH gene to the brain through 1-time direct-to-CNS administration | Mucopolysaccharidosis type IIIA | FDA granted fast track status | 2/25/20 | Endocrine/Metabolic |
| Prevail Therapeutics Inc., of New York | PR-001 | AAV-based gene therapy | Gaucher disease | FDA granted orphan designation; agency also granted rare pediatric disease designation for neuronopathic Gaucher disease | 2/12/20 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa (PRX-102) | Alpha-galactosidase stimulator | Fabry disease | Reached agreement with the FDA on an initial pediatric study plan | 2/6/20 | Endocrine/Metabolic |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-41 | Oral thyroid hormone receptor-beta agonist | Nonalcoholic steatohepatitis | China’s NMPA accepted IND for clinical trials; drug is expected to be used in combination with ASC-40 an oral fatty acid synthase inhibitor | 2/17/20 | Gastrointestinal |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Vedolizumab SC | Subcutaneous version of alpha4beta7 integrin antagonist | Moderately to severely active ulcerative colitis or Crohn’s disease | EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for use as maintenance therapy in adults; Takeda proposes to make therapy available in both a prefilled syringe and a prefilled pen | 2/28/20 | Gastrointestinal |
| Vivus Inc., of Campbell, Calif. | Pancreaze (pancrelipase) | Amylase stimulator; pancreatic lipase stimulator; protease stimulator | Exocrine pancreatic insufficiency | FDA approved sNDA for improved formulation that extends shelf life to 36 months across all dosages | 2/5/20 | Gastrointestinal |
| Agile Therapeutics Inc., of Princeton, N.J. | Twirla (levonorgestrel and ethinyl estradiol) | Once-weekly hormonal contraceptive patch | Contraception | FDA approved for use in women with BMI < 30 kg/m2 for whom a combined hormonal contraceptive is appropriate | 2/14/20 | Genitourinary/Sexual Function |
| Therapeuticsmd Inc., of Boca Raton, Fla. | Bijuva (estradiol + progesterone) | Estradiol agonist; progesterone receptor agonist | Severe vasomotor symptoms | NDA prior-approval efficacy supplement submitted to FDA for 0.5 mg/100-mg dosage | 2/3/20 | Genitourinary/Sexual Function |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | AAV5 gene therapy | Hemophilia A | FDA accepted the BLA and granted priority review, setting PDUFA date of Aug. 21, 2020; FDA informed company no advisory meeting is currently planned | 2/20/20 | Hematologic |
| CTI Biopharma Corp., of Seattle | Pacritinib | FLT3/IRAK-1/JAK2 kinase inhibitor; CSF-1 antagonist | Thrombocytopenia | FDA agreed to accelerated approval pathway for treatment of myelofibrosis patients with severe disease (platelet counts <50,000/µL); pivotal phase III Pacifica trial protocol amendment will allow for primary analysis of SVR rates on first 168 participants, which, if met, will trigger company's NDA submission under FDA's subpart H regulations | 2/3/20 | Hematologic |
| Fibrogen Inc., of San Francisco | Roxadustat | Oral, small-molecule hypoxia-inducible factor prolyl hydroxylase inhibitor | Anemia of chronic kidney disease | FDA completed its filing review of NDA seeking approval for use in both nondialysis-dependent and dialysis-dependent patients and set PDUFA date of Dec. 20, 2020 | 2/11/20 | Hematologic |
| Forma Therapeutics Inc., of Watertown, Mass. | FT-4202 | Pyruvate kinase-R activator | Sickle cell disease | FDA granted fast track and rare pediatric disease designations | 2/20/20 | Hematologic |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa. | PB-2452 | Monoclonal antibody antigen-binding fragment targeting ticagrelor | Antiplatelet reversal | Granted PRIME designation by the EMA | 2/11/20 | Hematologic |
| Amgen Inc., of Thousand Oaks, Calif. | Otezla (apremilast) | PDE4 inhibitor | Behçet’s disease | EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion to extend use for treating ulcers in patients with Behçet’s | 2/28/20 | Immune |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-108 (sublingual epinephrine) | Hormone | Anaphylaxis | During a pre-IND meeting, the FDA confirmed that the drug would be reviewed under 505(b)(2); plans to start pivotal pharmacokinetic studies by the end of 2020 | 2/6/20 | Immune |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut | Epicutaneous immunotherapy | Peanut allergy | FDA will hold Allergenic Products Advisory Committee meeting May 15, 2020, to discuss BLA | 2/21/20 | Immune |
| Mesoblast Ltd., of Melbourne, Australia | Ryoncil (remestemcel-L) | Allogeneic cell therapy | Graft-vs.-host disease | Completed BLA submitted to FDA for treatment of children with steroid-refractory acute disease, with request for priority review based on fast track designation in indication | 2/3/20 | Immune |
| Novartis AG, of Basel, Switzerland, and Genmab A/S, of Copenhagen, Denmark | Ofatumumab | Monoclonal antibody targeting CD20 | Relapsing forms of multiple sclerosis | FDA accepted the sBLA and gave the application a priority review with a decision expected in June 2020; EMA accepted the MAA with a decision expected by the second quarter of 2021 | 2/24/20 | Immune |
| Allovir Inc., of Cambridge, Mass. | Viralym-M (ALVR-105) | Allogeneic, off-the-shelf, multivirus-specific T-cell therapy | Infection | EMA granted PRIME designation for treating serious infections with BK virus, cytomegalovirus, human herpes virus-6, Epstein-Barr virus and/or adenovirus in allogeneic hematopoietic stem cell transplantation recipients | 2/12/20 | Infection |
| Cerovene Inc., of Valley Cottage, N.Y. | Generic of Daraprim (pyrimethamine) tablets | Dihydrofolate reductase inhibitor | Toxoplasmosis | FDA approved first generic for treating infection caused by Toxoplasma gondii when used with a sulfonamide | 2/28/20 | Infection |
| Contrafect Corp., of Yonkers, N.Y. | Exebacase | Lysin | Methicillin-resistant Staphylococcus aureus bloodstream infections | FDA granted breakthrough therapy designation | 2/24/20 | Infection |
| Cormedix Inc., of Berkeley Heights, N.J. | Neutrolin (citrate + heparin + taurolidine) | Antimicrobial catheter-lock solution | Catheter-related blood stream infections | FDA granted rolling review of NDA for prevention of CRBSIs in hemodialysis patients | 2/3/20 | Infection |
| Enochian Biosciences Inc., of Los Angeles | ENOB-HV-01 | Autologous stem cell transplantation | HIV infection | FDA scheduled an Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting for early June | 2/24/20 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Recarbrio (imipenem + cilastatin + relebactam) | Beta lactamase inhibitor; dehydropeptidase-1 inhibitor | Hospital-acquired and ventilator-associated bacterial pneumonia | FDA accepted review sNDA to treat infections caused by susceptible gram-negative microorganisms, granting priority review and setting PDUFA date of June 4, 2020 | 2/3/20 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. (also known as MSD outside the U.S. and Canada) | Ervebo | Ebola Zaire vaccine, live | Zaire ebolavirus | Confirmed 4 African countries, including Democratic Republic of Congo, approved vaccine | 2/14/20 | Infection |
| Seqirus Inc., of Holly Springs, N.C., unit of CSL Ltd., of Melbourne, Australia | Audenz (influenza A [H5N1] monovalent vaccine) | MF-59 adjuvanted cell-culture vaccine | Influenza virus infection | FDA approved pandemic influenza vaccine to treat individuals 6 months and older | 2/3/20 | Infection |
| Seqirus Inc., of Summit, N.J., a unit of CSL Ltd., of Melbourne, Australia | Fluad Quadrivalent | Influenza vaccine plus MF59 adjuvant | Influenza infection prophylaxis | FDA approved the drug for adults 65 years and older | 2/24/20 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | Fetcroja (cefiderocol) | Siderophore cephalosporin antibiotic | Aerobic gram-negative infections | EMA’s Committee for Medicinal Products for Human Use adopted positive opinion for use in treating adults with limited treatment options | 2/28/20 | Infection |
| Zai Lab Ltd., of Shanghai and San Francisco | Omadacycline | Tetracycline antibiotic | Community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections | China’s NMPA accepted the NDA | 2/10/20 | Infection |
| Eli Lilly Canada, a unit of Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | IL-17 antagonist; interleukin 17A ligand inhibitor | Ankylosing spondylitis | Health Canada granted approval for individuals who responded inadequately or are intolerant to conventional therapy | 2/11/20 | Musculoskeletal |
| Mereo Biopharma Group plc, of London | Setrusumab | Anti-sclerostin antibody | Osteogenesis imperfecta | Completed type B end-of-phase II meeting with FDA, which agreed to design of phase III pediatric study prior to submission of a BLA | 2/28/20 | Musculoskeletal |
| Anavex Life Sciences Corp., of New York | Anavex2-73 (blarcamesine) | Activates sigma-1 receptor | Rett syndrome | FDA granted fast track designation | 2/3/20 | Neurology/Psychiatric |
| Aquestive Therapeutics Inc., of Warren, N.J. | Libervant (diazepam) | Buccal film version of benzodiazepine | Management of seizure clusters | FDA accepted NDA and assigned a PDUFA date of Sept. 27, 2020 | 2/10/20 | Neurology/Psychiatric |
| Avenue Therapeutics Inc., of New York | Tramadol (intravenous) | Opioid receptor mu agonist | Pain | FDA accepted NDA for drug to manage moderate to moderately severe pain in adults in medically supervised health care setting, setting Oct. 10, 2020, PDUFA date | 2/13/20 | Neurology/Psychiatric |
| Baudax Bio Inc., of Malvern, Pa. | Anjeso (meloxicam injection) | 24-hour, intravenous COX-2 preferential nonsteroidal anti-inflammatory | Management of moderate or severe pain | Approved by FDA for use alone or in combination with other non-NSAID analgesics | 2/20/20 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Nurtec ODT (rimegepant) | CGRP receptor antagonist formulated in orally disintegrating tablet | Acute migraine | Approved by FDA for use in adults | 2/27/20 | Neurology/Psychiatric |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous mesenchymal stem cell-neurotrophic factor-producing cell therapy | Amyotrophic lateral sclerosis | Representatives from FDA's CBER confirmed during planned meeting that fully enrolled trial is collecting relevant data to assess efficacy; agency agreed to work collaboratively to identify regulatory pathway, including opportunities to expedite statistical review of phase III data | 2/11/20 | Neurology/Psychiatric |
| Cortexyme Inc., of South San Francisco | COR-388 | Gingipain inhibitor | Alzheimer's disease | FDA agreed during type C meeting to interim analysis of ongoing phase II/III Gain trial to assess evidence of overwhelming efficacy after about 100 participants in each of study's 3 arms complete 6 months of dosing, expected by year-end 2020; co-primary endpoints for interim analysis will be change from baseline in ADAS-Cog11 and CDR-SB vs. placebo | 2/13/20 | Neurology/Psychiatric |
| Eli Lilly and Co., of Indianapolis | Emgality (galcanezumab) | Monoclonal antibody targeting CGRP | Episodic cluster headache | EMA’s Committee for Medicinal Products for Human Use adopted a negative opinion to extend product’s indication for prevention of attacks in adults with episodic cluster headache | 2/28/20 | Neurology/Psychiatric |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | ET-105 | Oral liquid formulation of lamotrigine | Epilepsy | FDA requested changes to dosage and administration section of prescribing information to simplify dosing; agency also requested company conduct a human factors validation study with the revised labeling to show intended users can prepare and administer the oral suspension safely and effectively | 2/19/20 | Neurology/Psychiatric |
| Glaxosmithkline plc, of London | Voltaren (diclofenac sodium topical gel) | Nonsteroidal anti-inflammatory treatment | Arthritis pain | FDA approved 1% gel as over-the-counter product | 2/17/20 | Neurology/Psychiatric |
| GW Pharmaceuticals Ltd., of London, and U.S. subsidiary Greenwich Biosciences Inc., of Carlsbad, Calif. | Epidiolex (cannabidiol) | Dual cannabinoid CB1/CB2 receptor modulator | Tuberous sclerosis complex | sNDA submitted to FDA to treat seizures associated with TSC | 2/3/20 | Neurology/Psychiatric |
| H. Lundbeck A/S, of Valby, Denmark | Vyepti (eptinezumab-jjmr) | Monoclonal antibody targeting CGRP ligand | Migraine prevention | FDA approved the drug, which is expected to be available in April 2020 | 2/22/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | HTX-011 (bupivacaine + meloxicam) | Cyclooxygenase 2 inhibitor; sodium channel inhibitor | Postoperative pain | FDA extended NDA review period by up to 3 months, resetting PDUFA date to June 26, 2020 | 2/19/20 | Neurology/Psychiatric |
| Innocoll Holdings Ltd., of Athlone, Ireland | Xaracoll (bupivacaine HCl collagen-matrix implants) | Drug-device anesthetic | Postsurgical pain | Resubmitted NDA to FDA seeking approval for management of postsurgical pain after open inguinal hernia surgery; company anticipates a 6-month review | 2/27/20 | Neurology/Psychiatric |
| Lyndra Therapeutics Inc., of Watertown, Mass. | Ultra-long-acting oral therapy | Designed to open inside the stomach and deliver drugs over time | Schizophrenia | Filed an IND with the FDA | 2/11/20 | Neurology/Psychiatric |
| Neurosense Therapeutics, of Herzliya, Israel | PrimeC | Targets neuroinflammation and dysregulation of RNA metabolism | Amyotrophic lateral sclerosis | FDA granted orphan drug designation | 2/6/20 | Neurology/Psychiatric |
| Realta Life Sciences Inc., of Norfolk, Va. | PIC-1 | Anti-inflammatory, antioxidant | Hypoxic-ischemic encephalopathy | FDA granted orphan drug designation | 2/6/20 | Neurology/Psychiatric |
| Retrotope Inc., of Los Altos, Calif. | RT-001 | Chemically modified polyunsaturated fatty acid drug | Progressive supranuclear palsy | FDA granted orphan designation | 2/18/20 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Oliceridine | G protein-selective mu-opioid receptor agonist | Moderate to severe acute pain | Resubmitted NDA to FDA; company anticipates 6-month review period | 2/10/20 | Neurology/Psychiatric |
| Zhittya Genesis Medicine Inc., of Las Vegas | Undisclosed | Biologic candidate | Parkinson’s disease | Received approval from COFEPRIS, the Mexican regulator, for a phase I study | 2/13/20 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (ZX-008, fenfluramine oral solution) | Antiseizure drug | Dravet syndrome | FDA extended review period for NDA seeking approval to treat seizures associated with Dravet syndrome by 3 months; new PDUFA date is June 25, 2020 | 2/27/20 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab) | Anti-VEGF antibody | Wet age-related macular degeneration | Approved by European Commission | 2/17/20 | Ocular |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | ION-373 | Designed to stop a mutated gene from producing excess glial fibrillary acidic protein | Alexander disease | EMA granted orphan drug designation | 2/11/20 | Other/Miscellaneous |
| Arrevus Inc., of Raleigh, N.C. | ARV-1801 (sodium fusidate) | Oral antibiotic | Cystic fibrosis | FDA granted orphan status | 2/18/20 | Respiratory |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | Nintedanib | Antifibrotic | Systemic sclerosis-associated interstitial lung disease | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending granting marketing authorization in adults | 2/28/20 | Respiratory |
| Glaxosmithkline plc, of London, and Innoviva Inc., of South San Francisco | Trelegy Ellipta (fluticasone furoate/umeclidinium/vilanterol) | Combination of inhaled corticosteroid, long-acting muscarinic antagonist and long-acting beta2-adrenergic agonist | Asthma | EMA accepted the regulatory submission seeking additional indication for the use of once-daily, single-inhaler triple therapy in adults | 2/27/20 | Respiratory |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | INO-3107 | DNA medicine | Recurrent respiratory papillomatosis | FDA accepted the IND for a phase I/II trial | 2/10/20 | Respiratory |
| Translate Bio Inc., of Lexington, Mass. | MRT-5005 | mRNA therapeutic | Cystic fibrosis | FDA granted fast track designation | 2/26/20 | Respiratory |
| Acacia Pharma Group plc, of Cambridge, U.K. | Barhemsys (amisulpride injection) | Selective dopamine D2 and D3 antagonist | Postoperative nausea and vomiting | FDA approved drug for prevention and treatment of PONV in adults | 2/27/20 | Toxicity and Intoxication |
| Adamis Pharmaceuticals Corp., of San Diego | Zimhi | High-dose naloxone injection | Opioid overdose | During type A meeting with FDA to discuss 2019 complete response letter, company obtained concurrence from agency on CMC information required for resubmission of NDA and discussed potential public health role of drug in opioid epidemic; company said it believes it can generate necessary information and resubmit NDA early in second quarter of 2020 | 2/19/20 | Toxicity and Intoxication |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 (repurposed ondansetron) | 5-HT 3 receptor antagonist | Alcohol use disorder | Bulgarian Drug Agency and Bulgarian Ethics Committee for Clinical Trials approved initiation of sites in Bulgaria for ongoing phase III study in individuals with target genotypes related to serotonin transporter and receptor genes | 2/19/20 | Toxicity and Intoxication |
| Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 | Alpha 2A adrenoceptor agonist | Opioid withdrawal | FDA cleared IND for initiation of phase Ib/II Release trial | 2/5/20 | Toxicity and Intoxication |
| Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. | Pedmark | Formulation of sodium thiosulfate | Prevention of cisplatin-induced ototoxicity in pediatric patients | Received EMA validation for the MAA and it is now under evaluation | 2/28/20 | Toxicity and Intoxication |
| Soligenix Inc., of Princeton, N.J. | Rivax | Protein subunit vaccine | Ricin intoxication | FDA granted fast track designation | 2/13/20 | Toxicity and Intoxication |
| Abbvie Inc., of North Chicago | Venclyxto (venetoclax) | Bcl-2 inhibitor | Chronic lymphocytic leukemia | European Commission approved use in combination with obinutuzumab for previously untreated patients; approval valid in all 27 member states of the EU, as well as Iceland, Liechtenstein, Norway and the U.K. | 3/12/20 | Cancer |
| Agenus Inc., of Lexington, Mass. | Balstilimab and zalifrelimab | Anti-PD-1 antibody and anti-CTLA4 antibody | Relapsed/refractory metastatic cervical cancer | FDA granted fast track designation for combination | 3/12/20 | Cancer |
| Anheart Therapeutics Co. Ltd., of Hangzhou, China | Taletrectinib | ROS1 and NTRK inhibitor | Non-small-cell lung cancer | Chinese Center for Drug Evaluation cleared IND and issued clinical trial authorizations for 2 phase II trials in China in patients with ROS1 mutations and locally advanced or metastatic solid tumors with NTRK mutations | 3/23/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 selective inhibitor | Relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma | FDA cleared IND for phase Ib/II trial testing drug as single agent or in combination | 3/9/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 selective inhibitor | Waldenström macroglobulinemia | FDA cleared IND for a phase Ib/II trial testing the drug as a single agent or in combination | 3/9/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 selective inhibitor | Relapsed/refractory acute myeloid leukemia | China’s National Medical Products Administration approved start of phase Ib trial testing the drug as a single agent or in combination | 3/9/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | PD-L1-inhibiting antibody | Small-cell lung cancer | FDA approved use as first-line treatment for adults with extensive-stage disease in combination with standard-of-care chemotherapies etoposide and either carboplatin or cisplatin | 3/30/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza | PARP inhibitor | Maintenance treatment of germline BRCA-mutated, curatively unresectable, pancreatic cancer | Japanese Ministry of Health, Labor and Welfare granted orphan drug designation | 3/19/20 | Cancer |
| Aveo Pharmaceuticals Inc. (Aveo Oncology), of Cambridge, Mass. | Fotivda (tivozanib) | VEGF receptor-tyrosine kinase inhibitor | Renal cell carcinoma | NDA submitted to FDA to treat individuals with relapsed/refractory disease | 3/31/20 | Cancer |
| Ayala Pharmaceuticals Inc., of Rehovot, Israel | AL-101 | Selective inhibitor of gamma secretase-mediated Notch signaling | Recurrent or metastatic adenoid cystic carcinoma | FDA granted fast track designation | 3/3/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. | Idecabtagene vicleucel (bb-2121) | CAR T targeting B-cell maturation antigen | Multiple myeloma | Submitted a BLA to the FDA | 3/31/20 | Cancer |
| Bristol-Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 inhibitor | Hepatocellular carcinoma | FDA granted accelerated approval, in combination with Yervoy (ipilimumab), to treat individuals previously treated with sorafenib (Nexavar, Bayer AG) | 3/11/20 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China | Avapritinib | Inhibitor of KIT and PDGFRA mutant kinases | Gastrointestinal stromal tumor | NDA submitted | 3/27/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | Antibody-drug conjugate targeting HER2 | HER2-positive unresectable or recurrent breast cancer after prior chemotherapy | Japanese Ministry of Health, Labor and Welfare approved the drug under the conditional early approval system | 3/25/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Axicabtagene ciloleucel | CAR T-cell therapy | Relapsed/refractory diffuse large B-cell lymphoma and related lymphomas | Submitted NDA to Japan’s Ministry of Health, Labour and Welfare | 3/30/20 | Cancer |
| Eisai Co. Ltd., of Tokyo | Denileukin diftitox (genetic recombinant) | Fusion protein of the receptor-binding portion of interleukin-2 and diphtheria toxin that binds to the IL-2 receptor | Relapsed or refractory cutaneous T-cell lymphoma and peripheral T-cell lymphoma | Submitted a marketing authorization application in Japan | 3/27/20 | Cancer |
| EMD Serono, part of Merck KGaA, of Darmstadt, Germany | Tepmetko (tepotinib) | MET inhibitor | Unresectable, advanced or recurrent non-small-cell lung cancer with MET exon 14 skipping alterations | Japanese Ministry of Health, Labor and Welfare approved the drug | 3/25/20 | Cancer |
| Epicentrx Inc., of La Jolla, Calif. | AIM-001 | Oncolytic adenovirus enhanced with TGF-beta trap transgene | Cancer | FDA cleared the IND; clinical trials to begin this year | 3/3/20 | Cancer |
| Essa Pharmaceuticals Inc., of Vancouver, British Columbia | EPI-7386 | Inhibits the N-terminal domain of the androgen receptor | Metastatic castration-resistant prostate cancer | Submitted an IND to the FDA for a phase I study | 3/31/20 | Cancer |
| Exelixis Inc., of Alameda, Calif., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Cabometyx (cabozantinib) | Kinase inhibitor | Curatively unresectable or metastatic renal cell carcinoma | Japanese Ministry of Health, Labor and Welfare approved the drug | 3/25/20 | Cancer |
| Hansoh Pharmaceutical Group Co. Ltd., of Shanghai | Ameile (almonertinib) | EGFR tyrosine kinase inhibitor | Metastatic non-small-cell lung cancer | National Medical Products Administration in China granted marketing authorization for use in patients with an EGFR T790M mutation after progression on other EGFR TKI | 3/19/20 | Cancer |
| Immunomic Therapeutics Inc., of Rockville, Md. | ITI-1001 | Plasmid DNA vaccine therapy | Glioblastoma multiforme | Completed a pre-IND meeting with FDA | 3/3/20 | Cancer |
| Immutep Ltd., of Sydney | Eftilagimod alpha (IMP-321) | HLA class II antigen stimulator | Metastatic breast cancer | FDA approved IND for planned phase I AIPAC-002 combination study with paclitaxel in 24 participants | 3/9/20 | Cancer |
| Infinity Pharmaceuticals Inc., of Cambridge, Mass. | IPI-549 | PI3K-gamma inhibitor | Advanced urothelial cancer | FDA granted fast track designation in combination with Opdivo (nivolumab, Bristol Myers Squib Co.) | 3/25/20 | Cancer |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | JNJ-6372 | EGFR-mesenchymal epithelial transition factor bispecific antibody | Non-small-cell lung cancer | FDA granted breakthrough therapy designation to treat metastatic disease in people with EGFR exon 20 insertion mutations whose disease progressed on or after platinum-based chemotherapy | 3/10/20 | Cancer |
| Junshi Biosciences Ltd., of Shanghai | Toripalimab | Monoclonal antibody targeting PD-1 | Mucosal melanoma | FDA granted orphan designation for use in combination with axitinib (Pfizer Inc.) | 3/30/20 | Cancer |
| Kura Oncology Inc., of San Diego | Tipifarnib | Inhibitor of farnesyl transferase | T-cell lymphomas | Received fast track designation | 3/3/20 | Cancer |
| Morphosys AG, of Planegg, Germany | Tafasitamab | Anti-CD19 antibody | Relapsed or refractory diffuse large B-cell lymphoma | BLA accepted by FDA, with agency granting priority review; PDUFA date of Aug. 30, 2020 | 3/2/20 | Cancer |
| Mylan NV, of Hertfordshire, U.K. | MYL-1402O (bevacizumab biosimilar) | VEGF ligand inhibitor | Solid tumors | FDA accepted BLA for review under 351(k) pathway for first- and second-line metastatic colorectal cancer in combination with fluorouracil-based chemotherapy; first-line use in nonsquamous non-small-cell lung cancer; and in recurrent glioblastoma, metastatic renal cell carcinoma in combination with interferon alfa and persistent, recurrent or metastatic cervical cancer; BsUFA date is Dec. 27, 2020 | 3/9/20 | Cancer |
| Nucana plc, of Edinburgh, U.K. | Acelarin | Derived from nucleoside analogue gemcitabine | Biliary tract cancer | EMA’s Committee for Orphan Medicinal Products issued positive opinion for orphan designation | 3/4/20 | Cancer |
| Oncology Venture A/S, of Horsholm, Denmark | Dovitinib | Pan-tyrosine kinase inhibitor | Renal cell carcinoma | Attended a pre-NDA meeting with the FDA, in which the FDA indicated it would accept the NDA filing if submitted; the company’s proposal is to seek approval based on noninferiority against the already-approved compound sorafenib (Bayer AG), based on prior phase III trial results | 3/20/20 | Cancer |
| Orion Corp., of Espoo, Finland | Nubeqa (darolutamide) | Oral androgen receptor inhibitor | Non-metastatic castration-resistant prostate cancer | European Commission granted marketing authorization | 3/30/20 | Cancer |
| Ryvu Therapeutics SA, of Krakow, Poland | SEL-120 | Oral, selective inhibitor of CDK8 kinase | Acute myeloid leukemia | FDA granted orphan designation | 3/28/30 | Cancer |
| Samsung Bioepis Co. Ltd., of Incheon, Korea | Ontruzant (trastuzumab-dttb) | ERBB2 tyrosine kinase receptor inhibitor | Breast/metastatic breast cancer; metastatic gastric cancer; gastroesophageal junction adenocarcinoma | FDA approved 420-mg multidose vial of the biosimilar as adjuvant treatment | 3/24/20 | Cancer |
| Sanofi SA, of Paris | Sarclisa (isatuximab-irfc) | CD38-targeting monoclonal antibody | Relapsed multiple myeloma | Approved by FDA for use in patients who have received at least 2 prior therapies, including lenalidomide and a proteasome inhibitor | 3/2/20 | Cancer |
| Sanofi SA, of Paris | Sarclisa | Isatuximab | Relapsed and refractory multiple myeloma | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion | 3/27/20 | Cancer |
| Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Rethio (thiotepa) | Alkylating agent | Malignant lymphoma | Japan’s Ministry of Health, Labor, and Welfare approved the partial change in the marketing approval to include conditioning treatment prior to autologous hematopoietic stem cell transplantation for malignant lymphoma | 3/25/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Alunbrig (brigatinib) | ALK-targeting tyrosine kinase inhibitor | ALK-positive non-small-cell lung cancer | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in patients not previously treated with an ALK inhibitor | 3/2/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, | Adcetris (brentuximab vedotin) | Antibody-drug conjugate targeting CD30 | Previously untreated systemic anaplastic large-cell lymphoma | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for the extension of the marketing authorization in combination with cyclophosphamide, doxorubicin and prednisone | 3/31/20 | Cancer |
| TG Therapeutics Inc., of New York | Umbralisib | Dual PI3K-delta/CK1-epsilon inhibitor | Follicular lymphoma | FDA granted orphan designation | 3/5/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Single-domain antibody against PD-L1 | Sarcoma | Submitted request for a type B meeting with FDA to discuss the trial design for a potential pivotal study | 3/16/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Naxitamab | Anti-GD2 3F8 monoclonal antibody | Neuroblastoma | Rolling BLA submission to FDA completed for treatment of those with relapsed/refractory high-risk disease | 4/1/20 | Cancer |
| Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Epithelial ovarian, fallopian tube or primary peritoneal cancer | China’s NMPA accepted the supplemental NDA for use as maintenance treatment of adults with advanced disease who are in complete or partial response to first-line platinum-based chemotherapy | 3/16/20 | Cancer |
| Cardiol Therapeutics Inc., of Oakville, Ontario | Cannabidiol formulation | Cannabidiol formulation | Heart failure | Received no objection letter from Health Canada for a phase I study in healthy volunteers | 3/5/20 | Cardiovascular |
| Arch Therapeutics Inc., of Framingham, Mass. | AC5 topical gel | Hemostatic agent | Pressure sores, leg ulcers, diabetic ulcers, surgical wounds | FDA cleared the marketing of the gel using an additional supplier and additional manufacturing processes | 3/23/20 | Dermatologic |
| Athenex Inc., of Buffalo, N.Y. | Tirbanibulin (formerly KX2-391 or KX-01) | SRC tyrosine kinase inhibitor; tubulin binding agent | Actinic keratosis | FDA accepted NDA filing, setting PDUFA date of Dec. 30, 2020, with no advisory committee meeting planned | 3/9/20 | Dermatologic |
| Athenex Inc., of Buffalo, N.Y., and Almirall SA, of Barcelona, Spain | Tirbanibulin ointment | Src kinase inhibitor | Actinic keratosis | EMA accepted filing of the MAA | 3/2/20 | Dermatologic |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Berotralstat (BCX-7353) | Oral, once-daily inhibitor of kallikrein in plasma | Prevention of hereditary angioedema attacks | EMA validated the marketing authorization application | 3/30/20 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Baricitinib | Oral JAK inhibitor | Alopecia areata | FDA granted breakthrough therapy designation | 3/16/20 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | Humanized IgG4 monoclonal antibody | Moderate to severe plaque psoriasis | FDA approved supplemental BLA for treating pediatric patients, ages 6 to under 18, who are candidates for systemic therapy or phototherapy | 3/30/20 | Dermatologic |
| Inmed Pharmaceuticals Inc., of Vancouver, British Columbia | INM-755 (cannabinol) | Keratin modulator | Epidermolysis bullosa | CTA filed in the Netherlands to initiate second phase I trial in healthy volunteers assessing 2 strengths of study drug in 8 adults over 14-day treatment period | 3/24/20 | Dermatologic |
| Pharming Group NV, of Leiden, the Netherlands | Ruconest (conestat alfa) | Recombinant human C1 esterase inhibitor | Hereditary angioedema | FDA approved addition of new Netherlands production facility, expanding product manufacturing capacity | 3/9/20 | Dermatologic |
| Pharming Group NV, of Leiden, the Netherlands | Ruconest (conestat alfa) | Recombinant analogue of human C1 esterase inhibitor | Hereditary angioedema | EMA’s Committee for Medicinal Products for Human Use recommended approval for treating acute angioedema attacks in children, expanding the age range to children 2 and older | 3/27/20 | Dermatologic |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Mitapivat | Allosteric activator of wild-type and a variety of mutated pyruvate kinase-R enzymes | Pyruvate kinase deficiency | EMA granted orphan drug designation | 3/30/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Givlaari (givosiran) | RNAi therapeutic targeting aminolevulinic acid synthase 1 | Acute hepatic porphyria | European Commission granted marketing authorization in adults and adolescents | 3/3/20 | Endocrine/Metabolic |
| APR Applied Pharma Research SA, of Balerna, Switzerland | APR-OD031 | Extended-release amino acid mix | Phenylketonuria | FDA granted orphan designation | 3/26/20 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | Lokelma (sodium zirconium cyclosilicate) | Binds potassium | Hyperkalemia on stable hemodialysis | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on a dosing and administration label update | 3/31/20 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | Lokelma (sodium zirconium cyclosilicate) | Potassium-removing medicine | Hyperkalemia | Approved for marketing by Japan Ministry of Health | 3/26/20 | Endocrine/Metabolic |
| Avrobio Inc., of Cambridge, Mass. | AVR-RD-04 | CTNS gene stimulator | Cystinosis | FDA granted orphan drug designation | 3/9/20 | Endocrine/Metabolic |
| Cyclo Therapeutics Inc., of Alachua, Fla. | Trappsol Cyclo | Formulation of hydroxypropyl beta cyclodextrin | Niemann-Pick disease type C1 | Received feedback from type C meeting with FDA on global pivotal trial design; plans to meet with EMA in the second quarter of 2020 | 3/2/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim GmbH, of Ingelheim, Germany | Jardiance (empagliflozin) | SGLT2 inhibitor | Type 1 diabetes | FDA issued a complete response letter | 3/23/20 | Endocrine/Metabolic |
| Freeline Therapeutics Ltd., of London | FLT-190 | Liver-directed AAV gene therapy | Fabry disease | European Commission granted orphan designation | 3/10/20 | Endocrine/Metabolic |
| Intrabio Inc., of Oxford, U.K. | IB-1001 | Modified amino-acid analogues/esters | GM2 gangliosidosis | FDA granted fast track designation | 3/25/20 | Endocrine/Metabolic |
| Intrabio Inc., of Oxford, U.K. | IB-1001 | Modified amino-acid analogues/esters | Niemann-Pick disease type C | FDA granted fast track designation | 3/25/20 | Endocrine/Metabolic |
| Novartis AG, of Basel, Switzerland | Isturisa (osilodrostat) | 11?beta?hyrdoxylase inhibitor | Cushing's disease | FDA approved drug to treat adults who either cannot undergo pituitary gland surgery or have undergone surgery but still have Cushing's disease | 3/6/20 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Type 2 diabetes | At the end-of-phase-II meeting, the FDA gave feedback on issues for drug product manufacturing; company plans an additional meeting to go over phase III design | 3/19/20 | Endocrine/Metabolic |
| Recordati SpA, of Milan, Italy | Osilodrostat | Oral inhibitor of 11-beta-hydroxylase | Cushing’s syndrome | Submitted NDA to Japan's Ministry of Health, Labor and Welfare | 3/26/20 | Endocrine/Metabolic |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Selective agonist of MC? receptor | Alstrom syndrome | FDA granted orphan designation | 3/18/20 | Endocrine/Metabolic |
| Stealth Biotherapeutics Corp., of Boston | Elamipretide | Mitochondrial-targeted compound | Barth syndrome | FDA granted rare pediatric disease designation | 3/3/20 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | Dasiglucagon Hypopal Rescue Pen | Glucagon analogue | Hypoglycemia in patients with diabetes | Submitted an NDA to the FDA | 3/31/20 | Endocrine/Metabolic |
| Intercept Pharmaceuticals Inc., of New York | Obeticholic acid | Binds to farnesoid X receptor | Liver fibrosis due to nonalcoholic steatohepatitis | FDA notified firm that, due to COVID-19 pandemic, it rescheduled advisory committee; originally set for April 22, 2020, the meeting is now tentatively scheduled for June 9, 2020; PDUFA date for the priority application remains June 26, 2020 | 3/26/20 | Gastrointestinal |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | SGLT2 inhibitor | Chronic kidney disease | FDA granted fast track designation for use in reducing risk of kidney disease progression and cardiovascular death in adults with CKD | 3/12/20 | Genitourinary/Sexual Function |
| Lipocine Inc., of Salt Lake City | Tlando | Oral testosterone | Hypogonadism | FDA acknowledged receipt of resubmitted NDA seeking approval of the TRT candidate for use in adult males for conditions associated with a deficiency of endogenous testosterone | 3/4/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Staines-upon-Thames, U.K. | Terlipressin | Vasopressin analogue selective for V1 receptors | Hepatorenal syndrome type 1 | Initiated rolling submission of NDA to FDA; expects to complete submission in the coming months | 3/2/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Staines-upon-Thames, U.K. | Terlipressin | Vasopressin analogue | Hepatorenal syndrome type 1 | Completed a rolling submission of an NDA to the FDA | 3/17/20 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland | Relugolix | GNRH receptor antagonist | Uterine fibroids | MAA submitted to EMA for combination tablet (relugolix 40 mg + estradiol 1 mg + norethindrone acetate 0.5 mg) to treat moderate to severe symptoms | 3/9/20 | Genitourinary/Sexual Function |
| Orphan Technologies Ltd., of Rapperswil, Switzerland | OT-58 | Modified recombinant enzyme therapy | Homocystinuria | FDA granted rare pediatric disease designation | 3/26/20 | Genitourinary/Sexual Function |
| Rockwell Medical Inc., of Wixom, Mich. | Triferic AVNU (ferric pyrophosphate citrate) | Iron maintenance therapy | Hemodialysis-dependent chronic kidney disease | FDA approved NDA for I.V. formulation to replace iron and maintain hemoglobin in adults | 3/27/20 | Genitourinary/Sexual Function |
| Urovant Sciences, part of New York-based Sumitovant Biopharma Ltd. | Vibegron | ?3-adrenoreceptor agonist | Overactive bladder | FDA accepted for review NDA seeking approval to treat patients with symptoms of urge urinary incontinence, urgency and urinary frequency; agency assigned PDUFA date of Dec. 26, 2020, and said it does not currently plan to hold and advisory committee meeting to discuss the application | 3/5/20 | Genitourinary/Sexual Function |
| Zydus Cadila Ltd., of Ahmedabad, India | Saroglitazar | Targets PPAR alpha and gamma | Noncirrhotic nonalcoholic steatohepatitis | NDA approved by Drug Controller General of India | 3/5/20 | Genitourinary/Sexual Function |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | AG-946 | Pyruvate kinase-R activator | Hemolytic anemias | FDA cleared the IND; company plans to start a phase I trial in healthy volunteers in mid-2020 | 3/23/20 | Hematologic |
| Intellia Therapeutics Inc., of Cambridge, Mass., and Novartis AG, of Basel, Switzerland | OTQ-923 | CRISPR/Cas9 expressing fetal hemoglobin | Sickle cell disease | FDA accepted the IND for a phase I/II study | 3/31/20 | Hematologic |
| Alpine Immune Sciences Inc., of Seattle | AlPN-101 | Dual T-cell co-stimulation inhibitor | Acute graft-vs.-host disease | FDA granted 2 orphan designations for prevention and treatment of acute GVHD | 3/18/20 | Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Voclosporin | Next-generation calcineurin inhibitor | Lupus nephritis | Initiated rolling NDA to FDA; expects to complete submission by end of the second quarter of 2020 | 3/16/20 | Immune |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Relapsing remitting multiple sclerosis | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in adults with active disease as defined by clinical or imaging features | 3/27/20 | Immune |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut | Epicutaneous immunotherapy | Peanut allergy | FDA said during its ongoing review of the BLA it identified questions regarding efficacy, including the impact of patch-site adhesion, and therefore, the Allergenic Products Advisory Committee meeting to discuss the BLA will no longer take place on May 15, 2020 | 3/17/20 | Immune |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Ponesimod | Sphingosine-1-phosphate receptor 1 modulator | Relapsing multiple sclerosis | NDA submitted to the FDA | 3/18/20 | Immune |
| Janssen Pharmaceuticals Cos., of Allschwil, Switzerland, part of Johnson & Johnson | Ponesimod | S1P1R modulator | Relapsing multiple sclerosis | Submitted MAA to EMA seeking approval for use in adults | 3/4/20 | Immune |
| Kadmon Holdings Inc., of New York | KD-025 | ROCK2 inhibitor | Chronic graft-vs.-host disease | Held pre-NDA meeting with FDA and anticipates receiving final minutes within next 30 days; remains on track to complete NDA submission in fourth quarter of 2020 | 3/13/20 | Immune |
| Mylan NV, of Hertfordshire, U.K., and Lupin Ltd., of Mumbai, India | Nepexto | Etanercept biosimilar | Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis, plaque psoriasis and pediatric plaque psoriasis | The EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval | 3/27/20 | Immune |
| Orchard Therapeutics plc, of London | OTL-101 (simoladagene autotemcel) | ADA gene stimulator | Severe combined immunodeficiency | COVID-19-related clinical and manufacturing impacts expected to delay initiation of rolling BLA to FDA, previously planned for first half of 2020 | 3/31/20 | Immune |
| Shenzhen Chipscreen Biosciences Co. Ltd., of Shenzhen, China | CS-112192 | Dual JAK1/JAK3 tyrosine kinase inhibitor; serine threonine protein kinase TBK1 inhibitor | Autoimmune diseases | China NMPA's Center for Drug Evaluation accepted IND | 3/24/20 | Immune |
| Virpax Pharmaceuticals Inc., of West Chester, Pa. | Epoladerm (diclofenac epolamine) | Metered-dose topical aerosol of diclofenac epolamine | Osteoarthritis of the knee | Completed pre-IND meeting with the FDA, which agreed that the 505(b)(2) pathway is a reasonable approach for approval | 3/19/20 | Immune |
| Abbvie Inc., of North Chicago | Maviret (glecaprevir/pibrentasvir) | NS3/4A protease inhibitor and NS5A inhibitor | Hepatitis C virus | European Commission approved change to marketing authorization to shorten once-daily treatment duration from 12 to 8 weeks in treatment-naïve, compensated cirrhotic, chronic HCV patients with genotype 3 infection | 3/6/20 | Infection |
| Adaptive Phage Therapeutics Inc., of Gaithersburg, Md. | Phagebank | Personalized bacteriophage therapeutic | Multidrug-resistant and complicated bacterial infections | FDA cleared IND for phase I/II trial in patients with complicated and chronic recurrent urinary tract infections | 3/12/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NDMA receptor glutamate receptor antagonist specifically targeting NMDA-type subunit 2B | Prevention of acute lung injury and acute respiratory distress syndrome associated with COVID-19 | Requested pre-IND meeting with FDA, asking for direction regarding use of injectable and slow-release formulation as well as currently available drug supply for a U.S. clinical trial on an emergency basis; filing also includes clarification of expanded access pathway | 3/13/20 | Infection |
| Allovir Inc., of Cambridge, Mass. | Viralym-M (ALVR-105) | Multivirus-specific T-cell therapy | Prevention of BK virus, cytomegalovirus, human herpes virus-6, Epstein-Barr virus, adenovirus and JC virus | EMA granted orphan drug designation | 3/26/20 | Infection |
| Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Nitric oxide, inhaled | COVID-19 | FDA granted emergency expanded access | 3/20/20 | Infection |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | COVID-19 | Submitted drug to the institutional review board at Rabin Medical Center for a compassionate use program | 3/23/20 | Infection |
| Cansino Biologics Inc., of Tianjin, China | Ad5-nCoV | Recombinant coronavirus vaccine | COVID-19 prophylaxis | Vaccine approved to enter phase I study in China | 3/17/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 | Filed modified IND and protocol for phase II trial for patients who experience respiratory complications as result of contracting COVID-19 | 3/16/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 | FDA granted emergency IND for 2 patients | 3/19/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | CCR5 antagonist | HIV infection | Filed another round of modifications to its IND and protocol for a phase II trial | 3/27/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | Monoclonal antibody targeting CCR5 | COVID-19 | FDA cleared the start of a phase II study in patients with mild to moderate indications | 3/31/20 | Infection |
| Dare Biosciences Inc., of San Diego | DARE-BV1 | Thermosetting bioadhesive hydrogel containing clindamycin phosphate 2% | Bacterial vaginosis | FDA granted fast track designation | 3/10/20 | Infection |
| F2G Ltd., of Manchester, U.K. | Olorofim (formerly F-901318) | Dihydroorotate dehydrogenase inhibitor | Invasive aspergillosis and lomentospora/scedosporium infections | FDA granted orphan drug designation | 3/9/20 | Infection |
| Genentech, of South San Francisco, a member of the Roche Group | Actemra (tocilizumab) | Interleukin-6 receptor antagonist | COVID-19 pneumonia | FDA approved a phase III trial testing the drug in combination with standard of care | 3/23/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Epclusa (sofosbuvir and velpatasvir) | HCV NS5B polymerase inhibitor and HCV NS5A inhibitor | Hepatitis C virus | FDA approved supplemental application to expand use for children, ages 6 and older and weighing at least 37 pounds, with any of the 6 HCV genotypes without cirrhosis or with mild cirrhosis; Epclusa plus ribavirin is indicated for treating pediatric patients, 6 and older and weighing at least 37 pounds, with severe cirrhosis | 3/19/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Remdesivir | RNA polymerase modulator | COVID-19 | Gilead submitted request to FDA to rescind orphan designation and said it is waiving gall benefits that accompany such designation | 3/25/20 | Infection |
| I-Mab Biopharma Co. Ltd., of Rockville, Md., and Shanghai | TJM-2 | Neutralizing antibody against human GM-CSF | Cytokine storm | Submitted IND to South Korea’s Ministry of Food and Drug Safety to evaluate drug in severe and critically ill patients with COVID-19 | 3/30/20 | Infection |
| Paratek Pharmaceuticals Inc., of Boston | Nuzyra (omadacycline) | Broad-spectrum aminomethylcycline antibiotic | Anthrax infection | Pre-emergency use authorization application submitted by BARDA to FDA to allow for potential treatment and prophylaxis use during declared emergency | 3/10/20 | Infection |
| Relief Therapeutics Holding AG, of Zurich, and Neurorx Inc., of Wilmington, Del. | RLF-100 (aviptadil) | Synthesized peptide | Acute and moderate respiratory distress in COVID-19 | Filed an IND with the FDA for a phase II trial | 3/26/20 | Infection |
| Spero Therapeutics Inc., of Cambridge, Mass. | SPR-720 | DNA gyrase inhibitor; topoisomerase IV inhibitor | Nontuberculous mycobacterial infection | FDA granted orphan drug designation | 3/11/20 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Inhaled formulation of interferon-beta-1a | COVID-19 | Received expedited approvals from U.K.’s Medicines and Healthcare Products Regulatory Agency and Health Research Authority to conduct a clinical trial | 3/18/20 | Infection |
| Takis Biotech Srl, of Rome, and Applied DNA Sciences Inc., of Stony Brook, N.Y. | COVID-19 vaccine | DNA vaccine | COVID-19 prophylaxis | Italy’s Ministry of Health approved the start of preclinical testing | 3/18/20 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1533 | Monovalent, single-dose, live-attenuated vaccine | Chikungunya | Completed end-of-phase II meeting with FDA and agreed on development plan based on immunology surrogate (accelerated approval pathway); company is preparing for phase III initiation as soon as the COVID-19 situation permits, currently assuming initiation in the fourth quarter of 2020 | 3/25/20 | Infection |
| Valneva SE, of Saint-Herblain, France, | Ixiaro (IC-51) | Cell-derived vaccine | Japanese encephalitis virus infection | FDA approved extension of product shelf-life from 24 to 36 months; separately, U.S. Department of Defense issued request for proposal covering 3-year period that could lead to supply contract during first half of 2020 | 3/8/20 | Infection |
| Viiv Healthcare Ltd., of London | Cabenuva | Cabotegravir and rilpivirine extended-release injectable suspensions | HIV-1 infection | Approved by Health Canada | 3/20/20 | Infection |
| Viiv Healthcare Ltd., of London | Vocabria | Cabotegravir | HIV-1 infection | Health Canada approved it as a short-term addition to Cabenuva | 3/20/20 | Infection |
| Genentech, of South San Francisco, a member of the Roche Group | Xofluza | Baloxavir marboxil | Acute uncomplicated influenza; post-exposure prophylaxis of influenza | FDA accepted an NDA for a new formulation as 1-dose granules for oral suspension; the FDA also accepted 2 sNDAs: for acute uncomplicated influenza in children and for post-exposure prophylaxis of influenza in people 1 and older | 3/27/20 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | GM-CSF ligand inhibitor | COVID-19 | Submitted an initial protocol synopsis to the FDA in support of its plans to initiate a multicenter, phase III study in the U.S. | 3/27/20 | Infection |
| Bone Therapeutics SA, of Gosselies, Belgium | JTA-004 | Mix of plasma proteins, hyaluronic acid, a natural component of knee synovial fluid and a fast-acting analgesic | Osteoarthritic knee pain | Denmark regulatory authorities approved the clinical trial application | 3/23/20 | Inflammatory |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor that targets mast cells and macrophages | Amyotrophic lateral sclerosis | FDA cleared the IND for a phase III study (AB19001); company aims to initiate study as soon as conditions in U.S. clinical sites stabilize post-COVID-19 pandemic | 3/31/20 | Musculoskeletal |
| Acer Therapeutics Inc., of Newton, Mass. | Edsivo (celiprolol) | Beta blocker | Vascular Ehlers-Danlos syndrome with a confirmed COL3A1 mutation | FDA denied company's appeal of the CRL issued in June 2019 | 3/18/20 | Musculoskeletal |
| Bone Therapeutics SA, of Gosselies, Belgium | Allob | Allogeneic cell therapy | Difficult tibial fractures | Belgian regulatory authorities approved the clinical trial application | 3/23/20 | Musculoskeletal |
| Cytokinetics Inc., of South San Francisco | Reldesemtiv | Fast skeletal muscle troponin activator | Amyotrophic lateral sclerosis | EMA granted orphan designation | 3/4/20 | Musculoskeletal |
| Novartis AG, of Basel, Switzerland | Cosentyx | Secukinumab; IL-17A inhibitor | Non-radiographic axial spondyloarthritis | Gained positive opinion from EMA's Committee for Medicinal Products for Human Use for a new indication in the axial spondyloarthritis spectrum | 3/27/20 | Musculoskeletal |
| Acacia Pharma Group plc, of Cambridge, U.K. | Byfavo (remimazolam) | Ultra-short-acting, reversible intravenous benzodiazepine | Sedative/anesthetic use during invasive medical procedures | FDA extended the review period by up to 90 days to complete review of additional data submitted in January and February 2020; new PDUFA date of July 5, 2020 | 3/12/20 | Neurology/Psychiatric |
| Acadia Pharmaceuticals Inc., of San Diego, and Neuren Pharmaceuticals Ltd., of Melbourne, Australia | Trofinetide | Synthetic analogue of the amino?terminal tripeptide of IGF-1 | Rett syndrome | FDA granted rare pediatric disease designation | 3/3/20 | Neurology/Psychiatric |
| Agex Therapeutics Inc., of Alameda, Calif., and Imstem Biotechnology Inc., of Farmington, Conn. | IMS-001 | hES-MSC-based allogeneic cell therapy | Multiple sclerosis | FDA lifted the clinical hold and cleared the IND | 3/20/20 | Neurology/Psychiatric |
| Arvelle Therapeutics GmbH, of Zug, Switzerland | Cenobamate | Voltage-gated sodium channel blocker | Focal-onset seizures | EMA accepted marketing authorization application for use as adjunctive treatment in adults with epilepsy | 3/30/20 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Verdiperstat | Oral, brain-penetrant irreversible myeloperoxidase inhibitor | Multiple system atrophy | FDA granted fast track designation | 3/18/20 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Vazegepant | Calcitonin gene-related peptide receptor antagonist | Acute migraine | Following a successful end-of-phase-II interaction with the FDA, company plans to start a phase III study with the 10-mg dose | 3/23/20 | Neurology/Psychiatric |
| Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | Sphingosine 1-phosphate receptor modulator | Relapsing-remitting forms of multiple sclerosis | Approved for marketing by FDA | 3/26/20 | Neurology/Psychiatric |
| Cadent Therapeutics Inc., of Cambridge, Mass. | CAD-9303 | Positive allosteric modulator of NMDA | Schizophrenia | FDA cleared the IND for a phase I trial in schizophrenia patients and healthy volunteers | 3/10/20 | Neurology/Psychiatric |
| GW Pharmaceuticals plc, of Carlsbad, Calif. | Epidiolex | Cannabidiol | Tuberous sclerosis complex | FDA accepted the supplemental NDA with a priority review; PDUFA date of July 21, 2020 | 3/31/20 | Neurology/Psychiatric |
| GW Pharmaceuticals plc, of London | Epidyolex | Cannabidiol oral solution | Seizures associated with tuberous sclerosis complex | Submitted type II variation application to EMA; if approved, it will be the third licensed indication for the drug in Europe | 3/13/20 | Neurology/Psychiatric |
| Innocoll Holdings Ltd., of Athlone, Ireland | Xaracoll (bupivacaine hydrochloride collagen-matrix implant) | Anesthetic | Management of postsurgical pain after open inguinal hernia surgery | FDA accepted the NDA resubmission and set PDUFA date of Aug. 26, 2020 | 3/26/20 | Neurology/Psychiatric |
| Intelgenx Technologies Corp., of Saint Laurent, Quebec | Rizaport Versafilm | Oral thin-film formulation of 5-HT1 receptor agonist rizatriptan | Acute migraine | FDA issued complete response letter for resubmitted 505(b)(2) NDA, stating the agency cannot approve the application in its present form and requesting additional information, though no new bioequivalence study was requested | 3/27/20 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | JZP-258 | Oxybate product | Excessive daytime sleepiness | FDA accepted NDA for filing with priority review; seeks approval for use in patients 7 and older with narcolepsy; PDUFA date is July 21, 2020 | 3/26/20 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-415 | D-methylphenidate prodrug | Attention deficit hyperactivity disorder | Submitted an NDA to the FDA | 3/3/20 | Neurology/Psychiatric |
| Newron Pharmaceuticals SpA, of Milan, Italy | Sarizotan | 5-HT 1a receptor agonist; selective dopamine receptor modulator | Rett syndrome | Final minutes from Feb. 5, 2020, meeting with FDA indicated agency agreed with statistical analysis plan, including primary and key secondary efficacy endpoints, for Stars study, expected to report in second quarter of 2020; clinical database remains locked and blinded | 3/11/20 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Mayzent | Siponimod | Secondary progressive multiple sclerosis with active disease | Received Health Canada approval | 3/3/20 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | Zolgensma (onasemnogene abeparvovec) | Gene therapy expressing SMN | Spinal muscular atrophy | Japanese Ministry of Health, Labor and Welfare approved the therapy in patients under the age of 2 | 3/19/20 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland, and its Avexis unit | Zolgensma (onasemnogene abeparvovec) | Gene therapy | Spinal muscular atrophy | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending conditional marketing authorization | 3/27/20 | Neurology/Psychiatric |
| Orchard Therapeutics plc, of London | OTL-200 | ARSA gene stimulator | Metachromatic leukodystrophy | Despite continuing engagement with EMA on submitted MAA under accelerated assessment, decision likely to be postponed due to COVID-19 pandemic; potential launch in Europe now expected in first half of 2021; BLA submission to FDA also postponed to first half of 2021 | 3/31/20 | Neurology/Psychiatric |
| Pear Therapeutics Inc., of Boston | Somryst | Digital therapeutic | Chronic insomnia | FDA granted authorization for use in patients 22 and older | 3/26/20 | Neurology/Psychiatric |
| Pfizer Inc., and New York, and Eli Lilly and Co., of Indianapolis | Tanezumab | NGF-inhibiting monoclonal antibody | Chronic pain due to moderate to severe osteoarthritis | FDA accepted BLA seeking approval for use in patients who have experienced inadequate pain relief with other analgesics | 3/2/20 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Oral fixed-dose combination of baclofen, naltrexone and sorbitol | Charcot-Marie-Tooth disease type 1A | U.K.’s Medicine and Healthcare products Regulatory Agency granted promising innovative medicine designation for use in patients 16 and older | 3/18/20 | Neurology/Psychiatric |
| Prevail Therapeutics Inc., of New York | PR-006 | AAV-based gene therapy | Frontotemporal dementia | FDA accepted IND for a phase I/II trial in patients with GRN mutation | 3/2/20 | Neurology/Psychiatric |
| Prevail Therapeutics Inc., of New York | PR-006 | GRN gene stimulator | Frontotemporal dementia | FDA granted fast track designation to slow disease progression | 3/24/20 | Neurology/Psychiatric |
| Sage Therapeutics Inc., of Cambridge, Mass. | Zuranolone | Positive allosteric modulator of GABAA receptor | Postpartum depression, major depression and major depressive syndrome | Following an FDA breakthrough therapy guidance meeting, identified 3 pathways supporting possible filing for approval treat postpartum depression, acute treatment of major depressive disorder when co-initiated with a new antidepressant and as an episodic therapy in major depressive syndrome patients; 3 short-term clinical studies set to start in 2020 | 3/18/20 | Neurology/Psychiatric |
| Seelos Therapeutics Inc., of New York | SLS-005 (I.V. trehalose) | Transcription factor EB stimulator | Sanfilippo syndrome (mucopolysaccharidosis type III) | Written response received from EMA, in lieu of face-to-face meeting, recommended use of natural history data, enabling design of open-label, non-placebo-controlled, pivotal phase IIb/III EU study in Sanfilippo syndrome types A and B | 3/11/20 | Neurology/Psychiatric |
| Stoke Therapeutics Inc., of Bedford, Mass. | STK-001 | Antisense oligonucleotide to up-regulate NaV1.1 protein expression | Dravet syndrome | FDA confirmed company can start part A of its phase I/IIa Monarch study; the agency placed part B testing a higher dose of the drug on a partial clinical hold, pending additional preclinical toxicology data | 3/23/20 | Neurology/Psychiatric |
| Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Latuda (lurasidone hydrochloride) | Atypical antipsychotic | Schizophrenia and bipolar depression | Japanese Ministry of Health, Labor and Welfare approved the drug for both diseases | 3/25/20 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | CGRP inhibitor | Chronic migraine | U.K.’s National Institute for Health and Care Excellence recommended drug in final appraisal document for prevention of migraine in adults with chronic migraine; recommendation is for patients who have not responded to at least 3 prior preventive drug treatments | 3/12/20 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Oliceridine IV | G protein-selective mu-opioid receptor agonist | Moderate to severe acute pain | PDUFA date of August 7, 2020 | 3/5/20 | Neurology/Psychiatric |
| Virpax Pharmaceuticals Inc., of Malvern, Pa. | NES-100 | Nanotechnology for delivery of metabolically labile intranasal peptide | Acute and chronic pain | Completed a pre-IND application meeting with FDA | 3/2/20 | Neurology/Psychiatric |
| Zosano Pharma Corp., of Fremont, Calif. | Qtrypta | Formulation of zolmitriptan delivered using intracutaneous microneedle system | Migraine | FDA accepted the NDA under the 505(b)(2) pathway, setting a PDUFA date of Oct. 20, 2020 | 3/4/20 | Neurology/Psychiatric |
| Allergan plc, of Dublin | Durysta (bimatoprost implant) | Prostaglandin analogue for intracameral administration | Open-angle glaucoma or ocular hypertension | FDA approved NDA | 3/4/20 | Ocular |
| Janssen Pharmaceutical Inc., of Raritan, N.J., part of Johnson & Johnson, and Meiragtx Holding plc, of London | AAV-RPGR | Gene therapy | X-linked retinitis pigmentosa | EMA granted both PRIME (Priority Medicines) and advanced therapy medicinal product designations | 3/2/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-470 | Second-generation nitric oxide-donating bimatoprost analogue | Open-angle glaucoma or ocular hypertension | Completed end-of-phase II meeting with FDA and agreed on design of phase III program, nonclinical and CMC plans for submission of an NDA | 3/6/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France | Vyzulta (latanoprostene bunod) | PGF2 alpha agonist | Ocular hypertension; open-angle glaucoma | Partner Bausch & Lomb Inc., a unit of Bausch Health Cos. Inc. received approval in Taiwan for ophthalmic solution (0.024%) to reduce intraocular pressure in either indication | 3/9/20 | Ocular |
| Outlook Therapeutics Inc., of Cranbury, N.J. | ONS-5010 | Ophthalmic formulation of VEGF inhibitor | Wet age-related degeneration and other retinal diseases | Received tentative FDA approval for trade name of Lytenava (bevacizumab-vikg), subject to further review at the time of planned BLA filing, expected in 2021 | 3/5/20 | Ocular |
| Dicerna Pharmaceuticals Inc., of Lexington, Mass. | DCR-A1AT | RNAi targeting SERPINA1 | Alpha-1 antitrypsin deficiency | FDA granted orphan drug designation | 3/18/20 | Other/Miscellaneous |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Lonafarnib | Farnesyltransferase inhibitor | Progeria and progeroid laminopathies | Submitted NDA to FDA | 3/23/20 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Pro-opiomelanotonin deficiency obesity and leptin receptor deficiency obesity | Completed rolling NDA to FDA | 3/30/20 | Other/Miscellaneous |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | N-methyl-D-aspartate receptor glutamate receptor antagonist | Idiopathic pulmonary fibrosis and chronic cough | Submitted for ethics approval in Australia for its planned phase II study | 3/30/20 | Respiratory |
| Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Pulsatile nitric oxide delivery system | Pulmonary hypertension associated with pulmonary fibrosis | Finalized, in consultation with FDA, key elements of planned pivotal phase III trial, including use of moderate to vigorous physical activity as the primary endpoint for approval, the population of PF subjects at risk of PH, as well as the dose of iNO45 (45 mcg/kg IBW/hr) | 3/10/20 | Respiratory |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | Ofev (nintedanib) | Small-molecule kinase inhibitor | Chronic fibrosing interstitial lung diseases | Approved by FDA for use in patients with a progressive phenotype | 3/9/20 | Respiratory |
| Genentech, of South San Francisco, a member of the Roche Group | Esbriet | Pirfenidone | Unclassifiable interstitial lung disease | FDA granted breakthrough therapy designation | 3/3/20 | Respiratory |
| Translate Bio Inc., of Lexington, Mass. | MRT-5005 | mRNA therapeutic | Cystic fibrosis | FDA granted rare pediatric disease designation | 3/27/20 | Respiratory |
| Virometix AG, of Schlieren, Switzerland | V-306 | Vaccine | Respiratory syncytial virus | Belgian health authority approved clinical trial application for phase I study in healthy volunteers | 3/12/20 | Respiratory |
| Beyondspring Inc., of New York | Plinabulin | Immune and stem cell modulator | Chemotherapy-induced neutropenia | Initiated rolling submission of NDA to China’s NMPA | 3/30/20 | Toxicity and Intoxication |
| Helsinn Healthcare SA, of Lugano, Switzerland | Akynzeo (fosnetupitant/palonosetron) | Netupitant prodrug | Chemotherapy-induced nausea and vomiting | European Commission approved the intravenous formulation of Akynzeo | 3/23/20 | Toxicity and Intoxication |
| Acceleron Pharma Inc., of Cambridge, Mass. | Sotatercept | Ligand trap for members of the TGF-beta superfamily | Pulmonary arterial hypertension | FDA granted breakthrough therapy designation | 4/8/20 | Cancer |
| Adaptimmune Therapeutics plc, of Philadelphia | ADP-A2M4 | SPEAR T-cells targeting MAGE-A4 | Soft tissue sarcomas | EMA’s Committee for Orphan Medicinal Products adopted a positive opinion for orphan designation | 4/28/20 | Cancer |
| Affimed NV, of Heidelberg, Germany | AFM-13 | CD30- and CD16A-binding innate cell engager | T-cell lymphoma | FDA granted orphan drug designation | 4/1/20 | Cancer |
| Agenus Inc., of Lexington, Mass. | Balstilimab | Anti-PD-1 antibody | Cervical cancer | FDA granted fast track designation | 4/7/20 | Cancer |
| Alphamab Oncology Co. Ltd., of Suzhou, China | KN-046 | PD-L1/CTLA4 bispecific antibody | Anti-PD-(L)1-refractory or relapsed non-small-cell lung cancer | FDA issued a safe to proceed letter for the phase II study | 4/16/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 selective inhibitor | Relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma | The NMPA approved the start of the phase Ib/II study in China | 4/7/20 | Cancer |
| Astellas Pharma Inc., of Tokyo | Xospata (gilteritinib) | FLT3 inhibitor | Relapsed/refractory acute myelogenous leukemia | NDA accepted for review by China's NMPA | 4/10/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Koselugo (selumetinib) | MEK1/2 inhibitor | Neurofibromatosis type 1 | FDA approved for use in pediatric patients, 2 and older, who have symptomatic, inoperable plexiform neurofibromas | 4/13/20 | Cancer |
| Athenex Inc., of Buffalo, N.Y. | Oral paclitaxel + encequidar | P-glycoprotein 1 inhibitor | Metastatic breast cancer | Following meeting with FDA to discuss clinical section of NDA, filing on track per agency guidance | 4/9/20 | Cancer |
| Autolus Therapeutics plc, of London | AUTO-1 | CD19-targeting CAR T-cell therapy | Acute lymphoblastic leukemia | FDA accepted the IND for the pivotal phase Ib/II AUTO1-AL1 study | 4/16/20 | Cancer |
| AVM Biotechnology Inc., of Seattle | AVM-0703 | Small molecule that supercharges and mobilizes immune cells | Non-Hodgkin lymphoma, acute lymphocytic leukemia and chronic lymphocytic leukemia | Received FDA approval to begin phase I/II trials | 4/14/20 | Cancer |
| Bayer AG, of Leverkusen, Germany | Vitrakvi (larotrectinib) | Tyrosine receptor kinase (TRK) inhibitor | Advanced solid tumors | NICE recommended approval to treat advanced neurotrophic TRK fusion-positive tumors in adults and children if disease is locally advanced, metastatic or surgery could cause severe health problems and patients have no satisfactory treatment options | 4/21/20 | Cancer |
| Beigene Co. Ltd., of Beijing, and Cambridge, Mass. | Tislelizumab | Anti-PD-1 antibody | Urothelial carcinoma | China’s NMPA approved for use in patients with locally advanced or metastatic disease with PD-L1 high expression, whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy | 4/10/20 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | PD-1 inhibitor | Non-small-cell lung cancer | China NMPA's Center for Drug Evaluation accepted sNDA filing to treat, in combination with chemotherapy regimens, first-line advanced squamous disease | 4/20/20 | Cancer |
| Bioeclipse Therapeutics Inc., of Mountain View, Calif. | CRX-100 | Immunotherapy | Therapy-refractory solid tumors | FDA cleared IND for a phase I study expected to start in 2020 | 4/28/20 | Cancer |
| Blaze Bioscience Inc., of Seattle | Tozuleristide (BLZ-100) | Targeting peptide and a fluorescent dye | Pediatric central nervous system tumors | FDA granted fast track designation | 4/7/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | Monoclonal antibodies targeting PD-1 and CTLA4 | First-line metastatic or recurrent non-small-cell lung cancer with no EGFR or ALK genomic tumor aberrations | FDA accepted the supplemental BLA for the drugs in combination with a limited course of chemotherapy and granted a priority review with a PDUFA date of Aug. 6, 2020; EMA validated the type II variation for the same indication | 4/8/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Acceleron Pharma Inc., of Cambridge, Mass. | Reblozyl (luspatercept-aamt) | Erythroid maturation agent | Myelodysplastic syndromes | Approved by FDA for treatment of anemia failing an erythropoiesis-stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adults with very low- to intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis | 4/3/20 | Cancer |
| Carsgen Therapeutics Co. Ltd., of Shanghai | CT-053 | Anti-BCMA CAR T-cell therapy | Multiple myeloma | EMA’s Committee for Orphan Medicinal Products adopted positive opinion for orphan designation | 4/27/20 | Cancer |
| Clarity Pharmaceuticals, of Sydney | 67Cu-Sartate | Radiopharmaceutical | Neuroblastoma | FDA granted orphan drug designation | 4/21/20 | Cancer |
| CNS Pharmaceuticals Ag Inc., of Houston, | Berubicin (previously RTA-744) | Topoisomerase II inhibitor | Glioblastoma | Orphan drug application submitted to FDA | 4/23/20 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China | Avapritinib | Inhibitor of KIT and PDGFRA mutant kinases | Gastrointestinal stromal tumors | China's NMPA accepted NDA to treat adults with unresectable or metastatic disease harboring platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations, and adults with unresectable or metastatic fourth-line disease | 4/23/20 | Cancer |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | CCR5 chemokine antagonist | Metastatic triple-negative breast cancer | Company requested meeting with FDA to discuss potential filing for breakthrough therapy designation | 4/3/20 | Cancer |
| EMD Serono, unit of Merck KGaA, of Darmstadt, Germany, and Pfizer Inc., of New York | Bavencio (avelumab) | PD-L1 inhibitor | Urothelial carcinoma | sBLA submission to FDA completed for first-line maintenance treatment of locally advanced or metastatic disease; FDA granted breakthrough therapy designation in indication and is reviewing submission under real-time oncology review pilot program | 4/9/20 | Cancer |
| Erytech Pharma SA, of Lyon, France | Eryaspase | L-asparaginase encapsulated inside donor-derived red blood cells | Metastatic pancreatic cancer | FDA granted fast track designation for use as second-line treatment | 4/28/20 | Cancer |
| Genentech Inc., of South San Francisco, a unit of Roche Holding AG, of Basel, Switzerland | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20 | Relapsing or primary progressive multiple sclerosis | FDA accepted the supplemental BLA and the EMA validated the application for a 2-hour infusion, dosed twice yearly | 4/20/20 | Cancer |
| Genmab A/S, of Copenhagen | Daratumumab | CD38-targeting antibody | Multiple myeloma | EMA’s Committee for Medicinal Products for Human Use recommended use of subcutaneous formulation for treating adults in front-line and relapsed/refractory settings | 4/30/20 | Cancer |
| Glaxosmithkline plc, of London | Zejula (niraparib) | PARP 1/2 inhibitor | Advanced ovarian cancer | FDA approved sNDA for first-line monotherapy maintenance of individuals with platinum-responsive disease regardless of biomarker status | 4/30/20 | Cancer |
| Gracell Biotechnologies Co. Ltd., of Shanghai | GC-007g | B-lymphocyte antigen CD19 modulator | B-cell acute lymphoblastic leukemia | China's NMPA accepted IND application to initiate phase I study in individuals with CD19-positive relapsed/refractory disease, expected to begin in second quarter of 2020 | 4/3/20 | Cancer |
| Grid Therapeutics LLC, of Durham, N.C. | GT-103 | Recombinant antibody | Solid tumors | FDA approved its IND application and the company plans to initiate a phase I trial | 4/14/20 | Cancer |
| Halozyme Therapeutics Inc., of San Diego, and Janssen Pharmaceutical KK, a unit of New Brunswick, N.J.-based Johnson & Johnson | Daratumumab | CD38-targeting antibody | Multiple myeloma | Janssen submitted NDA to Japan’s Ministry of Health, Labour and Welfare for approval of subcutaneous formulation using Halozyme’s Enhanze technology | 4/26/20 | Cancer |
| Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong | Surufatinib | CSF-1 antagonist; FGF1 receptor antagonist; pan-VEGF receptor antagonist | Pancreatic and non-pancreatic neuroendocrine tumors | FDA granted fast track designations | 4/17/20 | Cancer |
| Immunomedics Inc., of Morris Plains, N.J. | Sacituzumab govitecan | Antibody-drug conjugate | Locally advanced or metastatic urothelial cancer | FDA granted fast track designation | 4/7/20 | Cancer |
| Immunomedics Inc., of Morris Plains, N.J. | Trodelvy (sacituzumab govitecan-hziy) | Anti-Trop-2 antibody-drug conjugate | Metastatic triple-negative breast cancer | FDA approved drug to treat adults who received at least 2 prior therapies for metastatic disease | 4/22/20 | Cancer |
| Incyte Corp., of Wilmington, Del. | Pemazyre (pemigatinib) | Inhibitor of FGFR isoforms 1, 2 and 3 | Locally advanced or metastatic cholangiocarcinoma | FDA approved the drug | 4/20/20 | Cancer |
| Innovent Biologics Inc., of Suzhou, China, and Eli Lilly and Co., of Indianapolis | Tyvyt (sintilimab) | Anti-PD-1 monoclonal antibody | Nonsquamous non-small-cell lung cancer | China’s NMPA accepted the sNDA seeking approval for use in combination with Alimta (pemetrexed, Lilly) and platinum chemo as first-line therapy | 4/23/20 | Cancer |
| Janssen Biotech Inc., of Horsham, Pa., and Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | Bruton's tyrosine kinase inhibitor | Chronic lymphocytic leukemia or small lymphocytic lymphoma | FDA approved the drug in combination with rituximab | 4/21/20 | Cancer |
| Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | PD-1-targeting monoclonal antibody | Recurrent/metastatic nasopharyngeal carcinoma | China’s NMPA accepted a supplemental NDA seeking approval for use in patients who failed at least 2 lines of systemic therapy | 4/30/20 | Cancer |
| Kiadis Pharma NV, of Amsterdam | KNK-002 | Haploidentical natural killer cell therapy | Hematologic cancer | IND filed with FDA for phase I/II NK-Realm study to evaluate KNK-002 as adjunctive therapy, along with standard-of-care post-transplant cyclophosphamide protocol, in 63 participants undergoing haploidentical hematopoietic stem cell transplant; study designed to confirm proof-of-concept data suggesting potential to improve outcomes | 4/9/20 | Cancer |
| Marker Therapeutics Inc., of Houston | MT-401 | Multitumor-associated antigen-specific T-cell therapy | Acute myeloid leukemia | FDA granted orphan drug designation to treat individuals with disease following allogeneic stem cell transplant | 4/29/20 | Cancer |
| Medivir AB, of Stockholm | MIV-818 (liver-targeted troxacitabine prodrug) | DNA polymerase inhibitor | Hepatocellular carcinoma | EU's Committee for Orphan Medicinal Products issued positive opinion on orphan medicinal product designation | 4/30/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1 inhibitor | Various cancers | Following CRL issued in February 2020, sNDA resubmitted to FDA updating dosing frequency to include 400-mg dose infused over 30 minutes every 6 weeks in all mono and combo therapy indications in adults | 4/23/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 antibody | All approved indications | FDA approved additional recommended dosage of 400 mg every 6 weeks across all adult indications, including monotherapy and combination therapy; approved under accelerated pathway based on pharmacokinetic data, relationship of exposure to efficacy and relationship of exposure to safety | 4/28/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Unresectable or metastatic solid tumors | Received priority review from the FDA for its sBLA; the FDA has set a PDUFA date of June 16, 2020 | 4/7/20 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | Next-generation anthracycline | Acute myeloid leukemia | Authorized by Polish Department of Registration of Medicinal Products to accelerate phase I dose-escalation portion of trial | 4/28/20 | Cancer |
| Myovant Sciences Ltd., of Basel, Switzerland | Relugolix | GNRH receptor antagonist | Advanced prostate cancer | Submitted NDA to FDA for once-daily oral (120 mg) drug | 4/21/20 | Cancer |
| Neon Therapeutics Inc., of Cambridge, Mass. | NEO-PTC-01 | Neoantigen-stimulated autologous T-cell therapy | Metastatic melanoma | Dutch Health Authority accepted CTA for phase I dose-finding trial in individuals with disease that did not respond to checkpoint inhibitors; study expected to begin in third quarter of 2020 | 4/30/20 | Cancer |
| Novartis AG, of Basel, Switzerland | Kymriah (tisagenlecleucel) | CD19-targeting CAR T | Relapsed or refractory follicular lymphoma | FDA granted regenerative medicine advanced therapy designation | 4/22/20 | Cancer |
| Pfizer Inc., of New York | Ruxience (rituximab) | Biosimilar to Mabthera | Non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and pemphigus vulgaris | Approved by the European Commission | 4/2/20 | Cancer |
| Pfizer Inc., of New York | Braftovi (encorafenib) | Raf B protein kinase inhibitor | Colorectal cancer | FDA approved drug in combination with cetuximab (Erbitux, Merck & Co. Inc.) to treat adults with metastatic disease with BRAFV600E mutation, following prior therapy | 4/8/20 | Cancer |
| Precigen Inc., of Germantown, Md. | PRGN-2009 | HPV antigen | Recurrent or metastatic HPV-associated cancers | FDA cleared the IND for a phase I/II study | 4/20/20 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | CPI-613 (devimistat) | 2 oxoglutarate dehydrogenase inhibitor; pyruvate dehydrogenase inhibitor/kinase stimulator | Acute myeloid leukemia | India's Central Drugs Standard Control Organization and Health Canada approved opening of sites for phase III combination trial with cytarabine and mitoxantrone (HAM) vs. HAM alone in individuals 50 and older with relapsed or refractory disease; enrollment will begin when deemed safe by local officials due to COVID-19 pandemic | 4/23/20 | Cancer |
| Remegen Ltd., of Yantai, China | RC-48 (disitamab vedotin) | Anti-HER2 antibody-drug conjugate | HER2 positive metastatic or unresectable urothelial cancer | FDA cleared the IND for a phase II trial | 4/29/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Rozlytrek (entrectinib) | Tumor-agnostic treatment targeting NTRK | Extracranial solid tumors | Approved by Health Canada for use in patients with unresectable locally advanced or metastatic disease, including brain metastases, that have a NTRK gene fusion without a known acquired resistance mutation, and with no satisfactory treatment options | 4/28/20 | Cancer |
| Samsung Biologics Co. Ltd., of Seoul, and Immuneoncia Therapeutics Inc., of Yongin, South Korea | IMC-002 | CD47 antagonist | Advanced solid tumors; relapsed/refractory lymphoma | FDA approved IND for phase I trial that Immuneoncia will conduct | 4/22/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Padcev (enfortumab vedotin-ejfv) | Antibody-drug conjugate targeting Nectin-4 | Unresectable locally advanced or metastatic urothelial cancer | FDA said data from cohort K of the EV-103/Keynote-869 study could serve as a pivotal study testing drug in patients unable to receive cisplatin-based chemotherapy in the first-line setting | 4/2/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Tukysa (tucatinib) | Oral, small-molecule tyrosine kinase inhibitor of HER2 | Breast cancer | FDA approved for use in adults with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received 1 or more prior anti-HER2-based regimens in the metastatic setting; application was reviewed under real-time oncology review pilot program; NDA also part of Project Orbis | 4/17/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Alunbrig (brigatinib) | Next-generation tyrosine kinase inhibitor targeting ALK genetic alterations | ALK-positive advanced non-small-cell lung cancer | European Commission approved extension of marketing application to include use as monotherapy for treating patients not previously treated with an ALK inhibitor | 4/6/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Mobocertinib (TAK-788) | Tyrosine kinase inhibitor targeting EGFR and HER2 exon 20 insertion mutations | Metastatic non-small-cell lung cancer | FDA granted breakthrough therapy designation for use in patients with EGFR exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy | 4/27/20 | Cancer |
| Tetra Bio-Pharma Inc., of Ottawa | HCC-011 | Inhaled delta-9-tetrahydrocannabinol | Hepatocellular carcinoma | FDA granted request for type B meeting, set for May 29, 2020, to discuss clinical and premarketing requirements for use as adjunct treatment to first-line therapy to improve disease control in patients with inoperable, advanced disease | 4/6/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Single-domain antibody against PD-L1 | Sarcoma | FDA granted company a type B teleconference May 8, 2020, to discuss trial design for a potential pivotal study | 4/6/20 | Cancer |
| Urogen Pharma Inc., of Princeton, N.J. | Jelmyto (mitomycin gel) | Alkylating drug | Low-grade upper tract urothelial cancer | Approved by FDA under priority review | 4/15/20 | Cancer |
| Viracta Therapeutics Inc., of San Diego | Nanatinostat | Histone deacetylase inhibitor | Lymphoma | FDA provided feedback during type C meeting on March 31, 2020, on combination program with DNA polymerase inhibitor valganciclovir to treat relapsed/refractory Epstein-Barr virus-associated disease; meeting covered patient selection and clinical data needed to ensure readiness for registrational trial | 4/8/20 | Cancer |
| Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Epithelial ovarian, fallopian tube or primary peritoneal cancer | China’s National Medical Products Administration accepted the supplemental NDA seeking use as maintenance treatment of advanced epithelial ovarian, fallopian tube or primary peritoneal cancer after complete or partial response to first-line platinum-based chemotherapy | 4/16/20 | Cancer |
| Zentalis Pharmaceuticals Inc., of New York | ZN-d5 | BCL-2 inhibitor | Hematologic malignancies | FDA cleared IND application | 4/30/20 | Cancer |
| 7 Hills Pharma, of Houston | 7HP-349 | Oral integrin activator | Solid tumors and infectious disease | FDA cleared its IND and granted approval to proceed with a proposed phase I trial in healthy volunteers | 4/14/20 | Cancer, Infection |
| Biocardia Inc., of San Carlos, Calif. | Cardiamp cell therapy | Autologous bone marrow-derived stem cells | Cardiac failure | FDA-recommended modifications adopted for primary endpoint of Cardiamp-HF trial, revised to outcomes composite score based on 3-tiered Finkelstein-Schoenfeld hierarchical analysis, along with associated statistical analysis plan; tiers include all-cause death, non-fatal major adverse coronary and cerebrovascular events and change from baseline in 6-minute walk distance at 12 months | 4/30/20 | Cardiovascular |
| Liquidia Technologies Inc., of Research Triangle Park, N.C. | LIQ-861 | Inhaled dry powder formulation of treprostinil | Pulmonary arterial hypertension | FDA accepted NDA filing for the treatment of pulmonary arterial hypertension. The PDUFA date is November 24, 2020 | 4/8/20 | Cardiovascular |
| Arch Therapeutics Inc., of Framingham, Mass. | AC5 Topical Hemostat | Contains synthetic peptide that self-assembles into a contiguous physical-mechanical seal on tissue | Skin wounds | FDA granted CE mark authorization for use as a dressing and to control bleeding in skin wounds in both out- and in-patient settings | 4/13/20 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 (deuterium-modified ruxolitinib) | JAK1/JAK2 tyrosine kinase inhibitor | Alopecia areata | Completed end-of-phase II meeting with FDA, reaching agreement on planned phase III program to include 2 trials in adults at sites in the U.S., Canada and Europe to evaluate Severity of Alopecia Tool score after 24 weeks of dosing in participants with moderate to severe disease | 4/1/20 | Dermatologic |
| Histogen Inc., of San Diego | HST-001 | Hair-stimulating complex | Androgenic alopecia | Submitted IND to FDA for a phase Ib/IIa trial in men | 4/6/20 | Dermatologic |
| Inmed Pharmaceuticals Inc., of Vancouver, British Columbia | INM-755 (cannabinol) | Keratin modulator | Epidermolysis bullosa | Dutch regulatory and ethics board approved CTA for second phase I trial in healthy volunteers, expected to begin once COVID-19 pandemic-related restrictions are lifted sometime after May 20, 2020 | 4/30/20 | Dermatologic |
| Lifemax Laboratories Inc., of Palo Alto, Calif. | LM-030 | Kallikrein-related peptidase inhibitor | Netherton syndrome | FDA granted fast track designation | 4/14/20 | Dermatologic |
| Mallinckrodt plc, of Staines-upon-Thames, U.K. | Stratagraft | Regenerative skin tissue therapy | Deep partial-thickness thermal burns | Initiated rolling BLA submission to FDA | 4/6/20 | Dermatologic |
| Pharming Group NV, of Leiden, the Netherlands | Ruconest (conestat alfa) | Recombinant C1 esterase inhibitor | Hereditary angioedema | European Commission approved drug to treat acute disease attacks in children 2 and older | 4/30/20 | Dermatologic |
| Tetra Bio-Pharma Inc., of Ottawa | PPP-004 | Delta-9 tetrahydrocannabinol and cannabidiol | Epidermolysis bullosa | FDA granted orphan drug designation | 4/16/20 | Dermatologic |
| Acasti Pharma Inc., of Laval, Quebec | Capre | Omega-3 phospholipid | Hypertriglyceridemia | Meeting request submitted to FDA to discuss phase III Trilogy 1 study, which missed statistical significance for primary endpoint, and gain alignment on data interpretation; FDA input also will be sought on proposed revisions to prespecified statistical analysis plan for still-blinded Trilogy 2 trial and potential plan to pool data from Trilogy studies to support NDA filing; meeting expected in second half of June 2020 | 4/1/20 | Endocrine/Metabolic |
| Acasti Pharma Inc., of Laval, Quebec | Capre | Krill oil-derived omega-3 phospholipid | Hypertriglyceridemia | Trilogy 1 briefing package submitted to FDA to gain alignment on data interpretation, proposed revisions to prespecified statistical analysis plan for still-blinded Trilogy 2 and plan for pooling data from both studies to support NDA filing; formal response expected by June 30, 2020 | 4/30/20 | Endocrine/Metabolic |
| Aeglea Biotherapeutics Inc., of Austin, Texas | ACN-00177 | Recombinant enzyme that degrades homocysteine and homocystine dimers | Homocystinuria | U.K.’s Medicines and Healthcare Products Regulatory Agency approved the CTA | 4/8/20 | Endocrine/Metabolic |
| Aeterna Zentaris Inc., of Charleston, S.C. | Macimorelin | Ghrelin agonist | Growth hormone deficiency | EMA agreed to adjust the company's pediatric investigation plan, so study P02 can be used to meet requirements for FDA and EMA. | 4/7/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | Subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | Completed the rolling submission of an NDA to the FDA; also submitted an MAA to the EMA | 4/7/20 | Endocrine/Metabolic |
| Amidebio LLC, of Boulder, Colo. | ABG-023 (glucagon analog; AB-GLC-0035) | Glucagon receptor agonist | Congenital hyperinsulinism | FDA granted orphan drug designation | 4/23/20 | Endocrine/Metabolic |
| Arcturus Therapeutics Inc., of San Diego | ARCT-810 (Lunar-OTC) | mRNA therapeutic | Ornithine transcarbamylase deficiency | FDA cleared IND for a phase Ib study in OTC patients; New Zealand Medicines and Medical Devices Safety Authority cleared CTA for phase I study in healthy volunteers | 4/13/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon hGH (lonapegsomatropin) | Long-acting prodrug of somatropin | Growth hormone deficiency | FDA granted orphan drug designation | 4/15/20 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | Lokelma (sodium zirconium cyclosilicate) | Potassium binder | Hyperkalemia | FDA approved label update to treat hyperkalemia in patients with end-stage renal disease on chronic hemodialysis | 4/27/20 | Endocrine/Metabolic |
| Daiichi Sankyo Europe GmbH, of Munich, Germany | Nilemdo (bempedoic acid) | ATP citrate lyase inhibitor | Primary hypercholesterolemia or mixed dyslipidemia | Approved by European Commission for use in adults | 4/6/20 | Endocrine/Metabolic |
| Daiichi Sankyo Europe GmbH, of Munich, Germany | Nustendi (bempedoic acid/ezetimibe) | ATP citrate lyase inhibitor plus cholesterol absorption inhibitor | Primary hypercholesterolemia or mixed dyslipidemia | Approved by European Commission for use in adults | 4/6/20 | Endocrine/Metabolic |
| Diurnal Group plc, of London | Chronocort (modified release hydrocortisone) | Glucocorticoid receptor agonist | Congenital adrenal hyperplasia | MAA passed validation with EMA | 4/1/20 | Endocrine/Metabolic |
| Lannett Co. Inc., of Philadelphia | Insulin glargine biosimilar | Insulin ligand; insulin receptor agonist | Diabetes mellitus; non-insulin dependent diabetes | FDA approved request for biosimilar biological product development type II meeting, set for June 9, 2020, to discuss design and endpoint of any additional human studies required for BLA filing | 4/8/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Rybelsus (oral semaglutide) | GLP-1 analogue | Type 2 diabetes | European Commission granted marketing authorization for use in treating adults with insufficiently controlled disease to improve glycemic control as an adjunct to diet and exercise | 4/6/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBGM-01 | GLB1 gene stimulator | Infantile GM1 gangliosidosis | FDA granted orphan drug designation | 4/21/20 | Endocrine/Metabolic |
| Provention Bio Inc., of Oldwick, N.J. | Teplizumab | Anti-CD3 monoclonal antibody | Delay or prevention of clinical type 1 diabetes | Started rolling submission of a BLA; nonclinical module submitted; clinical module submission planned for third quarter of 2020; chemistry, manufacturing and controls module submission planned for fourth quarter of 2020 | 4/16/20 | Endocrine/Metabolic |
| Seelos Therapeutics Inc., of New York | SLS-005 | Trehalose | Sanfilippo syndrome | FDA granted orphan designation | 4/30/20 | Endocrine/Metabolic |
| Galectin Therapeutics Inc., of Norcross, Ga. | Belapectin (GR-MD-02) | Galectin-3 inhibitor | Nonalcoholic steatohepatitis cirrhosis | Protocol submitted to FDA for seamless adaptive design phase IIb/III NASH-RX trial assessing prevention of esophageal varices in individuals with disease; trials expected to begin in second quarter of 2020 | 4/30/20 | Gastrointestinal |
| Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Moderate to severe ulcerative colitis | Following discussions with the FDA and EMA, company plans to advance drug to phase III development with sequential studies that may include a higher dose than was tested in the phase IIb study | 4/16/20 | Gastrointestinal |
| Futura Medical plc, of Guilford, U.K. | MED-3000 | Volatile solvent | Erectile dysfunction | Filed for a pre-submission meeting with the FDA | 4/20/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Staines-upon-Thames, U.K. | Terlipressin | Vasopressin analogue selective for V1 receptors | Hepatorenal syndrome type 1 | FDA accepted the NDA and assigned a PDUFA date of Sept. 12, 2020 | 4/22/20 | Genitourinary/Sexual Function |
| Mayne Pharma Group Ltd., of Adelaide, Australia, and Mithra Pharmaceuticals SA, of Liege, Belgium | E4/DRSP | Estetrol and drospirenone | Prevention of pregnancy | Submitted NDA to FDA | 4/16/20 | Genitourinary/Sexual Function |
| Akarx Inc., unit of Dova Pharmaceuticals Inc., of Durham, N.C., a Swedish Orphan Biovitrum AB company | Doptelet (avatrombopag) | Thrombopoietin receptor agonist | Thrombocytopenia | China NMPA approved drug to treat adults with chronic liver disease scheduled to undergo a procedure | 4/30/20 | Hematologic |
| Beyondspring Inc., of New York | Plinabulin | Immune and stem cell modulator | Chemotherapy-induced neutropenia | Following discussions with FDA, company formally changed primary endpoint of Study 106 phase III trial to be the rate of prevention of grade 4 neutropenia in combination with G-CSF vs. G-CSF alone; previous endpoint had been duration of severe (grade 4) neutropenia | 4/29/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | Saudi Food & Drug Authority accepted request for priority review | 4/16/20 | Hematologic |
| Fera Pharmaceuticals LLC, of New York, and Nicox SA, of Sophia Antipolis, France | Naproxcinod | Cyclooxygenase-inhibiting, nitric oxide-donating naproxen | Sickle cell disease | Applied for orphan drug designation with the FDA | 4/2/20 | Hematologic |
| Forma Therapeutics Inc., of Watertown, Mass. | FT-4202 | Activator of pyruvate kinase-R | Sickle cell disease | FDA granted orphan designation | 4/15/20 | Hematologic |
| Harbour Biomed Therapeutics Ltd., of Cambridge, Mass. | NBM-9161 | Antibody targeting neonatal Fc receptor | Immune thrombocytopenia | Received IND approval from China’s NMPA to begin phase II/III trial in adults; approval provides accelerated development pathway, allowing phase III to proceed directly following phase II | 4/15/20 | Hematologic |
| LFB SA, of Paris | Sevenfact | Coagulation factor VIIa (recombinant)-njcw | Hemophilia A and B | FDA approved use to treat and control bleeding episodes in adults and adolescents 12 and older with inhibitors | 4/1/20 | Hematologic |
| Alk-Abello A/S, of Hørsholm, Denmark | Standardized allergen extract, white birch (betula verrucosa) sublingual tablet | Allergy desensitization vaccine | Seasonal allergic rhinitis | Health Canada approved once-daily sublingual allergy immunotherapy, branded Itulatek, to treat tree pollen allergy | 4/20/20 | Immune |
| Banner Life Sciences LLC, of High Point, N.C. | Bafiertam (monomethyl fumarate) | Nuclear erythroid 2-related factor 2 stimulator | Relapsing multiple sclerosis | Delayed-release drug, bioequivalent of Tecfidera (dimethyl fumarate, Biogen Inc.), granted final approval by FDA through 505(b)(2) pathway | 4/28/20 | Immune |
| Janssen Pharmaceutical Cos., of Horsham, Pa. | Simponi Aria (golimumab) | Anti-TNF-alpha biologic for intravenous injection | Polyarticular juvenile idiopathic arthritis | Submitted supplemental BLA to the FDA seeking approval for use in patients, 2 and older, in combination with methotrexate | 4/24/20 | Immune |
| Janssen Pharmaceutical Cos., of Horsham, Pa. | Simponi Aria (golimumab) | Anti-TNF-alpha biologic for intravenous injection | Juvenile psoriatic arthritis | Submitted supplemental BLA to the FDA seeking approval for use in patients, 2 and older, in combination with methotrexate | 4/24/20 | Immune |
| Kadmon Holdings Inc., of New York | KD-025 | Oral inhibitor of ROCK2 | Chronic graft-vs.-host disease | Received the final meeting minutes from the FDA following its NDA pre-submission meeting, and the company said it believes the proposed data package will be sufficient to support a submission | 4/14/20 | Immune |
| Mesoblast Ltd., of Sydney | Ryoncil (remestemcel-L) | Mesenchymal stem cell therapy | Graft-vs.-host disease | FDA accepted BLA filing to treat children with steroid-refractory acute disease, granting priority review and setting Sept. 30, 2020, PDUFA date | 4/1/20 | Immune |
| Mustang Bio Inc., of New York | MB-107 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | EMA granted advanced therapy medicinal product classification | 4/20/20 | Immune |
| Remegen Ltd., of Yantai, China | RC-18 | Recombinant transmembrane activator, calcium modulator and cyclophil ligand interactor fusion protein | Systemic lupus erythematosus | FDA granted fast track designation | 4/15/20 | Immune |
| Vitalis LLC, of New York | Monomethyl fumarate + aspirin; diroximel fumarate + aspirin | Fixed-dose combinations of fumarate salts + VTS-aspirin | Relapsing/remitting multiple sclerosis | FDA granted orphan drug designation to reduce flush | 4/21/20 | Immune |
| 4D Pharma plc, of Leeds, U.K. | MRx-4DP0004 | Live biotherapeutic | COVID-19 | Received expedited acceptance from the UK Medicines and Healthcare products Regulatory Agency to start a phase II study | 4/20/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | N-methyl-D-aspartate receptor glutamate receptor antagonist | COVID-19 | Company received positive feedback from Health Canada's office of clinical trials and plans to submit a CTA for a phase II study within the next week | 4/8/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NMDA receptor antagonist | COVID-19 | Received positive feedback from FDA regarding plans to conduct a phase II trial; plans to submit IND to study drug in patients who experience respiratory complications as a result of contracting COVID-19 | 4/13/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NMDA receptor antagonist | Acute lung injury, including COVID-19 | Received feedback from FDA regarding plans to reformulate repurposed drug into intravenous product suited for hospital and ICU use; agency advised that single animal 30-day study would be acceptable for toxicology program | 4/15/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | N-methyl-D-aspartate receptor antagonist | COVID-19 | Submitted CTA to Health Canada for a phase IIb/III study enrolling 100 patients in the phase IIb portion; data from the phase IIb will determine the size of the phase III; primary endpoint is improvement in the ordinal clinical scale; secondary endpoints include mortality, blood oxygen levels, time in the ICU and time to mechanical ventilation | 4/22/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NMDA receptor antagonist | COVID-19 | Received no objection letter from Health Canada for phase IIb/III trial; same study protocol being prepared for submission to U.S. and Australian regulators | 4/29/20 | Infection |
| Applied Therapeutics Inc., of New York | AT-001 | Aldose reductase inhibitor | COVID-19 | IND opened with FDA for multiple investigator-initiated studies | 4/2/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | COVID-19 | Submitted CTA to Health Canada for a phase II study to prevent lung and kidney inflammation in COVID-19 patients | 4/23/20 | Infection |
| Ascletis Inc., of Hangzhou, China | ASC-09F | Fixed-dose combo of HIV protease inhibitor and ritonavir | HIV | Received IND approval from China’s NMPA | 4/13/20 | Infection |
| Athersys Inc., of Cleveland | Multistem | Cell therapy | COVID-19 | FDA authorized start of phase II/III study to assess safety and efficacy in subjects with moderate to severe acute respiratory distress syndrome induced by COVID-19 | 4/13/20 | Infection |
| Atyr Pharma Inc., of San Diego | ATYR-1923 | Immuno-modulatory domain of histidyl tRNA synthetase fused to the FC region of a human antibody | COVID-19 with severe respiratory complications | FDA accepted the IND for a phase II study of 30 patients comparing two doses of the drug to placebo | 4/21/20 | Infection |
| Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Inhaled nitric oxide system | COVID-19 | Submitted IND to the FDA | 4/8/20 | Infection |
| Biontech SE, of Mainz, Germany, and Pfizer Inc., of New York | BNT-162 | mRNA COVID-19 vaccine | COVID-19 prophylaxis | German regulatory authority, the Paul-Ehrlich-Institut, approved the phase I/II study | 4/22/20 | Infection |
| Biosig Technologies Inc., of Westport, Conn., and subsidiary Viralclear Pharmaceuticals Inc. | Merimepodib | Broad-spectrum antiviral | COVID-19 | Viralclear submitted IND to FDA for phase II trial | 4/24/20 | Infection |
| Caladrius Biosciences Inc., of Basking Ridge, N.J. | CLBS-119 | CD34+ cell therapy | COVID-19-induced lung damage | FDA authorized IND application for study assessing therapy's ability to restore lung function in individuals with severe SARS-CoV-2 infection who required ventilation due to respiratory failure | 4/23/20 | Infection |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | COVID-19 | Received approval at Rabin Medical Center to start pilot study in treatment of moderate to severe symptoms in COVID-19-infected patients | 4/13/20 | Infection |
| Capricor Inc., of Los Angeles | CAP-1002 | Allogeneic cardiac cell therapy | COVID-19 | Following a review of available data, FDA approved expanded access protocol to treat up to 20 additional patients | 4/29/20 | Infection |
| Celularity Inc., of Warren, N.J. | CYNK-001 | Placenta-derived natural killer cell therapy | COVID-19 | FDA cleared the IND for a phase I/II trial | 4/2/20 | Infection |
| Chimerix Inc., of Durham, N.C. | Brincidofovir | Antiviral | Smallpox | Received clearance from FDA for rolling NDA seeking approval for use as medical countermeasure | 4/28/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab | CCR5 antagonist | COVID-19 infection | Additional clinical trial protocol filed with FDA to treat individuals with severe disease under same FDA-approved IND as recently initiated phase II trial in those with mild to moderate disease; new protocol is for 342 participants, double-blinded with 2-to-1 ratio, with primary endpoint of mortality rate at 14 days | 4/1/20 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab (PRO-140) | CCR5 antagonist | HIV | Submitted clinical and CMC portions of BLA to FDA, seeking approval for use as combination with HAART for highly treatment-experienced HIV patients | 4/27/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | TSC | Trans sodium crocetinate | COVID-19 | Pre-IND submission to FDA for a planned clinical program in patients displaying severe respiratory symptoms and low oxygen levels | 4/27/20 | Infection |
| Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J. | Ryanodex | Dantrolene sodium | COVID-19 | Submitted IND to FDA for a phase II trial in partnership with Hackensack University Medical Center to evaluate the efficacy of Ryanodex in patients infected with SARS-CoV-2 | 4/16/20 | Infection |
| Everest Medicines Inc., of New York | Xerava (eravacycline) | Fully synthetic fluorocycline | Complicated intra-abdominal infections | The Health Science Authority in Singapore approved it | 4/17/20 | Infection |
| Hope Biosciences Inc., of Houston | HB-adMSCs | Allogeneic, adipose-derived mesenchymal stem cells | COVID-19 | FDA approved phase II trial to test safety and efficacy in providing immune support against COVID-19 | 4/13/20 | Infection |
| Hope Biosciences Inc., of Houston | HB-adMSCs | Adipose-derived mesenchymal stem cell therapy | COVID-19 infection | FDA approved phase II study expected to enroll 110 hospitalized participants into 2 arms; those taking and those not taking hydroxychloroquine and azithromycin will be randomized into separate placebo or treatment groups, in addition to standard of care; those treated will receive 4 doses of 100 million HB-adMSCs over 10-day period | 4/21/20 | Infection |
| Hope Biosciences LLC, of Houston | HB-adMSCs | Autologous, adipose-derived mesenchymal stem cells | COVID-19 | FDA cleared the IND for a phase II trial for protection against COVID-19 | 4/6/20 | Infection |
| Humanigen Inc., of Burlingame, Calif. | Lenzilumab | Anti-human GM-CSF monoclonal antibody | COVID-19 | FDA cleared start of phase III trial testing drug for prevention of respiratory failure and/or death in hospitalized patients with pneumonia associated with SARS-CoV-2 infection | 4/15/20 | Infection |
| I-Mab Biopharma Co. Ltd., of Shanghai | TJM-2 | GM-CSF ligand inhibitor | COVID-19 infection | FDA cleared IND application for phase I study assessing treatment of cytokine release syndrome associated with severe infection; Western Institutional Review Board also provided approval; 3-arm study evaluating effect on reducing severity of complications and levels of multiple cytokines | 4/3/20 | Infection |
| Mateon Therapeutics Inc., of Agoura Hills, Calif. | OT-101 | Phosphorothioate antisense oligodeoxynucleotide targeting human TGF-?2 mRNA | COVID-19 | Submitted pre-IND package to FDA to allow referencing of OT-101’s oncology IND to streamline submission for testing in COVID-19 | 4/6/20 | Infection |
| Mateon Therapeutics Inc., of Agoura Hills, Calif. | OT-101 | Inhibitor of SAR-CoV2 replication | COVID-19 | Submitted IND to FDA for phase II study in adults hospitalized with COVID-19 and pneumonia | 4/27/20 | Infection |
| Mesoblast Ltd., of Melbourne, Australia | Remestemcel-L | Allogeneic mesenchymal stem cell product | COVID-19 | FDA cleared IND for testing in patients with acute respiratory distress syndrome caused by coronavirus infection | 4/6/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | mRNA vaccine candidate | COVID-19 | Submitted IND to FDA to evaluate vaccine in phase II and late-stage studies if supported by safety data from phase I study led by NIH’s National Institute of Allergy and Infectious Diseases; phase II study expected to begin in second quarter of 2020 | 4/27/20 | Infection |
| Novan Inc., of Morrisville, N.C. | SB-206 (berdazimer sodium) | Nitric oxide-releasing macromolecule | Molluscum contagiosum infection | During type C meeting, FDA said it will consider 1 additional pivotal trial (B-Simple4) as confirmatory study of B-Simple2 and provided guidance on trial design and expectations for potential NDA submission | 4/3/20 | Infection |
| Ortho Dermatologics, a unit of Bausch Health Cos. Inc., of Laval, Quebec | Jublia (efinaconazole) topical solution 10% | Antifungal | Onychomycosis | FDA approved a supplemental NDA extending the age range to children 6 and older | 4/29/20 | Infection |
| Oryzon Genomics SA, of Madrid | Vafidemstat | Oral, CNS optimized LSD1 inhibitor | COVID-19 | Received approval from Spanish drug agency (AEMPS) to conduct phase II study in seriously ill patients | 4/24/20 | Infection |
| Pharmamar SA, of Madrid | Aplidin (plitidepsin) | Cyclodepsipeptide | COVID-19 pneumonia | Submitted trial protocol for the Aplicov study to the Spanish Medicines and Healthcare Products Agency | 4/2/20 | Infection |
| Pharmamar SA, of Madrid, Spain | Aplidin (plitidepsin) | Compound extracted from ascidian Aplidium albicans | COVID-19 | Started trial authorized by Spanish Medicines and Healthcare Products Agency | 4/28/20 | Infection |
| Predictive Biotech, of Salt Lake City, a subsidiary of Predictive Technology Group | MSCs | Mesenchymal stem cells derived from umbilical cord tissue | COVID-19 | Submitted emergency use authorization application to FDA for immediate use of MSCs for treating acute respiratory distress syndrome secondary to SARS-CoV-2 disease | 4/13/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Yeliva (opaganib, ABC-294640) | Oral sphingosine kinase-2 selective inhibitor | COVID-19 | Submitted IND to FDA for phase IIa trial in up to 60 adults hospitalized with positive SARS-CoV-2 and pneumonia | 4/17/20 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGN-EB3 | Triple antibody cocktail | Ebola virus infection | FDA accepted BLA for priority review with a PDUFA date of Oct. 25, 2020 | 4/16/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Xanthine oxidase inhibitor | COVID-19 infection | Filed pre-IND meeting request with FDA seeking permission to proceed to phase II study in indication | 4/3/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine (repurposed) | Xanthine oxidase inhibitor | COVID-19 infection | Responding to pre-IND meeting, FDA recommended proceeding to confirmatory phase III trial in patients with mild to moderate infection and provided guidance on study design and outcome measures; IND filing expected within 60 days | 4/23/20 | Infection |
| Ridgeback Biotherapeutics LP, of Miami | EIDD-2801 | Oral direct acting antiviral nucleoside analogue | COVID-19 | U.K.’s Medicines and Healthcare products Regulatory Agency cleared for phase I testing | 4/13/20 | Infection |
| Sanofi SA, of Paris | Menquadfi | Meningococcal (groups A, C, Y, W) conjugate vaccine | Meningococcal disease | FDA approved BLA for the prevention of invasive disease in persons 2 and older | 4/24/20 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | Cefiderocol | Antibiotic | Infections due to aerobic gram-negative bacteria | European Commission granted approval for use in adults with limited treatment options | 4/28/20 | Infection |
| Sinovac Biotech Ltd., of Beijing | Vaccine | Inactivated SARS-CoV-2 vaccine | COVID-19 | Obtained approval to conduct human clinical trial in China | 4/14/20 | Infection |
| The KD Pharma Group SA, of Bioggio, Switzerland, and SLA Pharma AG, of Liestal, Switzerland | Epaspire | Oral formulation of highly purified eicosapentaenoic acid free fatty acid | COVID-19 | U.K.'s MHRA approved the trial; the companies have submitted an application to the FDA for a trial | 4/14/20 | Infection |
| Vitro Diagnostics Inc., of Golden, Co. | Allorx stem cells | Umbilical cord-derived stem cells | COVID-19 | Filed an IND with the FDA | 4/21/20 | Infection |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 | Targets mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways | Amyotrophic lateral sclerosis | EMA’s Committee for Orphan Medicinal Products recommended orphan designation | 4/29/20 | Musculoskeletal |
| Carmell Therapeutics Inc., of Pittsburgh | BHA | Bone healing accelerant | Bone fractures | FDA granted fast track status | 4/27/20 | Musculoskeletal |
| Epirium Bio Inc., of San Diego | EB-002 ((+)-epicatechin) | Synthetic flavanol | Duchenne and Becker muscular dystrophy | Received orphan drug designation from the FDA | 4/14/20 | Musculoskeletal |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Inhibits IL-17A | Active non-radiographic axial spondyloarthritis | Approved by European Commission | 4/29/20 | Musculoskeletal |
| Pfenex Inc., of San Diego | PF-708 | Therapeutic equivalent candidate to Forteo (teriparatide injection) | Osteoporosis | FDA said additional comparative use human factors data, specifically from Forteo-experienced users, would be required before therapeutic equivalence could be determined | 4/14/20 | Musculoskeletal |
| Seneca Biopharma Inc., of Germantown, Md. | NSI-566 | Human neural stem cell therapy | Amyotrophic lateral sclerosis | Feedback from type C meeting with FDA on March 10, 2020, indicated existing phase I and II trial results support moving into phase III study; protocol design underway using agency guidance | 4/9/20 | Musculoskeletal |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Vutrisiran (ALN-TTRsc02) | RNAi targeting TTR | Polyneuropathy of hereditary transthyretin-mediated amyloidosis | FDA granted fast track designation | 4/14/20 | Neurology/Psychiatric |
| Alpha Cognition Inc., of Vancouver, British Columbia | ALPHA-1062 | Acetylcholinesterase inhibitor; nicotinic acetylcholine receptor modulator | Alzheimer's disease | At pre-IND meeting, FDA suggested that single pivotal trial, scheduled to begin in mid-2021, may be sufficient to obtain marketing approval | 4/30/20 | Neurology/Psychiatric |
| Annovis Bio Inc., of Berwyn, Pa. | ANVS-401 | Acetylcholinesterase/beta-amyloid synthesis/huntingtin/synuclein alpha/tau protein inhibitor | Alzheimer's disease; Parkinson's disease | Phase II trial expected to enroll 68 individuals across both indications set to begin recruiting in May 2020 and treating participants in June 2020 after FDA accepted protocol without comments | 4/30/20 | Neurology/Psychiatric |
| Athersys Inc., of Cleveland | Multistem | Cell therapy | Severe traumatic injury | FDA authorized IND for phase II study for early treatment of traumatic injuries and the subsequent complications that result following severe trauma | 4/15/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Dayvigo (lemborexant) | Binds to orexin receptors OX1R and OX2R | Insomnia | Drug Enforcement Administration issued interim final rule placing lemborexant in schedule IV of the Controlled Substances Act | 4/6/20 | Neurology/Psychiatric |
| GW Pharmaceuticals plc, of Carlsbad, Calif. | Epidiolex | Cannabidiol | Lennox-Gastaut and Dravet syndromes | Received notification from U.S. Drug Enforcement Administration confirming drug is no longer subject to Controlled Substances Act; change takes effect immediately | 4/6/20 | Neurology/Psychiatric |
| Neuraptive Therapeutics Inc., of Philadelphia | NTX-001 | Drug-device combination | Peripheral nerve injuries | FDA cleared IND for testing in patients with acute single transected PNI | 4/29/20 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego | Ongentys (opicapone) | Oral, selective catechol-O-methyltransferase inhibitor | Parkinson’s disease | FDA approved as add-on treatment to levodopa/carbidopa in patients experiencing “off” episodes | 4/24/20 | Neurology/Psychiatric |
| Polaryx Therapeutics Inc., of Paramus, N.J. | PLX-200 | Binds to the retinoid X receptor-alpha | Juvenile neuronal ceroid lipofuscinosis | FDA approved the IND application | 4/8/20 | Neurology/Psychiatric |
| Polyneuron Pharmaceuticals AG, of Basel, Switzerland | PN-1007 | Mimics natural HNK-1 carbohydrate epitope found on myelin of peripheral nerves and binds to circulating disease-causing antibodies | Anti-MAG neuropathy | Regulatory authorities in France, the Netherlands and the U.K. approved CTA for a phase I/IIa study | 4/27/20 | Neurology/Psychiatric |
| Retrotope Inc., of Los Altos, Calif. | RT-001 | Chemically stabilized fatty acid | Progressive supranuclear palsy | FDA cleared the IND for a phase II/III trial | 4/25/20 | Neurology/Psychiatric |
| Roche Holding AG, of Basel Switzerland | Risdiplam | Survival motor neuron-2 splicing modifier | Spinal muscular atrophy | FDA extended PDUFA date to Aug. 24, 2020, to allow time to review data from part 2 of the Sunfish study, which was submitted in February 2020 | 4/7/20 | Neurology/Psychiatric |
| Shanghai Green Valley Pharmaceuticals Co. Ltd., of Shanghai | GV-971 | Oral mixture of acidic linear oligosaccharides derived from marine brown algae | Alzheimer’s disease | FDA cleared IND for a phase III study | 4/27/20 | Neurology/Psychiatric |
| Tissuetech Inc., of Miami | TTAX-02 | Cryopreserved human umbilical cord investigational biologic product | Spina bifida | FDA granted regenerative medicine advanced therapy designation for treatment of spina bifida in-utero | 4/16/20 | Neurology/Psychiatric |
| Gensight Biologics SA, of Paris | Lumevoq (GS-010; lenadogene nolparvovec) | Gene therapy | Leber hereditary optic neuropathy | Completed pre-submission meeting with EMA; confirmed intention to submit MAA in September 2020 | 4/15/20 | Ocular |
| Iveric Bio Inc., of New York | Zimura (avacincaptad pegol) | Complement C5 inhibitor | Geographic atrophy | FDA granted fast track designation to treat GA secondary to dry age-related macular degeneration | 4/3/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France | NCX-4251 | Ophthalmic suspension of fluticasone propionate that targets the glucocorticoid receptor | Blepharitis and dry eye disease | Reached agreement with FDA on phase IIb trial designs for both acute exacerbations of blepharitis and the reduction of signs and symptoms of dry eye disease | 4/8/20 | Ocular |
| Tetra Bio-Pharma Inc., of Ottawa | PPP-003 | Activates type 2 cannabinoid receptor | Prevention of proliferative vitreoretinopathy | FDA granted orphan designation | 4/15/20 | Ocular |
| ANI Pharmaceuticals Inc., of Baudette, Minn. | Cortrophin Gel | Repository corticotropin injection USP | Undisclosed | Received a refuse to file letter from FDA regarding its supplemental NDA | 4/29/20 | Other/Miscellaneous |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Lonafarnib | Orally active inhibitor of farnesyltransferase | Hutchinson-Gilford progeria syndrome and progeroid laminopathies | EMA validated MAA | 4/27/20 | Other/Miscellaneous |
| Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. | Pedmark | Formulation of sodium thiosulfate | Ototoxicity induced by cisplatin chemotherapy | FDA accepted for filing and granted priority review to NDA seeking approval for use in preventing ototoxicity in patients 1 month to under 18 years with localized, nonmetastatic solid tumors; The PDUFA date is August 10, 2020 | 4/13/20 | Other/Miscellaneous |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (repurposed ifenprodil) | NDMA receptor glutamate receptor antagonist | COVID-19-related pneumonia | Regulatory submission filed with South Korea's Ministry of Food and Drug Safety for investigator-led phase II study in 40 individuals with severe disease; primary endpoint is lung function improvement measured by blood oxygen levels; secondary endpoints include mortality, rate of mechanical ventilation and patient-reported effects on cough and dyspnea | 4/9/20 | Respiratory |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (repurposed ifenprodil) | NMDA receptor antagonist | COVID-19-related severe pneumonia | South Korea's Ministry of Food and Drug Safety approved investigator-led phase II study in 40 participants assessing lung function improvement; primary endpoint is blood oxygen levels (Pa02/Fi02); secondary endpoints include mortality, rate of mechanical ventilation and patient-reported effects on dyspnea; enrollment expected to begin May 8, 2020 | 4/23/20 | Respiratory |
| Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-ENaC | RNAi therapy | Cystic fibrosis | Filed an application for clearance to begin a phase I/IIa dose-escalation study to evaluate safety, tolerability and pharmacokinetic effects in healthy volunteers and to evaluate safety, tolerability and efficacy in patients with CF | 4/10/20 | Respiratory |
| Azurrx Biopharma Inc., of New York | MS-1819 | Recombinant lipase enzyme | Cystic fibrosis | Received approval to conduct phase II Option trial in patients with exocrine pancreatic insufficiency | 4/29/20 | Respiratory |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Vazegepant | CGRP receptor antagonist | COVID-19-related lung inflammation | FDA approved IND for placebo-controlled phase II study, expected to begin within weeks, of intranasal drug to treat individuals hospitalized with disease and requiring supplemental oxygen; primary outcome measure includes resolution of pulmonary symptoms vs. progression to ventilator support or death | 4/9/20 | Respiratory |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | Ofev (nintedanib) | Tyrosine kinase inhibitor | Systemic sclerosis-associated interstitial lung disease | EC approved drug to treat adult patients | 4/21/20 | Respiratory |
| Calcimedica Inc., of La Jolla, Calif. | CM-4620-IE | Ca2+ release activated Ca2+ channel 1 inhibitor | COVID-19-related pneumonia | FDA issued letter to proceed with open-label phase II study in combination with standard of care (n=40) vs. standard of care alone (n=20) in individuals with severe disease at risk to progress to acute respiratory distress syndrome; initial participants enrolled at first study site | 4/9/20 | Respiratory |
| Faron Pharmaceuticals Oy, of Turku, Finland | Interferon-beta-1a (intravenous; also Traumakine) | Interferon-beta ligand | COVID-19-related acute respiratory distress syndrome | Regulatory approval granted for global Remap-Cap (Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community-Acquired Pneumonia) program that includes Faron candidate, with primary outcome of 90-day all-cause mortality | 4/1/20 | Respiratory |
| Mallinckrodt plc, of Staines-upon-Thames, U.K., and Novoteris LLC, of Garden Grove, Calif. | Thiolanox | Nitric oxide inhalant | COVID-19-associated lung complications | Health Canada's Therapeutic Products Directorate cleared joint pilot trial assessing Novoteris' inhaled nitric oxide delivery device and Mallinckrodt's high-concentration nitric oxide gas for inhalation canisters; recruitment expected to begin within days at Vancouver Coastal Health Authority facilities | 4/1/20 | Respiratory |
| Proteostasis Therapeutics Inc., of Boston | Dirocaftor, posenacaftor and nesolicaftor | Triple-combination CF therapy | Cystic fibrosis | Received scientific advice from Dutch Medicines Evaluation Board for Choices personalized medicine-based study in CF | 4/13/20 | Respiratory |
| Zambon SpA, of Milan, Italy | Liposomal cyclosporine A for inhalation | Calcineurin inhibitor | Bronchiolitis obliterans syndrome | FDA granted fast track designation | 4/28/20 | Respiratory |
| Agenus Inc., of Lexington, Mass. | AgenT-797 | Allogeneic iNKT therapy | Cancer | FDA accepted IND; clinical study expected to start in second half of 2020 | 5/13/20 | Cancer |
| Alethia Biotherapeutics Inc., of Montreal | AB-16B5 | Inhibitor of epithelial to mesenchymal transition | Metastatic non-small-cell lung cancer | FDA cleared the IND for a phase II study testing combination with docetaxel in previously treated patients who have experienced disease progression following treatment with a platinum-containing doublet treatment and an anti-PD1 or PD-L1 immune checkpoint antibody | 5/21/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | HQP-1351 | BCR-ABL inhibitor | Chronic myeloid leukemia | FDA granted orphan drug designation | 5/4/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | HQP-1351 | BCR-ABL inhibitor | Chronic myeloid leukemia | FDA granted fast track designation for use in patients with certain genetic mutations who have failed to respond to treatments with existing tyrosine kinase inhibitors | 5/7/20 | Cancer |
| Astellas Pharma Inc., of Tokyo | Xtandi (enzalutamide) | Oral androgen receptor signaling inhibitor | Prostate tumor | Japan Ministry of Health, Labour and Welfare approved drug for the treatment of prostate cancer patients with distant metastasis | 5/29/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (fam-trastuzumab deruxtecan-nxki) | ERBB2 tyrosine kinase receptor inhibitor; topoisomerase I inhibitor | Non-small-cell lung cancer | FDA granted breakthrough therapy designation to treat metastatic disease in people whose tumors have HER2 mutation and disease progressed on or after platinum-based therapy | 5/18/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | HER2-directed antibody-drug conjugate | Gastric cancer, including gastroesophageal junction cancer | FDA granted orphan designation | 5/22/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., Merck & Co. Inc., of Kenilworth, N.J. and Foundation Medicine Inc., of Cambridge, Mass. | Lynparza (olaparib) | Homologous recombination repair gene-mutated metastatic castration-resistant prostate cancer | PARP inhibitor | FDA approved the expanded indication for the drug and the Foundationone companion diagnostic | 5/20/20 | Cancer |
| Astrazeneca plc, of Dublin, and Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | HER2-directed antibody-drug conjugate | Gastric or gastroesophageal junction adenocarcinoma | FDA granted breakthrough therapy designation for treatment of patients with HER2-positive unresectable or metastatic disease who have received 2 or more prior regimens, including trastuzumab | 5/11/20 | Cancer |
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Mantle cell lymphoma | Submitted a new drug application in Israel seeking approval for use in patients who have received at least one prior therapy | 5/21/20 | Cancer |
| Bexion Pharmaceuticals Inc., of Covington, Ky. | BXQ-350 | Composed of lysosomal activator protein Saposin C and phosphatidylserine | Malignant glioma | FDA granted orphan designation, including for use in diffuse intrinsic pontine glioma | 5/7/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Avapritinib (Ayvakit) | KIT tyrosine kinase inhibitor | Gastrointestinal stromal tumor | FDA issued CRL for NDA seeking to treat adults with unresectable or metastatic fourth-line disease; company halting development in GIST indications other than already approved PDGFRA exon 18 mutant disease | 5/15/20 | Cancer |
| Bridge Biotherapeutics Inc., of Seongnam, South Korea | BBT-176 | Epidermal growth factor receptor tyrosine kinase inhibitor | Advanced non-small-cell lung cancer | Korea's Ministry of Food and Drug Safety cleared the IND for a 90-patient phase I/II study designed to find the maximum tolerable dose, recommended phase II dose and to observe safety, tolerability and the antitumor efficacy in the phase I portion; phase II includes an expansion cohort measuring safety and efficacy | 5/7/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Lisocabtagene maraleucel | CAR T targeting CD19 | Relapsed or refractory large B-cell lymphoma after at least 2 prior therapies | FDA extended the PDUFA date to Nov. 16, 2020, after company submitted an additional data package that was deemed a major amendment | 5/6/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Pomalyst (pomalidomide) | TNF-alpha inhibitor | Kaposi sarcoma | FDA granted accelerated approval to treat AIDS-related disease resistant to HAART or disease in individuals who are HIV-negative | 5/15/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) + Yervoy (ipilimumab) | PD-1 inhibitor; CTLA4 inhibitor | Metastatic non-small-cell lung cancer | FDA approved combination as first-line treatment of adults whose tumors express PD-L1 (?1%) with no EGFR or ALK genomic aberrations | 5/15/20 | Cancer |
| Bristol Myers Squibb Co., of New York | CC-486 | Cytidine nucleoside analogue | Maintenance treatment of adult patients in remission with acute myeloid leukemia | FDA accepted the NDA and granted the application priority review; a PDUFA date is set for Sept. 3, 2020 | 5/1/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. | Idecabtagene vicleucel (ide-cel; BB-2121) | APRIL receptor modulator |
Multiple myeloma | FDA issued RTF on BLA, citing need for additional details on CMC module | 5/13/20 | Cancer |
| Bristol Myers Squibb Co., of Princeton, N.J. | CC-486 | Cytidine nucleoside analogue | Acute myeloid leukemia | EMA validated MAA seeking approval for use in maintenance treatment of adults who achieved complete remission (CR) or CR with incomplete blood count recovery following induction therapy, with or without consolidation treatment, and who are not candidates for, or who choose not to proceed to, hematopoietic stem cell transplantation | 5/22/20 | Cancer |
| Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) plus Yervoy (ipilimumab) | PD-1-targeting antibody plus CTLA4-targeting antibody | Metastatic or recurrent non-small-cell lung cancer | Approved by FDA for use in combination with 2 cycles of platinum-doublet chemotherapy for first-line treatment of adults whose disease has no EGFR or ALK genomic tumor aberrations; therapy is approved for patients with squamous or nonsquamous disease and regardless of PD-L1 expression; application reviewed under FDA’s real-time oncology review pilot program | 5/26/20 | Cancer |
| Bristol Myers Squibb Co., of Princeton, N.J., and Bluebird Bio Inc., of Cambridge, Mass. | Idecabtagene vicleucel (bb-2121) | BCMA-directed CAR T-cell immunotherapy | Multiple myeloma | EMA validated MAA submission seeking approval for use in adults who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody; application as accelerated assessment status | 5/22/20 | Cancer |
| Canbridge Pharmaceuticals Inc., of Beijing and Shanghai | Nerlynx (neratinib) | Kinase inhibitor binding to HER2, HER4 and EGFR | Breast cancer | China’s NMPA granted marketing approval for use in adults with early stage HER2-positive disease following adjuvant trastuzumab-based therapy | 5/7/20 | Cancer |
| Cardiff Oncology Inc., of San Diego | Onvansertib | Polo-like kinase 1 inhibitor | Metastatic colorectal cancer | FDA granted fast track designation to treat individuals with KRAS-mutated disease | 5/28/20 | Cancer |
| Carsgen Therapeutics Co. Ltd., of Shanghai | CAR-CLD18 T-cell therapy | Claudin 18 modulator | Advanced gastric or pancreatic cancer | FDA cleared IND for open-label phase Ib trial expected to begin in third quarter of 2020 | 5/18/20 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | Rubraca (rucaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | Approved by FDA under accelerated approval for the treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated disease who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy | 5/15/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Trastuzumab deruxtecan (DS-8201) | HER2-directed antibody-drug conjugate | Metastatic gastric cancer | Submitted supplemental NDA to Japan’s Ministry of Health, Labour and Welfare seeking approval for treatment of patients with HER2-positive disease | 5/7/20 | Cancer |
| Dcprime BV, of Leiden, the Netherlands | DCP-001 | Allogeneic dendritic cell vaccine | Acute myeloid leukemia | FDA accepted IND application to expand ongoing phase II Advance-II trial into U.S. in individuals with disease who are ineligible for hematopoietic stem cell transplantation | 5/13/20 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Qinlock (ripretinib) | Kinase inhibitor | Advanced gastrointestinal stromal tumor | FDA approved as fourth-line treatment, indicated for adults who who have received prior treatment with 3 or more kinase inhibitor therapies, including imatinib | 5/15/20 | Cancer |
| Diverse Biotech Inc., of Doylestown, Pa. | Unnamed therapy | Therapeutic using cannabidiol as a core component | Newly diagnosed glioblastoma | FDA granted orphan drug designation | 5/5/20 | Cancer |
| Eli Lilly and Co., of Indianapolis | Retevmo (selpercatinib) | Selective RET kinase inhibitor | Non-small-cell lung cancer and medullary thyroid cancer | FDA approved under the accelerated pathway for treatment of adults with metastatic RET fusion-positive NSCLC and for treatment of adult and pediatric patients, 12 and older, with advanced or metastatic RET-mutant MTC who require systemic therapy, or advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) | 5/8/20 | Cancer |
| Eli Lilly and Co., of Indianapolis | Cyramza (ramucirumab) | Monoclonal antibody targeting VEGF receptor | First-line metastatic non-small-cell lung cancer | FDA approved the drug in combination with Tarceva (erlotinib) | 5/29/20 | Cancer |
| Essa Pharmaceuticals Inc., of Vancouver, British Columbia | EPI-7386 | Inhibits the N-terminal domain of the androgen receptor | Prostate cancer | FDA has notified the company that it may proceed with its proposed clinical investigation; Essa previously announced on March 30, 2020, that it had filed an IND application | 5/1/20 | Cancer |
| Fate Therapeutics Inc., of San Diego | FT-538 | CRISPR-edited, iPSC-derived natural killer cell immunotherapy | Acute myeloid leukemia and multiple myeloma | FDA cleared the IND for a phase I study testing FT-538 as a monotherapy for AML and in combination with daratumumab for MM | 5/20/20 | Cancer |
| Genelux Corp., of San Diego | Olvi-Vec (olvimulogene nanivacirepvec) | Oncolytic virus | Ovarian cancer | Type C meeting with FDA provided guidance for phase III registrational trial in patients with platinum-refractory/resistant disease; company plans to meet with FDA after lock of phase II Viro-15 study in the fourth quarter of 2020, with plans to start phase III in 2021 | 5/7/20 | Cancer |
| Genentech Inc., a unit of Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | Monoclonal antibody targeting PD-L1 | Unresectable or metastatic hepatocellular carcinoma | FDA approved the drug in combination with Avastin (bevacizumab) | 5/29/20 | Cancer |
| Genentech Inc., of South San Francisco, a member of the Roche Group | Tecentriq (atezolizumab) | PD-L1 inhibitor | Metastatic non-small-cell lung cancer | Approved by FDA for use in treating adults whose tumors have high PD-L1 expression, as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations | 5/18/20 | Cancer |
| Genmab A/S, of Copenhagen, and Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex Faspro (daratumumab and hyaluronidase-fihi) | Subcutaneous CD38-directed antibody | Multiple myeloma | FDA approved BLA for subcutaneous formulation for treatment of adults: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant (ASCT); in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least 1 prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least 1 prior therapy; and as monotherapy, in patients who have received at least 3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent | 5/1/20 | Cancer |
| Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong | Savolitinib | MET inhibitor | Non-small-cell lung cancer | NDA for NSCLC with MET exon 14 skipping mutations accepted for review by China's NMPA | 5/29/20 | Cancer |
| I-Mab Biopharma Co. Ltd., of Shanghai, and Genexine Co. Ltd., of Seoul, South Korea | TJ-107/Hyleukin-7 (efineptakin alfa) | Long-acting recombinant human interleukin-7 | Glioblastoma multiforme | China’s NMPA cleared initiation of phase II trial in lymphopenic patients with newly diagnosed disease | 5/21/20 | Cancer |
| Immodulon Therapeutics Ltd., of Uxbridge, U.K. | IMM-101 | Heat-killed mycobacterial immunomodulator | Advanced sarcoma | FDA approved IND for phase I/II combination study with standard-of-care chemotherapy and checkpoint inhibitor in up to 42 individuals with advanced leiomyosarcoma or dedifferentiated liposarcoma; efficacy endpoints include overall and progression-free survival | 5/28/20 | Cancer |
| Immune-Onc Therapeutics, Inc., of Palo Alto, Calif. | IO-202 | Leukocyte Ig-like receptor-4 antagonist | Acute myeloid leukemia; chronic myelomonocytic leukemia | FDA approved IND for phase I dose-escalation study in AML patients with monocytic differentiation and CMML patients | 5/29/20 | Cancer |
| Immunicum AB, of Stockholm | Ilixadencel | Cell-based, off-the-shelf immune primer | Metastatic renal cell carcinoma | FDA granted regenerative medicine advanced therapy designation | 5/6/20 | Cancer |
| Incyte Corp., of Wilmington, Del., and Morphosys AG, of Martinsried, Germany | Tafasitamab | Anti-CD19 antibody | Relapsed or refractory diffuse large B-cell lymphoma | EMA validated the MAA seeking approval for use in combination with lenalidomide, followed by tafasitamab monotherapy, for treating adults, including those whose DLBCL arises from low-grade lymphoma, who are not candidates for autologous stem cell transplantation | 5/20/20 | Cancer |
| Interna Technologies BV, of Utrecht, The Netherlands | INT-1B3 | miR-193a-3p mimic | Advanced solid tumors | Received regulatory approvals in the Netherlands and Belgium for an 80-patient phase I study | 5/6/20 | Cancer |
| Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Monoclonal antibody targeting PD-1 | Second-line, locally advanced or metastatic urothelial carcinoma | China National Medical Products Administration accepted the supplemental NDA | 5/7/20 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | Oral selective inhibitor of nuclear transport | Multiple myeloma | Submitted a supplemental NDA to FDA seeking approval in patients with previously treated disease | 5/20/20 | Cancer |
| Kiadis Pharma NV, of Amsterdam | K-NK002 | Natural killer cell therapy | Blood cancer patients undergoing haploidentical hematopoietic stem cell transplant | FDA approved start of the NK-REALM phase II study | 5/6/20 | Cancer |
| Lidds AB, of Uppsala, Sweden | Liproca Depot |
Androgen receptor antagonist | Prostate cancer | Swedish MPA provided positive response on proposed design of phase III study in individuals with localized disease, within intermediate risk group; primary endpoint is time to disease progression, prompting radical treatment, over 24 months for study drug vs. active surveillance | 5/18/20 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Margetuximab | ERBB-2 tyrosine kinase receptor inhibitor | Metastatic breast cancer | During mid-cycle communication, FDA disclosed that it is no longer planning to hold advisory committee meeting to discuss BLA, reconfirming Dec. 18, 2020, PDUFA date | 5/28/20 | Cancer |
| Mateon Therapeutics Inc., of Agoura Hills, Calif. | CA4P | Fosbretabulin; a vascular-disrupting agent | Stage IIB-IV melanoma due to genetic mutations that disproportionately affect pediatric patients | FDA granted rare pediatric disease designation | 5/5/20 | Cancer |
| Medivir AB, of Stockholm | MIV-818 | Nucleotide DNA polymerase inhibitor | Hepatocellular carcinoma | FDA granted orphan drug designation | 5/6/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J., and Astrazeneca plc, of Cambridge, U.K. | Lynparza (olaparib) | PARP inhibitor | Advanced epithelial ovarian, fallopian tube or primary peritoneal cancer | FDA approved for use in combination with bevacizumab as first-line maintenance treatment of adults who are in complete or partial remission to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency-positive status defined by either a deleterious or suspected deleterious BRCA mutation, and/or genomic instability | 5/8/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Designed to destroy tumors when activated by radiotherapy | Pancreatic cancer | FDA deems phase I trial safe to proceed | 5/6/20 | Cancer |
| Nordic Nanovector ASA, of Oslo, Norway | Betalutin (177Lu lilotomab satetraxetan) | Antibody-radionuclide conjugate | Marginal zone lymphoma | Received positive opinion from EMA on orphan designation | 5/14/20 | Cancer |
| Novartis AG, of Basel, Switzerland | Tabrecta (capmatinib, formerly INC-280) | Selective MET inhibitor | Non-small-cell lung cancer | Approved by FDA for use in patients whose tumors have a mutation that leads to MET exon 14 skipping as detected by an FDA-approved test; approval granted under accelerated pathway based on overall response rate and duration of response | 5/6/20 | Cancer |
| Novartis AG, of Basel, Switzerland | Piqray (alpelisib) | Kinase inhibitor | Metastatic breast cancer | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval | 5/29/20 | Cancer |
| Novocure Ltd., of St. Helier, Jersey, U.K., and Zai Lab Ltd., of Shanghai | Optune | Device delivering tumor treating fields | Glioblastoma | China NMPA approved MAA in combination with temozolomide to treat newly diagnosed disease and as monotherapy to treat recurrent disease | 5/13/20 | Cancer |
| Onxeo SA, of Paris | Asidna | DDR inhibitor | Relapsed ovarian cancer | French National Agency approved phase Ib/II study | 5/29/20 | Cancer |
| Pierre Fabre SA, of Castres, France | Braftovi | Encorafenib | BRAFV600E-mutant metastatic colorectal cancer | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for Braftovi in combination with cetuximab for the treatment of adult patients | 5/1/20 | Cancer |
| Qurient Co. Ltd., of Seongnam-Si, Korea | Q-702 | Targets Axl, Mer and CSF1 receptor tyrosine kinases | Advanced solid tumors | FDA cleared IND for a phase I study scheduled to start in the third quarter of 2020 | 5/26/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Rozlytrek (entrectinib) | Targets NTRK1/2/3 or ROS1 gene fusions | Solid tumors | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval | 5/29/20 | Cancer |
| Sapience Therapeutics Inc., of Harrison, N.Y. | ST-101 | Peptide therapy | Solid tumors | U.K.’s Medicines and Healthcare products Regulatory Agency accepted the CTA for phase I/II study in adults with unresectable and metastatic disease who are not eligible for other therapies or have progressed on prior therapies | 5/7/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Tukysa (tucatinib) | Oral, small-molecule tyrosine kinase inhibitor of HER2 | Metastatic HER2-positive breast cancer | Swissmedic granted approval for Tukysa tablets in combination with trastuzumab and capecitabine, for the treatment of patients who have previously received 2 or more anti-HER2 regimens in any setting | 5/12/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Adcetris (brentuximab vedotin) | Antibody-drug conjugate directed to CD30 | Systemic anaplastic large-cell lymphoma | European Commission extended conditional marketing authorization to include treatment of adults with previously untreated disease, in combination with CHP (cyclophosphamide, doxorubicin, prednisone) | 5/14/20 | Cancer |
| Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | Wilms tumor protein modulator | Acute myeloid leukemia | Independent data monitoring committee formed to monitor pivotal phase III Regal trial | 5/18/20 | Cancer |
| Sesen Bio Inc., of Cambridge, Mass. | Vicinium (VB4-845) | Recombinant fusion protein targeting epithelial cell adhesion molecule antigens to deliver pseudomonas exotoxin A | High-risk, BCG-unresponsive non-muscle invasive bladder cancer | Scientific advice from EMA's Committee for Medicinal Products for Human Use suggests the data required for approval can be addressed with the completed phase III data; plans to file an MAA in early 2021 | 5/7/20 | Cancer |
| Sino Biopharmaceutical Ltd., of Hong Kong, and Akeso Inc., of Zhongshan, China | Penpulimab | Monoclonal antibody targeting PD-1 | Relapsed or refractory classical Hodgkin lymphoma | National Medical Products Administration of China accepted the NDA | 5/25/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Alunbrig (brigatinib) | ALK inhibitor | First-line ALK+ metastatic non-small-cell lung cancer | FDA approved the expanded indication | 5/22/20 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX591-CDx | 68 Ga-PSMA-11 injection | Prostate cancer imaging | Submitted an MAA in the EU | 5/1/20 | Cancer |
| Theralase Technologies Inc., of Toronto | TLD-1433 | Light-activated photodynamic compound | Non-muscle invasive bladder cancer | FDA granted IND authorization to conduct a phase II study in the U.S. | 5/19/20 | Cancer |
| Tolero Pharmaceuticals Inc., of Salt Lake City | TP-1454 | Small-molecule PKM2 activator | Advanced metastatic or progressive solid tumors | Submitted an IND application to the FDA | 5/19/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Single-domain antibody against PD-L1 | Soft tissue sarcoma, subtypes of undifferentiated pleomorphic sarcoma and myxofibrosarcoma | Completed type B pre-IND meeting with FDA, which agreed with company’s proposals for key elements of pivotal Envasarc trial; enrollment expected to start in second half of 2020 | 5/11/20 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif., and Cytokinetics Inc., of South San Francisco | Omecamtiv mecarbil | Cardiac myosin activator | Chronic heart failure with reduced ejection fraction | FDA granted fast track designation | 5/8/20 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | Sodium glucose co-transporter 2 inhibitor | Heart failure with reduced ejection fraction with and without type 2 diabetes | FDA approved the expanded indication | 5/6/20 | Cardiovascular |
| Pharmazz Inc., of Willowbrook, Ill. | Lyfaquin (centhaquine) | Hypotensive drug | Hypovolemic shock | Indian regulators granted marketing authorization | 5/14/20 | Cardiovascular |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Takhzyro (lanadelumab) | Fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity | Hereditary angioedema | EMA's Committee for Medicinal Products for Human Use adopted positive opinion recommending approval | 5/29/20 | Cardiovascular |
| Akari Therapeutics plc, of New York | Nomacopan | C5 complement inhibitor | Bullous pemphigoid | EMA issued a positive opinion on application for orphan status | 5/21/20 | Dermatologic |
| Azitra Inc., of Branford, Conn. | ATR-12 | Strain of Staphylococcus epidermidis engineered to express therapeutic levels of LEKTI protein | Netherton syndrome | FDA granted rare pediatric disease designation | 5/27/20 | Dermatologic |
| Cabaletta Bio Inc., of Philadelphia | DSG3-CAART | Targets B cells that express pathogenic anti-DSG3 autoantibodies | Mucosal pemphigus vulgaris | FDA granted fast track designation | 5/6/20 | Dermatologic |
| Histogen Inc., of San Diego | HST-001 | Hair stimulating complex | Androgenic alopecia in men | FDA approved the IND for a phase Ib/IIa trial, expected to start in the second quarter of 2020 | 5/11/20 | Dermatologic |
| Menlo Therapeutics Inc., of Bridgewater, N.J. | Zilxi (minocycline, formerly Foamix) | Tetracycline antibiotic | Rosacea | Approved by FDA | 5/29/20 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Monoclonal antibody targeting IL-4 and IL-13 | Moderate to severe atopic dermatitis | FDA approved use in children, ages 6 to 11, whose disease is not adequately controlled with topical prescription therapies or when those therapies are not available | 5/26/20 | Dermatologic |
| Regenerx Biopharmaceuticals Inc., of Rockville, Md. | Thymosin beta 4 | Promotes corneal would healing and modulates inflammatory mediator | Tissue protection and repair | Posted information on its website that FDA reclassified thymosin beta 4 from a drug to a biologic | 5/27/20 | Dermatologic |
| Twi Biotechnology Inc., of Taipei, Taiwan | AC-1101 | Topical JAK inhibitor | Vitiligo | Received CTA approval from Health Canada for a phase I trial in healthy volunteers | 5/7/20 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | RNAi targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | FDA accepted the NDA and granted a priority review with a PDUFA action date of Dec. 3, 2020 | 5/26/20 | Endocrine/Metabolic |
| Chiesi Global Rare Diseases, of Boston, a unit of Chiesi Farmaceutici SpA | Ferriprox (deferiprone) | Iron chelator | Transfusional iron overload due to thalassemia syndromes | Approved by FDA as twice-a-day tablets for use when current chelation therapy is inadequate | 5/21/20 | Endocrine/Metabolic |
| Cyclo Therapeutics Inc., of Gainesville, Fla. | Trappsol Cyclo | Cholesterol binding agent | Niemann-Pick disease type C1 | EMA provided final advice letter with positive feedback on proposed pivotal trial, including feedback from Committee for Orphan Medicinal Products that proposed development plan is sufficient to demonstrate benefit | 5/18/20 | Endocrine/Metabolic |
| Freeline Therapeutics Ltd., of London | FLT-190 | Liver-directed adeno-associated viral gene therapy expressing alpha-galactosidase A | Fabry disease | FDA granted orphan drug designation | 5/4/20 | Endocrine/Metabolic |
| Neurobo Pharmaceuticals Inc., of Boston | Gemcabene | Peroxisome proliferation-activated receptor agonist | Dyslipidemia | After reviewing an amendment to the FDA's partial hold letter, the FDA declined to lift the hold on the phase II study | 5/26/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBGM-01 | AAV-delivery gene therapy | GM1 gangliosidosis | FDA granted rare pediatric disease designation | 5/21/20 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, unit of Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa | Alpha-galactosidase stimulator | Fabry disease | Submitted BLA to FDA via accelerated approval pathway | 5/28/20 | Endocrine/Metabolic |
| PTC Therapeutics Inc., of South Plainfield, N.J. | Gene therapy | Gene therapy | Aromatic L-amino acid decarboxylase deficiency | Expects to file BLA with FDA in second half of 2020, a delay due to COVID-19 | 5/1/20 | Endocrine/Metabolic |
| Sorrento Therapeutics Inc., of San Diego | STI-6129 | CD38-targeting antibody-drug conjugate | Advanced relapsed and/or refractory systemic amyloid light chain amyloidosis | FDA cleared IND for a phase I study | 5/26/20 | Endocrine/Metabolic |
| Tolmar Pharmaceuticals Inc., of Buffalo Grove, Ill. | Fensolvi (leuprolide acetate) | Gonadotropin-releasing hormone receptor agonist | Central precocious puberty | FDA approved the NDA | 5/4/20 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | Dasiglucagon Hypopal rescue pen | Peptide analogue of human glucagon | Severe hypoglycemia | Filed NDA with FDA | 5/14/20 | Endocrine/Metabolic |
| Zealand Pharma A/S, of Copenhagen | Hypopal (dasiglucagon) | Glucagon analogue | Hypoglycemia | FDA accepted the NDA and assigned a PDUFA action date of March 27, 2021 | 5/22/20 | Endocrine/Metabolic |
| 9 Meters Biopharma Inc., of Raleigh, N.C. | Larazotide acetate | Tight junction regulator | Celiac disease | Received a thorough QT study waiver from FDA’s Center for Drug Evaluation and Research | 5/11/20 | Gastrointestinal |
| Ascletis Pharma Inc., of Hangzhou, China | ASC-41 | Thyroid hormone receptor beta agonist | Nonalcoholic steatohepatitis | China NMPA approved IND for phase I trial | 5/13/20 | Gastrointestinal |
| Bird Rock Bio Inc., of La Jolla, Calif. | Nimacimab (RYI-018) | Cannabinoid CB1 receptor antagonist | Renal disease | IND filed with FDA to initiate phase II trial in individuals with diabetic kidney disease, focal segmental glomerulosclerosis or IgA nephropathy | 5/28/20 | Gastrointestinal |
| Evoke Pharma Inc., of Solana Beach, Calif. | Gimoti | Nasal spray formulation of metoclopramide, which inhibits dopamine D2 and serotonin 5-HT3 receptors | Diabetic gastroparesis | FDA conditionally accepted the proprietary brand name | 5/20/20 | Gastrointestinal |
| Galectin Therapeutics Inc., of Norcross, Ga. | Belapectin (GR-MD-02) | Galectin-3 inhibitor | Prevention of esophageal varices in patients with nonalcoholic steatohepatitis cirrhosis | Submitted to the FDA its protocol for a seamless adaptively designed phase IIb/III study, the NASH-RX trial, evaluating the safety and efficacy of belapectin | 5/1/20 | Gastrointestinal |
| Intercept Pharmaceuticals Inc., of New York | Obeticholic acid | Farnesoid X receptor agonist | Liver fibrosis due to nonalcoholic steatohepatitis | FDA said tentatively scheduled June 9, 2020, advisory committee related to NDA has been postponed to accommodate review of additional data requested by agency that company intends to submit within the next week; company now anticipates FDA review will extend beyond PDUFA date of June 26, 2020 | 5/22/20 | Gastrointestinal |
| Lipocine Inc., of Salt Lake City | LPCN-1148 | Oral prodrug of bioidentical testosterone | Liver cirrhosis | FDA cleared the IND application for phase II proof-of-concept study in adult male cirrhotic patients | 5/5/20 | Gastrointestinal |
| Protara Therapeutics Inc., of New York | Intravenous choline chloride | Phospholipid substrate replacement therapy | Intestinal failure-associated liver disease | FDA granted fast track designation | 5/27/20 | Gastrointestinal |
| Scimount Pharmatech Co. Ltd., of Chengdu, China | SMP-100 | Selective serotonin receptor-3 partial agonist | Irritable bowel syndrome with diarrhea | FDA approved IND for a phase I trial in healthy volunteers | 5/13/20 | Gastrointestinal |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Entyvio (vedolizumab) | Monoclonal antibody targeting alpha4beta7 integrin | Moderately to severely active ulcerative colitis or Crohn’s disease | European Commission granted marketing authorization for the subcutaneous formulation | 5/7/20 | Gastrointestinal |
| Abbvie Inc., of North Chicago | Oriahnn (elagolix, estradiol and norethindrone; elagolix) | Hormones | Heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women | FDA approved the drug | 5/29/20 | Genitourinary/Sexual Function |
| Aspargo Laboratories Inc., of Englewood Cliffs, N.J. | Sildenafil oral spray | Phosphodiesterase type 5 inhibitor | Erectile dysfunction | During a pre-IND meeting, the FDA was supportive of a single-dose bioequivalent study comparing the drug to Viagra (sildenafil, Pfizer Inc.) with approval through the 505(b)(2) regulatory pathway; plans to file the IND in the third quarter of 2020 | 5/4/20 | Genitourinary/Sexual Function |
| Astellas Pharma Inc., of Tokyo | Vesicare LS (solifenacin) | Muscarinic M3 receptor antagonist | Neurogenic detrusor overactivity | FDA approved oral suspension formulation to treat form of bladder dysfunction related to neurological impairment in children 2 and older | 5/26/20 | Genitourinary/Sexual Function |
| Astellas Pharma Inc., of Tokyo, and Fibrogen Inc., of San Francisco | Roxadustat | Inhibitor of hypoxia-inducible factor prolyl¬ hydroxylase | Anemia in chronic kidney disease | Marketing authorization application accepted by EMA for regulatory review | 5/20/20 | Genitourinary/Sexual Function |
| Evofem Biosciences Inc., of San Diego | Phexxi vaginal gel | Regulates vaginal pH | Contraception | Approved by FDA | 5/22/20 | Genitourinary/Sexual Function |
| Ferring Pharmaceuticals SA, of Saint-Prex, Switzerland | Carbetocin Ferring (heat-stable formulation) | Oxytocin receptor agonist | Uterine hemorrhage | Swissmedic approved drug under procedure for scientific advice and Marketing Authorization for Global Health Products to prevent excessive bleeding after childbirth | 5/13/20 | Genitourinary/Sexual Function |
| Rockwell Medical Inc., of Wixom, Mich. | Triferic Avnu (ferric pyrophosphate citrate) | Iron replacement | Hemodialysis-dependent chronic kidney disease | Filed a new drug submission with Health Canada | 5/26/20 | Genitourinary/Sexual Function |
| Akebia Therapeutics Inc., of Cambridge, Mass. | Riona (ferric citrate) | Iron-based phosphate binder | Iron deficiency anemia | Collaboration partner Japan Tobacco Inc. filed sNDA with PMDA to add indication in Japan | 5/18/20 | Hematologic |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | Monoclonal antibody targeting C5 | Atypical hemolytic uremic syndrome | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion, recommending marketing authorization for patients with a body weight of 10 kg or above who are complement inhibitor treatment-naïve or have received Soliris (eculizumab) for at least 3 months and have evidence of response to eculizumab | 5/1/20 | Hematologic |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Synthetic cyclic peptide | Paroxysmal nocturnal hemoglobinuria | Completed pre-NDA meeting with FDA; plans to submit NDA in the second half of the year based on data from Pegasus trial | 5/21/20 | Hematologic |
| Bristol Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma Inc., of Cambridge, Mass. | Reblozyl | Luspatercept | Adults with anemia in beta thalassemia and myelodysplastic syndromes | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion, which will now be reviewed by the EC | 5/1/20 | Hematologic |
| Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Boston | CTX-001 | Autologous, gene-edited hematopoietic stem cell therapy | Sickle cell disease and transfusion-dependent beta-thalassemia | FDA granted regenerative medicine advanced therapy designation | 5/11/20 | Hematologic |
| Expression Therapeutics LLC, of Atlanta | ET-3 | Gene therapy expressing factor VIII | Hemophilia A | FDA cleared IND for a phase I study | 5/26/20 | Hematologic |
| Fresenius Kabi AG, of Bad Homburg, Germany | MSB-11455 | Pegfilgrastim biosimilar to Neulasta | Neutropenia | FDA accepted the BLA for review | 5/27/20 | Hematologic |
| Geron Corp., of Menlo Park, Calif. | Imetelstat | Telomerase inhibitor | Myelofibrosis | Following type C meeting with FDA, company plans phase III study in patients refractory to JAK inhibitor treatment; study to enroll about 320 patients with intermediate-2 or high-risk disease, with overall survival (OS) as primary endpoint; in interim analysis meets prespecified OS criterion, company expects data may support registration in refractory MF; trial expected to open for screening and enrollment in first quarter of 2021 | 5/22/20 | Hematologic |
| Glaxosmithkline plc, of London | Nucala (mepolizumab) | Monoclonal antibody targeting IL-5 | Hypereosinophilic syndrome | FDA granted priority review for the application seeking approval | 5/27/20 | Hematologic |
| Hanmi Pharmaceutical Co. Ltd., of South Korea | Rolontis (eflapegrastim) | G-CSF analogue | Neutropenia | Filed a new drug approval application with the Ministry of Food and Drug Safety | 5/11/20 | Hematologic |
| Aurinia Pharmaceutical Inc., of Victoria, British Columbia | Voclosporin | Calcineurin inhibitor | Lupus nephritis | Completed rolling submission of the NDA to the FDA with a request for a priority review | 5/26/20 | Immune |
| Bristol Myers Squibb Co., of Princeton, N.J. | Zeposia (ozanimod) | S1P receptor modulator | Relapsing-remitting multiple sclerosis | Approved by European Commission for use in adults with active disease as defined as clinical or imaging features | 5/27/20 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Imlifidase | Cleaves IgG-antibodies | Sensitized kidney transplant patients | Company submitted responses to outstanding questions from the EMA; CHMP decision expected at the June 22-25, 2020, meeting | 5/25/20 | Immune |
| Roche Holding AG, of Basel, Switzerland | Ocrevus (ocrelizumab) | B-lymphocyte antigen CD20 inhibitor |
Multiple sclerosis | EMA approved shorter 2-hour infusion time, dosed twice yearly, to treat relapsing or primary progressive disease | 5/28/20 | Immune |
| Stallergenes Greer plc, of London | STAGR-320 | Sublingual house dust mite immunotherapy | HDM-induced allergic rhinitis | Submitted MAA via a European decentralized procedure | 5/4/20 | Immune |
| Theraly Fibrosis Inc., of Germantown, Md. | TLY-012 | TRAIL receptor modulator | Systemic sclerosis | FDA granted orphan drug designation in the indication | 5/28/20 | Immune |
| AB Science SA, of Paris | Masitinib | Tyrosine kinase inhibitor | Moderate and severe COVID-19 | Gained authorization from French Medicine Agency, ANSM, and started 200-patient study testing drug in combination with isoquercetin | 5/6/20 | Infection |
| Abivax SA, of Paris | ABX-464 | Small-molecule antiviral | COVID-19 | Received clearance from National Agency for the Safety of Medicines (ANSM) and French Ethics Committee to initiate phase IIb/III trial to prevent severe inflammation that leads to acute respiratory distress syndrome in 1,034 COVID-19 elderly or high-risk patients (miR-AGE trial) | 5/14/20 | Infection |
| Abivax SA, of Paris | ABX-464 | Up-regulates miR-124 | Elderly or high-risk patients with COVID-19 | German regulators Bundesinstitut für Arnzeimittel und Medizinprodukte approved the start of the phase IIb/III study in 1,034 patients | 5/25/20 | Infection |
| Aim Immunotech Inc., of Ocala, Fla. | Ampligen (rintatolimod) | Immunomodulatory drug | Cancer patients with COVID-19 | FDA authorized phase I/IIb trial assessing combination with interferon alfa-2b | 5/14/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (repurposed ifenprodil) | NMDA receptor epsilon 2 subunit inhibitor | COVID-19 infection | Request for ethics approval of multinational phase IIb/III study submitted in Australia | 5/15/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | Ifenprodil | N-methyl-D-aspartate receptor antagonist | COVID-19 hospitalized patients | Submitted IND to FDA for a phase IIb/III study measuring mortality, blood oxygen levels, time spent in intensive care and time to mechanical ventilation | 5/26/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (repurposed ifenprodil) | NMDA receptor antagonist | COVID-19 infection | IND application submitted to FDA for phase IIb/III study | 5/27/20 | Infection |
| Amivas (USA) LLC, of Frederick, Md. | Intravenous artesunate | Artemisinin derivative | Severe malaria | Approved by FDA for use in adult and pediatric patients | 5/26/20 | Infection |
| Anivive Lifesciences Inc., of Long Beach, Calif. | GC-376 | Protease inhibitor | COVID-19 infection | Pre-IND request filed with FDA for human trial with repurposed veterinary drug | 5/26/20 | Infection |
| Antabio SAS, of Labege, France | MEM-ANT-3310 | Meropenem plus broad-spectrum serine beta-lactamase inhibitor ANT-3310 | Complicated urinary tract infections, hospital-acquired bacterial pneumonia, ventilator-associated bacterial pneumonia and complicated intra-abdominal infections | FDA granted qualified infectious disease product designation for the 4 indications | 5/6/20 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Favipiravir | RNA synthesis inhibitor | Prevention of COVID-19 | Filed CTA with Health Canada for phase II study | 5/11/20 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Favipiravir | Broad-spectrum antiviral | COVID-19 | Health Canada cleared phase II study testing drug from Fujifilm Toyama Chemical as preventive measure against COVID-19 outbreaks; recruitment expected to begin imminently | 5/21/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide that inhibits DPEP-1 | Organ inflammation in COVID-19 patients | Health Canada issued a no objection letter to the start of a 60-patient phase II study with the primary endpoint of frequency of acute respiratory distress syndrome; secondary endpoint is frequency of kidney inflammation and injury | 5/8/20 | Infection |
| Atea Pharmaceuticals Inc., of Boston | AT-527 | Nucleotide prodrug | Hospitalized patients with moderate COVID-19 | FDA cleared the IND for a phase II study | 5/20/20 | Infection |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland | Ceftobiprole | Penicillin binding protein inhibitor | Staphylococcus aureus bacteremia infection | FDA approved protocol amendment enabling pivotal phase III Eradicate study of intravenous (I.V.) ceftobiprole vs. I.V. daptomycin, with or without I.V. aztreonam, to advance to pre-planned second cohort and to extend maximum treatment duration from 4 to up to 6 weeks; overall target enrollment (n=390) unchanged | 5/28/20 | Infection |
| Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Inhaled nitric oxide therapy | COVID-19 | FDA accepted the IND for a phase III study in up to 500 patients infected with COVID-19 | 5/11/20 | Infection |
| Beyond Air Inc., of Garden City, N.Y. | Lungfit | Inhaled nitric oxide | COVID-19 | Health Canada approved use for a study in hospitalized patients diagnosed with COVID-19 | 5/26/20 | Infection |
| Biosig Technologies Inc., of Westport, Conn. | Merimepodib | Inosine monophosphate dehydrogenase inhibitor | COVID-19 infection | FDA cleared IND application from subsidiary Viralclear Pharmaceuticals Inc. for phase II study in adults hospitalized with infection who require supplemental oxygen or are on non-invasive ventilation or high flow oxygen devices | 5/18/20 | Infection |
| Calcimedica Inc., of La Jolla, Calif. | Auxora (CM-4620-IE) | Selective small-molecule CRAC channel inhibitor | COVID-19 pneumonia | Received guidance from FDA recommending transition from current randomized open-label study to a blinded placebo-controlled trial as soon as possible; FDA provided that recommendation following submission of recent interim analysis | 5/21/20 | Infection |
| Combioxin SA, of Geneva | CAL-02 | Liposomes that bind bacterial virulence effectors | COVID-19 at high risk of secondary bacterial infections | FDA reviewed the pre-IND | 5/4/20 | Infection |
| Cynata Therapeutics Ltd., of Melbourne, Australia | Cymerus | Mesenchymal stem cells | COVID-19 patients in intensive care | Received ethics approval for the 12-patient Mend study; recruitment expected to begin once agreements with study centers are finalized | 5/8/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 | Submitted a request to the FDA to grant compassionate use for patients not eligible for 2 ongoing studies | 5/4/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | Adrenergic receptor agonist | HIV infection | Remaining portions of BLA submitted to FDA for combination therapy with HAART | 5/13/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 infection | Company plans to submit protocol to FDA for factorial design trial to compare effectiveness of leronlimab vs. and in combination with remdesivir (Veklury, Gilead Sciences Inc.) | 5/18/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | TSC | Trans sodium crocetinate | COVID-19 | Received accelerated response from FDA regarding pre-IND meeting request seeking guidance for proposed development of TSC in patients displaying severe respiratory symptoms and low oxygen levels; FDA recommended study employ a double-blinded, controlled, randomized design to address wide variability in standard of care and also recommended incorporating, where available, the drug remdesivir, which has been newly approved for emergency use; FDA agreed with the safety and oxygenation marker endpoints proposed by the company for early trials and suggested a range of potential functional outcomes which might be used as primary endpoints in possible later-stage trials supporting approval | 5/26/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | TSC (trans sodium crocetinate) | Vitamin A analogue | Severe respiratory symptoms and low oxygen levels in COVID-19 patients | FDA will accelerate its review of the company’s clinical development plan under the Coronavirus Treatment Acceleration Program | 5/5/20 | Infection |
| Enzychem Lifesciences Inc., of Englewood, N.J. | EC-18 | Small molecule | Preventing acute respiratory distress syndrome due to COVID-19 pneumonia | Korea’s Ministry of Food and Drug Safety accepted the IND for a phase II study | 5/14/20 | Infection |
| Evelo Biosciences Inc., of Cambridge, Mass. | EDP-1815 | Anti-inflammatory agent | Hospitalized COVID-19 patients | Submitted IND to the FDA for a study of 60 patients; primary endpoint is reduced requirements for oxygen therapy; secondary endpoints include symptom duration, progression along the WHO scale of disease severity and mortality; data expected in second half of 2020 | 5/7/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Remdesivir | RNA polymerase modulator | COVID-19 | FDA issued an emergency use authorization | 5/1/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Remdesivir | Nucleotide analogue | COVID-19 | Japan’s Ministry of Health, Labour and Welfare granted regulatory approval of drug, branded Veklury, under exceptional approval pathway | 5/7/20 | Infection |
| Immunic Inc., of New York | IMU-838 | Oral DHODH inhibitor | COVID-19 | Germany’s BfArM (Bundesinstitut für Arzneimittel und Medizinprodukte) cleared firm to initiate phase II trial, expected to enroll 230 patients with moderate COVID-19; top-line data expected later this year | 5/13/20 | Infection |
| Immunitybio Inc., of Culver City, Calif. | Nogapendekin alfa inbakicept | IL-15 drug | COVID-19 | FDA authorized use of drug to treat patients prior to onset of severe disease | 5/27/20 | Infection |
| Johnson & Johnson, of New Brunswick, N.J. | Zabdeno (Ad26.Zebov) + MVABEA (MVA-BN-Filo) | Prime?boost vaccine regimen | Ebola virus infection | EMA's Committee for Medicinal Products for Human Use issued positive opinion | 5/29/20 | Infection |
| Laurent Pharmaceuticals Inc., of Montreal | LAU-7b | Oral formulation of synthetic retinoid fenretinide | COVID-19 | Health Canada approved start of the 200-patient, phase II Resolution study in hospitalized subjects with COVID-19 | 5/6/20 | Infection |
| Moleculin Biotech Inc., of Houston | WP-1122 | 2-deoxy-D-glucose ester-type prodrug | COVID-19 infection | FDA granted request for pre-IND meeting | 5/27/20 | Infection |
| Myr Pharmaceuticals GmbH, of Bad Homburg, Germany | Hepcludex (bulevirtide) | Entry inhibitor | Chronic hepatitis delta virus infection | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending the granting of a conditional marketing authorization | 5/29/20 | Infection |
| Nabriva Therapeutics Inc., of Dublin | Xenleta (lefamulin) | Semisynthetic pleuromutilin antibiotic | Community-acquired pneumonia | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion recommending approval | 5/29/20 | Infection |
| Novan Inc., of Morrisville, N.C. | SB-206 (berdazimer sodium) | Nitric oxide-releasing macromolecule | Molluscum contagiosum infection | Received meeting minutes from the April 1, 2020, type C meeting with the FDA, and based on the guidance, the company is preparing to conduct 1 additional pivotal trial; the FDA provided guidance with regard to the study design and expectations for a future NDA submission | 5/1/20 | Infection |
| Octapharma USA Inc., of Paramus, N.J. | Octagam 10% | Intravenous human immune globulin | COVID-19 with severe disease progression | FDA approved the IND for a phase III study | 5/20/20 | Infection |
| Organicell Regenerative Medicine Inc., of Miami | Organicell Flow | Acellular product derived from amniotic fluid | Moderate to severe acute respiratory syndrome due to COVID-19 infection | FDA approved the IND application | 5/5/20 | Infection |
| Organicell Regenerative Medicine Inc., of Miami | Organicell Flow | Acellular product derived from amniotic fluid | COVID-19 | FDA authorized 2 emergency compassionate use INDs; the severely ill patients received immediate administration of Organicell Flow | 5/19/20 | Infection |
| Orpheris Inc., subsidiary of Ashvattha Therapeutics LLC, of Redwood City, Calif. | OP-101 | Hydroxyl PAMAM dendrimer | COVID-19 infection | FDA agreed to phase II study in severe infection, in addition to standard-of-care therapy, to assess reduction of pro-inflammatory cytokine storm and oxidative stress | 5/28/20 | Infection |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD | Placental expanded cells | Acute respiratory distress syndrome caused by COVID-19 | FDA cleared IND for 140-patient phase II study; primary endpoint is ventilator-free days during the main 28-day study period; secondary endpoints include survival rate and days outside the intensive care unit | 5/8/20 | Infection |
| Pulmotect Inc., of Houston | PUL-042 | Combination of 2 Toll-like receptor agonists | COVID-19 prevention and treatment | FDA approved the start of 2 phase II studies | 5/5/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Opaganib | Sphingosine kinase-2 inhibitor | Moderate to severe COVID-19 with pneumonia | FDA approved the IND for a 40-patient phase IIa study; primary endpoint is reduction in total oxygen requirement over the course of treatment for up to 14 days; secondary endpoints include time to 50% reduction in oxygen requirements, proportion of patients without fever at day 14 and proportion with negative nasal swabs at day 14 | 5/8/20 | Infection |
| Relief Therapeutics Holding AG, of Geneva | RLF-100 | Formulation of vasoactive intestinal polypeptide | COVID-19 | Partner Neurorx Inc. filed investigational protocol with FDA under the Coronavirus Treatment Acceleration Program for a phase II/III study assessing inhaled drug in patients with non-acute lung injury due to COVID-19 | 5/11/20 | Infection |
| Restem LLC, of Corona, Calif. | Umbilical cord mesenchymal stem cells | Umbilical cord mesenchymal stem cells | COVID-19 | FDA cleared start of 60-patient phase I/IIa study in patients with severe cases | 5/14/20 | Infection |
| Sanofi SA, of Paris | Sutimlimab | Monoclonal antibody designed to inhibit complement C1s | Cold agglutinin disease | FDA granted priority review of BLA; target action date of Nov. 13, 2020 | 5/14/20 | Infection |
| Zia Lab Ltd., of Shanghai | Nuzyra (omadacycline) | Novel tetracycline | Community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections | China’s National Medical Products Administration granted priority review for the NDA | 5/6/20 | Infection |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | COVID-19 (moderate to severe symptoms) | Filed a pre-IND meeting request with the FDA | 5/15/20 | Infection |
| Kancera AB, of Stockholm | KAND-567 | Fractalkine blocker | COVID-19 | Submitted an application to the Swedish Medicines Agency for permission to conduct a phase II study | 5/15/20 | Infection |
| Leading Biosciences Inc., of Carlsbad, Calif. | LB-1148 (tranexamic acid) | Serine protease inhibitor | Pulmonary dysfunction associated with COVID-19 pneumonia | FDA cleared its IND application for a phase II study | 5/15/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | mRNA vaccine candidate | COVID-19 | FDA granted fast track designation | 5/12/20 | Infection |
| Cerecor Inc., of Rockville, Md. | CERC-002 | Tumor necrosis factor 14 ligand inhibitor | COVID-19-induced acute respiratory distress syndrome | FDA cleared IND for proof-of-concept study expected to enroll 82 individuals hospitalized with COVID-19 ARDS; primary outcome measure is respiratory failure and death vs. standard of care over 28 days; first participant expected to enroll in June 2020, with top-line data expected in fourth quarter of 2020 | 5/28/20 | Inflammatory |
| Alpha Cognition Inc., of Vancouver, British Columbia | Alpha-602 | Progranulin | Amyotrophic lateral sclerosis | FDA granted orphan drug designation | 5/12/20 | Musculoskeletal |
| Cytrx Corp., of Los Angeles, and Orphazyme A/S, of Copenhagen | Arimoclomol | Amplifies the production of heat-shock proteins | Amyotrophic lateral sclerosis | FDA granted fast track designation | 5/26/20 | Musculoskeletal |
| Novartis AG, of Basel, Switzerland, and its Avexis unit | Zolgensma (onasemnogene abeparvovec) | Gene therapy expressing SMN | Spinal muscular atrophy | European Commission granted conditional approval for babies and young children with SMA up to 21 kg | 5/19/20 | Musculoskeletal |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Amplifies production of heat-shock proteins | Amyotrophic lateral sclerosis | Received FDA fast track designation | 5/22/20 | Musculoskeletal |
| Radius Health Inc., of Waltham, Mass., and Teijin Pharma Ltd., of Tokyo | Abaloparatide-SC | Parathyroid hormone receptor 1 agonist | Osteoporosis | NDA filed in Japan to treat men and women at high risk of fractures | 5/27/20 | Musculoskeletal |
| Santhera Pharmaceuticals AG, of Pratteln, Switzerland | Puldysa (idebenone) | Short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase | Duchenne muscular dystrophy | EMA's Committee for Medicinal Products for Human Use requested extension of the clock-stop in the regulatory procedure for the conditional marketing authorization | 5/29/20 | Musculoskeletal |
| Alume Biosciences Inc., of La Jolla, Calif. | ALM-488 | Peptide-dye conjugate targeting nerves | Head and neck surgery | FDA issued a study-may-proceed letter for the phase I/II study of up to 36 patients to determine the safety and pharmacokinetics of the drug; study expected to start in the second quarter of 2020 | 5/4/20 | Neurology/Psychiatric |
| Anavex Life Sciences Corp., of New York | ANAVEX 3-71 | Small molecule targeting sigma-1 and M1 muscarinic receptors | Neurodegenerative diseases, initially frontotemporal dementia | Australia’s Human Research Ethics Committee cleared start of first-in-human study; phase I trial will enroll at least 36 healthy male and female subjects | 5/21/20 | Neurology/Psychiatric |
| Arbor Pharmaceuticals LLC, of Atlanta | AR-19 (amphetamine sulfate) | Adrenergic receptor agonist | Attention deficit hyperactivity disorder | FDA accepted NDA filing, setting PDUFA date of Nov. 15, 2020 | 5/13/20 | Neurology/Psychiatric |
| Eli Lilly and Co., of Indianapolis | Tauvid (flortaucipir F18) | Radioactive diagnostic agent | Alzheimer's disease | FDA approved for PET imaging of the brain to estimate the density and distribution of aggregated tau neurofibrillary tangles in adults with cognitive impairment being evaluated for AD | 5/28/20 | Neurology/Psychiatric |
| Genetx Biotherapeutics LLC, of Sarasota, Fla., and Ultragenyx Pharmaceutical Inc., of Novato, Calif. | GTX-102 | Antisense oligonucleotide targeting UBE3A-AS | Angelman syndrome | FDA granted fast track designation | 5/4/20 | Neurology/Psychiatric |
| Intelgenx Corp., of Saint Laurent, Quebec | Rizaport Versafilm | Oral thin-film formulation of 5-HT1 receptor agonist | Acute migraine | FDA granted request for type A meeting relating to obtain clarification on resubmitted NDA; application received complete response letter March 27, 2020; type A meeting set for June 10, 2020 | 5/27/20 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-415 | Serdexmethylphenidate, a prodrug of d-methylphenidate | Attention deficit hyperactivity disorder | FDA accepted the NDA; PDUFA date in March 2021 | 5/4/20 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-415 | Serdexmethylphenidate, a prodrug of d-methylphenidate | Attention deficit hyperactivity disorder | Received a Day-74 letter from the FDA stating that the NDA is sufficiently complete to permit a substantive review by the FDA; the target PDUFA date is March 2, 2021 | 5/19/20 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ganaxolone | Positive allosteric modulator of GABAA receptors | Status epilepticus | Based on feedback from an end-of-phase-II meeting with the FDA, company has designed a 125-patient phase III study with co-primary endpoints that focus on status cessation within 30-minutes and suppression of status for at least 24 hours; study scheduled to start in the third quarter of 2020 and generate data in the first half of 2022 | 5/4/20 | Neurology/Psychiatric |
| Neuraptive Therapeutics Inc., of Philadelphia | NTX-001 | Drug-device combination | Peripheral nerve injuries | FDA granted fast track designation | 5/15/20 | Neurology/Psychiatric |
| Novaremed AG, of Basel, Switzerland | NRD135S.E1 | Allosteric modulator of Lyn kinase | Painful diabetic peripheral neuropathy | Submitted IND to FDA for a phase II study | 5/25/20 | Neurology/Psychiatric |
| Relmada Therapeutics Inc., of New York | REL-1017 (dextromethadone) | Non-competitive N-methyl-D-aspartate receptor antagonist | Major depressive disorder | Completed end-of-phase II meeting with FDA on development program for use as adjunctive treatment; company can proceed into phase III, which will consist of 2 studies set to start in the fourth quarter of 2020 | 5/27/20 | Neurology/Psychiatric |
| Seelos Therapeutics Inc., of New York | SLS-005 (trehalose) | Transcription factor EB stimulator | Sanfilippo syndrome | Granted a rare pediatric disease designation by the FDA | 5/15/20 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Marlborough, Mass. | Dasotraline | Dopamine/norepinephrine reuptake inhibitor | Binge eating disorder/attention deficit hyperactivity disorder | NDAs withdrawn on eve of May 14, 2020, PDUFA date; company halting program, citing presumed need for additional studies to support approval | 5/13/20 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Marlborough, Mass. | Kynmobi (apomorphine HCl) sublingual film | Dopamine agonist | Parkinson’s disease | FDA approved for use in acute, intermittent treatment of off episodes | 5/21/20 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Austedo (deutetrabenazine) | Synaptic vesicular amine transporter inhibitor | Huntington's chorea; tardive dyskinesia | China NMPA approved drug following priority review | 5/18/20 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | CNS neuroactive nasal spray | COVID-19-related anxiety | Protocol submitted to FDA through Coronavirus Treatment Acceleration Program for phase IIa study | 5/18/20 | Neurology/Psychiatric |
| Kala Pharmaceuticals Inc., of Watertown, Mass. | Eysuvis (loteprednol etabonate) | Corticosteroid | Dry eye disease | Resubmitted NDA to FDA; company believes the application will be considered a class 2 resubmission | 5/4/20 | Ocular |
| Kala Pharmaceuticals Inc., of Watertown, Mass. | Eysuvis (loteprednol etabonate) | Corticosteroid | Dry eye disease | FDA accepted the NDA resubmission and set a PDUFA action date of Oct. 30, 2020 | 5/26/20 | Ocular |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Zokinvy (lonafarnib) | Farnesyltransferase inhibitor | Progeria and progeroid laminopathies | FDA acceptance of NDA for filing with priority review; the target PDUFA date is November 20, 2020 | 5/19/20 | Other/Miscellaneous |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Heat-shock protein amplifier | Niemann-Pick disease type C | Started submission of NDA | 5/29/20 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Obesity | FDA accepted NDA for treatment of pro-opiomelanocortin deficiency and leptin receptor deficiency obesity, granting priority review and setting Nov. 27, 2020, PDUFA date; no advisory committee meeting expected | 5/13/20 | Other/Miscellaneous |
| Astrazeneca plc, of Cambridge, U.K. | Bevespi Aerosphere (glycopyrronium/formoterol fumarate) | Beta 2 adrenoceptor agonist; muscarinic receptor antagonist | Chronic obstructive pulmonary disease | China NMPA approved as maintenance treatment, including chronic bronchitis and/or emphysema | 5/18/20 | Respiratory |
| Boehringer Ingelheim International GmbH | Ofev (nintedanib) | Tyrosine kinase inhibitor | Interstitial lung disease | EMA's Committee for Medicinal Products for Human Use issued positive opinion for treatment of adults with other chronic fibrosing interstitial lung diseases with a progressive phenotype beyond idiopathic pulmonary fibrosis | 5/29/20 | Respiratory |
| Microbion Corp., of Bozeman, Mont. | Pravibismane | Inhaled delivery of BisEDT antimicrobial suspension | Pulmonary infections in cystic fibrosis | FDA granted orphan designation | 5/27/20 | Respiratory |
| Nantkwest Inc., of El Segundo, Calif. | BM-Allo.MSC | Allogeneic mesenchymal stem cell therapy | COVID-19-related acute respiratory distress syndrome | FDA cleared IND application for phase Ib study of therapy vs. current supportive care in individuals with severe disease who require ventilator support | 5/18/20 | Respiratory |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa. | PB-1046 | VIP 2 receptor agonist | COVID-19 infection | FDA authorized IND for potentially pivotal Vangard trial expected to enroll about 210 hospitalized COVID-19 patients at high risk for clinical deterioration and acute respiratory distress syndrome; primary endpoint is days alive and free of respiratory failure | 5/28/20 | Respiratory |
| Sosei Heptares, part of Sosei Group Corp., of Tokyo, and Novartis AG, of Basel, Switzerland | Enerzair Breezhaler (QVM-149) | Indacaterol acetate, glycopyrronium bromide and mometasone furoate | Uncontrolled asthma | EMA's Committee for Medicinal Products for Human Use recommended approval | 5/1/20 | Respiratory |
| United Therapeutics Corp., of Silver Spring, Md. | Trevyent (treprostinil) | Preservative-free, parenteral formulation of prostacyclin analogue | Pulmonary arterial hypertension | Disclosed in its first-quarter earnings that FDA issued complete response letter (CRL); company has 1 year from date of CRL to resubmit the NDA | 5/1/20 | Respiratory |
| Verona Pharma plc, of London | Nebulized ensifentrine | PDE3/4 inhibitor | Chronic obstructive pulmonary disease | Received written comments from FDA in response to end-of-phase II briefing package for drug as maintenance treatment, which supports company’s plan for phase III program, expected to start later in 2020 | 5/14/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | Affects the gating of cystic fibrosis transmembrane conductance regulator | Cystic fibrosis | Received a positive opinion from EMA's Committee for Medicinal Products for Human Use for the label extension to include children and adolescents with CF between the ages 6 months and 18 years with the R117H mutation in the CFTR gene | 5/1/20 | Respiratory |
| Adamis Pharmaceuticals Corp., of San Diego | Zimhi (naloxone HCI) | Opioid receptor antagonist | Opioid overdose | NDA resubmitted to FDA following CRL | 5/18/20 | Toxicity and Intoxication |
| Cellphire Inc., of Rockville, Md. | Thrombosomes | Platelet-based freeze-dried hemostatic | Acute radiation syndrome | FDA granted orphan designation | 5/14/20 | Toxicity and Intoxication |
| Aadi Bioscience Inc., of Pacific Palisades, Calif. | Nab-sirolimus (sirolimus albumin-bound nanoparticles for injectable suspension) | mTOR inhibitor | Advanced malignance PEComa (perivascular epithelioid-cell tumor) | Initiated rolling NDA to the FDA seeking approval to the rare form of sarcoma driven by mTOR activation; submission to be completed in the fourth quarter of 2020 | 6/25/20 | Cancer |
| Aptose Biosciences Inc., of San Diego | CG-806 | BTK/FLT3 tyrosine kinase inhibitor | Acute myeloid leukemia | FDA cleared IND to initiate phase Ia/b trial at starting dose of 450 mg twice daily; trial expected to open enrollment in second half of 2020 in individuals with relapsed, resistant or refractory disease | 6/29/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | HQP-1351 | T315I-mutant chronic phase chronic myeloid leukemia and accelerated phase CML | Bcr-Abl inhibitor | Submitted NDA to the Center for Drug Evaluation of China National Medical Products Administration | 6/18/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic adenocarcinoma of the pancreas with BRCA1/2 germline mutations | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on use of the drug in patients who have not progressed after at least 16 weeks of a first-line platinum-based treatment | 6/1/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Selumetinib | Mitogen-activated protein kinase kinases 1 and 2 inhibitor | Neurofibromatosis type 1 | Japanese Ministry of Health, Labour and Welfare granted orphan drug designation | 6/30/20 | Cancer |
| Aveo Oncology Inc., of Boston | Tivozanib | Next-generation VEGFR tyrosine kinase inhibitor | Relapsed or refractory renal cell carcinoma | FDA accepted for filing the NDA, assigning a PDUFA date of March 31, 2021; agency also indicated it does not plan to convene Oncology Drug Advisory Committee to discuss the application | 6/1/20 | Cancer |
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Chronic lymphocytic leukemia/small lymphocytic lymphoma | Approved by China’s NMPA for use in patients who have received at least 1 prior therapy | 6/3/20 | Cancer |
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Mantle cell lymphoma | Approved by China’s NMPA for use in patients who have received at least 1 prior therapy | 6/3/20 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | Monoclonal antibody targeting PD-1 | First-line treatment nonsquamous non-small-cell lung cancer | Center for Drug Evaluation of the China National Medical Products Administration accepted the supplemental NDA | 6/19/20 | Cancer |
| Bioinvent International AB, of Lund, Sweden | BI-1808 | Monoclonal antibody targeting tumor necrosis factor receptor 2 | Ovarian cancer, non-small-cell lung cancer and cutaneous T-cell lymphoma | Submitted a CTA to the EMA to test the drug as a monotherapy and in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 6/30/20 | Cancer |
| Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-1706 | Biosimilar to Avastin (bevacizumab, Roche Holding AG) | Advanced, metastatic or relapsed non-small-cell lung cancer and metastatic colorectal cancer | China National Medical Products Administration accepted the BLA | 6/22/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Monoclonal antibody targeting PD-1 | Unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma after prior fluoropyrimidine- and platinum-based chemotherapy | FDA approved the expanded indication | 6/10/20 | Cancer |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | A3 adenosine receptor agonist | Hepatocellular carcinoma | Concluded a meeting with the EMA's Scientific Advice Working Party; plans to run 1 pivotal trial for registration in the U.S. and EU | 6/4/20 | Cancer |
| CASI Pharmaceuticals Inc., of Rockville, Md. | CID-103 | Monoclonal antibody targeting CD38 | Multiple myeloma | Submitted a CTA for a phase I study to the U.K.'s Medicines and Healthcare Products Regulatory Agency | 6/8/20 | Cancer |
| Clarity Pharmaceuticals Pty Ltd., of Sydney | 67Cu-Sartate | Radiopharmaceutical | Neuroblastoma | FDA granted rare pediatric disease designation | 6/3/20 | Cancer |
| CNS Pharmaceuticals Inc., of Houston | Berubicin | Anthracycline that interferes with topoisomerase II | Malignant gliomas | FDA granted orphan drug designation | 6/11/20 | Cancer |
| Compugen Ltd., of Holon, Israel | COM-701 | Anti-PVRIG antibody | Advanced solid tumors | FDA cleared the IND for a study testing COM-701 plus Opdivo (nivolumab, Bristol Myers Squibb Co.) and BMS-986207; study scheduled to start in the second half of 2020 | 6/1/20 | Cancer |
| Curis Inc., of Lexington, Mass. | CI-8993 | Monoclonal antibody targeting VISTA | Cancer | FDA cleared the IND; phase Ia/Ib study to start in second half of 2020 | 6/10/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Pexidartinib | Oral small molecule inhibiting CSF1R | Severe tenosynovial giant cell tumor | EMA’s Committee for Medicinal Products for Human Use adopted negative opinion on MAA for treating this subset of adults | 6/26/20 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Qinlock (ripretinib) | Switch-control tyrosine kinase inhibitor | Advanced gastrointestinal stromal tumor in patients who have received prior treatment | Health Canada authorized product for sale | 6/22/20 | Cancer |
| EMD Serono, a unit of Darmstadt, Germany-based Merck KGaA, and Pfizer Inc., of New York | Bavencio (avelumab) | Monoclonal antibody targeting PD-L1 | Urothelial carcinoma | FDA approved the supplemental BLA for use in treating locally advanced or metastatic disease that has not progressed with first-line platinum-containing chemotherapy | 6/30/20 | Cancer |
| Epizyme Inc., of Cambridge, Mass. | Tazverik (tazemetostat) | Methyltransferase inhibitor | Follicular lymphoma | FDA approved supplemental NDA expanding use for adults with relapsed or refractory FL whose tumors are positive for EZH2 mutation as detected by FDA-approved test and who have received at least 2 prior systemic therapies; and for adults with relapsed or refractory FL who have no satisfactory alternative treatment options; accelerated approvals may be contingent upon data from confirmatory trials | 6/18/20 | Cancer |
| ERC Belgium SA, of Isnes, Belgium | Sitoiganap (ERC-1671) | Immunotherapy based on freshly extracted tumor cells and lysates | Recurrent glioblastoma | Submitted MAA to EMA seeking conditional approval | 6/26/20 | Cancer |
| Genentech, of South San Francisco, a member of the Roche Group | Phesgo (pertuzumab/trastuzumab) | Fixed-dose combination of HER2-targeting antibodies with hyaluronidase | HER2-positive breast cancer | FDA approved for use in combination with intravenous chemotherapy for early and metastatic disease; administered via single subcutaneous injection | 6/29/20 | Cancer |
| Genequantum Healthcare (Suzhou) Co. Ltd., of Suzhou, China | GQ-1001 | Site-specific antibody-drug conjugate | HER2-positive solid tumors | Received U.S. IND approval | 6/11/20 | Cancer |
| Genmab A/S, of Copenhagen, Denmark | Darzalex (daratumumab) | Monoclonal antibody targeting CD38 | Multiple myeloma | European Commission granted marketing authorization for the subcutaneous fixed-dosed formulation of the drug | 6/4/20 | Cancer |
| Genocea Biosciences Inc., of Cambridge, Mass. | GEN-011 | T-cell therapy | Tumors | Submitted IND to FDA for a phase I/IIa study; preliminary data expected in mid-2021 | 6/15/20 | Cancer |
| Genprex Inc., of Austin, Texas | GPX-001 | TUSC2 gene stimulator | Non-small-cell lung cancer | United States Adopted Names Council approved non-proprietary name quaratusugene ozeplasmid for GPX-001, formerly called Oncoprex | 6/25/20 | Cancer |
| Heat Biologics Inc., of Durham, N.C. | PTX-35 | Agonist antibody targeting TNFRSF25 | Solid tumors | FDA cleared the IND for a phase I study | 6/8/20 | Cancer |
| Helsinn Healthcare SA, of Lugano, Switzerland, and Taiho Pharmaceutical Co. Ltd., of Tokyo | TAS-0953/HM-06 | RET inhibitor | Advanced solid tumors with RET gene abnormalities | FDA cleared the IND for a phase I/II study; plans to start study in the third quarter of 2020 | 6/8/20 | Cancer |
| Hookipa Pharma Inc., of New York | HB-202 | Arenavirus expressing E7/E6 fusion protein derived from HPV16 | Human papillomavirus 16-positive cancers | FDA cleared the IND, which will allow the company to add an arm testing HB-202 plus HB-201 to the phase I/II study of HB-201 | 6/17/20 | Cancer |
| Hutchison China Meditech Ltd., of London | Surufatinib | Angio-immuno kinase inhibitor | Advanced neuroendocrine tumors | FDA agreed that completed studies can form the basis to support an NDA submission; plans to start a rolling submission in late 2020 | 6/1/20 | Cancer |
| Hutchison China Meditech Ltd., of London | Fruquintinib | Inhibits vascular endothelial growth factor receptor 1/2/3 | Metastatic colorectal cancer | FDA granted fast track designation for the drug as a treatment after fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy and, if RAS wild-type, an anti-EGFR therapy | 6/18/20 | Cancer |
| Incyte Corp., of Wilmington, Del., and Novartis AG, of Basel, Switzerland | Tabrecta (capmatinib) | Kinase inhibitor that targets MET | Non-small-cell lung cancer | Japan’s Ministry of Health, Labour and Welfare approved for use in MET exon 14 skipping mutation-positive advanced and/or recurrent unresectable NSCLC; Tabrecta approved for first-line and previously treated patients, regardless of prior treatment type | 6/29/20 | Cancer |
| Inmune Bio Inc., of La Jolla, Calif. | Inkmune | Therapy designed to prime NK cells | High-risk myelodysplastic syndrome | U.K.’s Medicines and Healthcare products Regulatory Agency cleared start of phase I trial; study expected to begin in second half of 2020 | 6/11/20 | Cancer |
| Innate Pharma SA, of Paris | Lacutamab (IPH-4102) | Anti-KIR3DL2 humanized cytotoxicity-inducing antibody | Advanced T-cell lymphomas | FDA lifted partial clinical hold placed on phase II Tellomak trial, based on quality assessment of new GMP-certified batch manufactured for clinical development program; recruitment can now resume for new patients with relapsed/refractory Sézary syndrome and mycosis fungoides who have received at least 2 prior systemic therapies | 6/24/20 | Cancer |
| Innovent Biologics Inc., of Suzhou, China | Byvasda (bevacizumab) | Biosimilar to Avastin (bevacizumab), an anti-VEGF monoclonal antibody | Advanced non-small-cell lung cancer and metastatic colorectal cancer | Center for Drug Evaluation of the China National Medical Products Administration approved the drug | 6/19/20 | Cancer |
| Ipsen SA, of Paris | Onivyde (liposomal irinotecan) | Topoisomerase I inhibitor | Previously untreated, unresectable, locally advanced and metastatic pancreatic ductal adenocarcinoma | FDA granted fast track designation for the drug in combination with 5-fluorouracil/leucovorin and oxaliplatin | 6/17/20 | Cancer |
| Jazz Pharmaceuticals plc, of Dublin, and Pharmamar SA, of Madrid | Zepzelca (lurbinectedin) | Alkylating drug that binds guanine residues within DNA | Metastatic small-cell lung cancer | FDA approved the drug for use in patients with disease progression on or after platinum-based chemotherapy | 6/15/20 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | SINE compound | Relapsed or refractory diffuse large B-cell lymphoma | Approved by FDA for use in adults with DLBCL, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy | 6/22/20 | Cancer |
| Kite Pharma, a unit of Gilead Sciences Inc., of Foster City, Calif. | Yescarta (axicabtagene ciloleucel) | CAR T therapy targeting CD19 | Cancer | Received approval to implement a variation to the marketing authorization from the EMA for end-to-end manufacturing; with this approval, Kite’s European manufacturing facility is now fully operational | 6/12/20 | Cancer |
| Leap Therapeutics Inc., of Cambridge, Mass. | DKN-01 | Humanized monoclonal antibody targeting DKK1 protein | Gastric and gastroesophageal junction cancer | FDA granted orphan drug designation | 6/12/20 | Cancer |
| Linnaeus Therapeutics Inc., of Haddonfield, N.J. | LNS-8801 | Agonist of GPER | Metastatic or unresectable melanoma who have progressed on or after anti-PD-1/L1 therapy | FDA granted fast track designation | 6/18/20 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Margetuximab | Fc-engineered monoclonal antibody targeting HER2 | Gastric and gastroesophageal junction cancer | FDA granted orphan drug designation | 6/5/20 | Cancer |
| Marker Therapeutics Inc., of Houston | Zelenoleucel (MT-401) | Multitumor-associated antigen-specific T-cell therapy | Acute myeloid leukemia | Received approval from the U.S. Adopted Names (USAN) Council for zelenoleucel as the nonproprietary name for MT-401 | 6/23/20 | Cancer |
| Medigene AG, of Martinsried, Germany | MDG-1021 | T-cell receptor-modified T-cell immunotherapy targeting antigen HA-1 | Relapsed or persistent blood cancers | Received approval from Dutch regulators to begin phase I study enrolling patients after allogeneic hematopoietic stem cell transplantation | 6/24/20 | Cancer |
| Medivir AB, of Stockholm | MIV-818 (liver-targeted troxacitabine prodrug) | DNA polymerase inhibitor | Hepatocellular carcinoma | EC granted orphan medicinal product designation in the EU | 6/25/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Gardasil9 | HPV vaccine | Prevention of oropharyngeal and other head and neck cancers | FDA approved the expanded indication | 6/12/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Unresectable or metastatic, mutational burden-high solid tumors | FDA approved the expanded indication regardless of tumor type; FDA also approved the Foundationone Cdx (Foundation Medicine Inc.) as a companion diagnostic for Keytruda in patients with mutational burden-high tumors | 6/17/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Metastatic squamous non-small-cell lung cancer | Approved by the National Medical Products Administration (NMPA) in China for use in combination with carboplatin and paclitaxel for the first-line treatment | 6/22/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1 inhibitor | Cutaneous squamous cell carcinoma | FDA approved drug as monotherapy to treat recurrent or metastatic disease not curable by surgery or radiation, based on data from phase II Keynote-629 trial | 6/24/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Unresectable or metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer | FDA approved the supplemental BLA for use in first-line treatment | 6/29/20 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | Next-generation anthracycline | Acute myeloid leukemia | Submitted requests to Polish regulatory authorities for approval to open 2 additional clinical sites for the ongoing phase I/II study | 6/4/20 | Cancer |
| Myovant Sciences Ltd., of Basel, Switzerland | Relugolix | Oral gonadotropin-releasing hormone receptor antagonist | Advanced prostate cancer | NDA for once-daily, oral relugolix (120 mg) was granted priority review by the FDA; PDUFA action date is December 20, 2020 | 6/23/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Designed to destroy tumors when activated by radiotherapy | Head and neck cancer | Received feedback from FDA to advanced phase III study design and CMC development plan for NDA; 500-patient Nanoray-312 study set to start after company makes protocol refinements and secures requisite financing | 6/17/20 | Cancer |
| Nkmax America Inc., of Santa Ana, Calif. | SNK-01 | Autologous natural killer cell immunotherapy | Solid tumors | FDA cleared IND for phase I/IIa trial in combination with trastuzumab or cetuximab to treat advanced/metastatic HER2- or EGFR-expressing cancer; trial expected to begin in third quarter of 2020 | 6/2/20 | Cancer |
| Nordic Nanovector ASA, of Oslo, Norway | Betalutin (177Lu lilotomab satetraxetan) | CD37-targeting antibody-radionuclide-conjugate | Relapsed or refractory marginal zone lymphoma | FDA granted fast track designation for use in adults who have received at least 2 prior therapies | 6/29/20 | Cancer |
| Oncolys Biopharma Inc., of Tokyo | Telomelysin | Genetically modified type 5 adenovirus targeting cancer cells | Esophageal cancer | FDA granted orphan drug designation | 6/3/20 | Cancer |
| Oncopeptides AB, of Stockholm | Melflufen | Aminopeptidase-targeting peptide-drug conjugate | Multiple myeloma | Submitted NDA to the FDA seeking approval for use in patients refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent and 1 anti-CD38 monoclonal antibody | 6/30/20 | Cancer |
| Oncternal Therapeutics Inc., of San Diego | Cirmtuzumab | Monoclonal antibody targeting ROR1 | Mantle cell lymphoma and chronic lymphocytic leukemia/small lymphocytic lymphoma | FDA granted orphan designations | 6/30/20 | Cancer |
| Pharmacyclics LLC, of Sunnyvale, Calif., an Abbvie company | Imbruvica (ibrutinib) | Bruton's tyrosine kinase inhibitor | Waldenström’s macroglobulinemia | Submitted an sNDA to the FDA for Imbruvica in combination with rituximab | 6/23/20 | Cancer |
| Philogen SpA, of Siena, Italy | Nidlegy (bifikafusp alfa and onfekafusp alfa) | Combination of immunocytokines | Melanoma and nonmelananoma skin cancers | EMA agreed to request for marketing the product as a combination pack for use as neoadjuvant intralesional treatment for resectable stage III melanoma and locally advanced, not metastatic nonmelanoma skin cancers | 6/24/20 | Cancer |
| Pierre Fabre SA, of Castres, France | Braftovi (encorafenib) | Small-molecule BRAF kinase inhibitor | BRAFV600E-mutant metastatic colorectal cancer | European Commission approved for use in combination with Erbitux (cetuximab) for treating adults who have received prior systemic therapy | 6/3/20 | Cancer |
| Polynoma LLC, of San Diego, unit of CK Life Sciences International (Holdings) Inc. | Seviprotimut-L | Polyvalent, shed melanoma antigen vaccine | Melanoma | Received FDA fast track designation | 6/23/20 | Cancer |
| Prestige Biopharma Ltd., of Singapore | PBP-1510 | Monoclonal antibody targeting pancreatic adenocarcinoma up-regulated factor | Pancreatic cancer | FDA granted orphan designation | 6/30/20 | Cancer |
| Rakuten Medical Inc., of San Mateo, Calif. | ASP-1929 | Antibody-drug conjugate comprising EGFR-targeting cetuximab and Irdye 700DX, a light activatable dye | Recurrent head and neck cancer | Japan’s Ministry of Health, Labour and Welfare said application will be reviewed under conditional early approval system; Rakuten submitted Japanese BLA in March 2020 based on phase I/II results | 6/29/20 | Cancer |
| Renovorx Inc., of Los Altos, Calif. | Renovocath + gemcitabine | Drug/device | Cholangiocarcinoma | FDA granted orphan drug designation in indication | 6/2/20 | Cancer |
| Rigenerand SRL, of Modena, Italy | RR-001 | TNFSF10 gene stimulator | Pancreatic cancer | Italian Medicine Authority granted authority to produce gene therapy products for clinical purposes, including RR-001 for use in phase I trial expected to start in first quarter of 2021 | 6/2/20 | Cancer |
| Samsung Bioepis Co. Ltd., of Incheon, Korea | Aybintio (bevacizumab biosimilar) | Anti-VEGF antibody | Multiple cancers | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in same types of cancer as reference bevacizumab in EU, including metastatic carcinoma of the colon or rectum, metastatic breast cancer, non-small-cell lung cancer, advanced and/or metastatic renal cell cancer, epithelial ovarian, fallopian tube and primary peritoneal cancer and cervical cancer | 6/26/20 | Cancer |
| Sapience Therapeutics Inc., of Harrison, N.Y. | ST-101 | CCAAT enhancer binding protein beta inhibitor | Advanced solid tumors | FDA accepted IND application for phase I/II trial in adults and granted orphan drug designation to treat glioma | 6/2/20 | Cancer |
| Shanghai Henlius Biotech Inc., of Shanghai, and Accord Healthcare Ltd., of Durham, N.C. | HLX-02 | Biosimilar to Herceptin (trastuzumab) | HER2-positive early breast cancer, HER2-positive metastatic breast cancer and previously untreated HER2-positive metastatic adenocarcinoma of the stomach or gastroesophageal junction | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the biosimilar | 6/1/20 | Cancer |
| Specialised Therapeutics Asia Pty Ltd., of Singapore, a unit of Specialised Therapeutics | Lurbinectedin (PM-1183) | Synthetic compound that inhibits oncogenic transcription in tumor-associated macrophages | Solid tumors, including small-cell lung cancer | Australia’s TGA granted provisional designation; drug will now be reviewed concurrently by FDA and other international regulators, including TGA, under Project Orbis initiative | 6/3/20 | Cancer |
| Sun Biopharma Inc., of Minneapolis | SBP-101 | Polyamine analogue | First-line metastatic pancreatic ductal adenocarcinoma | FDA granted fast track designation | 6/30/20 | Cancer |
| Tetra Bio-Pharma Inc., of Ottawa, Ontario | HCC-011 | Inhaled THC | Hepatocellular carcinoma | During type B meeting, FDA provided guidance on nonclinical safety and human clinical pharmacology, safety and efficacy requirements and agreed with proposed nonclinical safety program to support development; agency also agreed that food effect study not required | 6/2/20 | Cancer |
| TG Therapeutics Inc., of New York | Umbralisib | Inhibitor of PI3K-delta and CK1-epsilon | Previously treated marginal zone lymphoma and follicular lymphoma | Completed rolling submission of the NDA | 6/17/20 | Cancer |
| Tizona Therapeutics Inc., of South San Francisco | TTX-080 | Anti-HLA-G antibody | Advanced cancer | FDA cleared IND for phase I study, expected to begin in third quarter of 2020, evaluating TTX-080 both as monotherapy and in combination therapy | 6/25/20 | Cancer |
| Treadwell Therapeutics Inc., of New York | CFI-402411 | HPK1 inhibitor | Solid tumors | FDA accepted IND; Health Canada issued no objection letter to CTA; phase I/II study to test drug as monotherapy and in combination with PD-1 pathway blockade set to start in 2020 | 6/2/20 | Cancer |
| Viracta Therapeutics Inc., of San Diego | Nanatinostat + valganciclovir | Histone deacetylase inhibitor | T-cell lymphoma | FDA granted orphan drug designation in the indication | 6/24/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Danyelza (naxitamab) | 3F8 IgG1 monoclonal antibody | Neuroblastoma | FDA accepted BLA and granted priority review, setting PDUFA date of Nov. 30, 2020; no advisory committee meeting expected | 6/2/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Omburtamab | Monoclonal antibody targeting B7-H3 | Central nervous system/leptomeningeal metastases from neuroblastoma | Started rolling submission of the BLA to the FDA | 6/30/20 | Cancer |
| Algo Therapeutix SAS, of Suresnes, France | ATX-01 | Topical amitriptyline | Erythromelalgia | FDA granted orphan designation | 6/8/20 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K | Brilinta (ticagrelor) | P2Y12 receptor antagonist | Coronary artery disease | FDA approved the drug to reduce the risk of a first heart attack or stroke in high-risk patients | 6/1/20 | Cardiovascular |
| Mezzion Pharma Co. Ltd., of Seoul, South Korea | Udenafil | PDE5 inhibitor | Single ventricle heart disease after a Fontan operation | Submitted an NDA to the FDA with a request for a priority review | 6/30/20 | Cardiovascular |
| Novartis AG, of Basel, Switzerland | Entresto (sacubitril/valsartan) | Inhibits neprilysin and blocks angiotensin II type-I receptor | Heart failure with preserved ejection fraction | FDA accepted supplemental NDA; decision expected in first half of 2021 | 6/24/20 | Cardiovascular |
| Novartis AG, of Basel, Switzerland | Entresto (sacubitril valsartan sodium hydrate) | Inhibits neprilysin and blocks angiotensin II type-I receptor | Chronic heart failure | Approved by Japan’s Ministry of Health, Labour and Welfare | 6/29/20 | Cardiovascular |
| Resverlogix Corp., of Calgary, Alberta | Apabetalone | Targets bromodomain and extra terminal domain proteins | Atherosclerosis | FDA accepted BETonMACE2 clinical plan as a registration-enabling study | 6/22/20 | Cardiovascular |
| Valeo Pharma Inc., of Montreal | Ethacrynate sodium | Loop diuretic | Congestive heart failure, acute pulmonary edema, renal edema, hepatic cirrhosis with ascites | FDA approved the abbreviated NDA | 6/16/20 | Cardiovascular |
| Leo Pharma A/S, of Ballerup, Denmark | Tralokinumab | Fully human monoclonal antibody targeting interleukin-13 cytokine | Moderate to severe atopic dermatitis | EMA validated marketing authorization application | 6/11/20 | Dermatologic |
| Mediwound Ltd., of Yavne, Israel, and Vericel Corp., of Cambridge, Mass. | Nexobrid | Proteolytic enzymes | Eschar removal in deep partial-thickness and/or full-thickness thermal burns | Submitted BLA to the FDA | 6/30/20 | Dermatologic |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Inhibits interleukin-17A | Moderate to severe plaque psoriasis | EMA’s Committee for Medicinal Products for Human Use adopted positive opinion for approval in treating children and adolescents, ages 6 to under 18 | 6/26/20 | Dermatologic |
| Philogen SpA, of Siena, Italy | Nidlegy (bifikafusp alfa and onfekafusp alfa) | Combination of immunocytokines | Skin cancer | Swissmedic cleared approval for phase II study in patients with locally advanced, not metastatic nonmelanoma skin cancer | 6/26/20 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Fully human monoclonal antibody targeting IL-4 and IL-13 | Atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis | FDA approved a 300-mg single-dose prefilled pen | 6/19/20 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Monoclonal antibody that inhibits the signaling of IL-4 and IL-13 | Moderate to severe atopic dermatitis | Center for Drug Evaluation of the China National Medical Products Administration approved the drug | 6/19/20 | Dermatologic |
| SFA Therapeutics Inc., of Philadelphia | SFA-002 | Microbiome-derived metabolite | Mild to moderate psoriasis | FDA cleared IND for open-label, 30-subject trial | 6/29/20 | Dermatologic |
| Acasti Pharma Inc., of Laval, Quebec | Capre (omega-3 phospholipid) | Phospholipid derived from krill oil | Hypertriglyceridemia | In a written response to a type C meeting, the FDA said post-hoc analysis from the Trilogy 1 study may be used for exploratory purposes; depending on the outcome of Trilogy 2 study, an additional clinical trial may be required before submission of an NDA | 6/19/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon hGH (lonapegsomatropin) | Long-acting, once-weekly prodrug of human growth hormone | Pediatric growth hormone deficiency | Submitted BLA to FDA | 6/26/20 | Endocrine/Metabolic |
| Chiasma Inc., of Needham, Mass. | Mycapssa (octreotide) | Oral somatostatin analogue | Acromegaly | Approved by FDA for use as long-term maintenance treatment in patients who have responded to and tolerated treatment with octreotide or lanreotide | 6/26/20 | Endocrine/Metabolic |
| Dicerna Pharmaceuticals Inc., of Lexington, Mass. | Nedosiran | RNAi targeting lactate dehydrogenase | Primary hyperoxaluria | FDA granted rare pediatric disease designation | 6/18/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Lyumjev (insulin lispro-aabc) | Rapid-acting insulin | Type 1 and type 2 diabetes | FDA approved the drug | 6/15/20 | Endocrine/Metabolic |
| Hemoshear Therapeutics Inc., of Charlottesville, Va. | HST-5040 | Oral, small molecule | Methylmalonic acidemia and propionic acidemia | FDA cleared IND for phase II study | 6/24/20 | Endocrine/Metabolic |
| Lannett Co. Inc., of Philadelphia | Insulin glargine biosimilar | Long-acting insulin | Diabetes | FDA provided feedback during type II meeting; company plans 351(k) biosimilar application filed in 2022 | 6/11/20 | Endocrine/Metabolic |
| Mylan NV, of Hertfordshire, U.K. | Semglee | Long-acting insulin | Diabetes | Approved by FDA to improve glycemic control in adult and pediatric patients with type 1 diabetes mellitus and in adults with type 2 diabetes mellitus; approved as BLA under section 351(a) of Public Health Service Act and can be a reference product for a proposed insulin glargine biosimilar or interchangeable biosimilar product | 6/11/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Rybelsus (oral semaglutide) | Oral GLP-1 receptor agonist | Type 2 diabetes | Japan’s Ministry of Health, Labour and Welfare approved for use in adults | 6/29/20 | Endocrine/Metabolic |
| Rezolute Inc., of Redwood City, Calif. | RZ-358 | Monoclonal antibody targeting the insulin receptor | Congenital hyperinsulinism | FDA granted rare pediatric disease designation | 6/10/20 | Endocrine/Metabolic |
| Ultragenyx Pharmaceutical Inc., of Novato, Calif. | Dojolvi (triheptanoin) | Odd-carbon medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone | Molecularly confirmed long-chain fatty acid oxidation disorders | FDA approved the marketing application | 6/30/20 | Endocrine/Metabolic |
| Ultragenyx Pharmaceutical Inc., of Novato, Calif., and Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Recombinant fully human monoclonal IgG1 antibody against FGF23 | FGF23-related hypophosphatemia in tumor-induced osteomalacia | FDA approved for use in TIO associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 and older | 6/18/20 | Endocrine/Metabolic |
| Bacainn Therapeutics Inc., of Concord, Mass. | BT-051 | Controls neutrophil migration into the lumen of the gastrointestinal tract | Healthy volunteers (eventually ulcerative colitis) | FDA cleared the IND for a phase I study testing the safety, tolerability and pharmacokinetics of the drug; data expected by the end of 2020 | 6/9/20 | Gastrointestinal |
| Biovie Inc., of Santa Monica, Calif. | BIV-201 | Continuous infusion terlipressin | Refractory ascites due to liver cirrhosis | Received guidance from FDA and plans 24-patient phase II study this year, with larger pivotal phase III trial to begin first half of 2021; agency noted that pending positive phase II study results, a sufficiently large and well-controlled phase III trial, with supportive data from the phase II study (statistical significance not required), could potentially yield the data needed to apply for marketing approval | 6/11/20 | Gastrointestinal |
| Cancer Prevention Pharmaceuticals Inc., of Tucson, Ariz. | CPP-1X (eflornithine) + sulindac | Cyclooxygenase inhibitor; ornithine decarboxylase inhibitor | Familial adenomatous polyposis | NDA submitted to FDA, seeking accelerated approval to treat indication in adults | 6/29/20 | Gastrointestinal |
| Evoke Pharma Inc., of Solana Beach, Calif. | Gimoti (metoclopramide) | Works via antagonist activity at D2 receptors | Acute and recurrent diabetic gastroparesis | FDA approved the drug | 6/19/20 | Gastrointestinal |
| Goliver Therapeutics SA, of Nantes, France | Undisclosed | Cell therapy | Acute liver failure | EMA provided scientific advice on clinical development, pharmaco-toxicological and production strategy plans; company is working with Paul-Brousse Hospital on design and implementation of a phase I/IIa trial | 6/29/20 | Gastrointestinal |
| Intercept Pharmaceuticals Inc., of New York | Obeticholic acid | FXR agonist | Fibrosis due to nonalcoholic steatohepatitis | FDA issued complete response letter, recommending submission of additional post-interim analysis of efficacy and safety data from ongoing Regenerate study in support of potential accelerated approval and said long-term outcomes phase of the study should continue | 6/29/20 | Gastrointestinal |
| Plakous Therapeutics Inc., of Winston-Salem, N.C. | Protego-PD | Acellular biotherapeutic from post-delivery placentas | Necrotizing enterocolitis prophylaxis | FDA granted orphan drug designation | 6/19/20 | Gastrointestinal |
| Vivet Therapeutics SAS, of Paris | VTX-803 | Gene therapy | Progressive familial intrahepatic cholestasis type 3 | Both FDA and European Commission granted orphan designation | 6/1/20 | Gastrointestinal |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-2191 | Inhibits dihydrofolate reductase | Proliferative vitreoretinopathy | European Commission granted orphan medicinal product designation | 6/15/20 | Genitourinary/Sexual Function |
| Ardelyx Inc., of Fremont, Calif. | Tenapanor | Inhibits sodium hydrogen exchanger 3 | Control of serum phosphorus in adult patients with chronic kidney disease on dialysis | Submitted NDA to the FDA | 6/30/20 | Genitourinary/Sexual Function |
| Mayne Pharma Group Ltd., of Adelaide, Australia | E4/DRSP | Combined oral pill containing 15 mg estetrol and 3 mg drospirenone | Contraception | NDA accepted for review by the FDA; decision expected in first half of 2021 | 6/24/20 | Genitourinary/Sexual Function |
| Mundipharma International Ltd., of Cambridge, U.K., and Janssen-Cilag International NV, a unit of New Brunswick, N.J.-based Johnson & Johnson | Invokana (canagliflozin) | Sodium glucose co-transporter 2 inhibitor | Diabetic kidney disease | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the expanded indication | 6/1/20 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland | Relugolix/estradiol/norethindrone | Gonadotropin-releasing hormone receptor antagonist and hormones | Heavy menstrual bleeding associated with uterine fibroids | Submitted an NDA to the FDA | 6/1/20 | Genitourinary/Sexual Function |
| Nymox Pharmaceutical Corp., of Hasbrouck Heights, N.J. | Fexapotide triflutate | Pro-apoptotic protein | Benign prostatic hyperplasia | On track to file marketing applications in the U.S. and EU approximately by the end of the third quarter or early fourth quarter of 2020 | 6/19/20 | Genitourinary/Sexual Function |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Mitapivat | Pyruvate kinase stimulator | Thalassemia | FDA granted orphan drug designation | 6/8/20 | Hematologic |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | C5 complement inhibitor | Atypical hemolytic uremic syndrome | European Commission granted marketing authorization to treat adults and children with body weight of 10 kg or above | 6/29/20 | Hematologic |
| Beigene Ltd., of Beijing, China | Brukinsa (zanubrutinib) | BTK inhibitor | Waldenström’s macroglobulinemia | EMA accepted the MAA | 6/18/20 | Hematologic |
| Bristol Myers Squibb Co., of New York, and Acceleron Pharma Inc., of Cambridge, Mass. | Reblozyl (luspatercept) | Erythroid maturation agent | Anemia | European Commission approved for use in adults with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes with ring sideroblasts, who had unsatisfactory response or are ineligible for erythropoietin-based therapy, and for adults with transfusion-dependent anemia associated with beta-thalassemia | 6/26/20 | Hematologic |
| Elixirgen Therapeutics Inc., of Baltimore | EXG-34217 | Autologous cell therapy | Telomere biology disorders with bone marrow failure | FDA approved the IND for the phase I/II study | 6/4/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | Issued license from Israeli Ministry of Health granting marketing authorization Endari to reduce acute complications of sickle cell disease in adult and pediatric patients 5 and older | 6/29/20 | Hematologic |
| Global Blood Therapeutics Inc., of South San Francisco | Oxbryta (voxelotor) | Hemoglobin alpha subunit modulator | Sickle cell disease | Following type B meeting with FDA, company plans to submit NDA for age-appropriate formulation to treat 4- to 11-year-olds under accelerated approval pathway | 6/8/20 | Hematologic |
| Global Blood Therapeutics Inc., of South San Francisco | Oxbryta (voxelotor) | Inhibits hemoglobin polymerization | Hemolytic anemia in sickle cell disease | Based on discussion with EMA, plans to submit MAA by mid-2021, seeking approval for use in patients 12 and older; company also intends to initiate early access program in Europe prior to potential marketing authorization | 6/24/20 | Hematologic |
| Imara Inc., of Boston | IMR-687 | Small-molecule inhibitor of PDE9 | Beta-thalassemia | FDA granted orphan designation | 6/24/20 | Hematologic |
| Pfizer Inc., of New York | Nyvepria (pegfilgrastim-apgf) | Biosimilar to G-CSF therapy Neulasta | Febrile neutropenia | Approved by FDA for use to decrease incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive cancer drugs associated with a clinically significant incidence of febrile neutropenia; company expects drug to be available to patients later in 2020 | 6/11/20 | Hematologic |
| Pharmaessentia Corp., of Burlington, Mass. | Ropeginterferon alfa-2b (P-1101) | Pegylated interferon | Polycythemia vera | FDA accepted the BLA; decision expected in early 2021 | 6/4/20 | Hematologic |
| Protagonist Therapeutics Inc., of Newark, Calif. | PTG-300 | Synthetic peptide mimetic of hepcidin | Polycythemia vera | FDA granted orphan drug designation | 6/17/20 | Hematologic |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | Selective and reversible JAK inhibitor | Active psoriatic arthritis | Submitted applications to FDA and EMA for use in treating adults | 6/1/20 | Immune |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-108 (epinephrine, sublingual soluble film) | Adrenergic receptor agonist | Anaphylaxis | FDA cleared IND for pharmacokinetic trials, expected to begin by year-end 2020; program will be reviewed under 505(b)(2) pathway | 6/29/20 | Immune |
| Baylx Inc., of Irvine, Calif. | BX-U001 | Umbilical cord tissue mesenchymal stem cells | Rheumatoid arthritis | FDA cleared the IND | 6/5/20 | Immune |
| Genmab A/S, of Copenhagen, and Novartis AG, of Basel, Switzerland | Subcutaneous ofatumumab | Fully human monoclonal antibody targeting CD20 | Relapsing forms of multiple sclerosis | FDA notified Novartis that agency extended review of supplemental BLA; action now anticipated in September 2020 | 6/2/20 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Cleaves IgG-antibodies | Kidney transplant | EMA’s Committee for Medicinal Products for Human Use adopted positive opinion, recommending conditional approval for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor; endorsement by the European Commission expected in the third quarter of 2020 | 6/25/20 | Immune |
| Novartis AG, of Basel, Switzerland | Ilaris (canakinumab) | Monoclonal antibody targeting interleukin-1beta | Active Still’s disease | FDA approved the expanded indication | 6/16/20 | Immune |
| Novartis AG, of Basel, Switzerland | Mayzent (siponimod fumaric acid) | Sphingosine 1-phosphate receptor modulator | Secondary progressive multiple sclerosis | Approved by Japan’s Ministry of Health, Labour and Welfare | 6/29/20 | Immune |
| Osmotica Pharmaceuticals plc, of Bridgewater, N.J. | Arbaclofen | Active R-enantiomer of baclofen | Spasticity in multiple sclerosis | Resubmitted NDA to FDA | 6/30/20 | Immune |
| TFF Pharmaceuticals Inc., of Austin, Texas | Tacrolimus inhalation powder | Immunosuppressant | Prophylaxis of lung allograft rejection | FDA granted orphan drug designation | 6/18/20 | Immune |
| Mylan NV, of Hertfordshire, U.K., and Lupin Ltd., of Mumbai | Nepexto | Biosimilar to Enbrel (etanercept) | Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis, plaque psoriasis and pediatric plaque psoriasis | European Commission granted marketing authorization for the biosimilar | 6/4/20 | Immune/Dermatalogic |
| Aclaris Therapeutics Inc., of Wayne, Pa. | ATI-450 | MK2 inhibitor | Hospitalized patients with COVID-19 | FDA cleared the IND for a phase IIa study with the primary endpoint of proportion of patients who are free from respiratory failure by day 14 | 6/17/20 | Infection |
| Agenus Inc., of Lexington, Mass. | AgenT-797 | Allogeneic invariant natural killer T cell-based immunotherapy | COVID-19 infection | FDA cleared IND to assess study drug in infected individuals with moderate to severe respiratory distress | 6/2/20 | Infection |
| Aicuris Anti-infective Cures GmbH, of Wuppertal, Germany | Pritelivir | Inhibits viral helicase-primase complex | Acyclovir-resistant mucocutaneous herpes simplex virus | FDA granted breakthrough therapy designation | 6/5/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | NMDA receptor antagonist | COVID-19 | FDA cleared IND for phase IIb/III study | 6/4/20 | Infection |
| Aligos Therapeutics Inc., of South San Francisco | ALG-010133 | Oligonucleotide S-antigen transport inhibiting oligonucleotide polymer | Chronic hepatitis B | Submitted clinical trial application for phase Ia/b study | 6/18/20 | Infection |
| Altimmune Inc., of Gaithersburg, Md. | T-COVID | Replication-deficient adenovirus 5 | Early COVID-19 | FDA cleared the IND for a 100-patient phase I/II study with a primary efficacy endpoint of patients with clinical worsening, defined as a 4% decrease in pulse oxygen saturation, or need for hospitalization; study scheduled to start in June with data in the fourth quarter of 2020 | 6/1/20 | Infection |
| Altimmune Inc., of Gaithersburg, Md. | Heptcell | Peptide-based immunotherapeutic | Chronic hepatitis B | FDA cleared IND for phase II trial | 6/22/20 | Infection |
| Ampio Pharmaceuticals Inc., of Englewood, Colo. | Ampion | Low-molecular-weight fraction of 5% human serum albumin | COVID-19 patients on supplemental oxygen | FDA cleared the IND for a phase I study | 6/19/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | Acute organ inflammation and injury in patients with COVID-19 | Submitted an IND for a phase II study to the FDA | 6/8/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | COVID-19 | FDA cleared firm to proceed with phase II trial testing drug in preventing acute lung and kidney injury in patients with COVID-19 | 6/16/20 | Infection |
| Basilea Pharmaceutical Ltd., of Basel, Switzerland | Cresemba (isavuconazole) | Antifungal | Invasive aspergillosis; mucormycosis in adult patients for whom amphotericin B is inappropriate | Russian Federation granted marketing authorization | 6/22/20 | Infection |
| Biophytis SA, of Paris | Sarconeos (BIO-101) | Proto-oncogene Mas agonist | Acute respiratory failure associated with COVID-19 | Received approval from the U.K. Medicines and Healthcare Products Regulatory Agency to start the study | 6/11/20 | Infection |
| Cellenkos Inc., of Houston | CK-0802 | Cryopreserved cord blood derived T regulatory cells | COVID-19 infection | FDA cleared IND to initiate phase I study in intubated individuals with acute respiratory distress syndrome; co-primary endpoints are toxicity and 28-day treatment success | 6/2/20 | Infection |
| Celltex Inc., of Houston | AdMSCs | Autologous adipose tissue-derived mesenchymal stem cells | COVID-19 | FDA cleared IND for phase II study testing safety and prophylactic efficacy; trial will enroll 200 patients who have never been infected with COVID-19 | 6/26/20 | Infection |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Lok (minocycline + edetate + ethyl alcohol) | 30S ribosomal protein inhibitor |
Bloodstream infections | FDA provided positive guidance on proposed catheter compatibility studies to treat central line-associated and catheter-related infections | 6/2/20 | Infection |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Induced mesenchymal stem cells | Induced mesenchymal stem cells | COVID-19 | Received written response from FDA regarding pre-IND to test iMSCs for treating and reducing severity of acute respiratory syndrome in COVID-19; agency said firm could apply for fast track designation and provided CMC requirements for proposed trials; company plans to initiate actions on FDA’s recommendations and follow up with IND application under the Coronavirus Treatment Acceleration Program | 6/26/20 | Infection |
| Curevac AG, of Tubingen, Germany | SARS-CoV-2 vaccine | mRNA vaccine | COVID-19 prophylaxis | German Health Authority Paul-Ehrlich-Institute and the Belgian Federal Agency for Medicines and Health Products approved the dose-escalation phase I trial in 168 subjects | 6/17/20 | Infection |
| Cytocom Inc., of Winter Park, Fla. | CYTO-201 | Inhibits replication of SARS-CoV-2 virus | COVID-19 | Completed type B pre-IND meeting with FDA in relation to planned phase II trial; based on agency’s feedback, company plans to revise protocol and quickly initiate enrollment | 6/29/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO 140) | CCR5 antagonist | HIV indication | Filed BLA with the FDA; requested a priority review | 6/1/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | Highly treatment-experienced HIV patients | Company expects to receive a PDUFA action date from the FDA on July 10 | 6/8/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | TSC (trans sodium crocetinate) | Oxygenation-enhancing product | COVID-19 | Submitted CTA to Romanian National Agency for Medicines and Medical Devices for a phase Ia/Ib trial in hospitalized COVID-19 patients displaying severe respiratory symptoms and low oxygen levels; assuming approval, 24-patient phase Ia portion set to start by end of second quarter of 2020 | 6/11/20 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody targeting Toll-like receptor 4 | Moderate to severe COVID-19 | Health Canada approved the start of the phase II/III study | 6/15/20 | Infection |
| Elixirgen Therapeutics Inc., of Baltimore | EXG-5003 | Self-replicating RNA vaccine expressing the receptor binding domain of the SARS-CoV-2 spike protein | COVID-19 prophylaxis | Completed pre-IND meeting with the FDA | 6/4/20 | Infection |
| EMD Serono, unit of Merck KGaA, of Darmstadt, Germany | M-5049 | Dual TLR-7/TLR-8 antagonist | COVID-19 infection | FDA cleared IND for phase II study assessing effect on preventing or ameliorating hyper-inflammatory response in COVID-19 pneumonia and preventing progression to cytokine storm; study expected to enroll ~150 participants in U.S. and Brazil, with top-line data expected by year-end 2020 | 6/25/20 | Infection |
| Enochian Biosciences Inc., of Los Angeles | ENOB-HV-01 | Genetically modified autologous stem cell | HIV infection | Company had an initial targeted engagement for regulatory advice meeting with the FDA | 6/8/20 | Infection |
| F2G Ltd., of Manchester, U.K. | Olorofim (formerly F-901318) | Dihydroorotate dehydrogenase inhibitor | Coccidioidomycosis and invasive aspergillosis, scedosporiosis, lomentosporiosis, fusariosis and disease due to scopulariopsis species | FDA granted qualified infectious disease product designation | 6/10/20 | Infection |
| FSD Pharma Inc., of Toronto | FSD-201 (ultramicronized palmitoylethanolamide) | Analgesic and anti-inflammatory molecule | COVID-19 | FDA cleared firm to submit IND to test FSD-201’s ability to avoid cytokine storm associated with acute lung injury in hospitalized COVID-19 patients | 6/3/20 | Infection |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | p38alpha/beta mitogen activated protein kinase inhibitor | COVID-19 | Submitted an IND to the FDA for a phase III study | 6/10/20 | Infection |
| Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | Oral p38?/? MAPK inhibitor | COVID-19 | Received early notification from FDA that it may proceed with initiating phase III trial in higher-risk hospitalized adults with COVID-19; Losvid trial will enroll about 400 patients; primary endpoint is proportion who progress to death or respiratory failure by day 28; an interim analysis will be conducted in the fourth quarter of 2020 for futility, and top-line data are expected in the first quarter of 2021 | 6/24/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Remdesivir | COVID19 non-structural protein 8 modulator/replicase polyprotein 1AB inhibitor; RNA polymerase | COVID-19 | EMA’s Committee for Medicinal Products for Human Use recommended conditionally approving the drug for use in patients, 12 and older, who require oxygen support | 6/26/20 | Infection |
| Glenmark Pharmaceuticals Ltd., of Mumbai, India | Favipiravir | RNA synthesis inhibitor | Mild to moderate COVID-19 | Drug got approved by Drug Controller General of India | 6/22/20 | Infection |
| Immune Therapeutics Inc., of Winter Park, Fla. | CYTO-201 (metenkefalin) | Immunotherapy | COVID-19 infection | Partner and former subsidiary Cytocom Inc. submitted IND application to FDA for open-label phase II study to assess CYTO-201 as adjunct to standard of care to mitigate disease progression | 6/9/20 | Infection |
| Immunic Inc., of New York | IMU-838 (vidofludimus) | Dihydroorotate dehydrogenase inhibitor | COVID-19 infection | FDA authorized initiation of phase II Calvid-1 trial at U.S. sites | 6/9/20 | Infection |
| Immunovative Therapies Ltd., of Jerusalem, and Mirror Biologics Inc., of Phoenix, Ariz. | Allostim | Universal antiviral vaccine | Respiratory viral infections, including COVID-19 and influenza | FDA cleared the phase I/II trial for healthy elderly adults | 6/23/20 | Infection |
| Immuron Ltd., of Melbourne, Australia | Undisclosed | Undisclosed | Acute infectious diarrhea | Research collaborator U.S. DoD Naval Medical Research Center requested pre-IND meeting with FDA on phase II development of oral therapeutic targeting Campylobacter and E. coli-based infections; IND application expected later this year, with 2 studies to commence during first half of 2021 | 6/9/20 | Infection |
| Kamada Ltd., of Rehovot, Israel | Plasma-derived IgG product | Plasma-derived IgG product | COVID-19 | Proposed protocol for a phase I/II trial submitted to the Israeli Ministry of Health; company expects to initiate the study during the third quarter of 2020 | 6/17/20 | Infection |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Mavrilimumab | Monoclonal antibody targeting granulocyte macrophage colony-stimulating factor receptor alpha | Severe COVID-19 pneumonia and hyperinflammation | FDA approved the IND for the phase II/III study | 6/8/20 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | Recarbrio (imipenem + cilastatin + relebactam) | Beta lactamase inhibitor; dehydropeptidase-1 inhibitor | Hospital-acquired and ventilator-associated bacterial pneumonia | FDA approved the expanded indication | 6/4/20 | Infection |
| Nabriva Therapeutics plc, of Dublin | Contepo (fosfomycin) | Antibiotic | Complicated urinary tract infections | Received complete response letter from FDA | 6/19/20 | Infection |
| Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holding AG, of Geneva | RLF-100 | Synthetic form of human vasoactive intestinal peptide | COVID-19 | FDA granted fast track designation for treatment of acute lung injury/acute respiratory distress syndrome associated with COVID-19 | 6/24/20 | Infection |
| Nuvox Pharma LLC of Tucson, Ariz. | NanO2 | Emulsion for oxygen delivery | COVID-19 with acute hypoxic respiratory failure | Health Canada issued a no objection letter for the phase IIa study | 6/30/20 | Infection |
| Opko Health Inc., of Miami | Rayaldee (calcifediol) | Prohormone of calcitriol | Mild to moderate COVID-19 | FDA authorized the phase II study | 6/1/20 | Infection |
| PTC Therapeutics Inc., of South Plainfield, N.J. | PTC-299 | Dihydroorotate dehydrogenase inhibitor | COVID-19 | FDA authorized starting the phase II/III study; CTA submitted in Spain; plans to file in Europe, Brazil and Australia | 6/17/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Yeliva (opaganib) | Sphingosine kinase-2 inhibitor | Hospitalized severe COVID-19 with pneumonia | Submitted a CTA to the Ministry of Health of the Russian Federation for a phase II/III study | 6/10/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Yeliva (opaganib) | Sphingosine kinase-2 inhibitor | Hospitalized COVID-19 patients with pneumonia | Submitted CTAs to the U.K. Medicines & Healthcare Products Regulatory Agency and the Italian Medicines Agency for a phase II/III study | 6/18/20 | Infection |
| Renibus Therapeutics Inc., of Dallas | RBT-6 | Stannous protoporphyrin | COVID-19 | The FDA granted fast track designation | 6/23/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative | COVID-19 | Filed a CTA with Health Canada and is finalizing IND with FDA for proposed phase III confirmatory trial in patients with mild to moderate COVID-19 due to the SARS-CoV-2 infection | 6/3/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative | COVID-19 | Pre-CTA meeting with Health Canada provided guidance for proposed clinical study design and information required for CTA submission; company aims to file IND with FDA in June 2020, followed by CTA in Canada for phase III study | 6/9/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative with 2 thiol groups | Mild to moderate COVID-19 | Submitted IND to the FDA for an 800-patient phase III study scheduled to start in the third quarter of 2020 | 6/30/20 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | Fetroja (cefiderocol) | Cephalosporin antibiotic | Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible gram-negative pathogens | FDA accepted supplemental NDA, granting priority review; PDUFA date of Sept. 27, 2020 | 6/1/20 | Infection |
| Sinovac Biotech Ltd., of Beijing | Quadrivalent influenza vaccine | Split virion influenza vaccine | Influenza infection | NMPA approved product in China to protect against 2 A strains and 2 B strains of influenza in individuals 3 years and older | 6/24/20 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Pa. | VP-102 (cantharidin 0.7% topical solution) | Protein phosphatase 2A inhibitor | Molluscum contagiosum | Received letter from FDA stating there are deficiencies in NDA that preclude discussion of labeling and postmarketing requirements/commitments at this time, though that does not reflect a final decision on the information under review; FDA previously assigned a PDUFA date of July 13, 2020, for completion of NDA review | 6/29/20 | Infection |
| Viiv Healthcare, of London | Tivicay PD (dolutegravir) | Integrase strand transfer inhibitor | HIV infection | FDA approved the tablets for oral suspension | 6/12/20 | Infection |
| Cynata Therapeutics Ltd., of Melbourne, Australia | CYP-004 | Mesenchymal stem cells | Osteoarthritis | Therapeutic Goods Administration advised the company that the study can be conducted under the Clinical Trial Notification scheme | 6/18/20 | Inflammatory |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Monoclonal antibody targeting interleukin-17A | Active non-radiographic axial spondyloarthritis | FDA approved the expanded indication | 6/17/20 | Inflammatory |
| Viela Bio Inc., of Gaithersburg, Md. | Uplizna (inebilizumab-cdon) | Humanized anti-CD19 monoclonal antibody | Neuromyelitis optica spectrum disorder | FDA approved for the treatment of adult patients who are anti-AQP4 antibody-positive as a twice-a-year maintenance regimen following initial doses | 6/12/20 | Inflammatory |
| Abbvie Inc., of North Chicago | Botox (onabotulinumtoxinA) | Acts on motor neurons to reduce muscle activity | Pediatric patients with neurogenic detrusor overactivity | FDA accepted sBLA. The Prescription Drug User Fee Act date is expected to be in the first quarter of 2021 following a standard 10-month review | 6/22/20 | Musculoskeletal |
| Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | Monoclonal antibody targeting IL-17A | Active non-radiographic axial spondyloarthritis | FDA approved the supplemental BLA | 6/1/20 | Musculoskeletal |
| Lupin Ltd., of Zug Switzerland | Mexiletine | Antimyotonic agent | Myotonic disorders | FDA granted orphan drug designation | 6/8/20 | Musculoskeletal |
| Merz Pharmaceuticals GmbH, of Frankfurt, Germany, and Teijin Pharma Ltd., of Tokyo | Xeomin (incobotulinumtoxinA) | Botulinum toxin type A | Upper limb spasticity | Teijin received marketing approval from Japan’s Ministry of Health, Labor and Welfare | 6/29/20 | Musculoskeletal |
| Pfenex Inc., of San Diego | Livogiva (PF-708) | Biosimilar to teriparatide injection Forsteo | Osteoporosis | EMA’s Committee for Medicinal Products for Human Use recommended approval, including for use in postmenopausal women with osteoporosis who are at high risk for having bone fractures | 6/26/20 | Musculoskeletal |
| PTC Therapeutics Inc., of South Plainfield, N.J. | Translarna (ataluren) | Protein restoration therapy | Duchenne muscular dystrophy | EMA’s Committee for Medicinal Products for Human Use recommended by a majority of votes to remove the statement "efficacy has not been demonstrated in non-ambulatory patients”; opinion is subject to final approval by European Commission | 6/29/20 | Musculoskeletal |
| Reneo Pharmaceuticals Inc., of San Diego | REN-001 | PPAR-delta agonist | Primary mitochondrial myopathies | FDA granted orphan drug designation | 6/23/20 | Musculoskeletal |
| Rocket Pharmaceuticals Inc., of New York | RPL-401 | Lentiviral vector-based gene therapy | Infantile malignant osteopetrosis | FDA cleared the IND for a phase I trial to enroll 2 pediatric patients, 1 month of age or older | 6/29/20 | Musculoskeletal |
| Santhera Pharmaceuticals AG, of Pratteln, Switzerland | Idebenone | Oxidoreductase inhibitor | Duchenne muscular dystrophy | U.K.’s MHRA renewed for a further year the Early Access to Medicines Scheme scientific opinion | 6/23/20 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | Casimersen (SRP-4045) | Phosphorodiamidate morpholino oligomer | Duchenne muscular dystrophy | Completed submission of rolling NDA to FDA seeking accelerated approval for use in DMD patients who have genetic mutations amenable to skipping exon 45 of the Duchenne gene | 6/26/20 | Musculoskeletal |
| Acadia Pharmaceuticals Inc., of San Diego | Nuplazid (pimavanserin) | Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors | Hallucinations and delusions associated with dementia-related psychosis | Submitted supplementary NDA to the FDA | 6/15/20 | Neurology/Psychiatric |
| Adamas Pharmaceuticals Inc., of Emeryville, Calif. | Gocovri (amantadine) | Reduces the amount of glutamate hyperactivity | Parkinson’s disease | FDA accepted the supplemental NDA as treatment of off episodes in patients receiving levodopa; PDUFA action date is Feb. 1 2021 | 6/4/20 | Neurology/Psychiatric |
| Akili Interactive Labs Inc., of Boston | Endeavorrx | Game-based digital therapeutic device | Prescription treatment for children with attention-deficit hyperactivity disorder | FDA cleared the digital therapeutic | 6/15/20 | Neurology/Psychiatric |
| Alterity Therapeutics Ltd., of Melbourne, Australia | ATH-434 | Inhibitor of alpha-synuclein aggregation | Multiple system atrophy | Reached agreement with the FDA on the nonclinical studies required for the phase II study; FDA agreed to key aspects of the study design, including the patient population, safety monitoring plan and strategy for evaluating drug exposure during the study | 6/29/20 | Neurology/Psychiatric |
| Anavex Life Sciences Corp., of New York | Anavex 2-73 (blarcamesine) | Activates sigma-1 receptor | Early Alzheimer’s disease | Health Canada issued a no objection letter for the phase IIb/III ANAVEX2-73-AD-004 study; Medicines and Healthcare products Regulatory Agency in the U.K. approved the CTA for the study | 6/4/20 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-05 | Oral NMDA receptor antagonist | Alzheimer’s disease agitation | FDA granted breakthrough therapy designation | 6/26/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Equfina (safinamide mesylate) | Selective MAO-B inhibitor | Parkinson’s disease | Eisai Korea Inc. subsidiary received marketing approval in South Korea for treating idiopathic Parkinson's disease as adjunctive therapy with levodopa-containing products in patients with end of dose motor fluctuations | 6/25/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | HTX-011 (bupivacaine + meloxicam) | Cyclooxygenase 2 inhibitor; sodium channel inhibitor | Management of postoperative pain | FDA issued complete response letter, seeking additional nonclinical information; company plans to request type A meeting to obtain agreement with the FDA for NDA resubmission | 6/29/20 | Neurology/Psychiatric |
| Intelgenx Corp., of Saint Laurent, Quebec | Rizaport Versafilm | Rizatriptan oral disintegrating film | Acute migraine | Obtained clarity from FDA during type A meeting to discuss March 27, 2020, complete response letter, regarding CMC information required for resubmission of NDA | 6/11/20 | Neurology/Psychiatric |
| Karuna Therapeutics Inc., of Boston | Karxt | Oral co-formulation of xanomeline (muscarinic receptor agonist) and trospium (muscarinic receptor antagonist) | Acute psychosis in patients with schizophrenia | Announced the completion of end-of-phase II meeting with the FDA that supports the progression of Karxt into phase III development | 6/23/20 | Neurology/Psychiatric |
| Neoimmunetech Inc., of Rockville, Md. | NT-I7 (efineptakin alfa) | Long-acting human IL-7 | Progressive multifocal leukoencephalopathy | FDA granted orphan drug designation | 6/15/20 | Neurology/Psychiatric |
| Novaremed AG, of Basel, Switzerland | NRD-135S.E1 | Allosteric modulator of Lyn kinase | Painful diabetic peripheral neuropathy | FDA approved the IND application to initiate a phase II study | 6/23/20 | Neurology/psychiatric |
| Ovid Therapeutics Inc., of New York | OV-101 (gaboxadol) | Delta-selective GABAA | Angelman syndrome | FDA granted rare pediatric disease designation | 6/19/20 | Neurology/Psychiatric |
| Pharnext SA, of Paris | PXT-3003 | Fixed-dose combination of baclofen, naltrexone and sorbitol | Charcot-Marie-Tooth type 1A | FDA agreed with company that a single pivotal phase III study plus previous studies would be sufficient to support an NDA; primary endpoint will be Overall Neuropathy Limitations Scale | 6/10/20 | Neurology/Psychiatric |
| Polyneuron Pharmaceuticals AG, of Basel, Switzerland | PN-1007 | Mimics the HNK-1 carbohydrate epitope found on myelin of peripheral nerves | Anti-MAG neuropathy | FDA granted orphan drug designation | 6/5/20 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland | Enspryng (satralizumab) | IL-6-targeting monoclonal antibody | Neuromyelitis optica spectrum disorder | Japan’s Ministry of Health, Labour and Welfare approved for the prevention of relapses of NMOSD, including NMO, for aquaporin-4 antibody seropositive adults and children | 6/29/20 | Neurology/Psychiatric |
| Sunovion Pharmaceuticals Inc., of Mississauga, Ontario | Kynmobi (apomorphine hydrochloride) | Dopamine agonist | Off episodes in patients with Parkinson’s disease | Health Canada approved the drug | 6/15/20 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | I.V. oliceridine | Mu receptor G protein pathway selective modulator | Moderate to severe acute pain | Chinese partner Jiangsu Nhwa Pharmaceutical Co. Ltd. said NMPA cleared start of clinical trials | 6/3/20 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (fenfluramine) | Antiseizure drug | Dravet syndrome | FDA approved for use in treating seizures associated with Dravet syndrome in patients, 2 and older | 6/25/20 | Neurology/Psychiatric |
| ZZ Biotech LLC, of Houston | 3K3A-APC | Genetically engineered variant of the naturally occurring activated protein C | Acute ischemic stroke | FDA granted fast track designation | 6/12/20 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Reactive aldehyde species inhibitor | Dry eye disease | FDA agreed to the use of reactive aldehyde species as an objective sign in clinical trials | 6/4/20 | Ocular |
| Allergan, a unit of North Chicago-based Abbvie, and Molecular Partners AG, of Zurich, Switzerland | Abicipar pegol | DARPin therapy | Wet age-related macular degeneration | FDA issued completed response letter to BLA, indicating the rate of intraocular inflammation observed following administration of 2 mg/0.05 mL results in unfavorable benefit-risk ratio | 6/26/20 | Ocular |
| Eyegate Pharmaceuticals Inc., of Waltham, Mass. | Ocular Bandage Gel | Modified form of natural polymer hyaluronic acid | Wound repair post photorefractive keratectomy and for dry eye with punctate epitheliopathies | Received feedback from FDA regarding requested packaging for eye drop; agency has provided path forward using the multi-dose preservative-free bottle, requesting some additional tests prior to fully approving bottle for use; tests expected to be completed in second half of 2020 | 6/11/20 | Ocular |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab) | Single-chain antibody fragment | Wet age-related macular degeneration | FDA approved label update to include additional safety information regarding retinal vasculitis and retinal vascular occlusion under “Warnings and Precautions”; update also specifies that those adverse reactions are part of a spectrum of intraocular inflammation rates from phase III Hawk and Harrier trials | 6/11/20 | Ocular |
| Novartis AG, of Basel, Switzerland | Xiidra (lifitegrast) | Small-molecule integrin antagonist | Moderate to severe dry eye disease | Withdrew EMA MAA | 6/26/20 | Ocular |
| Reneuron Group plc, of London | hRPC stem cell therapy | Retinal progenitor cells | Retinitis pigmentosa | FDA approved the expansion of the phase IIa study to add an additional 9 patients at higher doses; U.K.'s Medicines and Healthcare products Regulatory Agency approved opening a clinical site for the study; plans to seek regulatory approval in the second half of 2021 | 6/17/20 | Ocular |
| Sparingvision SAS, of Paris | SPVN-06 | NXNL1 gene stimulator; AAV-based gene therapy | Inherited retinal dystrophies | European Commission granted orphan designation | 6/30/20 | Ocular |
| Longeveron LLC, of Miami | Mesenchymal stem cells | Stem cells from bone marrow of young healthy adult donors | Aging frailty | Japan's Pharmaceutical and Medical Devices Agency approved the clinical trial notification for a phase II study | 6/1/20 | Other/Miscellaneous |
| Novartis AG, of Basel, Switzerland | Xolair (omalizumab) | Anti-IgE antibody | Severe chronic rhinosinusitis with nasal polyps | EMA’s Committee for Medicinal Products for Human Use recommended approval as add-on therapy with intranasal corticosteroids (INCs) to treat adults for whom therapy with INC does not provide adequate disease control | 6/26/20 | Other/Miscellaneous |
| Saniona AB, of Copenhagen | Tesomet (tesofensine + metoprolol) | Beta 1 adrenoceptor antagonist | Prader-Willi syndrome | During pre-IND meeting, FDA endorsed proposed filing via 505(b)2 pathway and indicated agreement on proposed design, doses and duration of potential phase IIb trial | 6/9/20 | Other/Miscellaneous |
| Sound Pharmaceuticals Inc., of Seattle | SPI-1005 (ebselen) | Glutathione peroxidase stimulator | Meniere's disease | FDA approved the IND for the pivotal phase III STOPMD-3 study | 6/8/20 | Other/Miscellaneous |
| Insmed Inc., of Bridgewater, N.J. | Brensocatib (INS-1007) | Dipeptidyl peptidase 1 inhibitor | Non-cystic fibrosis bronchiectasis | FDA granted breakthrough therapy designation | 6/8/20 | Respiratory |
| Novartis AG, of Basel, Switzerland | Atectura | Fixed-dose combination of indacaterol acetate, mometasone furoate | Asthma | Approved by Japan’s Ministry of Health, Labour and Welfare | 6/29/20 | Respiratory |
| Novartis AG, of Basel, Switzerland | Enerzair | Fixed-dose combination of glycopyrronium bromide, indacaterol acetate, mometasone furoate | Asthma | Approved by Japan’s Ministry of Health, Labour and Welfare | 6/29/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | Cystic fibrosis transmembrane conductance regulator potentiator | Cystic fibtosis | European Commission granted approval of the label extension to include children 6 months and older with a R117H mutiation in CFTR | 6/10/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kaftrio (ivacaftor/tezacaftor/elexacaftor) | Combination of CFTR potentiator and 2 CFTR correctors | Cystic fibrosis | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in a combination regimen with Kalydeco (ivacaftor) 150 mg to treat patients, 12 and older, with 1 F508del mutation and 1 minimal function mutation or 2 F508del mutations in the CFTR gene | 6/26/20 | Respiratory |
| Braeburn Inc., of Plymouth Meeting, Pa. | Brixadi (buprenorphine) | ORL1 receptor agonist; opioid receptor mu modulator | Opioid use disorder | Final approval requested from FDA of NDA covering weekly and monthly extended-release formulations of drug; 6-month review cycle expected | 6/1/20 | Toxicity and Intoxication |
| Helsinn Healthcare SA, of Lugano, Switzerland | Akynzeo (fosnetupitant + palonosetron) | 5-HT 3 receptor antagonist; NK1 receptor antagonist | Chemotherapy-induced nausea and vomiting | FDA approved ready-to-dilute liquid formulation | 6/2/20 | Toxicity and Intoxication |
| Pear Therapeutics Inc., of Boston | Reset | Prescription digital therapeutic | Substance use disorder | Health Science Authority of Singapore authorized the treatment | 6/18/20 | Toxicity and Intoxication |
| Synthetic Biologics Inc., of Rockville, Md. | SYN-020 | Purified recombinant bovine intestinal alkaline phosphatase formulated for oral delivery to intestines | Radiation enteropathy secondary to pelvic cancer therapy | Submitted IND to FDA for phase I single ascending-dose study in healthy volunteers | 6/30/20 | Toxicity and Intoxication |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 (repurposed ondansetron) | 5-HT 3 receptor antagonist | Alcohol use disorder | Received all necessary approvals to commence the Onward pivotal phase III trial in Poland | 6/12/20 | Toxicity/intoxication |
| ANI Pharmaceuticals, of Baudette, Minn. | Cortrophin Gel | Repository corticotropin | Undisclosed | Company forgoing a request for a type A meeting with the FDA and plans to address items identified in the refuse to file letter in a resubmission of its supplementary NDA | 6/1/20 | Unknown |
| Adaptimmune Therapeutics plc, of Oxfordshire, U.K. | ADP-A2M4 | Melanoma associated antigen 4 modulator | Synovial sarcoma | EMA granted PRIME designation | 7/23/20 | Cancer |
| ADC Therapeutics SA, of Lausanne, Switzerland | Camidanlumab tesirine | IL-2 receptor alpha subunit inhibitor | Hodgkin lymphoma | FDA lifted partial hold on pivotal phase II study in individuals with relapsed or refractory disease who failed 3 prior lines of therapy | 7/6/20 | Cancer |
| Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1702 (Cevira) | Hexaminolevulinate photodynamic drug-device therapy | Cervical dysplasia | China NMPA granted CTA for global phase III trial for non-surgical treatment of high-grade disease | 7/8/20 | Cancer |
| Astellas Pharma Inc., of Tokyo | Xospata (gilteritinib) | FLT3 inhibitor | Relapsed/refractory FLT3 mutation-positive acute myelogenous leukemia | NICE recommended coverage of the drug | 7/16/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | First-line extensive-stage small-cell lung cancer | EMA's Committee for Medicinal Products for Human Use issued a positive opinion for use of the drug in combination with a choice of chemotherapies, etoposide plus either carboplatin or cisplatin | 7/27/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Tagrisso (osimertinib) | EGFR antagonist | Early stage EGFR-mutated non-small-cell lung cancer | FDA granted breakthrough therapy designation for use in patients after complete tumor resection with curative intent | 7/30/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Pancreatic cancer | EU approved drug as monotherapy for maintenance treatment of adults with germline BRCA1/2 mutations and metastatic disease who did not progress after minimum of 16 weeks of platinum treatment within first-line chemotherapy regimen | 7/8/20 | Cancer |
| Beigene Ltd., of Beijing | Tislelizumab | PD-1 inhibitor | Hepatocellular carcinoma | China NMPA's Center for Drug Evaluation accepted sNDA for treatment of previously treated unresectable disease | 7/1/20 | Cancer |
| Beigene Ltd., of Beijing | Pamiparib | PARP1/2 inhibitor | Ovarian, fallopian tube or primary peritoneal cancer | China’s NMPA accepted for review the NDA seeking approval to treat patients with deleterious or suspected deleterious germline BRCA-mutated advanced disease who have been treated with 2 or more lines of chemotherapy | 7/17/20 | Cancer |
| Beigene Ltd., of Beijing | Pamiparib | PARP1 and PARP2 inhibitor | Third-line advanced ovarian, fallopian tube or primary peritoneal cancer with deleterious or suspected deleterious germline BRCA mutations | Center for Drug Evaluation of the China National Medical Products Administration granted a priority review | 7/27/20 | Cancer |
| Black Diamond Therapeutics Inc., of Cambridge, Mass. | BDTX-189 | Irreversible small-molecule inhibitor | Solid tumors | FDA granted fast track for use in patients with solid tumors harboring an allosteric HER2 mutation or an EGFR or HER2 exon 20 insertion mutation who have progressed following prior treatment and who have no satisfactory treatment options | 7/28/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Pralsetinib | RET inhibitor | Advanced or metastatic RET mutant medullary thyroid cancer and RET fusion-positive thyroid cancers | Filed NDA with the FDA | 7/1/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | KIT tyrosine kinase inhibitor | Gastrointestinal stromal tumors | EMA's Committee for Medicinal Products for Human use issued positive opinion for monotherapy treatment of adults with unresectable or metastatic disease harboring PDGFRA D842V mutation | 7/24/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Lisocabtagene maraleucel | CD19-directed CAR T-cell therapy | Relapsed or refractory diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma and follicular lymphoma grade 3B | EMA validated the MAA seeking approval for use in adults after at least 2 prior therapies; centralized review process begins | 7/17/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. | Idecabtagene vicleucel (bb-2121) | BCMA-directed CAR T-cell immunotherapy | Relapsed and refractory multiple myeloma | Submitted BLA to FDA; submission provides further details on the CMC module to address outstanding regulatory requests from the agency in May 2020 refuse-to-file letter following original BLA submission in March 2020 | 7/30/20 | Cancer |
| Cellectis Inc., of New York | UCARTCS-1A | Allogeneic, gene-edited T-cell product targeting CS1/SLAMF7 | Relapsed and refractory multiple myeloma | FDA placed the Melani-01 study on clinical hold after a patient treated at dose level 2 experienced a fatal treatment-emergent adverse event of cardiac arrest; immediate and underlying causes of the event are being collected | 7/6/20 | Cancer |
| Celyad Oncology SA, of Mont-Saint-Guibert, Belgium | CYAD-211 | Short hairpin RNA-based allogeneic CAR T targeting BCMA | Relapsed/refractory multiple myeloma | FDA cleared IND for phase I trial | 7/14/20 | Cancer |
| Checkmate Pharmaceuticals Inc., of Cambridge, Mass. | CMP-001 | Toll-like receptor 9 agonist | Melanoma | FDA granted fast track designation for the drug in combination with a PD-1-blocking antibody for initial treatment of patients with unresectable stage III or stage IV melanoma and for unresectable or metastatic melanoma refractory to prior anti-PD-1 blockade | 7/27/20 | Cancer |
| Cybrexa Therapeutics Inc., of New Haven, Conn. | CBX-12 | Peptide-drug conjugate targeting acidic cell surfaces | Advanced solid tumors | After a pre-IND meeting with the FDA, company plans to submit an IND in the fourth quarter of 2020 and start a phase I/II study in the first half of 2021 | 7/7/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Trastuzumab deruxtecan | ERBB2 tyrosine kinase receptor inhibitor; topoisomerase I inhibitor | HER2-positive breast cancer | EMA validated MAA to treat adults with unresectable or metastatic disease who received 2 or more prior anti-HER2 regimens; EMA's CHMP to review under accelerated assessment | 7/6/20 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Qinlock (ripretinib) | Switch-control tyrosine kinase inhibitor | Advanced gastrointestinal stroma tumor | Australia’s TGA approved for use in adults with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib; application approved under Project Orbis | 7/14/20 | Cancer |
| Denovo Biopharma LLC, of San Diego | DB-102 (enzastaurin) | Small-molecule serine/threonine kinase inhibitor of PKC beta, PI3K and AKT pathways | Newly diagnosed glioblastoma | FDA granted fast track designation | 7/17/20 | Cancer |
| Eisai Co. Ltd., of Tokyo, and Merck & Co. Inc., of Kenilworth, N.J. | Lenvima (lenvatinib mesylate) | Multiple receptor tyrosine kinase inhibitor | Unresectable thymic carcinoma | Submitted a marketing application in Japan | 7/31/20 | Cancer |
| Fate Therapeutics Inc., of San Diego | FT-819 | Allogeneic CAR T targeting CD19 | Advanced B-cell leukemias and lymphomas | FDA cleared the IND for a phase I study | 7/9/20 | Cancer |
| Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan | Camcevi (FP-001) | 6-month depot formulation of leuprolide mesylate | Advanced prostate cancer | Submitted NDA to FDA under 505(b)(2) pathway, seeking approval as palliative treatment | 7/27/20 | Cancer |
| Genor Biopharma Co. Inc., of Hong Kong | Geptanolimab | PD-1 monoclonal antibody | Peripheral T-cell lymphoma | China’s NMPA accepted NDA | 7/21/20 | Cancer |
| Geron Corp., of Menlo Park, Calif. | Imetelstat | Telomerase inhibitor | Myelodysplastic syndrome | EMA Committee for Orphan Medicinal Products issued positive opinion on orphan drug designation in indication | 7/1/20 | Cancer |
| Glaxosmithkline plc, of London | Belantamab mafodotin | Antibody-drug conjugate targeting B-cell maturation antigen | Relapsed or refractory multiple myeloma | FDA's Oncologic Drugs Advisory Committee voted 12-0 that the benefit of the treatment outweigh the risks for use after at least 4 prior therapies including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody | 7/14/20 | Cancer |
| Hutchison China Meditech Ltd., of Hong Kong | Savolitinib | Small-molecule MET inhibitor | Non-small-cell lung cancer | China’s NMPA granted priority review status to the NDA seeking approval for use in treating NSCLC with MET exon 14 skipping mutations | 7/28/20 | Cancer |
| Isa Pharmaceuticals BV, Leiden, the Netherlands | ISA-101b | Immunotherapy targeting oncogenic E6 and E7 proteins of HPV16 | Human papillomavirus type 16-positive cervical cancer | FDA granted orphan drug designation | 7/1/20 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | Exportin 1 inhibitor | Multiple myeloma | FDA accepted sNDA; decision expected by end of first quarter of 2021 | 7/20/20 | Cancer |
| Kite Pharma Inc., a unit of Foster City, Calif.-based Gilead Sciences Inc. | Tecartus (brexucabtagene autoleucel) | Autologous, anti-CD19 CAR T-cell therapy | Relapsed or refractory mantle cell lymphoma | FDA granted accelerated approval for the treatment | 7/24/20 | Cancer |
| Kite Pharma, a unit of Gilead Sciences Inc., of Foster City, Calif. | Tecartus (brexucabtagene autoleucel, KTE-X19) | Cell-based gene therapy | Mantle cell lymphoma | FDA approved for adults with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment | 7/24/20 | Cancer |
| Lintonpharm Co. Ltd., of Guangzhou, China | Catumaxomab | T-cell engaging bispecific antibody | Peritoneal carcinomatosis | China’s NMPA cleared the start of a phase III trial | 7/15/20 | Cancer |
| Medivir AB, of Stockholm | MIV-818 (liver-targeted troxacitabine prodrug) | DNA polymerase inhibitor | Hepatocellular carcinoma | European Commission granted orphan medicinal product designation | 7/3/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Relapsed or refractory classical Hodgkin lymphoma | FDA accepted the supplemental BLA and granted a priority review; PDUFA action date is Oct. 30, 2020 | 7/9/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | MK-6482 | HIF-2-alpha inhibitor | Renal cell carcinoma | FDA granted breakthrough therapy designation for treatment of Von Hippel-Lindau (VHL) disease-associated RCC with certain nonmetastatic tumors and of VHL disease | 7/29/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 antibody | Triple-negative breast cancer | FDA accepted for priority review the BLA seeking approval in combination with chemotherapy to treat patients with locally recurrent unresectable or metastatic disease whose tumors express PD-L1 | 7/30/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J., and Eisai Co. Ltd., of Tokyo | Keytruda (pembrolizumab) + Lenvima (lenvatinib) | PD-1 inhibitor + FGF1/2/3/4 receptor antagonist | Hepatocellular carcinoma | FDA issued CRL for application seeking accelerated approval of drug combination to treat first-line unresectable disease, citing no meaningful advantage over available therapies; companies plan to continue advancing combo through phase III program | 7/8/20 | Cancer |
| Merus NV, of Utrecht, the Netherlands | Zenocutuzumab | Bispecific antibody targeting HER2 and HER3 receptors | Pancreatic cancer | FDA granted orphan drug designation | 7/27/20 | Cancer |
| Metvital Inc., of San Diego | Anhydrous enol-oxaloacetate | AMP activated protein kinase stimulator | Glioblastoma | FDA granted fast track designation to treat people with newly diagnosed disease | 7/1/20 | Cancer |
| Miragen Therapeutics Inc., of Boulder, Colo. | Cobomarsen | miR-155 inhibitor | T-cell lymphoma | FDA granted orphan drug designation | 7/23/20 | Cancer |
| Neoimmunetech Inc., of Rockville, Md. | NT-I7 (efineptakin alfa) | Long-acting human IL-7 | Gastric, gastroesophageal junction or esophageal adenocarcinoma | FDA cleared IND application for phase II combination study with Opdivo (nivolumab, Bristol Myers Squibb Co.) in people with advanced or metastatic disease | 7/20/20 | Cancer |
| Neoimmunetech Inc., of Rockville, Md. | NT-I7 (efineptakin alfa) | Long-acting human IL-7 | Locally recurrent squamous cell carcinoma of head and neck | FDA cleared the IND for a window-of-opportunity study | 7/27/20 | Cancer |
| Novartis AG, of Basel, Switzerland | Piqray (alpelisib) | Phosphoinositide-3 kinase alpha inhibitor | Breast cancer | European Commission approved in combination with fulvestrant to treat men and postmenopausal women with HR+/HER2- locally advanced or metastatic disease with PIK3CA mutation after disease progression following endocrine therapy | 7/29/20 | Cancer |
| Oncoceutics Inc., of Philadelphia | ONC-201 | Dopamine receptor D2 antagonist; ClpP agonist | Glioma | FDA granted rare pediatric disease designation to treat H3 K27M-mutant disease | 7/21/20 | Cancer |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo | Inqovi (decitabine and cedazuridine) | Tablet formulation of hypomethylating agent and cytidine deaminase inhibitor | Myelodysplastic syndromes and chronic myelomonocytic leukemia | Approved by FDA; application was granted priority review and reviewed as part of Project Orbis | 7/7/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cypcaps | Cell-in-a-box encapsulated cell product | Locally advanced, inoperable pancreatic cancer | Manufacturing partner Austrianova submitted a drug master file to the FDA | 7/27/20 | Cancer |
| Pieris Pharmaceuticals Inc., of Boston | PRS-343 | 4-1BB/HER2 fusion protein | HER2-positive solid tumors | FDA placed partial clinical hold on phase I studies, while company conducts additional in-use and compatibility study requested by agency; currently enrolled patients may continue to receive treatment but no additional patients are to be enrolled until resolution of partial hold | 7/20/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Ventana HER2 Dual ISH DNA Probe Cocktail assay | Companion diagnostic | Breast cancer | Approved by FDA for use in Herceptin (trastuzumab) therapy for detecting HER2 biomarker in breast cancer | 7/29/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Tecentriq plus Cotellic and Zelboraf | Atezolizumab plus cobimetinib and vemurafenib | BRAF V600 mutation-positive advanced melanoma | FDA approved it | 7/31/20 | Cancer |
| Senhwa Biosciences Inc., of Taipei, Taiwan | Silmitasertib | Casein kinase 2 inhibitor | Recurrent sonic hedgehog medulloblastoma | FDA granted rare pediatric disease designation | 7/7/20 | Cancer |
| Specialised Therapeutics Asia Pte Ltd., of Singapore, and Puma Biotechnology Inc., of Los Angeles | Nerlynx (neratinib) | HER2-targeting therapy | Breast cancer | Approved in Malaysia for use in women who have had surgery, chemotherapy and prior trastuzumab-based therapy | 7/16/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Pevonedistat | NEDD8-activating enzyme inhibitor | Higher-risk myelodysplastic syndromes | FDA granted breakthrough therapy designation | 7/30/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Single-domain antibody targeting PD-L1 | Undifferentiated pleomorphic sarcoma and myxofibrosarcoma | Filed IND with the FDA for the pivotal Envasarc study | 7/16/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Brilinta (ticagrelor) | P2Y12 ADP-receptor inhibitor | Acute ischemic stroke or transient ischemic attack | FDA accepted the supplemental NDA for the reduction of subsequent strokes and granted a priority review; PDUFA action date in fourth quarter of 2020 | 7/9/20 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Acute myocardial infarction or heart attack | FDA granted fast track designation | 7/16/20 | Cardiovascular |
| Chemocentryx Inc., of Mountain View, Calif. | Avacopan | C5a receptor inhibitor | ANCA-associated vasculitis | Submitted NDA to the FDA | 7/9/20 | Cardiovascular |
| Janone Inc., of Las Vegas | TV-1001SR | Sodium nitrate tablets | Peripheral artery disease | Received confirmation from FDA of the transfer of the IND previously held by Soin Neuroscience | 7/21/20 | Cardiovascular |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Rilonacept | Recombinant fusion protein that blocks interleukin-1 alpha and beta signaling | Pericarditis | FDA granted orphan drug designation | 7/16/20 | Cardiovascular |
| Merck & Co. Inc., of Kenilworth, N.J., and Bayer AG, of Leverkusen, Germany | Vericiguat | Guanylate cyclase stimulator | Symptomatic chronic heart failure with reduced ejection fraction | FDA accepted the NDA with a priority review; PDUFA target date is Jan. 20, 2021 | 7/16/20 | Cardiovascular |
| Milestone Pharmaceuticals Inc., of Montreal | Etripamil | Calcium channel inhibitor | Paroxysmal supraventricular tachycardia | FDA agreed with plan to modify ongoing NODE-301B (Rapid) study, including primary endpoint of time to conversion within 30 minutes, to complete pivotal program efficacy requirements for NDA filing; enrollment expected to reopen by year-end, with results expected by early 2022 | 7/23/20 | Cardiovascular |
| Myokardia Inc., of Brisbane, Calif. | Mavacamten | Myosin inhibitor | Hypertrophic cardiomyopathy | FDA granted breakthrough therapy designation | 7/23/20 | Cardiovascular |
| Sanifit Therapeutics SA, of Palma, Spain | SNF-472 (myo-inositol hexaphosphate) | Calcification inhibitor | Peripheral artery disease | Based on FDA feedback, single pivotal study in individuals with end-stage kidney disease with PAD planned for 2021 following small cross-sectional study expected to begin in third quarter of 2020 | 7/20/20 | Cardiovascular |
| Scpharmaceuticals Inc., of Burlington, Mass. | Furoscix | Furosemide solution formulated for use with a wearable subcutaneous injector | Congestion in patients with heart failure | Resubmitted the 505(b)(2) NDA to the FDA; addressed concerns raised in the June 2018 complete response letter | 7/1/20 | Cardiovascular |
| Scpharmaceuticals Inc., of Burlington, Mass. | Furoscix | Furosemide solution formulated to a neutral pH | Worsening heart failure due to congestion | FDA accepted the resubmitted NDA as a class 2 response and assigned a PDUFA action date of Dec. 30, 2020 | 7/27/20 | Cardiovascular |
| Aclaris Therapeutics Inc., of Wayne, Pa. | ATI-1777 | JAK 1/3 kinase inhibitor | Atopic dermatitis | IND application to treat moderate to severe disease submitted to FDA | 7/7/20 | Dermatologic |
| Anaptysbio Inc., of San Diego | Imsidolimab | Monoclonal antibody targeting interleukin-36 receptor | Generalized pustular psoriasis | FDA granted orphan drug designation | 7/8/20 | Dermatologic |
| Botanix Pharmaceuticals Ltd., of Philadelphia and Sydney | BTX-1503 | Transdermal gel | Acne | Completed end-of-phase II meeting with FDA, which provided feedback on development program and reached agreement on required co-primary endpoints for phase III studies | 7/17/20 | Dermatologic |
| Concert Pharmaceuticals Inc., of Lexington, Mass. | CTP-543 (deuterium-modified ruxolitinib analog) | JAK 1/2 kinase inhibitor | Alopecia areata | FDA granted breakthrough therapy designation | 7/8/20 | Dermatologic |
| Croma-Pharma GmbH, of Leobendorf, Austria | LetibotulinumtoxinA | Botulinum toxin A stimulator | Frown lines | Drug file submitted to Germany's BfArM | 7/8/20 | Dermatologic |
| Endo International plc., of Dublin | Qwo (collagenase clostridium histolyticum-aaes) | Combination of bacterial collagenases | Moderate to severe cellulite in the buttocks | FDA approved the drug | 7/6/20 | Dermatologic |
| Janssen Pharmaceutical Cos., of Beerse, Belgium, part of Johnson & Johnson | Stelara (ustekinumab) | Targets IL-23/IL-12 pathway | Moderate to severe plaque psoriasis | FDA approved for the treatment of pediatric patients with moderate to severe plaque psoriasis | 7/31/20 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Tralokinumab | Monoclonal antibody targeting interleukin-13 | Moderate to severe atopic dermatitis | FDA accepted the BLA; PDUFA action date in the second quarter of 2021 | 7/9/20 | Dermatologic |
| MC2 Therapeutics A/S, of Copenhagen | Wynzora (calcipotriol/betamethasone) | Vitamin D/steroid | Plaque psoriasis | Submitted MAA to the EMA | 7/16/20 | Dermatologic |
| MC2 Therapeutics A/S, of Copenhagen | Wynzora (calcipotriene + betamethasone dipropionate) | Vitamin D3 receptor agonist | Plaque psoriasis | FDA approved to treat adults 18 and older | 7/20/20 | Dermatologic |
| Acasti Pharma Inc., of Laval, Québec | Capre (omega-3 phospholipid) | Purified from krill oil | Severe hypertriglyceridemia | Filed the prespecified statistical analysis plan for the phase III Trilogy 2 study with the FDA | 7/31/20 | Endocrine/Metabolic |
| Adocia SA, of Lyon, France | BC Lispro | Insulin | Type 1 diabetes | Cleared by FDA and German Federal Institute for Drugs and Medical Devices to initiate comparative study to demonstrate safety and efficacy of BC Lispro with insulin lispro manufactured by Tonghua Dongbao | 7/30/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | RNAi therapeutic | Primary hyperoxaluria type 1 | U.K.’s Medicines and Healthcare products Regulatory Agency granted a positive scientific opinion trough EAMS (early access to medicines scheme), allowing eligible PH1 patients to gain access to drug prior to marketing authorization | 7/13/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Growth hormone deficiency | EMA's pediatric committee issued positive opinion on agreement with proposed pediatric investigation plan to treat individuals from 6 months to <18 years of age; MAA submission on track for third quarter of 2020, with no additional studies required | 7/6/20 | Endocrine/Metabolic |
| Crinetics Pharmaceuticals Inc., of San Diego | Paltusotine | Nonpeptide-biased agonist of the somatostatin type 2 receptor | Acromegaly | FDA granted orphan drug designation | 7/8/20 | Endocrine/Metabolic |
| Diurnal Group plc, of London | Ditest | Native oral testosterone formulation | Male primary and secondary hypogonadism | Received FDA feedback from pre-IND meeting, confirming that drug can progress to NDA via 505(b)(2) pathway | 7/30/20 | Endocrine/Metabolic |
| Fortress Biotech Inc., of New York | CUTX-101 | Copper histidinate | Menkes disease | Received a positive opinion on orphan drug designation from the EMA | 7/31/20 | Endocrine/metabolic |
| Hemoshear Therapeutics Inc., of Charlottesville, Va. | HST-5040 | Oral, small molecule | Methylmalonic acidemia and propionic acidemia | Received FDA fast track and rare disease designations | 7/28/20 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin | Diabetes | Based on an end-of-phase II meeting with the FDA, the company plans to run 2 concurrent phase III studies | 7/15/20 | Endocrine/Metabolic |
| Orchard Therapeutics plc, of London | OTL-203 | IDUA gene stimulator |
Mucopolysaccharidosis type I | FDA granted orphan drug and rare pediatric disease designations in indication | 7/20/20 | Endocrine/Metabolic |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Chaperonin/Hsp70 stimulator | Niemann-Pick disease type C | Completed rolling submission of NDA to FDA | 7/20/20 | Endocrine/Metabolic |
| Poxel SA, of Lyon, France, and Sumitomo Dainippon Pharma Ltd., of Osaka, Japan | Imeglimin | Tetrahydrotriazine compound | Type 2 diabetes | Sumitomo submitted Japanese NDA | 7/30/20 | Endocrine/Metabolic |
| Protokinetix Inc., of Marietta, Ohio | PKX-001 | AAGP molecule | Type 1 diabetes | Submitted amendments to Health Canada; upon approval, company will deliver 75 grams of drug to University of Alberta to accommodate ongoing trial; study will treat islet cells prior to transplantation in type 1 diabetes patients | 7/13/20 | Endocrine/Metabolic |
| Rhythm Pharmaceuticals, Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Pro-opiomelanocortin deficiency obesity and leptin receptor deficiency obesity | Submitted MAA to the EMA; FDA granted granted rare pediatric disease designation | 7/1/20 | Endocrine/Metabolic |
| Seelos Therapeutics Inc., of New York | SLS-005 | Transcription factor EB stimulator | Sanfilippo syndrome | EMA’s Committee for Orphan Medicinal Products issued positive opinion on orphan designation | 7/21/20 | Endocrine/Metabolic |
| Chemomab Ltd., of Tel Aviv, Israel | CM-101 | Humanized monoclonal antibody target CCL24 | Primary sclerosing cholangitis | Received approvals in the U.K. and Israel for a phase IIa trial | 7/21/20 | Gastrointestinal |
| Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Nonalcoholic steatohepatitis; primary biliary cholangitis; primary sclerosing cholangitis | FDA lifted clinical hold on INDs; development to resume initially in PBC | 7/23/20 | Gastrointestinal |
| Immuron Ltd., of Melbourne | Campylobacter jejuni/Escherichia coli vaccine | Not disclosed | Moderate to severe diarrhea | Naval Medical Research Center received written guidance from FDA on clinical development pathway, supporting planned IND submission for 2 phase II trials expected to be conducted in 2021 | 7/20/20 | Gastrointestinal |
| Napo Pharmaceuticals Inc., wholly owned subsidiary of Jaguar Health Inc., of San Francisco | Mytesi (crofelemer) | Calcium activated chloride channel inhibitor | Diarrhea | IND filing to FDA completed for planned indication of prophylaxis and symptomatic relief for cancer therapy?related condition in adults | 7/6/20 | Gastrointestinal |
| Vanda Pharmaceuticals Inc., of Washington | Tradipitant | NK-1R antagonist | Gastroparesis | FDA authorized individual patient expanded access protocol | 7/14/20 | Gastrointestinal |
| Celltrion Healthcare, of Incheon, South Korea | Remsima (infliximab, CT-P13) | Subcutaneous biosimilar of Remicade (Johnson & Johnson) | Ankylosing spondylitis, Crohn’s disease, ulcerative colitis, psoriatic arthritis and psoriasis | European Commission granted marketing application for the drug | 7/27/20 | Gastrointestinal/Muskuloskeletal/Immune |
| Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Voclosporin | Calcineurin inhibitor | Lupus nephritis | FDA accepted NDA filing, granting priority review and assigning PDUFA date of Jan. 22, 2021 | 7/22/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Dublin | Terlipressin | Vasopressin analogue selective for V1 receptors | Hepatorenal syndrome type 1 | FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 8-7 in favor of recommending approval; PDUFA date is Sept. 12, 2020 | 7/16/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc., of Staines-upon-Thames, U.K. | Terlipressin | Vasopressin analogue selective for V1 receptor | Hepatorenal syndrome type 1 | FDA plans to hold a virtual meeting of the Cardiovascular and Renal Drugs Advisory Committee to review the NDA | 7/7/20 | Genitourinary/Sexual Function |
| Mundipharma International Ltd., of Cambridge, U.K., and Napp Pharmaceuticals Ltd., of Cambridge, U.K. | Invokana (canagliflozin) | SGLT2 inhibitor | Diabetic kidney disease | European Commission approved extended indication in people with type 2 diabetes | 7/1/20 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva, and Hangzhou Yuyuan Bioscience Technology Co. Ltd., of Hangzhou, China | Nolasiban | Oxytocin receptor antagonist | In vitro fertilization | Submitted a pre-IND meeting request to the Center for Drug Evaluation at the Chinese National Medical Products Administration | 7/1/20 | Genitourinary/Sexual Function |
| Regulus Therapeutics Inc., of La Jolla, Calif. | RGLS-4326 | Anti-miR-17 antisense oligonucleotide inhibitor | Autosomal dominant polycystic kidney disease | FDA granted orphan drug designation | 7/29/20 | Genitourinary/Sexual Function |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Non-absorbed, oral polymer | Metabolic acidosis in chronic kidney disease | FDA found deficiencies in the NDA that preclude discussion of labeling and postmarketing requirements/commitments at this time | 7/15/20 | Genitourinary/Sexual Function |
| Ascentage Pharma Group International, of Suzhou, China, | APG-2575 | Bcl-2 inhibitor | Waldenström macroglobulinemia | FDA granted orphan drug designation | 7/15/20 | Hematologic |
| CSL Behring LLC, of Hattersheim, Germany | Idelvion (albutrepenonacog alfa) | Coagulation factor modulator; factor IX agonist | Factor IX deficiency |
EMA updated routine prophylaxis regimen, approving 21-day dosing intervals for adults with hemophilia B | 7/3/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Xyndari | L-glutamine oral powder | Sickle cell disease | EMA issued scientific advice regarding the clinical development pathway | 7/29/20 | Hematologic |
| Imago Biosciences Inc., of South San Francisco | IMG-7289 (bomedemstat) | LSD1 inhibitor | Myelofibrosis | EMA granted access to PRIME scheme for treating intermediate-2 and high-risk patients who have become intolerant of, resistant to or are ineligible or a JAK inhibitor | 7/30/20 | Hematologic |
| Imara Inc., of Boston | IMR-687 | Small-molecule PDE9 inhibitor | Beta-thalassemia | FDA granted fast track and rare pediatric disease designations | 7/30/20 | Hematologic |
| Momenta Pharmaceuticals Inc., of Cambridge, Mass. | Nipocalimab | Fully human aglycosylated, effectorless IgG1 anti-FcRn monoclonal antibody | Prevention of hemolytic disease of the fetus and newborn | FDA granted rare pediatric disease designation; agency also granted orphan status | 7/28/20 | Hematologic |
| Novartis AG, of Basel, Switzerland | Adakveo (crizanlizumab) | P selectin antagonist | Vaso-occlusive crisis | EMA's Committee for Medicinal Products for Human Use issued positive option for prevention of recurrent crises in individuals 16 years and older with sickle cell disease | 7/24/20 | Hematologic |
| Allergy Therapeutics plc, of Worthing, U.K. | Birch allergy candidate | Chemically modified allergen with monophosphoryl lipid A adjuvant | Birch pollen allergy | Reported outcome of scientific advice from German regulator, the Paul Ehrlich Institute (PEI), regarding invalidation of the primary endpoint data of the Birch MATA MPL pivotal phase III trial, which reported in March 2019; technical issues encountered in the study made it impossible to reconstruct the primary endpoint data and PEI agreed the trial cannot be considered for assessment of clinical efficacy and a new pivotal phase III study will be conducted within the therapy allergens ordinance time frame | 7/9/20 | Immune |
| Elsalys Biotech SAS, of Lyon, France, unit of Mediolanum Farmaceutici Spa, of Milan | Leukotac (inolimomab) | IL-2 receptor alpha subunit inhibitor | Graft-vs.-host disease | BLA submitted to FDA under Real-Time Oncology Review pilot program | 7/23/20 | Immune |
| Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium | Jyseleca (filgotinib) | JAK1 inhibitor | Rheumatoid arthritis | EMA's Committee for Medicinal Products for Human use issued positive opinion for treatment of adults with moderate to severe disease who responded inadequately or are intolerant to disease-modifying drugs | 7/24/20 | Immune |
| Janssen Pharmaceutical Cos., of Horsham, Pa., part of Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody inhibiting IL-23 receptor | Active psoriatic arthritis | FDA approved use in adults, alone or in combination with conventional disease-modifying anti-rheumatic drug | 7/14/20 | Immune |
| Mesoblast Ltd., of Melbourne, Australia | Ryoncil (remestemcel-L) | Therapy comprising culture-expanded mesenchymal stem cells derived from bone marrow of unrelated donor | Steroid-refractory acute graft-vs.-host disease | U.S. FDA’s Oncology Drugs and Advisory Committee scheduled a meeting Aug. 13, 2020,to review data supporting the BLA, seeking approval for use in children; BLA has action date of Sept. 30, 2020 | 7/21/20 | Immune |
| Mylan NV, of Hertfordshire, U.K., and Fujifilm Kyowa Kirin Biologics Co. Ltd., of Tokyo | Hulio (adalimumab-fkjp) | Biosimilar to Humira (adalimumab, Abbvie Inc.) | Rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn's disease, ulcerative colitis and plaque psoriasis | FDA approved the biosimilar | 7/9/20 | Immune |
| Protara Therapeutics Inc., of New York | TARA-002 | Cell-based therapy based on immunopotentiator OK-432 | Lymphatic malformations | FDA granted rare pediatric disease designation | 7/28/20 | Immune |
| Talaris Therapeutics Inc., of Boston | FCR-001 | Allogeneic cell therapy | Scleroderma | FDA cleared IND to initiate phase I/IIa study in diffuse systemic sclerosis | 7/8/20 | Immune |
| Apogenix AG, of Heidelberg, Germany | Asunercept | Extracellular domain of CD95 receptor and Fc domain of IgG1 antibody fusion protein | Severe COVID-19 | Received regulatory approval to start the 400-patient phase II Asunctis study in Russia; study will compare 3 doses of drug plus standard of care (SOC) to SOC alone; plans to expand into other European countries, including Spain | 7/28/20 | Infection |
| Arcturus Therapeutics Holdings Inc., of San Diego, and Duke-NUS Medical School in Singapore | Lunar-COV19 | Vaccine | COVID-19 | Clinical trial application approved by Singapore Health Sciences Authority; dosing to begin shortly in up to 108 adults, including older adults, to evaluate several dose levels | 7/21/20 | Infection |
| Ascletis Pharma Inc., of Hangzhou, China | RDV/DNV regimen | Ravidasvir and danoprevir | Hepatitis C infection | China's National Medical Products Administration approved the treatment | 7/31/20 | Infection |
| Assembly Biosciences Inc., of South San Francisco | ABI-H2158 | Hepatitis B virus core inhibitor | Hepatitis B virus infection | FDA granted fast track designation | 7/9/20 | Infection |
| Bavarian Nordic A/S, of Copenhagen, and Janssen Vaccines & Prevention BV, a unit of New Brunswick, N.J.-based Johnson & Johnson | Mvabea (MVA-BN filo) and Zabdeno (Ad26.ZEBOV) | Modified vaccinia virus ankara- and adenovirus serotype 26-based vaccines | Ebola virus infection prophylaxis | European Commission granted marketing authorization for the 2-dose regimen | 7/1/20 | Infection |
| Bioaegis Therapeutics Inc., of Morristown, N.J. | Rhu-pGSN | Recombinant human plasma gelsolin | COVID-19 | Received clearance from the Spanish Agency for Medicines and Health Products for phase II study in hospitalized patients with severe COVID-19 pneumonia | 7/15/20 | Infection |
| Biocon Ltd., of Bangalore, India | Itolizumab | Biologic therapy | COVID-19 | Received the Indian drug regulator's approval for restricted emergency use of its psoriasis biologic, itolizumab, to treat patients with severe cases of COVID-19 in need of ventilator support | 7/20/20 | Infection |
| Biophytis SA, of Paris | Sarconeos (BIO-101) | Small molecule | COVID-19 | FDA accepted IND for phase II/III program assessing drug as potential treatment for acute respiratory failure associated with COVID-19 | 7/1/20 | Infection |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | Moderate COVID-19 | Submitted IND to FDA for a 40-patient phase II study | 7/27/20 | Infection |
| Carisma Therapeutics Inc., of Philadelphia | CT-0508 | Anti-HER2-targeted chimeric antigen receptor macrophage | Recurrent or metastatic HER2-overexpressing solid tumors after failure of approved HER2-targeted agents | FDA cleared the IND for a phase I study | 7/27/20 | Infection |
| Celltrion Group, of Incheon, South Korea | CT-P59 | Antiviral antibody | COVID-19 | U.K.’s Medicines and Healthcare products Regulatory Agency approved clinical trial authorization for phase I study in patients with mild symptoms of SARS-CoV-2 infection | 7/30/20 | Infection |
| Cormedix Inc., of Berkeley Heights, N.J. | Defencath/neutrolin (catheter lock solution) | Drug-device combination with antibacterial/antifungal solution | Catheter-related bloodstream infections | Submission of NDA to FDA completed for treatment of individuals with end-stage renal disease who receive hemodialysis via central venous catheter; priority review requested | 7/8/20 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab | CCR5 antagonist | HIV | Received a refuse to file letter from FDA regarding BLA seeking approval as combination therapy with HAART for highly treatment-experienced patients; company intends to request type A meeting with FDA to discuss request for additional information; FDA request does not require any additional clinical trials | 7/13/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | Trans sodium crocetinate | Increases oxygen availability | COVID-19 with symptoms of impaired respiratory function and low oxygen levels | Filed IND application with the FDA for a phase Ib/IIb study in 424 patients | 7/7/20 | Infection |
| Diffusion Pharmaceuticals Inc., of Charlottesville, Va. | Trans sodium crocetinate | Increases oxygen availability | COVID-19 | FDA provided guidance on the IND for a phase Ib study, suggesting the company change endpoints and statistical considerations; company plans to submit the same changes to the Romanian National Agency for Medicines and Medical Devices for the Romanian part of the study; first patient now expected to be enrolled by the end of August | 7/27/20 | Infection |
| Direct Biologics LLC, of Austin, Texas | Exoflo | Bone marrow-derived extracellular vesicle and exosome product | COVID-19-associated acute respiratory distress syndrome | FDA cleared IND for phase II Exit COVID-19 trial; patient enrollment expected to start in next 30 days | 7/30/20 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | TLR4-targeting monoclonal antibody | COVID-19 | Filed IND with FDA for a phase II/III study in hospitalized patients | 7/30/20 | Infection |
| Enzychem Lifesciences Inc., of Englewood, N.J. | EC-18 | CCL26 gene inhibitor; TLR modulator | Acute respiratory distress syndrome due to COVID-19 pneumonia | Filed IND with FDA for a phase II trial testing use as preventive therapeutic | 7/13/20 | Infection |
| Eusa Pharma Inc., of Hemel Hempstead, U.K. | Sylvant (siltuximab) | IL-6 receptor antagonist | COVID-19-associated acute respiratory distress syndrome | FDA cleared protocol for phase III study of intravenous Sylvant plus standard of care (SOC) vs. placebo plus SOC in about 400 hospitalized participants; primary endpoint is all-cause mortality at 28 days | 7/2/20 | Infection |
| Generex Biotechnology Corp., of Miramar, Fla. | Ii-Key-SARS-CoV-2 peptide vaccine | Multivalent peptide vaccine | COVID-19 infection | Pre-IND briefing package submitted to FDA requesting guidance on phase I/II trial protocol and clinical development plan | 7/20/20 | Infection |
| Generex Biotechnology Corp., of Miramar, Fla. | Ii-Key-SARS-CoV-2 | Vaccine | COVID-19 prophylaxis | FDA accepted the pre-IND briefing package and is expected to issue a written response by Aug. 24, 2020 | 7/27/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | COVID-19 nonstructural protein 8 modulator; COVID-19 replicase polyprotein 1ab inhibitor | COVID-19 infection | European Commission granted conditional marketing authorization to treat people 12 and older and weighing at least 40 kg who have pneumonia requiring supplemental oxygen | 7/3/20 | Infection |
| Golden Biotechnology Corp., of Taipei, Taiwan | Hocena (antroquinonol) | Isoprenyl transferase inhibitor | Mild to moderate pneumonia in COVID-19 patients | FDA approved the IND for a phase II study | 7/1/20 | Infection |
| Immodulon Therapeutics Ltd., of Uxbridge, U.K. | IMM-101 | Heat-killed bacteria | COVID-19 prophylaxis in cancer patients | Health Canada approved the start of the study | 7/9/20 | Infection |
| IMV Inc., of Dartmouth, Nova Scotia | DPX-COVID-19 | DPX-based vaccine | COVID-19 | Reached agreement with Health Canada on design of phase I study, to enroll 84 healthy adults across 2 age cohorts, between 18-55 and 56 and older; study will test 25-?g and 50-?g doses | 7/14/20 | Infection |
| Inotrem SA, of Paris | Nangibotide | TREM receptor 1 antagonist | COVID-19 infection | French, Belgian and U.S. regulators authorized phase IIa trial in about 60 critically ill people showing features of systemic inflammation; outcome measures include impact on severity of respiratory failure, duration of mechanical ventilation, ICU length of stay and mortality | 7/23/20 | Infection |
| Medicinova Inc., of La Jolla, Calif. | MN-166 (ibudilast) | PDE4 and 10 inhibitor | Prevention of acute respiratory distress syndrome in patients with COVID-19 | FDA approved the IND for a phase II study with primary endpoints of the proportion of patients free of respiratory failure, change in clinical status measured by NIAID scale and plasma cytokine levels after 7 days of treatment | 7/1/20 | Infection |
| Mylan NV, of Hertfordshire, U.K. | Remdesivir lyophilized powder for injection (Desrem) | COVID-19 nonstructural protein 8 modulator; COVID-19 replicase polyprotein 1ab inhibitor | COVID-19 infection | Drug Controller General of India approved formulation of 100 mg/vial, under license from Gilead Sciences Inc. for restricted emergency use in India under accelerated approval to treat suspected or laboratory confirmed infection in adults and children hospitalized with severe disease | 7/6/20 | Infection |
| Nabriva Therapeutics plc, of Dublin | Xenleta (lefamulin) | Semisynthetic pleuromutilin antibiotic | Community-acquired pneumonia | European Commission approved MAA for use in adults when considered inappropriate to use antibacterial agents that are commonly recommended for initial treatment or when those agents have failed | 7/28/20 | Infection |
| Nabriva Therapeutics plc, of Dublin, and Sunovion Pharmaceuticals Canada Inc., of Mississauga, Ontario | Xenleta (lefamulin) | Semisynthetic pleuromutilin antibiotic | Community-acquired pneumonia | Oral and I.V. formulations approved by Health Canada | 7/16/20 | Infection |
| Neoimmunetech Inc., of Rockville, Md. | NT-I7 (efineptakin alfa) | Long-acting human IL-7 | Mild COVID-19 | FDA cleared the IND for a phase I study | 7/15/20 | Infection |
| Neoimmunetech Inc., of Rockville, Md. | NT-I7 (efineptakin alfa) | Long-acting human IL-7 | COVID-19 | FDA cleared the IND for an investigator-initiated phase I study | 7/27/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b1 and BNT-162b2 | mRNA-based vaccines | COVID-19 | FDA granted fast track designation to both | 7/13/20 | Infection |
| Realta Life Sciences Inc., of Norfolk, Va. | RLS-0071 | Peptide designed to inhibit complement and anti-inflammatory immune responses | Acute lung injury secondary to COVID-19 | FDA accepted IND; phase I study in adults with pneumonia and early respiratory failure set to start in the third quarter of 2020 | 7/28/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Opaganib (Yeliva, ABC-294640) | Sphingosine kinase-2 inhibitor | COVID-19 infection | CTA submitted to Mexican Federal Committee for Protection against Sanitary Risks, COFEPRIS, and submission process initiated with Brazilian Health Regulatory Agency, ANVISA, for phase II/III study in individuals hospitalized with severe infection and pneumonia | 7/22/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | RHB-204 | Fixed-dose oral capsule containing a combination of clarithromycin, rifabutin and clofazimine | Pulmonary nontuberculous mycobacteria infections | FDA cleared its IND application for a pivotal phase III study to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium complex infection | 7/31/20 | Infection |
| Redhill Biopharma Ltd., of Tel-Aviv, Israel | Yeliva (opaganib) | Sphingosine kinase-2 inhibitor | Hospitalized, severe COVID-19 | Ministry of Health of the Russian Federation approved the CTA | 7/16/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative with 2 thiol groups | Mild to moderate COVID-19 | FDA approved it to proceed with randomized, double-blind, placebo-controlled confirmatory phase III trial protocol to evaluate safety and efficacy | 7/31/20 | Infection |
| Ridgeback Biotherapeutics LP, of Miami | Ansuvimab (mAb-114) | Monoclonal antibody | Ebola | FDA accepted BLA, granting priority review | 7/29/20 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | 1,3 beta glucan synthase inhibitor | Vulvovaginal candidiasis infection | Pre-NDA meetings with FDA completed; based on feedback, regulatory package considered sufficient for submission and filing expected in fourth quarter of 2020 | 7/29/20 | Infection |
| Shanghai Fosun Pharmaceutical (Group) Co. Ltd., of Shanghai | BNT-162b1 | Vaccine | COVID-19 | Clinical trial application accepted by China’s NMPA | 7/14/20 | Infection |
| Siga Technologies Inc., of New York | Oral tecovirimat | Small-molecule antiviral | Smallpox, monkeypox, cowpox, and complications from Vaccinia infection | Filed MAA with EMA for same formulation approved under brand Tpoxx by FDA in July 2018 | 7/30/20 | Infection |
| Sinovac Biotech Ltd., of Beijing | Coronavac | Adjuvant inactivated vaccine | COVID-19 infection | Brazil's Anvisa granted approval for pivotal phase III trial sponsored by Instituto Butantan; enrollment expected to begin in July 2020 | 7/6/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | Abivertinib | Tyrosine kinase inhibitor targeting EGFR and BTK | COVID-19 | FDA cleared start of phase II trial in patients with COVID-19 who have moderate to severe pulmonary symptoms | 7/20/20 | Infection |
| Sound Pharmaceuticals Inc., of Seattle | SPI-1005 (ebselen) | Glutathione peroxidase stimulator | COVID-19 | Filed an IND application with the FDA to begin phase II studies | 7/31/20 | Infection |
| TC Biopharm Ltd., of Glasgow, U.K. | Gamma-delta T | Immunotherapy targeting SARS-CoV-2-infected cells | COVID-19 | Submitted documents to U.K. regulators to start safety trials | 7/1/20 | Infection |
| Tetra Bio-Pharma Inc., of Ottawa | ARDS-003 | Cannabinoid CB2 receptor agonist | COVID-19 patients at risk for developing acute respiratory distress syndrome | FDA reviewed the pre-IND application | 7/16/20 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Pa. | VP-102 (cantharidin 0.7% topical solution) | Blistering agent delivered via single-use precision device | Molluscum contagiosum | FDA issued complete response letter for NDA seeking additional information regarding certain aspects of chemistry, manufacturing and controls process for the drug/device combination, as well as human factors validation; FDA did not identify any clinical deficiencies | 7/14/20 | Infection |
| Viiv Healthcare Ltd., of London | Rukobia (fostemsavir) | HIV-1 attachment inhibitor | HIV-1 infection | FDA approved drug in combination with other antiretroviral therapies to treat heavily treatment-experienced adults with multidrug-resistant infection who are failing current regimen due to resistance, intolerance or safety considerations | 7/2/20 | Infection |
| Inflazome Ltd., of Dublin | Inzomelid | Small-molecule inhibitor of NLRP3 inflammasome | Cryopyrin-associated periodic syndrome | FDA granted orphan designation | 7/14/20 | Inflammatory |
| Mesoblast Ltd., of Melbourne, Australia | Remestemcel-L | Allogeneic mesenchymal stem cell therapy | COVID-19-related multisystem inflammatory syndrome | Expanded access protocol initiated in U.S. for compassionate use in infected children 2 months to 17 years with cardiovascular and other MIS-C complications | 7/6/20 | Inflammatory |
| Abbvie Inc., of North Chicago | Botox (onabotulinumtoxinA) | Botulinum toxin type A | Spasticity | FDA approved supplemental BLA for use in pediatric patients, 2 and older, including those with lower limb spasticity caused by cerebral palsy | 7/8/20 | Musculoskeletal |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Vosoritide | C-type natriuretic peptide ligand | Achondroplasia | MAA for treatment in children submitted to EMA; NDA submission to FDA remains on track for third quarter of 2020 | 7/23/20 | Musculoskeletal |
| Clinigen Group plc, of Burton Upon Kent, U.K. | Aldesleukin | Non-glycosylated interleukin-2 product | Amyotrophic lateral sclerosis | FDA granted orphan designation | 7/13/20 | Musculoskeletal |
| Dynacure SAS, of Strasbourg, France | DYN-101 | Antisense medicine designed to modulate expression of dynamin 2 | Myotubular and centronuclear myopathies | FDA granted rare pediatric disease designation | 7/30/20 | Musculoskeletal |
| Ipsen SA, of Paris | Dysport (abobotulinumtoxinA) | Botulinum toxin type A | Spasticity | FDA approved expanded use in pediatric patients | 7/9/20 | Musculoskeletal |
| Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Fibroblast growth factor 23 ligand inhibitor | X-linked hypophosphatemia | EMA's Committee for Medicinal Products for Human Use recommended expanding label to include treatment of older adolescents and adults | 7/24/20 | Musculoskeletal |
| Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Monoclonal antibody targeting phosphaturic hormone fibroblast growth factor 23 | X-linked hypophosphatemia | EMA's Committee for Medicinal Products for Human Use issued a positive opinion for expanded use of the drug | 7/24/20 | Musculoskeletal |
| Osmotica Pharmaceuticals plc, of Bridgewater, N.J. | Arbaclofen extended release | GABA B receptor agonist | Spasticity | FDA deemed NDA resubmission complete, setting PDUFA date of Dec. 29, 2020 | 7/20/20 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | SRP-9001 | DMD gene stimulator | Duchenne muscular dystrophy | FDA granted fast track designation | 7/24/20 | Musculoskeletal |
| Solid Biosciences Inc., of Cambridge, Mass. | SGT-001 | Gene therapy expressing microdystrophin | Duchenne muscular dystrophy | FDA issued written communication requesting additional information on manufacturing, updated safety and efficacy data for all patients dosed and requested plans for total viral load to be administered per patient; Ignite DMD study remains on clinical hold | 7/24/20 | Musculoskeletal |
| Vita Therapeutics Inc., of Baltimore | VTA-110 | Allogenic iPSC-based therapy | Duchenne muscular dystrophy | FDA granted orphan drug designation | 7/23/20 | Musculoskeletal |
| Acacia Pharma Group plc, of Cambridge, U.K. | Byfavo (remimazolam) | Ultra-short-acting, reversible intravenous benzodiazepine | Sedative/anesthetic use during invasive medical procedures | Approved by FDA for the induction and maintenance of procedural sedation in adults undergoing procedures lasting 30 minutes or less | 7/2/20 | Neurology/Psychiatric |
| Acadia Pharmaceuticals Inc., of San Diego | Nuplazid (pimavanserin) | Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors | Dementia-related psychosis | FDA accepted for filing supplemental NDA seeking approval to treat hallucinations and delusions associated with DRP | 7/20/20 | Neurology/Psychiatric |
| Aytu Bioscience Inc., of Englewood, Colo. | Zolpimist (zolpidem tartrate oral spray) | Benzodiazepine receptor agonist | Sleep aid | Sublicensee Suda Pharmaceuticals Ltd. received marketing approval from Australia’s Therapeutic Good Administration | 7/30/20 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Aducanumab | Beta-amyloid antagonist | Alzheimer's disease | BLA submission to FDA completed; priority review requested | 7/8/20 | Neurology/Psychiatric |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | Zonisamide oral suspension (ET-104) | Calcium channel inhibitor; sodium channel inhibitor | Epilepsy | NDA for treatment of partial seizures submitted to FDA | 7/30/20 | Neurology/Psychiatric |
| Grunenthal GmbH, of Aachen, Germany, and subsidiary Averitas Pharma Inc. | Qutenza (capsaicin) 8% patch | TRPV1 agonist | Neuropathic pain associated with diabetic peripheral neuropathy of the feet | Approved by FDA | 7/21/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | Zynrelef (bupivacaine + meloxicam, formerly HTX-011) | Cyclooxygenase 2 inhibitor; sodium channel inhibitor | Postoperative pain | EMA's Committee for Medicinal Products for Human Use issued positive opinion for treatment of pain from small to medium-sized surgical wounds in adults | 7/24/20 | Neurology/Psychiatric |
| Jazz Pharmaceuticals plc, of Dublin | Xywav (calcium, magnesium, potassium and sodium oxybates) | GABA B receptor agonist | Cataplexy; excessive daytime sleepiness | FDA approved to treat individuals 7 and older with narcolepsy | 7/21/20 | Neurology/Psychiatric |
| Knopp Biosciences LLC, of Pittsburgh | KB-3061 | Activator of voltage-gated Kv7.2/7.3 potassium channels | KCNQ2 epileptic encephalopathy | FDA granted rare pediatric disease designation | 7/15/20 | Neurology/Psychiatric |
| Larimar Therapeutics Inc., of Bala Cynwyd, Pa. | CTI-1601 | Recombinant fusion protein intended to deliver human frataxin into mitochondria | Friedreich’s ataxia | EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending orphan designation | 7/28/20 | Neurology/Psychiatric |
| Marinus Pharmaceuticals Inc., of Radnor, Pa. | Ganaxolone | Positive allosteric modulator of GABAA receptors | CDKL5 deficiency disorder | FDA granted rare pediatric disease designation for rare, refractory form of epilepsy | 7/30/20 | Neurology/Psychiatric |
| Melt Pharmaceuticals Inc., of Boston | MELT-100 (midazolam + ketamine, sublingual) | Benzodiazepine receptor agonist; NMDA receptor antagonist | Pain | FDA cleared IND to begin phase I pharmacokinetics study, with data expected in fourth quarter of 2020; drug advancing through 505(b)(2) regulatory pathway to provide sedation and analgesia for individuals undergoing cataract surgery | 7/29/20 | Neurology/Psychiatric |
| Neumentum Inc., of Palo Alto, Calif. | NTM-006 | Non-opioid, non-NSAID analgesic | Neuropathic and moderate to severe chronic pain | Type C meeting with FDA scheduled for October 2020 to discuss development pathway | 7/23/20 | Neurology/Psychiatric |
| Neumentum Inc., of Palo Alto, Calif. | NTM-001 (pre-mixed bag ketorolac) | Cyclooxygenase/oxidoreductase inhibitor | Postsurgical pain | Guidance letter from FDA authorized rapid phase III development, with pivotal post-bunionectomy efficacy study expected to begin after company's next equity financing | 7/23/20 | Neurology/Psychiatric |
| Neurogene Inc., of New York | Adeno-associated virus vector with engineered transgene encoding the human CLN5 gene | Gene therapy | CLN5 Batten disease | FDA granted orphan drug designation | 7/7/20 | Neurology/Psychiatric |
| Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | CGRP receptor antagonist | Migraine | Licensee Otsuka Pharmaceutical Co. Ltd. submitted NDA for preventive treatment to Japan PMDA | 7/29/20 | Neurology/Psychiatric |
| Vistagen Therapeutics Inc., of South San Francisco | PH-94B | Synthetic neurosteroid nasal spray | Anxiety | FDA agreed on key features of initial pivotal phase III trial involving single-event, laboratory-simulated public speaking challenge; study expected to enroll about 182 adults with social anxiety disorder | 7/23/20 | Neurology/Psychiatric |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Reactive aldehyde species inhibitor | Dry eye disease | Based on FDA meeting minutes, company plans to run 2 clinical trials testing the reduction of reactive aldehyde species in tears to support the NDA | 7/7/20 | Ocular |
| Applied Genetic Technologies Corp. (AGTC), of Gainesville, Fla. | AAV-RPGR gene therapy | Undisclosed | X-linked retinitis pigmentosa | Following written feedback on development plan from FDA, expanding ongoing phase I/II trial to dose about 20 patients in 2 masked arms to collect additional functional data; for late-stage studies, FDA indicated change in visual sensitivity of 7 decibels or > in at least 5 loci seen as clinically meaningful; phase II/III trial, expected to begin in first quarter of 2021, will have visual sensitivity as primary endpoint | 7/22/20 | Ocular |
| Molecular Partners AG, of Zurich-Schlieren, Switzerland | Abicipar pegol | Ankyrin modulator; VEGF-A ligand inhibitor | Wet age-related macular degeneration | Reported that partner Abbvie Inc., of North Chicago, disclosed plans to withdraw filings with EMA and PMDA following CRL from FDA in June 2020 | 7/20/20 | Ocular |
| Ocugen Inc., of Malvern, Pa. | OCU-400 | Gene therapy expressing NR2E3 | RHO mutation-associated retinal degeneration | FDA granted orphan drug designation | 7/27/20 | Ocular |
| Oculis SA, of Lausanne, Switzerland | OCS-01 | Dexamethasone in a solubilizing nanoparticle | Diabetic macular edema and inflammation and pain following cataract surgery | Completed end-of-phase-II meeting; plans to start phase III studies for both indications in the first half of 2021 | 7/27/20 | Ocular |
| Osmotica Pharmaceuticals plc, of Bridgewater, N.J. | Upneeq (oxymetazoline hydrochloride) | Alpha adrenergic receptor agonist | Ptosis | FDA approved the drug | 7/9/20 | Ocular |
| Esperare Foundation, of Geneva | ER-004 | Ectodysplasin A ligand | X linked hypohidrotic ectodermal dysplasia | FDA granted breakthrough therapy designation for prenatal treatment | 7/6/20 | Other/Miscellaneous |
| Otonomy Inc., of San Diego | Otividex | Sustained-exposure formulation of dexamethasone | Ménière’s disease | In response to questions from the FDA, company submitted a revised statistical analysis plan that uses the Negative Binomial model for the primary analysis of the ongoing trial | 7/6/20 | Other/Miscellaneous |
| Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin-4 receptor agonist | Obesity | MAA submitted to EMA, which granted accelerated assessment, for treatment of pro-opiomelanocortin deficiency and leptin receptor deficiency obesity | 7/1/20 | Other/Miscellaneous |
| Synthetic Biologics Inc., of Rockville, Md. | SYN-020 | Oral formulation of recombinant intestinal alkaline phosphatase | Multiple indications, including radiation enteropathy secondary to pelvic cancer therapy | FDA cleared IND for phase I study in healthy volunteers | 7/30/20 | Other/Miscellaneous |
| Astrazeneca plc, of Cambridge, U.K. | Breztri Aerosphere (budesonide + glycopyrrolate + formoterol fumarate) | Beta 2 adrenoceptor agonist; muscarinic receptor antagonist | Chronic obstructive pulmonary disease | FDA approved for maintenance treatment | 7/24/20 | Respiratory |
| Boehringer Ingelheim International GmbH, of Ingelheim, Germany | Nintedanib | Tyrosine kinase inhibitor | Chronic fibrosing interstitial lung diseases with a progressive phenotype beyond idiopathic pulmonary fibrosis | European Commission approved the additional indication | 7/15/20 | Respiratory |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | INO-3107 | DNA plasmid vaccine | Recurrent respiratory papillomatosis | FDA granted orphan drug designation | 7/29/20 | Respiratory |
| Insmed Inc., of Bridgewater, N.J. | Arikayce (amikacin) | 30S ribosomal protein inhibitor | Non-tuberculous mycobacterial lung infection | EMA's Committee for Medicinal Products for Human Use issued positive opinion for treatment of infections caused by Mycobacterium avium complex in adults | 7/24/20 | Respiratory |
| Novartis AG, of Basel, Switzerland, Sosei Group Corp., of Tokyo, and Propeller Health, of Madison, Wis. | Enerzair Breezhaler (indacaterol acetate, glycopyrronium bromide and mometasone furoate) | Long?acting beta2?agonist/long-acting muscarinic antagonist/inhaled corticosteroid | Uncontrolled asthma | European Commission approved the drug/device | 7/7/20 | Respiratory |
| Relief Therapeutics Holding AG, of Geneva, and Neurorx Inc., of Radnor, Pa. | RLF-100 (aviptadil) | VIP receptor agonist | COVID-19-related respiratory failure | FDA granted expanded access protocol to those ineligible for enrollment in clinical trial, including pregnant women, who exhausted approved therapies | 7/29/20 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 (repurposed ondansetron) | 5-HT 3 receptor antagonist | Alcohol use disorder | Regulators in Sweden and Estonia cleared initiation of sites in pivotal phase III Onward trial in people with target genotypes related to serotonin transporter and receptor genes | 7/6/20 | Toxicity and Intoxication |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 (repurposed ondansetron) | 5-HT 3 receptor antagonist |
Alcohol use disorder | EMA accepted pediatric investigation plan and will not require additional trials for individuals ages 12 to 17 | 7/22/20 | Toxicity and Intoxication |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | DS-100 | Dehydrated alcohol | Methanol poisoning | Submitted NDA to the FDA | 7/27/20 | Toxicity and Intoxication |
| Akeso Biopharma Inc., of Los Angeles | AK-104 | Anti-PD-1/CTLA4 bispecific antibody | Recurrent or metastatic squamous cervical cancer after platinum-based chemotherapy | FDA granted fast track designation | 8/13/20 | Cancer |
| Antengene Corp., of Shanghai | ATG-017 | Inhibitor of extracellular signal-regulated kinase 1 and 2 | Advanced solid tumors and hematological malignancies | Australian Therapeutic Goods Administration authorized the start of a phase I study | 8/12/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | First-line extensive-stage small cell lung cancer | Japanese Ministry of Health, Labour and Welfare approved the drug in combination with etoposide plus a choice of platinum chemotherapy (either carboplatin or cisplatin) | 8/21/20 | Cancer |
| Biomark Diagnostics Inc., of Vancouver, British Columbia | Acetyl-amantadine | Biomarker | Glioblastoma | Health Canada issued a letter of no objection for the CTA | 8/27/20 | Cancer |
| Biosight Ltd., of Airport City, Israel | BST-236 (aspacytarabine) | Antimetabolite | Acute myeloid leukemia | FDA granted fast track designation for use in adults 75 or older or with co-morbidities that preclude use of intensive induction chemotherapy | 8/4/20 | Cancer |
| Bristol-Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 inhibitor | Unresectable advanced, recurrent or metastatic esophageal cancer after prior chemotherapy | Positive scientific opinion issued in U.K. for early access to medicines scheme as monotherapy treatment of adult patients after prior fluoropyrimidine- and platinum-based chemotherapy. | 8/20/20 | Cancer |
| Canbridge Pharmaceuticals Inc., of Beijing | Nerlynx (neratinib) | EGFR antagonist; Erbb2/Erbb4 tyrosine kinase receptor inhibitor | Early stage HER2-positive breast cancer | Received marketing approval from the Taiwan Food and Drug Administration for the extended adjuvant treatment of adult patients following adjuvant trastuzumab-based therapy | 8/7/20 | Cancer |
| Carsgen Therapeutics Co. Ltd., of Shanghai | CT-041 | Humanized CAR-claudin18.2 T-cell therapy | Claudin18.2-positive adenocarcinoma of the stomach, gastroesophageal junction or pancreas | China's NMPA cleared its IND application | 8/20/20 | Cancer |
| Cerecor Inc., of Rockville, Md. | CERC-006 | Dual mTOR complex 1/2 inhibitor | Lymphatic malformations | FDA granted orphan drug designation | 8/3/20 | Cancer |
| Cstone Pharmaceuticals Co. Ltd., of Suzhou, China | Sugemalimab (CS-1001) | Anti-PD-L1 monoclonal antibody | Relapsed or refractory extranodal natural killer/T-cell lymphoma | FDA issued a study may proceed letter for the IND for a phase II study | 8/31/20 | Cancer |
| Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Ripretinib | Switch-control tyrosine kinase inhibitor that inhibits KIT and PDGFR-alpha | Advanced gastrointestinal stromal tumors | China's NMPA granted priority review status to the NDA for the treatment of adults who have received prior treatment with 3 or more kinase inhibitors, including imatinib | 8/4/20 | Cancer |
| EMD Serono, part of Merck KGaA, of Darmstadt, Germany | Tepotinib | MET inhibitor | Metastatic non-small cell lung cancer with MET exon 14 skipping alterations | The FDA accepted the NDA and granted priority review | 8/25/20 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Tyrosine kinase inhibitor | Advanced renal cell carcinoma | Submitted supplemental NDA to use the drug in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) | 8/24/20 | Cancer |
| Genmab A/S, of Copenhagen, Denmark, and Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex (daratumumab) | Monoclonal antibody targeting CD38 | Relapsed/refractory multiple myeloma after 1-3 previous lines of therapy. | FDA approved the drug in combination with carfilzomib and dexamethasone | 8/20/20 | Cancer |
| Glaxosmithkline plc, of London | Blenrep (belantamab mafodotin-blmf) | Monoclonal antibody targeting BCMA | Relapsed or refractory multiple myeloma | FDA granted an accelerated approval contingent upon verification and description of clinical benefit in confirmatory trials, for use after at least 4 prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor and an immunomodulatory agent | 8/6/20 | Cancer |
| Glaxosmithkline plc, of London | Blenrep (belantamab mafodotin) | Anti-BCMA (B-cell maturation antigen) | Multiple myeloma | EC granted conditional marketing authorization for adult patients who have received at least 4 prior therapies and whose disease is refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy | 8/27/20 | Cancer |
| Henlius Biotech Inc., of Shanghai | HLX-02 | Trastuzumab biosimilar | HER2-positive early breast cancer, HER2-positive metastatic breast cancer and HER2-positive metastatic gastric cancer | National Medical Products Administration in China approved the biosimilar | 8/17/20 | Cancer |
| Hutchison China Meditech Ltd., of Hong Kong and Shanghai | Surufatinib | Oral angio-immuno kinase inhibitor targeting VEGFR and FGFR | Advanced neuroendocrine tumors | Received scientific advice from EMA’s Committee for Medicinal Products for Human Use and concluded that completed SANET-ep (non-pancreatic NET) and SANET-p (pancreatic NET) | 8/10/20 | Cancer |
| Immatics NV, of Tuebingen, Germany | IMA-203 | Targets peptides derived from preferentially expressed antigen in melanoma | Solid tumors expressing preferentially expressed antigen in melanoma | Germany's Paul-Ehrlich-Institute approved the CTA for a phase I study | 8/18/20 | Cancer |
| Immunomedics Inc., of Morris Plains, N.J. | Trodelvy (sacituzumab govitecan-hziy) | Monoclonal antibody targeting Trop-2 | Metastatic triple-negative breast cancer | FDA approved the prior approval supplement, allowing the antibody to be produced by manufacturing partner Samsung Biologics | 8/17/20 | Cancer |
| Innovent Biologics Inc., of Suzhou, China, and Eli Lilly and Co., of Indianapolis | Tyvyt (sintilimab) | Monoclonal antibody targeting PD-1 | First-line squamous non-small-cell lung cancer | National Medical Products Administration of China accepted the supplemental NDA for use of the drug in combination with Gemzar (gemcitabine, Lilly) and platinum | 8/12/20 | Cancer |
| Jiangsu Hengrui Medicine Co. Ltd., of Lianyungang, China | Camrelizumab and rivoceranib | Combination therapy of PD-1 inhibitor and VEGFR-2 inhibitor | Advanced or metastatic hepatocellular carcinoma | Korea’s Ministry of Food and Drug Safety (MFDS) granted approval to begin a phase III trial | 8/18/20 | Cancer |
| Kahr Medical Ltd., of Jerusalem | DSP-107 | Targets CD47 and 41BB | Advanced solid tumors | FDA cleared the IND for a phase I/II study testing the drug as a monotherapy and in combination with Tecentriq (atezolizumab, Roche Holding AG) | 8/12/20 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib | PI3K pathway inhibitor | Diffuse intrinsic pontine glioma | FDA granted rare pediatric disease designation | 8/7/20 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib (GDC-0084) | PI3K pathway inhibitor | Glioblastoma | FDA granted fast track designation | 8/20/20 | Cancer |
| Kazia Therapeutics Ltd., of Sydney | Paxalisib (GDC-0084) | PI3K pathway inhibitor | Malignant glioma | FDA granted orphan drug designation | 8/24/20 | Cancer |
| Lidds AB, of Uppsala, Sweden | Liproca Depot | Androgen receptor antagonist | Prostate cancer | Licensee, Jiangxi Puheng Pharma, is preparing documentation for an application targeting a newly issued regulatory guideline in China on conditional market approval, for unmet medical needs based on less comprehensive data than normally required | 8/20/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Esophageal squamous cell carcinoma | Japan Pharmaceuticals and Medical Devices Agency granted approval to treat PD-L1-positive, radically unresectable, advanced or recurrent esophageal squamous cell carcinoma after progression on chemotherapy; agency also approved use of 400 mg dose every six weeks administered as an intravenous infusion over 30 minutes across all adult indications | 8/24/20 | Cancer |
| Mersana Therapeutics Inc., of Cambridge, Mass. | XMT-1536 | Sodium phosphate cotransporter 2B inhibitor | Ovarian cancer | FDA granted fast track designation to treat platinum-resistant high-grade serous disease | 8/11/20 | Cancer |
| Mesoblast Ltd., of Melbourne, Australia | Ryoncil (remestemcel-L) | Mesenchymal stem cell therapy | Graft-vs.-host disease | FDA Oncologic Drugs Advisory Committee meeting scheduled for Aug. 13, 2020, will include non-voting morning session to discuss characterization and quality attributes of study drug and afternoon session to discuss phase III results and supporting data in BLA submission, with vote on whether available data support efficacy in pediatric patients with steroid-refractory acute disease | 8/11/20 | Cancer |
| Miragen Therapeutics Inc., of Boulder, Colo. | Cobomarsen | miR-155 inhibitor | Adult T-cell leukemia/lymphoma | Received guidance from the FDA in a type C meeting on running a pivotal study with a primary endpoint of progression free survival | 8/20/20 | Cancer |
| Morphosys AG, of Planegg, Germany | Monjuvi (tafasitamab-cxix) | Anti-CD19 antibody | Diffuse large B-cell lymphoma | FDA approved in combination with lenalidomide to treat adults with relapsed/refractory disease, including that arising from low-grade lymphoma, ineligible for autologous stem cell transplant | 8/1/20 | Cancer |
| Nkmax America Inc., of Santa Ana, Calif. | SNK-01 | Autologous natural killer cell immunotherapy | Refractory solid tumors | FDA granted permission to amend the phase I study to add a fourth cohort testing up to 18 patients with SNK-O1 plus Keytruda (pembrolizumab, Merck & Co. Inc.) or Bavencio (avelumab, Merck KGaA/Pfizer Inc.) | 8/31/20 | Cancer |
| Oncopeptides AB, of Stockholm | Melflufen flufenamide | Peptide-drug conjugate targeting aminopeptidases | Multiple myeloma refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent and 1 anti-CD38 monoclonal antibody | FDA granted a priority review for the NDA; PDUFA action date is Feb. 28, 2021 | 8/29/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cypcaps | Cell-in-a-box encapsulated cell product | Locally advanced, inoperable pancreatic cancer | FDA accepted the drug master file from manufacturing partner Austrianova | 8/5/20 | Cancer |
| Precision Biosciences Inc., of Durham, N.C. | PBCAR-0191 | CAR T-cell therapy | Advanced B-cell precursor acute lymphoblastic leukemia | FDA granted fast track designation | 8/19/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Rozlytrek (entrectinib) | ROS1/TRKA/B/C kinase inhibitor | Solid tumors; non-small-cell lung cancer | European Commission granted conditional marketing authorization to treat adults and children 12 and older with solid tumors expressing neurotrophic tyrosine receptor kinase (NTRK) gene fusion and locally advanced or metastatic disease who did not receive prior NTRK inhibitor and also to treat adults with ROS1-positive, advanced NSCLC not previously treated with ROS1 inhibitor | 8/3/20 | Cancer |
| Samsung Bioepis Co. Ltd., of Incheon, Korea | Aybintio (bevacizumab biosimilar) | Anti-VEGF antibody | Multiple cancers | The European Commission granted marketing authorization | 8/20/20 | Cancer |
| Seattle Genetics Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | Advanced unresectable or metastatic HER2-positive breast cancer | Australian regulatory authorities approved use of the drug in combination with Herceptin (trastuzumab, Roche Holding AG) | 8/12/20 | Cancer |
| Sirtex Medical US Holdings Inc., of Woburn, Mass. | SIR-Spheres Y-90 | Resin microspheres | Liver cancer | China's National Medical Products Administration granted a notice of drug clinical trial approval | 8/27/20 | Cancer |
| TG Therapeutics Inc., of New York | Umbralisib | Inhibitor of PI3K-delta and CK1-epsilon | Marginal zone lymphoma after at least 1 prior anti-CD20-based regimen and follicular lymphoma after at least 2 prior systemic therapies | FDA accepted the NDA for a priority review; PDUFA action date of Feb. 15, 2021 | 8/13/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Monoclonal antibody targeting PD-L1 | Undifferentiated pleomorphic sarcoma and myxofibrosarcoma | FDA cleared the IND for the Envasarc pivotal study | 8/17/20 | Cancer |
| Turning Point Therapeutics Inc., of San Diego | Repotrectinib | ROS1 and TRK inhibitors | Advanced solid tumors with an NTRK gene fusion | FDA granted fast track designation for patients who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK tyrosine kinase inhibitors and have no satisfactory alternative treatments | 8/24/20 | Cancer |
| Tyme Technologies Inc., of Bedminster, N.J. | SM-88 (racemetyrosine) | Cytochrome P450 2A6 inhibitor; cytochrome P450 3A4 stimulator | Pancreatic cancer | FDA granted orphan drug designation | 8/3/20 | Cancer |
| Velosbio Inc., of San Diego | VLS-101 | Antibody-drug conjugate targeting receptor tyrosine kinase-like orphan receptor 1 | Mantle cell lymphoma | FDA granted fast track and orphan drug designation | 8/31/20 | Cancer |
| Y-mabs Therapeutics, Inc., of New York | Omburtamab | Monoclonal antibody targeting B7-H3 | Pediatric CNS/leptomeningeal metastasis from neuroblastoma | Completed rolling submission of the BLA | 8/6/20 | Cancer |
| Stempeutics Research Pvt Ltd., of Bangalore, India | Stempeucel | Bone-marrow derived allogeneic mesenchymal stem cell therapy | Critical limb ischemia (CLI) due to Buerger’s disease and atherosclerotic peripheral arterial disease | Drug Controller General of India (DCGI) approved the drug | 8/24/20 | Cardiovascular |
| Akari Therapeutics plc, of New York | Nomacopan | Complement C5 factor inhibitor; leukotriene BLT receptor antagonist | Bullous pemphigoid | At end-of-phase-II meeting, FDA agreed to phase III design for treatment of moderate to severe disease, with primary endpoint of complete remission on minimal oral corticosteroids (OCS) and secondary endpoint of reduction in cumulative OCS; pivotal trial expected to begin in first half of 2021 | 8/12/20 | Dermatologic |
| Cassiopea SpA, of Ainate, Italy | Winlevi (clascoterone cream 1%) | Androgen receptor inhibitor | Acne | FDA approved the product | 8/27/20 | Dermatologic |
| Hoth Therapeutics Inc., of New York | Biolexa | Antimicrobial | Mild to moderate atopic dermatitis | Submitted study for ethics approval from the Bellberry Human Research Ethics Committee in Australia | 8/13/20 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib cream | JAK inhibitor | Moderate to severe chronic hand eczema | FDA granted fast track designation for the treatment of adults | 8/4/20 | Dermatologic |
| Mallinckrodt plc, of Staines-upon-Thames, U.K. | Stratagraft | Regenerative skin tissue therapy | Deep partial-thickness thermal burns | FDA accepted for review the BLA and granted priority review; PDUFA date of Feb. 2, 2021 | 8/10/20 | Dermatologic |
| Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | IL-17 antagonist | Plaque psoriasis | European Commission approved to treat moderate to severe disease in children and adolescents 6 to <18 years | 8/3/20 | Dermatologic |
| 4D Molecular Therapeutics Inc., of Emeryville, Calif | 4D-310 | Gene therapy expressing GLA | Fabry disease | FDA granted fast track designation | 8/13/20 | Endocrine/Metabolic |
| Amidebio LLC, of Boulder, Colo. | ABG-023 (glucagon analog; AB-GLC-0035) | Glucagon receptor agonist | Congenital hyperinsulinism | The FDA granted rare pediatric disease designation | 8/20/20 | Endocrine/Metabolic |
| Applied Therapeutics Inc., of New York | AT-007 | CNS-penetrant aldose reductase inhibitor | Galactosemia | FDA placed a partial clinical hold on the Action-Kids study and requested information showing every patient has the potential to benefit from the drug | 8/17/20 | Endocrine/Metabolic |
| Diurnal Group plc, of Israel | Alkindi (hydrocortisone granules in capsules for opening) | Steroid | Adrenal insufficiency | Australia’s TGA approved as replacement therapy for adrenal insufficiency, with no age restriction | 8/10/20 | Endocrine/Metabolic |
| Diurnal Group plc., of Israel | Alkindi (hydrocortisone granules) | Steroid | Adrenal insufficiency | Ministry of Health in Israel approved the drug for infants, children and adolescents | 8/5/20 | Endocrine/Metabolic |
| I-Mab Biopharma Co. Ltd., of Shanghai | Eftansomatropin | Long-acting recombinant human growth hormone | Growth hormone deficiency in pediatric patients | China's NMPA accepted its pivotal trial application | 8/4/20 | Endocrine/Metabolic |
| Intellia Therapeutics Inc., of Cambridge, Mass. | NTLA-2001 | Systemically delivered CRISPR/Cas9 therapy | Transthyretin amyloidosis | Submitted a CTA to the U.K.’s MHRA to initiate a phase I trial | 8/6/20 | Endocrine/Metabolic |
| Lipocine Inc., of Salt Lake City, Calif. | Tlando | Oral testosterone | Hypogonadism | FDA needs additional time to complete its review of the NDA, which had a PDUFA action date of Aug. 28, 2020; agency expects to complete its review in the coming weeks | 8/28/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Sogroya (somapacitan) | Weekly human growth hormone therapy | Growth hormone deficiency | Approved by FDA for use in adults | 8/28/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBGM-01 | Gene therapy expressing b-gal | GM1 gangliosidosis | Submitted IND to FDA for a phase I/II study; agency placed the application on clinical hold pending additional risk assessments of the intra-cisterna magna injection delivery method | 8/13/20 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, unit of Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa | Alpha-galactosidase stimulator | Fabry disease | FDA accepted BLA, granting priority review and setting Jan. 27, 2021, PDUFA date; no advisory committee meeting expected | 8/11/20 | Endocrine/Metabolic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Evinacumab | Monoclonal antibody targeting angiopoietin-like 3 | Homozygous familial hypercholesterolemia | FDA accepted the BLA for a priority review; PDUFA action date is Feb. 11, 2021 | 8/12/20 | Endocrine/Metabolic |
| Taysha Gene Therapies Inc., of Dallas | TSHA-101 | AAV9-based gene therapy | GM2 gangliosidosis | FDA granted orphan drug and rare pediatric disease designations | 8/27/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | ALN-HSD | HSD17B3 gene modulator | Nonalcoholic steatohepatitis | CTA application submitted to U.K. MHRA to initiate a phase I study, expected to begin in late 2020 | 8/3/20 | Gastrointestinal |
| Metacrine Inc., of San Diego | MET-409 | Farnesoid X receptor agonist | Nonalcoholic steatohepatitis | The FDA granted fast track designation | 8/25/20 | Gastrointestinal |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | FDA granted orphan drug designation | 8/31/20 | Genitourinary/Sexual Function |
| Knight Therapeutics Inc., of Montreal, and Therapeuticsmd Inc., of Boca Raton, Fla. | Imvexxy | Estradiol vaginal inserts | Postmenopausal moderate to severe dyspareunia, a symptom of vulvar and vaginal atrophy | Health Canada approved it | 8/25/20 | Genitourinary/Sexual Function |
| Myovant Sciences Ltd., of Basel, Switzerland | Relugolix combination tablet | Relugolix, estradiol and norethindrone acetate | Heavy menstrual bleeding associated with uterine fibroids | FDA accepted the NDA; PDUFA action date is June 1, 2021 | 8/17/20 | Genitourinary/Sexual Function |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Non-absorbed, oral polymer | Metabolic acidosis in chronic kidney disease | FDA issued a complete response letter | 8/24/20 | Genitourinary/Sexual Function |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | BCX-9930 | Complement factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | FDA granted fast track designation | 8/3/20 | Hematologic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec | Gene therapy | Severe hemophilia A | FDA issued CRL for BLA, adding a new recommendation for 2 years of data from the ongoing 270-301 phase III study to provide substantial evidence of a durable effect using annualized bleeding rate as primary endpoint | 8/19/20 | Hematologic |
| Editas Medicine Inc., of Cambridge, Mass. | EDIT-301 | Autologous CD34+ cells with a CRISPR/Cas12a modified HBG1/2 promoter region in the beta-globin locus | Sickle cell disease | FDA granted rare pediatric disease designation | 8/24/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | Submitted an MAA to the Saudi Food and Drug Authority | 8/18/20 | Hematologic |
| Imara Inc., of Boston | IMR-687 | Small-molecule inhibitor of PDE9 | Sickle cell disease | The European Commission granted orphan drug designation | 8/25/20 | Hematologic |
| Omeros Corp., of Seattle | OMS-906 | Monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 | Healthy subjects (eventually paroxysmal nocturnal hemoglobinuria) | FDA cleared an IND for a phase I study testing the safety, tolerability, pharmacodynamics and pharmacokinetics of OMS-906 | 8/31/20 | Hematologic |
| Trinity Biotech plc, of Dublin | Ultra2 | HPLC ion exchange analyzer | Beta-thalassemia screening | Company's subsidiary, Primus Corp., received an FDA warning letter for its manufacturing facility in Kansas City, Mo. | 8/17/20 | Hematologic |
| Aquestive Therapeutics Inc., of Warren, N.J. | AQST-108 | Sublingual film formulation of epinephrine | Allergic reactions including anaphylaxis | FDA granted fast track designation | 8/10/20 | Immune |
| Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon C-type natriuretic peptide | Induces natriuresis | Achondroplasia | European Commission granted orphan drug designation | 8/12/20 | Immune |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Vosoritide | Analogue of C-type natriuretic peptide | Achondroplasia | EMA validated the MAA | 8/13/20 | Immune |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Vosoritide | Analog of C-type natriuretic peptide | Achondroplasia | Submitted an NDA to the FDA | 8/20/20 | Immune |
| Catalyst Pharmaceuticals Inc., of Coral Gables, Fla. | Firdapse (amifampridine) | Voltage-dependent, potassium channel blocker | Lambert-Eaton myasthenic syndrome | Health Canada approved use of the drug | 8/6/20 | Immune |
| Catalyst Pharmaceuticals, Inc., of Coral Gables, Fla. and KYE Pharmaceuticals Inc., of Mississauga, Ontario | Firdapse (amifampridine) | Potassium channel inhibitor | Lambert-Eaton Myasthenic | Challenging Health Canada’s issuance of a Notice of Compliance on Aug. 10 for Medunik’s new drug submission for Ruzurgi (amifampridine) | 8/26/20 | Immune |
| Cerecor Inc., of Rockville, Md. | CERC-006 | Inhibitor of mTOR complexes 1 and 2 | Lymphatic malformations | FDA granted rare pediatric disease designation | 8/4/20 | Immune |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut (DBV-712) | Allergy immunotherapy | Peanut allergies | FDA issued a complete response letter for the BLA due to potential impact of patch-site adhesion on efficacy | 8/4/20 | Immune |
| Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif. | Jyseleca (filgotinib) | JAK1 inhibitor | Rheumatoid arthritis | FDA issued CRL for NDA, citing concerns about overall benefit/risk profile of the 200-mg dose and requesting completed data from the Manta and Manta-Ray studies | 8/19/20 | Immune |
| Genmab A/S, of Copenhagen, and Novartis AG, of Basel, Switzerland | Subcutaneous ofatumumab | Fully human monoclonal antibody targeting CD20 | Relapsing forms of multiple sclerosis | Received FDA clearance for the anti-CD20 monoclonal antibody Kesimpta (ofatumumab) in relapsing forms of multiple sclerosis (MS) in adults, with a label that includes clinically isolated syndrome and relapsing-remitting as well as active secondary progressive disease | 8/20/20 | Immune |
| Hansa Biopharma AB, of Lund, Sweden | Idefirix | Cleaves IgG-antibodies | Desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor | European Commission has granted conditional approval | 8/26/20 | Immune |
| Mesoblast Ltd., of New York | Ryoncil (remestemcel-L) | Culture-expanded mesenchymal stem cells derived from bone marrow of an unrelated donor | Acute graft-vs.-host disease | FDA’s Oncologic Drugs Advisory Committee voted 8-2 that data support efficacy of Ryoncil in pediatric patients with steroid-refractory aGVHD; FDA expected to make decision by Sept. 30, 2020, PDUFA date | 8/14/20 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-107 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | FDA granted rare pediatric disease designation | 8/17/20 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-207 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | FDA granted rare pediatric disease designation | 8/31/20 | Immune |
| Mylan NV, of Hertfordshire, U.K | Dimethyl fumarate | Nuclear erythroid 2-related factor 2 stimulator; Kelch-like ECH-associated protein 1 modulator | Multiple sclerosis | FDA approved the drug | 8/19/20 | Immune |
| Adamis Pharmaceuticals Inc., of San Diego | Tempol | Superoxide dismutase modulator | COVID-19 | In response to pre-IND filing, FDA provided detailed comments regarding prospective use of Tempol in randomized, placebo-controlled trial; Adamis can proceed to next step of formally submitting IND | 8/14/20 | Infection |
| Aligos Therapeutics Inc., San Francisco | ALG-000184 | Small molecule class II capsid assembly modulator | Hepatitis B virus | Submitted a clinical trial application to the New Zealand Medicines and Medical Devices Safety Authority for a first-in-human phase Ia/b proof-of-concept trial (ALG-000184-201) | 8/24/20 | Infection |
| American Gene Technologies International Inc., of Rockville, Md. | AGT103-T | Lentiviral vector-based gene therapy | HIV infection | FDA cleared IND for phase I study; enrollment expected to begin in September 2020, with initial data by year-end 2020 | 8/11/20 | Infection |
| Ana Therapeutics, of Foster City, Calif. | ANA-001 (niclosamide) | Antiviral | COVID-19 | FDA cleared the IND for a study of at least 400 patients | 8/4/20 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Favipiravir | Broad-spectrum antiviral | COVID-19 | Received FDA IND clearance to expand phase II study into U.S. testing favipiravir tablets in controlling outbreaks following exposure to COVID-19 in long-term care facilities | 8/10/20 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | AB-201 | Tissue factor inhibitor | COVID-19 infection | Following pre-IND consultation with FDA under coronavirus treatment acceleration program, IND submission for phase IIb trial expected in September 2020, with trial to begin as early as fourth quarter of 2020 | 8/12/20 | Infection |
| Bayer AG, of Basel, Switzerland | Lampit | Nifurtimox | Chagas disease | FDA approved it for the treatment of children | 8/7/20 | Infection |
| Biophytis SA, of Paris | Sarconeos (BIO-101) | Activates the MAS receptor | COVID-19 with respiratory failure | Brazilian regulatory agency, Agencia Nacional de Vigilancia Sanitaria, approved the start of the 310-patient Cova study | 8/4/20 | Infection |
| Calidi Biotherapeutics Inc., of San Diego | Adipose-derived mesenchymal stem cells | Stem cell therapy | COVID-19 infection | FDA cleared IND for phase I trial by partner Personalized Stem Cells Inc. in 20 hospitalized patients | 8/3/20 | Infection |
| Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Piclidenoson | A3 adenosine receptor agonist | COVID-19 | FDA issued a safe to proceed notice for the IND for a 40-patient phase II study | 8/31/20 | Infection |
| Capricor Therapeutics Inc., of Los Angeles | CAP-1002 | Allogeneic cardiac cell therapy | COVID-19 | The FDA accepted its IND application for a phase II clinical trial | 8/25/20 | Infection |
| Cormedix Inc., of Berkeley Heights, N.J. | Defencath | Antibacterial and antifungal catheter lock | Prevention of catheter-related bloodstream infections in hemodialysis patients | FDA accepted the NDA; PDUFA action date is Feb. 28, 2021 | 8/31/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | HIV and COVID-19 | Announced it will submit requests for pre-submission meetings in the U.K. for leronlimab as an HIV treatment in combination with HAART for highly treatment-experienced HIV patients, as well as for emergency approval for COVID-19 patients with mild to moderate symptoms | 8/7/20 | Infection |
| Enzychem Lifesciences Corp., of Englewood, N.J. | EC-18 | CCL26 gene inhibitor; TLR modulator | Acute respiratory disease syndrome due to COVID-19 pneumonia | FDA accepted the IND for a 60-patient phase II study with a primary endpoint of patients alive and free of respiratory failure through day 28 | 8/13/20 | Infection |
| Equillium Inc., of La Jolla, Calif. | Itolizumab (EQ-001) | Monoclonal antibody targeting CD6-ALCAM pathway | COVID-19 | Submitted request to FDA for pre-IND meeting to review proposal for initiating clinical trial in hospitalized patients with COVID-19 | 8/10/20 | Infection |
| FSD Pharma Inc., of Toronto | FSD-201 (ultramicronized palmitoylethanolamide) | Anti-inflammatory | COVID-19 | Submitted an IND to the FDA for a phase II study testing 2 dose levels of the drug | 8/31/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Antiviral | COVID-19 | FDA expanded the EUA to include all hospitalized patients with COVID-19, in addition to the previous authorization for patients hospitalized with severe COVID-19 | 8/28/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Antiviral | COVID-19 | Submitted NDA to FDA; final tier of rolling NDA initiated on April 8, 2020 | 8/10/20 | Infection |
| Health and Human Services Office of the Assistant Secretary for Preparedness and Response, of Washington | COVID-19 convalescent plasma | Antibodies from recovered patients | Hospitalized COVID-19 patients | FDA granted an emergency use authorization | 8/23/20 | Infection |
| Hifibio Therapeutics Inc., of Cambridge, Mass. | HFB-30132A | COVID-19 spike glycoprotein inhibitor; ACE2 inhibitor | COVID-19 | Submitted an IND for phase I ascending dose study | 8/26/20 | Infection |
| Kimera Labs Inc., of Miramar, Fla. | Xoglo | Isolated placental mesenchymal stem cell-based exosomes | Acute respiratory distress syndrome secondary to COVID-19 infection | Filed an IND application with the FDA | 8/18/20 | Infection |
| Laurent Pharmaceuticals Inc., of Montreal | LAU-7b | Oral formulation of synthetic retinoid fenretinide | COVID-19 | FDA approved the start of a phase II study | 8/27/20 | Infection |
| Micurx Pharmaceuticals Inc., of Foster City, Calif. | MRX-8 | Polymyxin antimicrobial | Infections caused by multidrug-resistant gram-negative pathogens | FDA cleared the IND for a phase I study | 8/13/20 | Infection |
| Myr Pharmaceuticals GmbH, of Bad Homburg, Germany | Hepcludex (bulevirtide) | Entry inhibitor | Chronic hepatitis delta virus infection | Received conditional marketing authorization from the European Commission | 8/4/20 | Infection |
| Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holdings AG, of Geneva | RLF-100 (aviptadil) | Vasoactive intestinal polypeptide | Prevention of respiratory failure in moderate and severe COVID-19 | FDA approved the IND for the phase II/III study | 8/6/20 | Infection |
| Organicell Regenerative Medicine Inc., of Miami | Organicell Flow | Acellular product derived from amniotic fluid | Mild to moderate respiratory distress due to COVID-19 | FDA approved emergency INDs to treat 2 patients | 8/6/20 | Infection |
| Pliant Therapeutics Inc., of South San Francisco | PLN-74809 | Dual integrin alpha-V/beta-1/6 antagonist | Acute respiratory distress syndrome associated with COVID-19 | FDA cleared the IND for a phase II study to be started in the second half of 2020 | 8/11/20 | Infection |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD | Allogeneic mesenchymal-like cells | Severe COVID-19 | Germany’s health regulatory agency cleared the company’s phase II protocol for patients hospitalized with severe cases of COVID-19 complicated by acute respiratory distress syndrome | 8/10/20 | Infection |
| Polypid Ltd., of Petah Tikva, Israel | D-Plex100 | Drug-device using controlled-release antibiotic | Post-abdominal surgical site infection | FDA granted fast track designation | 8/3/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Yeliva (opaganib) | Sphingosine kinase-2 inhibitor | COVID-19 and pneumonia | Mexican Federal Committee for the Protection against Sanitary Risks approved the CTA for a phase II/III study in up to 270 patients; primary endpoint of the study is the proportion of patients requiring intubation and mechanical ventilation by day 14 | 8/6/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Yeliva (opaganib) | Sphingosine kinase-2 inhibitor | Hospitalized COVID-19 patients with pneumonia | Mexican Federal Committee for the Protection against Sanitary Risks approved the CTA for the 270-patient phase II/III study | 8/11/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Xanthine oxidase inhibitor | COVID-19 | Submitted its clinical trial protocol for IRB approval; exploring the FDA expanded access program | 8/26/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Xanthine oxidase inhibitor | Mild to moderate COVID-19 | Phase III study received institutional review board approval | 8/31/20 | Infection |
| Revive Therapeutics Ltd., of Toronto | Bucillamine | Cysteine derivative | Mild or moderate COVID-19 | FDA approved the start of the phase III, which is scheduled to begin in September | 8/5/20 | Infection |
| Sonoran Biosciences Inc., of Tempe, Ariz. | SBG-003 (tobramycin and vancomycin hydrogel) | Antibiotic combination | Post-abdominal surgical site infection | FDA granted qualified infectious disease product designation | 8/3/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | STI-1499 (Covi-guard) | Antibody | Hospitalized COVID-19 patients | Filed IND with FDA | 8/19/20 | Infection |
| Sound Pharmaceuticals Inc., of Seattle | Ebselen (SPI-1005) | Glutathione peroxidase stimulator | Moderate or severe COVID-19 | FDA allowed the start of 2 phase II studies of 120 adults | 8/31/20 | Infection |
| Spero Therapeutics Inc., of Cambridge, Mass. | SPR-720 | Antimicrobial agent | Nontuberculous mycobacterial pulmonary disease | FDA accepted the IND for a phase IIa study, which is scheduled to start before the end of 2020 | 8/31/20 | Infection |
| TLC Biopharmaceuticals Inc., of South San Francisco | TLC-19 | Hydroxychloroquine liposome inhalation suspension | COVID-19 | Submitted IND to Taiwan FDA | 8/14/20 | Infection |
| TLC Biopharmaceuticals Inc., of South San Francisco and 3Sbio Inc., of Shenyang, China | Ampholipad (amphotericin B liposome) | Complex generic | Systemic fungal infections | NMPA's Center for Drug Evaluation accepted its marketing authorization application for Ampholipad | 8/26/20 | Infection |
| Vaxart Inc., of South San Francisco | Oral COVID-19 vaccine candidate | Vaccine | COVID-19 | Filed IND with the FDA for a phase I trial | 8/10/20 | Infection |
| Verona Pharma plc, of London | Ensifentrine | CFTR stimulator; PDE 4 inhibitor; PDE 3 inhibitor | COVID-19 | Disclosed on second-quarter earnings that FDA cleared IND to proceed with randomized, double-blind, placebo-controlled pilot study to evaluate ensifentrine delivered via pressurized metered-dose inhaler formulation in patients hospitalized with COVID-19 | 8/14/20 | Infection |
| Viiv Healthcare Ltd., of London | Dovato (dolutegravir/lamivudine) | Once-daily, single pill combining integrase strand transferase inhibitor and nucleoside analogue reverse transcriptase inhibitor | HIV-1 infection | FDA approved an expanded indication, as a complete regimen for the treatment of HIV-1 infection to replace the current antiretroviral regimen in adults who are virologically suppressed on a stable ARV regimen with no history of treatment failure and no known resistance to the individual components of Dovato | 8/7/20 | Infection |
| Xortx Therapeutics Inc., of Calgary, Alberta | XRx-101 (oxypurinol) | Xanthine oxidase inhibitor | Acute kidney injury associated with COVID-19 | Requested a pre-IND meeting with the FDA | 8/31/20 | Infection |
| Epirium Bio Inc., of San Diego | EPM-01 | Mitochondrial complex I stimulator | Becker muscular dystrophy | European Commission granted orphan designation | 8/12/20 | Musculoskeletal |
| NS Pharma Inc., of Paramus, N.J., a unit of Nippon Shinyaku Co. Ltd. | Viltepso (viltolarsen) | DMD gene modulator | Duchenne muscular dystrophy amenable to exon 53 skipping | FDA granted an accelerated approval for the drug | 8/12/20 | Musculoskeletal |
| Rocket Pharmaceuticals Inc., of New York | RP-L401 | Lentiviral vector-based gene therapy | Infantile malignant osteopetrosis | FDA granted fast track designation | 8/27/20 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | Casimersen | Binds to exon 45 of dystrophin pre-mRNA | Duchenne muscular dystrophy amenable to skipping exon 45 | The FDA accepted the NDA | 8/25/20 | Musculoskeletal |
| Seelos Therapeutics Inc., of New York | SLS-005 | Transcription factor EB stimulator | Amyotrophic lateral sclerosis | FDA cleared start of phase IIb/III trial | 8/10/20 | Musculoskeletal |
| Alkermes plc., of Dublin | ALKS-3831 | Atypical antipsychotic plus an opioid antagonist | Schizophrenia and bipolar I disorder | FDA scheduled a joint meeting of the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee to review the NDA on 10/9/20 | 8/21/20 | Neurology/Psychiatric |
| Amneal Pharmaceuticals Inc., of Bridgewater Township, N.J. | Lidocaine Patch, 5% | Generic Lidoderm patch, 5% | Post-herpetic neuralgia | The FDA approved its ANDA | 8/25/20 | Neurology/Psychiatric |
| Anavex Life Sciences Corp., of New York | ANAVEX2-73 (blarcamesine) | Activates the Sigma-1 receptor | Alzheimer’s disease | Australian Government Department of Health – Therapeutic Goods Administration approved the compassionate use special access scheme | 8/5/20 | Neurology/Psychiatric |
| Arvelle Therapeutics GmbH, of Zug, Switzerland | Cenobamate | Sodium channel inhibitor | Epilepsy | U.K. MHRA granted promising innovative medicine designation to treat drug-resistant focal-onset seizures in adults | 8/11/20 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-12 | Norepinephrine reuptake inhibitor | Narcolepsy | FDA issued breakthrough designation | 8/5/20 | Neurology/Psychiatric |
| Axsome Therapeutics Inc., of New York | AXS-07 (meloxicam + rizatriptan) | Dual 5-HT 1b/1d receptor agonist; cyclooxygenase 2 inhibitor | Migraine | Completed a successful FDA pre-NDA meeting; based on feedback the company's regulatory data package will be sufficient to support an NDA; on track to submit NDA in 4Q of 2020 | 8/20/20 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Aducanumab | Beta-amyloid antagonist | Alzheimer's disease | FDA accepted the BLA and the application was granted priority review, with a PDUFA date of March 7, 2021; the FDA has stated that, if possible, it plans to act early on this application under an expedited review | 8/7/20 | Neurology/Psychiatric |
| Cannformatics Inc., of San Francisco | Medical cannabis | Personalized treatment through identification and application of cannabis-responsive biomarkers found in saliva | Autism spectrum disorder in children | Received institutional review board approval to conduct pilot study | 8/19/20 | Neurology/Psychiatric |
| GW Pharmaceuticals plc, of London, and U.S. subsidiary Greenwich Biosciences Inc., of Carlsbad, Calif. | Epidiolex (cannabidiol) | Cannabinoid CB1/2 receptor modulator | Epilepsy | FDA approved to treat seizures associated with tuberous sclerosis complex in individuals 1 older and expanded age range to treat seizures associated with Lennox-Gastaut or Dravet syndromes to those 1 and older | 8/3/20 | Neurology/Psychiatric |
| India Globalization Capital Inc., of Potomac, Md. | IGC-AD1 | Cannabinoid formulation | Mild to severe dementia due to Alzheimer’s disease | FDA authorized the IND for a phase I study; plans to start 12-subject multiple ascending-dose study | 8/11/20 | Neurology/Psychiatric |
| Innocoll Holdings Ltd., of Athlone, Ireland | Xaracoll (bupivacaine hydrochloride collagen-matrix implant) | Anesthetic | Management of postsurgical pain after open inguinal hernia surgery | FDA approved the drug-device pain therapy | 8/31/20 | Neurology/Psychiatric |
| Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. | Spravato (esketamine, intranasal) | NMDA receptor antagonist | Major depressive disorder | FDA approved sNDA to treat depressive symptoms in adults with MDD with acute suicidal ideation or behavior | 8/3/20 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-415 | Serdexmethylphenidate co-formulated with d-methylphenidate | Attention deficit hyperactivity disorder | At midcycle communication meeting, FDA raised no substantive issues and indicated no major safety concerns noted; PDUFA date of March 2, 2021 was reaffirmed | 8/14/20 | Neurology/Psychiatric |
| Kineta Inc., of Seattle | KCP-506 | Alpha9alpha10 nicotinic acetylcholine receptor antagonist | Healthy subjects (eventually neuropathic pain) | CTA for a phase I study was approved in the Netherlands; plans to start the single and multiple ascending-dose study in the fourth quarter of 2020 | 8/12/20 | Neurology/Psychiatric |
| Neurogene Inc., of New York | Adeno-associated viral vector-based gene therapy | CLN5 gene stimulator | Batten disease | FDA granted orphan drug designation to treat CLN7 form of disease | 8/11/20 | Neurology/Psychiatric |
| Polaryx Therapeutics Inc., of Paramus, N.J. | PLX-200 | Repurposed drug that binds to the retinoid X receptor-alpha | Juvenile neuronal ceroid lipofuscinosis | Received FDA fast track designation | 8/20/20 | Neurology/Psychiatric |
| PTC Therapeutics Inc., of South Plainfield, N.J., and Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | Small-molecule SMN2-directed RNA splicing modifier | Spinal muscular atrophy | Approved by FDA under priority review; application granted a rare pediatric disease voucher | 8/7/20 | Neurology/Psychiatric |
| PTC Therapeutics Inc., of South Plainfield, N.J., and Roche Holding, of Basel, Switzerland | Evrysdi (risdiplam) | Survival motor neuron 2-directed RNA splicing modifier | Spinal muscular atrophy | EMA accepted the MAA | 8/17/20 | Neurology/Psychiatric |
| Reata Pharmaceuticals Inc., of Plano, Texas | Omaveloxolone | Activator of Nrf2 | Friedreich’s ataxia | Completed type C meeting with FDA, which said it was not convinced Moxie part 2 results will support single study approval without additional evidence and stated need for second pivotal trial to confirm results with a similar magnitude of effect; given difficulties due to slow progression rate of FA, limited number of patients for enrollment and impact of COVID-19, FDA agreed second pivotal study could result in delay of treatment to patients and asked company to submit proposed a crossover study design | 8/10/20 | Neurology/Psychiatric |
| Roche Holding, of Basel, Switzerland | Enspryng (satralizumab-mwge) | Monoclonal antibody targeting interleukin-6 | Anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder | FDA approved the drug | 8/17/20 | Neurology/Psychiatric |
| Scholar Rock Holding Corp., of Cambridge, Mass. | SRK-015 | Inhibits activation of myostatin | Spinal muscular atrophy | FDA granted rare pediatric disease designation | 8/12/20 | Neurology/Psychiatric |
| Theranexus SAS, of Lyon, France | BBDF-101 | Combination product | Batten disease | FDA granted orphan drug designation and rare pediatric disease designation | 8/11/20 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Olinvyk (oliceridine) | Opioid agonist | Management of acute pain | Approved by FDA for use in managing acute pain severe enough to require intravenous opioid analgesic and for whom alternative treatments are inadequate | 8/7/20 | Neurology/Psychiatric |
| Vanda Pharmaceuticals Inc., of Washington | Hetlioz (tasimelteon) | Melatonin MT1/MT2 receptor agonist | Smith-Magenis syndrome | FDA accepted sNDA for capsule formulation and NDA to treat adults and children, respectively, with SMS, granting priority review and setting PDUFA date of Dec. 1, 2020 | 8/3/20 | Neurology/Psychiatric |
| Vivoryon Therapeutics AG, of Halle, Germany | Varoglutamstat (PQ-912) | Targets glutaminyl cyclase | Alzheimer's disease | FDA cleared the company's IND application, enabling it to initiate its U.S. phase II trial program | 8/4/20 | Neurology/Psychiatric |
| Alimera Sciences Inc., of Atlanta | Iluvien (fluocinolone acetonide) | Intraocular implant of glucocorticoid receptor agonist | Diabetic macular edema; Uveitis | Iluvien received pricing and reimbursement approvals for both diabetic macular edema and posterior uveitis indications simultaneously | 8/26/20 | Ocular |
| Clearside Biomedical Inc., of Alpharetta, Ga. | CLS-AX (axitinib injectable suspension) | Tyrosine kinase inhibitor; pan-VEGF inhibitor | Neovascular age-related macular degeneration | FDA cleared IND for phase I/IIa study, expected to begin by year-end 2020 | 8/10/20 | Ocular |
| Eyegate Pharmaceuticals Inc., of Waltham, Mass. | Moxigel | Antibiotic in ocular bandage gel | Bacterial conjunctivitis | Received positive feedback from the pre-IND meeting with the FDA; plans to file an IND in first half of 2021 | 8/12/20 | Ocular |
| Gyroscope Therapeutics Ltd., of London | Orbit Subretinal Delivery System | Microinjection procedure designed to avoid damaging the structure of the eye by preventing the need for a vitrectomy or retinotomy | Subretinal microinjection of balanced salt solution or BSS Plus at the back of the eye | The FDA granted 510(k) clearance | 8/25/20 | Ocular |
| Ocugen Inc., of Malvern, Pa. | OCU-400 | Gene therapy consisting of functional copy of nuclear hormone receptor gene NR2E3 | PDE6B gene mutation-associated retinal diseases | FDA granted orphan drug designation | 8/10/20 | Ocular |
| Opthea Ltd., of Melbourne, Australia | OPT-302 | Soluble form of vascular endothelial growth factor receptor 3 | Neovascular (wet) age-related macular degeneration | In end of phase II meetings with the FDA and EMA, the agencies agreed with company's proposed phase III plan for 2 studies, one testing OPT-302 with Lucentis (ranibizumab, Roche Holding AG) and the second testing OPT-302 with Eylea (aflibercept, Regeneron Pharmaceuticals Inc.) | 8/21/20 | Ocular |
| Regenxbio Inc., of Rockville, Md. | RGX-314 | NAV AAV8 vector encoding an antibody fragment targeting VEGF | Diabetic retinopathy using suprachoroidal delivery | The FDA cleared the IND for a phase II trial | 8/25/20 | Ocular |
| Kubota Vision Inc., of Seattle, a subsidiary of Kubota Pharmaceutical Holdings Co. Ltd. | Emixustat hydrochloride | Synthetic small-molecule non-retinoid drug | Stargardt disease | Awarded FDA orphan products clinical trial grant to support ongoing phase III study | 8/20/20 | Ocular |
| Atyr Pharma Inc., of San Diego, and Kyorin Pharmaceutical Co. Ltd., of Tokyo | ATYR-1923 (KRP-R120) | Fusion protein comprising immuno-modulatory domain of histidyl tRNA synthetase fused to FC region of a human antibody | Interstitial lung disease | Kyorin received Japanese approval for start of phase I study in healthy male volunteers in Japan | 8/11/20 | Respiratory |
| Eloxx Pharmaceuticals Inc., of Waltham, Mass. | ELX-02 | CFTR gene modulator; ribosomal protein modulator | Cystic fibrosis | FDA granted orphan drug designation | 8/4/20 | Respiratory |
| Galecto Inc., of Boston | GB-0139 | Galectin-3 inhibitor | Idiopathic pulmonary fibrosis | FDA and EMA issued orphan drug designation | 8/27/20 | Respiratory |
| Genentech, of South San Francisco, a unit of Roche Holding AG | Xolair (omalizumab) | Monoclonal antibody targeting immunoglobulin E | Moderate to severe persistent asthma and chronic idiopathic urticaria | FDA accepted the supplementary BLA for prefilled syringes across all approved U.S. indications; decision expected by the first quarter of 2021 | 8/13/20 | Respiratory |
| Glaxosmithkline plc, of London | Trelegy Ellipta (fluticasone furoate, umeclidinium, and vilanterol inhalation powder for oral inhalation) | Muscarinic receptor antagonist; beta 2 adrenoceptor agonist | Chronic obstructive pulmonary disease | FDA’s Pulmonary-Allergy Drugs Advisory Committee voted 14-1 against supplemental NDA seeking a labeling change to include reduction in all-cause mortality in COPD; panel agreed efficacy data submitted did not support that claim | 9/1/20 | Respiratory |
| Krystal Biotech Inc., of Pittsburgh, Pa. | KB-407 | Inhaled, repeat-dose gene therapy product | Cystic fibrosis | Granted orphan drug designation by the FDA | 8/18/20 | Respiratory |
| Reviral Ltd., of London | Sisunatovir | RSV fusion inhibitor | Serious respiratory syncytial virus infection | FDA granted fast track designation | 8/4/20 | Respiratory |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso (treprostinil) | Prostacyclin vasodilator | Pulmonary hypertension associated with interstitial lung disease | FDA accepted the supplementary NDA; decision expected in April 2021 | 8/17/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kaftrio (ivacaftor/tezacaftor/elexacaftor) | Combination of CFTR potentiator and 2 CFTR correctors | Cystic fibrosis ages 12 years and older with 1 F508del mutation and 1 minimal function mutation or 2 F508del mutations in the cystic fibrosis transmembrane conductance regulator gene | European Commission granted marketing authorization for the drug in combination with ivacaftor | 8/21/20 | Respiratory |
| BTG Specialty Pharmaceuticals, of London, a unit of Boston Scientific Corp. | Voraxaze (glucarpidase) | Methotrexate antidote | Methotrexate toxicity due to delayed methotrexate elimination | EMA accepted the MAA | 8/17/20 | Toxicity and Intoxication |
| Emergent Biosolutions Inc., of Gaithersburg, Md. | Narcan (naloxone) | Naloxone nasal spray | Opioid overdose | FDA approved the extension of shelf-life from 24 months to 36 months | 8/17/20 | Toxicity and Intoxication |
| Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. | Pedmark (sodium thiosulfate) | Otoprotectant | Ototoxicity | FDA issued CRL for NDA covering intravenous administration to prevent ototoxicity associated with cisplatin chemotherapy in patients ?1 month to 18 years of age with localized, non-metastatic solid tumors; company plans to request type A meeting | 8/11/20 | Toxicity and Intoxication |
| G1 Therapeutics Inc., of Research Triangle Park, N.C. | Trilaciclib | Myelopreservation agent | Small-cell lung cancer being treated with chemotherapy | FDA accepted the NDA and granted a priority review; PDUFA action date is Feb. 15, 2021 | 8/17/20 | Toxicity and Intoxication |
| Jaguar Health Inc., of San Francisco, CA | Mytesi (crofelemer) | Antidiarrheal | Prophylaxis and symptomatic relief of diarrhea in adult patients with solid tumors receiving targeted therapy with or without standard chemotherapy | FDA approved the IND | 8/6/20 | Toxicity and Intoxication |
| Tonix Pharmaceuticals Holding Corp., of New York | TNX-102 SL (cyclobenzaprine HCl sublingual tablets) | Non-opioid, centrally acting analgesic | Alcohol use disorder | FDA cleared the IND for a phase II study | 8/6/20 | Toxicity and Intoxication |
| ADC Therapeutics SA, of Lausanne, Switzerland | Loncastuximab tesirine (ADCT-402) | CD19-targeted antibody-drug conjugate | Relapsed or refractory diffuse large B-cell lymphoma | Submitted a BLA to the FDA | 9/22/20 | Cancer |
| Advaxis Inc., of Princeton, N.J. | ADXS-504 | Listeria monocytogenes secreting antigen-adjuvant fusion proteins | Prostate cancer | FDA cleared IND for investigator-sponsored phase I monotherapy study, expected to begin before year-end 2020 | 9/24/20 | Cancer |
| Agenus Inc., of Lexington, Mass. | Balstilimab | Anti-PD-1 monoclonal antibody | Recurrent/metastatic cervical cancer | Initiated rolling BLA to the FDA | 9/18/20 | Cancer |
| Apollomics Inc., of Foster City, Calif. | APL-106 (uproleselan) | Inhibitor of E-selectin | Relapsed/refractory acute myeloid leukemia | Received IND approval from the China National Medical Products Administration for a phase I pharmacokinetics and tolerability study, which includes acceptance of a phase III bridging study of APL-106 | 9/28/20 | Cancer |
| Apros Therapeutics Inc., of San Diego | APR-003 | Oral gastrointestinal- and liver-targeted TLR7 agonist | Colorectal cancer | FDA cleared IND for a phase I dose-escalation study in patients with advanced unresectable CRC with malignant liver lesions | 9/9/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 inhibitor | Chronic lymphocytic leukemia | FDA granted orphan drug designation | 9/7/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-115 | MDM2-p53 inhibitor | MDM2-p53 inhibitor | FDA granted orphan drug designation | 9/14/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Koselugo (selumetinib) | Mitogen-activated protein kinase kinases 1 and 2 inhibitor | Neurofibromatosis type 1 | FDA issued a rare pediatric disease priority review voucher; to be published in Federal Register Sept. 16, 2020 | 9/15/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Antibody targeting PD-L1 | Small-cell lung cancer | European Union approved for use in first-line treatment in adults with extensive-stage disease in combination with etoposide plus either carboplatin or cisplatin | 9/1/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the application seeking use in patients with BRCA1/2 mutations after progression following a prior therapy that included a new hormonal agent | 9/21/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Advanced, high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the application to approve the drug in combination with bevacizumab in patients with tumors that have a BRCA1/2 mutation and/or genomic instability who have responded to a completed first-line platinum-based chemotherapy | 9/21/20 | Cancer |
| Atara Biotherapeutics Inc., of South San Francisco | ATA-2271 | Autologous CAR T therapy targeting mesothelin | Advanced mesothelioma | FDA accepted the IND for a single-arm phase I study | 9/8/20 | Cancer |
| Athenex Inc., of Buffalo, N.Y. | Oral paclitaxel and encequidar | Oral taxane with P-gp inhibitor to increase absorption | Metastatic breast cancer | FDA accepted for filing the NDA, granting priority review; PDUFA date set for Feb. 28, 2021 | 9/1/20 | Cancer |
| Athenex Inc., of Buffalo, N.Y. | TCRT-ESO-A2 | Autologous T-cell receptor T-cell therapy | Solid tumors | FDA cleared IND for phase I trial targeting NY-ESO-1-positive tumors in people who are HLA-A 02:01-positive | 9/23/20 | Cancer |
| Beigene Ltd., of Cambridge, Mass., and Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Waldenström's macroglobulinemia | New drug submission accepted by Health Canada and granted priority review status | 9/9/20 | Cancer |
| Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakyt (avapritinib) | Kinase inhibitor | Gastrointestinal stromal tumors | European commission granted conditional marketing authorization for use as monotherapy in adults with unresectable or metastatic disease harboring PGFRA D842V mutation | 9/25/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | PD-1 inhibitor and CTLA4 inhibitor | Malignant pleural mesothelioma | EMA validated a type II variation application seeking use in patients with previously untreated, unresectable disease; validation begins the EMA’s centralized review process | 9/15/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Onureg (azacitidine 300-mg tablets; CC-486) | Oral hypomethylating agent | Acute myeloid leukemia | Approved by FDA for use in continued treatment of adult patients who achieved first complete remission (CR) or CR with incomplete blood count recovery following intensive induction chemotherapy and who are not able to complete intensive curative therapy | 9/1/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | Anti-PD-1 antibody and anti-CTLA4 antibody | Metastatic non-small-cell lung cancer | EMA’s Committee for Medicinal Products for Human Use recommended approval of combo for use with 2 cycles of platinum-based chemotherapy in first-line treatment in adults whose tumors have no sensitizing EGFR mutation or ALK translocation | 9/18/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Bluebird Bio Inc., of Cambridge, Mass. | Idecabtagene vicleucel (bb-2121) | BCMA-directed CAR T-cell immunotherapy | Relapsed and refractory multiple myeloma | FDA accepted the BLA for priority review; PDUFA goal date of March 27, 2021 | 9/22/20 | Cancer |
| Canbridge Pharmaceuticals Inc., of Beijing and Cambridge, Mass. | Hunterase (idursulfase beta injection) | Enzyme replacement therapy | Mucopolysaccharidosis II (Hunter syndrome) | Approved by China’s NMPA for long-term treatment | 9/9/20 | Cancer |
| Caribou Biosciences, of Berkeley, Calif. | CB-010 | Allogeneic CAR T targeting CD19 | Relapsed/refractory B-cell non-Hodgkin lymphoma | FDA cleared the IND for a phase I study | 9/8/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Enhertu (trastuzumab deruxtecan) | HER2-directed antibody-drug conjugate | Gastric cancer | Approved by Japan’s MHLW for use in patients with HER2-positive unresectable advanced or recurrent disease that has progressed after chemotherapy | 9/25/20 | Cancer |
| Eagle Pharmaceuticals Inc., of Woodcliff Lake, N.J., and Symbio Pharmaceuticals Ltd., of Tokyo | Treakisym (bendamustine hydrochloride) | PARP modulator | Non-Hodgkin’s lymphoma; mantle cell lymphoma; chronic lymphocytic leukemia | Japan PMDA approved ready-to-dilute liquid formulation to treat approved indications, replacing lyophilized formulation at launch in January 2021 | 9/23/20 | Cancer |
| Epimab Biotherapeutics Inc., of Shanghai | EMB-02 | Bispecific antibody targeting PD-1 and LAG-3 | Solid tumors | Received study may proceed letter from FDA in response to IND | 9/15/20 | Cancer |
| Essa Pharmaceuticals Inc., of Houston | EPI-7386 | N-terminal domain inhibitor of the androgen receptor | Metastatic castration-resistant prostate cancer resistant to standard-of-care treatment | FDA granted fast track designation | 9/14/20 | Cancer |
| Gan & Lee Pharmaceuticals Co. Ltd., of Beijing | GLR-2007 | Cyclin-dependent kinase 4/6 inhibitor | Malignant glioma | FDA granted orphan drug designation | 9/11/20 | Cancer |
| Genocea Biosciences Inc., of Cambridge, Mass. | GEN-011 | T-cell therapy | Tumors | FDA accepted the IND application | 9/22/20 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Magrolimab | Anti-CD47 monoclonal antibody | Myelodysplastic syndrome | FDA granted breakthrough therapy designation for treating newly diagnosed MDS | 9/15/20 | Cancer |
| Glaxosmithkline plc, of London | Zejula (niraparib) | PARP inhibitor | Advanced ovarian cancer | EMA’s Committee for Medicinal Products for Human Use recommended approval for use as first-line maintenance treatment in women who responded to platinum-based chemotherapy, regardless of biomarker status | 9/18/20 | Cancer |
| Harbour Biomed Therapeutics Ltd., of Suzhou, China | HBM-4003 | Anti-CTLA4 antibody | Advanced solid tumors | China National Medical Products Administration approved 2 INDs for phase I studies of the drug as a monotherapy and in combination with Tuoyi (toripalimab) | 9/21/20 | Cancer |
| Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong | Surufatinib | Angio-immuno kinase inhibitor | Pancreatic neuroendocrine tumors | China's NMPA accepted NDA covering treatment of advanced disease | 9/17/20 | Cancer |
| Huya Bioscience International LLC, of San Diego | HBI-8000 | Targets class I histone deacetylase | Relapsed or refractory adult T-cell leukemia-lymphoma | Japanese Ministry of Health, Labour and Welfare granted orphan drug designation | 9/28/20 | Cancer |
| I-Mab Biopharma Co. Ltd., of Shanghai | Lemzoparlimab | Anti-CD47 monoclonal antibody | Relapsed or refractory advanced lymphoma | Center for Drug Evaluation of the China National Medical Products Administration cleared the IND for a phase I study | 9/21/20 | Cancer |
| Infinity Pharmaceuticals Inc., of Cambridge, Mass. | Eganelisib (IPI-549) | PI3K-gamma inhibitor | Inoperable locally advanced or metastatic triple-negative breast cancer | FDA granted fast track designation in combination with a checkpoint inhibitor and chemotherapy | 9/29/20 | Cancer |
| Innova Therapeutics Inc., of Charleston, S.C. | IVT-8086 | Secreted frizzled-related protein 2 antagonist | Osteosarcoma | FDA granted rare pediatric disease designation | 9/24/20 | Cancer |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Uptravi (selexipag) | Oral prostacyclin receptor agonist | Pulmonary arterial hypertension | Submitted NDA to the FDA for intravenous version of the drug for patients who are currently taking the Uptravi but are temporarily unable to take oral therapy | 9/30/20 | Cancer |
| Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Monoclonal antibody targeting PD-1 | Nasopharyngeal carcinoma | FDA granted breakthrough therapy designation | 9/10/20 | Cancer |
| Junshi Biosciences Inc., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Soft tissue sarcoma | FDA granted orphan designation | 9/17/20 | Cancer |
| Kite Pharma Inc., a unit of Foster City, Calif.-based Gilead Sciences Inc. | Yescarta (axicabtagene ciloleucel) | Autologous CAR T-cell therapy targeting CD19 | Relapsed or refractory follicular lymphoma and marginal zone lymphoma after 2 or more lines of systemic therapy | Submitted a supplemental BLA to the FDA | 9/4/20 | Cancer |
| Kleo Pharmaceuticals Inc., of New Haven, Conn. | KP-1237 | CD38-targeting antibody recruiting molecule | Multiple myeloma | FDA granted orphan drug designation | 9/8/20 | Cancer |
| Lantheus Holdings Inc., of North Billerica, Mass. | Pyl (18F-DCFPyL) | Prostate-specific membrane antigen-targeted positron emission tomography imaging agent | Prostate cancer | Submitted NDA to the FDA with a request for a priority review | 9/30/20 | Cancer |
| Leap Therapeutics Inc., of Cambridge, Mass. | DKN-01 | Dickkopf-1 ligand inhibitor | Gastric and gastroesophageal junction cancer | FDA granted fast track designation | 9/24/20 | Cancer |
| Mateon Therapeutics, of Agoura Hills, Calif. | Oxi-4503 | Cis-combretastatin A1 dipotassium diphosphate | Acute myeloid leukemia | FDA granted rare pediatric disease designation | 9/16/20 | Cancer |
| Menarini Group, of Florence, Italy | Elzonris (tagraxofusp) | Targeted therapy directed to CD123 | Blastic plasmacytoid dendritic cell neoplasm | Applicant requested a re-examination of the EMA’s July 2020 negative opinion on the MAA | 9/18/20 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | Anthracycline | Soft tissue sarcomas | Completed pre-IND meeting with FDA regarding development plan, including study design and dosing strategy for initial phase Ib/II protocol for STS with lung metastases | 9/9/20 | Cancer |
| Morphosys AG, of Planegg, Germany, and I-Mab Biopharma Co. Ltd., of Shanghai | MOR-210/TJ-210 | Anti-C5aR1 antibody | Relapsed or refractory advanced solid tumors | FDA cleared the IND for phase I trial | 9/17/20 | Cancer |
| Nanomab Technology Ltd., of London | NM-01 | Imaging agent; PD-L1 modulator | Cancer | Submitted request to U.K.’s MHRA to start phase II study assessing PD-L1 expression using 99mTc-NM-01 as a SPECT/CT radiotracer and its correlation to PD-L1 expression results based on routine immunohistochemical testing in biopsy diagnostic specimens | 9/9/20 | Cancer |
| Oblato Inc., of Oklahoma City, a subsidiary of Gtreebnt Co. Ltd. | OKN-007 | Sulfatase inhibitor | Diffuse intrinsic pontine glioma | FDA granted rare pediatric disease designation | 9/2/20 | Cancer |
| Oblato Inc., of Oklahoma City, a subsidiary of Gtreebnt Co. Ltd. | OKN-007 | Sulfatase inhibitor | Malignant glioma | FDA granted orphan designation | 9/2/20 | Cancer |
| Oncoheroes Biosciences Inc., of Boston | Volasertib | PLK1 inhibitor | Rhabdomyosarcoma | FDA granted rare pediatric disease designation | 9/25/20 | Cancer |
| Pfizer Inc., of New York | Xalkori (crizotinib) | ALK tyrosine kinase inhibitor | Anaplastic large cell lymphoma | FDA accepted sNDA, granting priority review, to treat children with relapsed or refractory systemic disease that is ALK positive, setting PDUFA date in January 2021 | 9/23/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cypcaps | Genetically engineered human cells encapsulated using the Cell-in-a-Box technology | Pancreatic cancer | Submitted IND to FDA for a phase IIb trial in patients with locally advanced, inoperable disease | 9/2/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cypcaps | Genetically engineered human cells encapsulated using the Cell-in-a-Box technology | Locally advanced, inoperable pancreatic cancer | Completed 6-month stability testing required by the FDA | 9/14/20 | Cancer |
| Plus Therapeutics Inc., of Austin, Texas | Rhenium Nanoliposomes | Nanoparticle radioisotope | Recurrent glioblastoma | FDA granted orphan designation | 9/1/20 | Cancer |
| Plus Therapeutics Inc., of Austin, Texas | Rhenium Nanoliposomes | Nanoliposome-encapsulated radionuclide | Recurrent glioblastoma | FDA granted fast track designation | 9/15/20 | Cancer |
| Precision Biosciences Inc., of Durham, N.C. | PBCAR-269A | CAR T-cell therapy; APRIL receptor modulator | Relapsed/refractory multiple myeloma | FDA granted fast track designation | 9/9/20 | Cancer |
| Protara Therapeutics Inc., of New York | TARA-002 | Based on broad immunopotentiator OK-432 | Non-muscle invasive bladder cancer and lymphatic malformations | Following pre-IND meeting with FDA, agency agreed company has demonstrated initial manufacturing comparability between TARA-002 and OK-432 and that company was on track with its plans to conduct 3 large-scale batch runs to confirm comparability; also reached alignment with FDA on proposed clinical development plan in NMIBC, with phase I study expected to begin in 2021; plans to request meeting with FDA by year-end to discuss regulatory path in LMs | 9/8/20 | Cancer |
| Rakuten Medical Inc., of San Mateo, Calif. | Akalux I.V. infusion 250 mg (cetuximab sarotalocan sodium) | EGFR inhibitor | Head and neck cancer | Japan’s MHLW approved for use in patients with unresectable locally advanced or recurrent disease | 9/25/20 | Cancer |
| Remegen Co. Ltd., of Yantai, China | Disitamab vedotin (RC-48) | Humanized anti-HER2 antibody-drug conjugate | Urothelial cancer | FDA granted breakthrough therapy designation for second-line treatment of patients with HER2-positive, locally advanced or metastatic disease who have also previously received platinum-containing chemotherapy | 9/25/20 | Cancer |
| Roche Group, of Basel Switzerland | Gavreto | Pralsetinib | Metastatic RET fusion-positive non-small-cell lung cancer | FDA approved for the treatment of adults, and granted priority review for the treatment of people with advanced or metastatic RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer | 9/8/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | Anti-PD-L1 inhibitor | Advanced or unresectable hepatocellular carcinoma | EMA’s Committee for Medicinal Products for Human Use recommended approval for use in adults who have not received prior systemic therapy | 9/18/20 | Cancer |
| Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | Targets WT1 protein | Acute myeloid leukemia | France’s regulatory authority cleared the start of the pivotal phase III Regal study in patients with AML wo have achieved complete remission after second-line anti-leukemic therapy | 9/9/20 | Cancer |
| Seneca Therapeutics Inc., of Philadelphia | SVV-001 | Naturally occurring Seneca Valley virus active against cancer cells | Neuroendocrine cancer | Received positive feedback from the FDA on the reactivation of the IND; plans to start a phase I/II study in the second quarter of 2021 | 9/14/20 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX591-CDx (68Ga-PSMA-11) | Imaging agent | Prostate cancer | NDA submitted to FDA for radiopharmaceutical targeting prostate-specific membrane antigen using PET | 9/24/20 | Cancer |
| Tokyo-based Chugai Pharmaceutical Co. Ltd., of Tokyo (subsidiary of Roche Holding AG) | Tecentriq (atezolizumab) and Avastin (bevacizumab): | Drug combination | Hepatocellular carcinoma | Received MHLW approval | 9/29/20 | Cancer |
| Xcures Inc., of San Francisco, and Biomed Valley Discoveries Inc., of Kansas City, Mo. | Ulixertinib (BVD-523) | ERK inhibitor | Cancer | FDA granted IND for intermediate expanded access program open to adolescent and adult cancer patients who cannot access an open clinical trial | 9/28/20 | Cancer |
| Xiangxue Life Sciences Ltd., of Nanjing, China | TAEST-16001 | TCR-T program targeting tumors that are NY-ESO-1-positive | Solid tumors | Partner Genscript Probio said FDA cleared IND for phase I study | 9/24/20 | Cancer |
| Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Ovarian cancer | NMPA approved sNDA for first-line maintenance treatment of advanced epithelial ovarian, fallopian tube or primary peritoneal cancer in adults with complete or partial response to first-line platinum-based chemotherapy | 9/10/20 | Cancer |
| Ziopharm Oncology Inc., of Boston | Ad-RTS-hIL-12 with veledimex | Controlled IL-12 | Diffuse intrinsic pontine glioma | FDA granted rare pediatric disease designation | 9/14/20 | Cancer |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis | Jardiance (empagliflozin) | SGLT2 inhibitor | Heart failure | FDA granted fast track designation for development to prevent hospitalization for heart failure and reduce risk of mortality in patients, with and without diabetes, who have had an acute myocardial infarction | 9/15/20 | Cardiovascular |
| Chemocentryx Inc., of Mountain View, Calif. | Avacopan | Complement 5a receptor inhibitor | Vasculitis | FDA accepted NDA covering treatment of ANCA-associated disease, setting July 7, 2021, PDUFA date; agency did not issue determination on advisory committee meeting | 9/17/20 | Cardiovascular |
| Medeor Therapeutics Inc., of San Mateo, Calif. | MDR-101 | Uses the organ donor’s immune cells transfused into the organ recipient to create a mixed chimeric immune state in the recipient | Kidney transplant rejection | FDA granted regenerative medicine advanced therapy designation | 9/22/20 | Cardiovascular |
| Silence Therapeutics plc, of London | SLN-360 | Silences the apolipoprotein(a) component of lipoprotein(a) | Cardiovascular disease associated with high lipoprotein(a) levels | FDA approved the IND application, and company aims to start dosing healthy volunteers in the phase I trial by the end of the year | 9/8/20 | Cardiovascular |
| Amryt plc, of Dublin | AP-103 | Gene therapy | Dystrophic epidermolysis bullosa | EMA’s Committee for Orphan Medicinal Products adopted a positive opinion for orphan designation | 9/15/20 | Dermatologic |
| Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | ARQ-151 (topical roflumilast cream) | PDE4 inhibitor | Atopic dermatitis | After meeting with the FDA, company has decided not to run a planned phase IIb study and instead start a phase III program in late 2020 or early 2021 | 9/8/20 | Dermatologic |
| Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., Tokyo | Sofpironium bromide gel, 5% | Anticholinergic | Primary axillary hyperhidrosis | Kaken received marketing approval in Japan under brand name Ecclock | 9/25/20 | Dermatologic |
| CSL Behring, of King of Prussia, Pa. | Haegarda | C1 esterase inhibitor | Hereditary angioedema prophylaxis | FDA approved the expanded indication for the prevention of hereditary angioedema | 9/28/20 | Dermatologic |
| Eli Lilly and Co., of Indianapolis | Baricitinib | JAK1/2 inhibitor | Moderate to severe atopic dermatitis | EMA’s Committee for Medicinal Products for Human Use issued positive opinion for treating adults who are candidates for systemic therapy | 9/18/20 | Dermatologic |
| Histogen Inc., of San Diego | HST-002 | Human-derived collagen and extracellular matrix dermal filler | Facial folds and wrinkles | FDA’s Office of Combination Products deemed product a drug-biologic-device combination, which will be assigned to Center for Biologics Evaluation Research Office of Tissues and Advanced Therapies as the agency lead for premarket review and regulation | 9/29/20 | Dermatologic |
| Medimetriks Pharmaceuticals Inc., of Fairfield, N.J. | MM-36 (difamilast) | PDE4b inhibitor | Mild to moderate atopic dermatitis | Received type C meeting written response from FDA, and is now preparing to conduct single pivotal trial for NDA submission, to be supported by data from already-completed phase III trials conducted in Japan | 9/18/20 | Dermatologic |
| Mediwound Ltd., of Yavne, Israel | Nexobrid | Proteolytic enzymes enriched in bromelain | Eschar removal in deep partial-thickness and/or full-thickness thermal burns | FDA accepted the BLA; PDUFA target action date is June 29, 2021 | 9/16/20 | Dermatologic |
| Olix Pharmaceuticals Inc., of Suwon, Republic of Korea | OLX-10010 | CTGF gene inhibitor | Hypertrophic scars | Submitted IND to FDA to test as adjunct therapy to reduce recurrence of hypertrophic scars after scar revision surgery | 9/24/20 | Dermatologic |
| Otsuka Pharmaceutical Co. Ltd., of Tokyo, and Medimetriks Pharmaceuticals Inc., of Fairfield, N.J. | Difamilast (OPA-15406) | PDE4 inhibitor | Mild to moderate atopic dermatitis | Otsuka submitted an NDA for approval in Japan | 9/29/20 | Dermatologic |
| Sol-Gel Technologies Ltd., of Ness Ziona, Israel | Epsolay (benzoyl peroxide) | Microencapsulated benzoyl peroxide cream | Inflammatory lesions of rosacea | FDA accepted the NDA; PDUFA action date is April 26, 2021 | 9/10/20 | Dermatologic |
| UCB SA, of Brussels | Bimekizumab | Interleukin-17F ligand inhibitor | Moderate to severe plaque psoriasis | FDA accepted the BLA and the EMA accepted the MAA | 9/22/20 | Dermatologic |
| Applied Therapeutics Inc., of New York | PMM2-CDG | Aldose reductase inhibitor | Congenital disorder of glycosylation type Ia | FDA granted rare pediatric disease and orphan drug designations | 9/24/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon hGH (lonapegsomatropin) | Long-acting prodrug of somatropin | Pediatric growth hormone deficiency | FDA accepted the BLA and indicated that it doesn't currently plan to hold an advisory committee meeting to discuss the application; PDUFA action date is June 25, 2021 | 9/4/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Growth hormone deficiency | Clinical trial notification filed with Japan PMDA for phase III Right trial of long-acting somatropin produg to treat prepubertal treatment-naïve children | 9/23/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon PTH | Parathyroid hormone prodrug | Adult hypoparathyroidism | IND amendment submitted to FDA to initiate U.S. sites of phase III Pathway trial, with primary composite endpoint at 26 weeks of proportion of participants with serum calcium in normal range, independence from active vitamin D and taking ?600 mg/day of calcium supplements | 9/29/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon hGH (lonapegsomatropin) | Long-acting, once-weekly prodrug of somatropin (human growth hormone) | Growth hormone deficiency | Submitted MAA to EMA seeking approval for treatment of pediatric patients diagnosed with GHD | 9/8/20 | Endocrine/Metabolic |
| Avrobio Inc., of Cambridge, Mass. | AVR-RD-02 | Autologous hematopoietic stem cells, genetically modified to express glucocerebrosidase | Gaucher disease | European Commission granted orphan drug designation | 9/28/20 | Endocrine/Metabolic |
| Bridgebio Pharma Inc., of Boston, and subsidiary Origin Biosciences | Fosdenopterin (BBP-870/ORGN-001) | cPMP substrate replacement therapy | Molybdenum cofactor deficiency type A | FDA accepted its NDA and granted priority review designation | 9/29/20 | Endocrine/Metabolic |
| Crinetics Pharmaceuticals Inc., of San Diego | CRN-04777 | Non-peptide somatostatin receptor type 5 agonist | Congenital hyperinsulinism | FDA granted rare pediatric disease designation | 9/21/20 | Endocrine/Metabolic |
| Cycle Pharmaceuticals Ltd., of Boston | Nityr (nitisinone) | Inhibits catabolism of tyrosine | Hereditary tyrosinemia type 1 | FDA approved once-daily dosing of the drug | 9/14/20 | Endocrine/Metabolic |
| Diurnal Group plc, of London | Alkindi Sprinkle (hydrocortisone oral granules) | Steroid | Adrenocortical insufficiency | FDA approved the drug; launch expected in the fourth quarter of 2021 | 9/30/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Trulicity (dulaglutide) | Glucagon-like peptide-1 analogue | Type 2 diabetes | FDA approved 2 higher doses of the drug | 9/3/20 | Endocrine/Metabolic |
| Eli Lilly and Co., of Indianapolis | Trulicity (dulaglutide) | Glucagon-like peptide-1 analogue | Diabetes mellitus | Health Canada approved the new indication to reduce the risk of nonfatal stroke in adults with type 2 diabetes mellitus who have multiple cardiovascular risk factors or established cardiovascular disease, as an adjunct to diet, exercise and standard-of-care therapy | 9/22/20 | Endocrine/Metabolic |
| Genmab A/S, of Copenhagen, and Halozyme Therapeutics Inc., of San Diego | Darzalex Faspro (daratumumab + hyaluronidase-fihj) | ADP ribosyl cyclase-1 inhibitor | Light-chain amyloidosis | Partner Janssen Biotech Inc. submitted sBLA to FDA for use in combination with bortezomib, cyclophosphamide and dexamethasone; agency to review under Real-Time Oncology Review pilot program | 9/10/20 | Endocrine/Metabolic |
| I-Mab Biopharma Co. Ltd., of Shanghai | Eftansomatropin (TJ-101) | Long-acting recombinant human growth hormone | Growth hormone deficiency in pediatric patients | China Center for Drug Evaluation approved the pivotal phase III trial application | 9/30/20 | Endocrine/Metabolic |
| Liminal Biosciences Inc., of Laval, Quebec | Ryplazim (plasminogen) | Recombinant protein | Congenital plasminogen deficiency | FDA received the BLA resubmission by its U.S. subsidiary, Prometic Biotherapeutics Inc., and assigned a PDUFA target action date March 5, 2021 | 9/21/20 | Endocrine/Metabolic |
| LNC Therapeutics SA, of Bordeaux, France | Xla1 | Single strain Live Biotherapeutic Product | Obesity and metabolic disorders | FDA approved the IND application | 9/8/20 | Endocrine/Metabolic |
| Matinas Biopharma Holdings Inc., of Bedminster, N.J. | MAT-9001 | Omega-3 therapy | Severe hypertriglyceridemia | Minutes from end-of-phase II meeting with FDA confirmed alignment on key next steps for phase III program and registration pathway; company remains on track to start phase III in first half of 2021 | 9/15/20 | Endocrine/Metabolic |
| Orchard Therapeutics Inc., of Boston and London | OTL-203 | Ex vivo autologous hematopoietic stem cell gene therapy | Mucopolysaccharidosis type I | EMA granted priority medicines (PRIME) designation | 9/28/20 | Endocrine/Metabolic |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Chaperonin/Hsp70 stimulator | Niemann-Pick disease type C | FDA accepted the NDA and granted a priority review; PDUFA target action date is March 17, 2021 | 9/16/20 | Endocrine/Metabolic |
| Provention Bio Inc., of Red Bank, N.J. | Teplizumab (PRV-031) | Anti-CD3 monoclonal antibody | Delay or prevention of type 1 diabetes | Submitted clinical module of BLA to FDA for use in at-risk individuals; final modules, including CMC module, expected to be submitted in the fourth quarter of 2020 | 9/30/20 | Endocrine/Metabolic |
| Regenxbio Inc., of Rockville, Md. | RGX-121 | Gene therapy expressing iduronate-2-sulfatase | Mucopolysaccharidosis type II | FDA cleared an IND for a phase I/II study of patients ages 5-18; study expected to start in the second half of 2020 | 9/30/20 | Endocrine/Metabolic |
| Swedish Orphan Biovitrum AB, of Stockholm | Orfadin (nitisinone) | Hydroxyphenylpyruvate dioxygenase inhibitor | Alkaptonuria | EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for treating adults | 9/18/20 | Endocrine/Metabolic |
| Gannex Pharma Co. Ltd., of Shanghai | ASC-42 | Farnesoid X receptor agonist | Nonalcoholic steatohepatitis | Filed an IND with the FDA | 9/13/20 | Gastrointestinal |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Monoclonal antibody targeting interleukin-4 | Eosinophilic esophagitis | FDA granted breakthrough designation | 9/14/20 | Gastrointestinal |
| Seed Health, of Boston | DS-01 | Broad-spectrum multispecies, multistrain probiotic | Irritable bowel syndrome | FDA authorized its IND application, and the company plans to initiate a phase II trial | 9/29/20 | Gastrointestinal |
| Terns Pharmaceuticals Inc., of Foster City, Calif. | TERN-201 | Vascular adhesion protein-1 inhibitor | Nonalcoholic steatohepatitis | FDA granted fast track designation | 9/10/20 | Gastrointestinal |
| Ardelyx Inc., of Fremont, Calif. | Tenapanor | Acts locally in the gut to inhibit sodium hydrogen exchanger 3 | Chronic kidney disease | FDA accepted NDA seeking approval to control serum phosphorus in adults with CKD on dialysis | 9/15/20 | Genitourinary/Sexual Function |
| Knight Therapeutics Inc., of Montreal, and Therapeuticsmd Inc., of Boca Raton, Fla. | Bijuva (estradiol + progesterone) | Estradiol agonist; progesterone receptor agonist | Moderate to severe vasomotor symptoms associated with menopause in women | Health Canada approved it | 9/22/20 | Genitourinary/Sexual Function |
| Mallinckrodt plc, of Dublin | Terlipressin | Vasopressin analogue selective for V1 receptors | Hepatorenal syndrome type 1 | FDA issued the company a third complete response letter | 9/14/20 | Genitourinary/Sexual Function |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on a new 100-mg/mL intravenous formulation | 9/21/20 | Hematologic |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Atypical hemolytic uremic syndrome | Approved by Japan’s MHLW for use in adults and children | 9/25/20 | Hematologic |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Targeted C3 inhibitor | Paroxysmal nocturnal hemoglobinuria | Submitted NDA to the FDA and MAA to the EMA | 9/15/20 | Hematologic |
| Beyondspring Inc., of New York | Plinabulin | Guanine nucleotide exchange factor stimulator; tubulin receptor antagonist | Chemotherapy-induced neutropenia | Received breakthrough designations from both the FDA and China’s Center for Drug Evaluation of the National Medical Products Administration | 9/8/20 | Hematologic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Roctavian (valoctocogene roxaparvovec) | Gene therapy | Severe hemophilia A | Company disclosed in 8-K filing that it received EMA’s joint assessment report related to ongoing review of MAA; EMA requests company to submit full 52-week results from 134 patients in ongoing phase III study with the 6e13 vg/kg dose; last patient expected to reach 52 weeks in November 2020, and Biomarin is working with EMA to enable potential submission of requested data by end of first quarter of 2021 | 9/9/20 | Hematologic |
| Bluebird Bio Inc., of Cambridge, Mass. | Lentiglobin (betibeglogene autotemcel) | HBB gene stimulator | Sickle cell disease | EMA granted PRIME designation | 9/23/20 | Hematologic |
| Bristol Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma Inc., of Cambridge, Mass. | Reblozyl | Luspatercept | Red blood cell transfusion-dependent anemia associated with beta-thalassemia | Health Canada approved it | 9/29/20 | Hematologic |
| Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Boston | CTX-001 | Autologous, gene-edited hematopoietic stem cell therapy | Sickle cell disease | EMA granted Priority Medicines (PRIME) designation | 9/22/20 | Hematologic |
| CTI Biopharma Corp., of Seattle | Pacritinib | JAK2, FLT3, IRAK1 and CSF1R kinase inhibitor | Myelofibrosis with severe thrombocytopenia | At a pre-NDA meeting, company and FDA reached an agreement to submit an NDA based on the phase III Persist-1 and -2 studies and the phase II PAC203 dose-ranging study; company plans to start the rolling submission in the first quarter of 2021; ongoing phase III Pacifica study will be completed as a postmarketing commitment | 9/29/20 | Hematologic |
| Glaxosmithkline plc, of London | Nucala (mepolizumab) | Monoclonal antibody targeting IL-5 | Hypereosinophilic syndrome | FDA approved the drug | 9/25/20 | Hematologic |
| Glaxosmithkline plc, of London | Nucala (mepolizumab) | Monoclonal antibody targeting IL-5 | Hypereosinophilic syndrome | FDA approved the supplemental NDA | 9/28/20 | Hematologic |
| Pfizer Inc., of New York | Nyvepria (pegfilgrastim) | Neulasta biosimilar | Neutropenia | EMA’s Committee for Medicinal Products for Human Use recommended approval to treat neutropenia and to help prevent febrile neutropenia after cytotoxic chemotherapy | 9/18/20 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm | Gamifant (emapalumab) | Interferon gamma-blocking antibody | Primary hemophagocytic lymphohistiocytosis | Applicant requested a re-examination of EMA’s July 2020 negative opinion for its MAA, seeking approval for use in children under 18 | 9/18/20 | Hematologic |
| Anokion SA, of Lausanne, Switzerland | ANK-700 | Immune-masked protein therapeutic | Multiple sclerosis | FDA cleared IND for phase I trial, expected to begin by year-end 2020 | 9/24/20 | Immune |
| Cipla Ltd., of Mumbai, India | Dimethyl fumarate DR | Generic of Tecfidera (Biogen Inc.) | Multiple sclerosis | FDA granted final approval of the abbreviated NDA | 9/28/20 | Immune |
| Gilead Sciences Inc., of Foster City, Calif. | Jyseleca (filgotinib) | JAK1 inhibitor | Rheumatoid arthritis | Japanese Ministry of Health, Labour and Welfare approved the drug | 9/25/20 | Immune |
| Gilead Sciences Inc., of Foster City, Calif., Galapagos NV, of Mechelen, Belgium, and Eisai Co. Ltd., of Tokyo | Jyseleca (filgotinib) | Oral JAK1 inhibitor | Rheumatoid arthritis | Approved by Japan’s MHLW for use in patients who have had an inadequate response to conventional therapies, including prevention of structural joint damage | 9/25/20 | Immune |
| Immupharma plc, of London, and Avion Pharmaceuticals LLC, of Atlanta | Lupuzor (forigerimod) | Modulates activation of autoreactive T cells | Lupus | FDA has not responded to the submission of a special protocol assessment more than 45 days after submission due to current workload at the agency | 9/11/20 | Immune |
| Innovent Biologics Inc., of San Francisco | Sulinno (adalimumab) | Biosimilar of Humira | Rheumatoid arthritis, ankylosing spondylitis and psoriasis | National Medical Products Administration of China approved the drug | 9/3/20 | Immune |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Simponi Aria (golimumab) | Anti-TNF-alpha monoclonal antibody | Polyarticular juvenile idiopathic arthritis and active psoriatic arthritis | FDA approved the drug for patients 2 and older | 9/29/20 | Immune |
| Kadmon Holdings Inc., of New York | Belumosudil (KD-025) | Oral inhibitor of Rho-associated coiled-coil kinase 2 | Systemic sclerosis | FDA granted orphan drug designation | 9/8/20 | Immune |
| Kadmon Holdings Inc., of New York | Belumosudil (KD-025) | ROCK2 inhibitor | Chronic graft-vs.-host disease | Submitted NDA to FDA; application reviewed under Real-Time Oncology Review program | 9/30/20 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-107 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | FDA granted orphan designation | 9/2/20 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-207 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | FDA granted orphan drug designation in previously transplanted patients | 9/22/20 | Immune |
| Pfizer Inc., of New York | Xeljanz (tofacitinib) | Janus kinase inhibitor | Active polyarticular course juvenile idiopathic arthritis | FDA approved it for the treatment of children and adolescents 2 and older | 9/29/20 | Immune |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Hyqvia (human normal immunoglobulin 10% and recombinant human hyaluronidase) | Subcutaneous immunoglobulin replacement therapy | Secondary immunodeficiencies | EMA approval label update for use in adults, adolescents and children with secondary immunodeficiencies who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven scientific antibody failure or serum IgG level of <4 g/l | 9/15/20 | Immune |
| VBL Therapeutics Ltd., of Tel Aviv, Israel | VB-601 | Anti-MOSPD2 antibody | Immune-inflammatory indications | Announced the successful completion of a pre-IND meeting with the FDA regarding proposed clinical development, and the company aims to start a first-in-human study in the second half of 2021 | 9/8/20 | Immune |
| Aldeyra Therapeutics Inc., of Lexington, Mass. | ADX-629 | Oral RASP inhibitor | COVID-19 | Received a study may proceed letter from the FDA for a phase II study in hospitalized adult patients | 9/9/20 | Infection |
| Aligos Therapeutics Inc., South San Francisco | ALG-000184-201 | Class II capsid assembly modulator | Hepatitis B virus infection | Received approval of a CTA for a phase Ia/Ib study in healthy volunteers and patients | 9/28/20 | Infection |
| Allovir Inc., of Cambridge, Mass. | ALVR-109 | Allogeneic virus-specific T-cell therapy | COVID-19 infection | FDA cleared IND application for proof-of-concept trial, expected to begin in fourth quarter of 2020 | 9/17/20 | Infection |
| Ampio Pharmaceuticals Inc., of Englewood, Colo. | Ampion | Aryl hydrocarbon receptor agonist; stem cell antigen-1 inhibitor | Respiratory distress due to COVID-19 | FDA cleared an IND for a phase I study of 40 patients | 9/21/20 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Favipiravir | Broad-spectrum antiviral | Mild to moderate COVID-19 | Submitted an IND to the FDA for a phase III study in 826 patients; study expected to launch in Fall of 2020 | 9/11/20 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | AB-201 | Inhibitor of tissue factor | Hospitalized COVID-19 | Submitted IND to FDA for a phase IIb/III study | 9/21/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | COVID-19 | Filed applications to Turkish Ministry of Health and local Ethics Committee to recruit patients in phase II trial | 9/1/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | COVID-19 infection | Istanbul University Ethics Committee cleared initiation of phase II trial to treat acute lung and kidney injury caused by inflammation in people with severe infection; regulatory review by Turkish Ministry of Health expected to finish by mid-September 2020 | 9/10/20 | Infection |
| Aridis Pharmaceuticals Inc., of San Jose, Calif. | AR-501 | Inhaled gallium citrate | Chronic lung infections associated with cystic fibrosis | Reached an agreement with the FDA to remove the single ascending-dose portion of the upcoming phase IIa study; plans to run a phase IIa/IIb study with multiple ascending-dose regimen followed by a sample size expansion at the selected dose for the phase IIb | 9/8/20 | Infection |
| AVM Biotechnology LLC, of Seattle | AVM-0703 | Formulation of high concentration of dexamethasone | Acute respiratory distress syndrome mediated by COVID-19 or influenza | FDA cleared the IND | 9/16/20 | Infection |
| Baylx Inc., of Irvine, Calif. | BX-U001 | Non-frozen human umbilical cord tissue mesenchymal stem cells | COVID-19 | FDA cleared the IND for a phase I/IIa trial in about 30 patients with moderate to severe acute respiratory distress syndrome | 9/30/20 | Infection |
| Cardiol Therapeutics Inc., of Oakville, Ontario | Cardiorx | Extra-strength cannabidiol formulation | COVID-19 | FDA cleared the IND for a phase II/III study in hospitalized patients with a prior history of, or risk factors for, cardiovascular disease | 9/25/20 | Infection |
| Celltrion Group, of Incheon, South Korea | CT-P59 | COVID-19 Spike glycoprotein inhibitor | COVID-19 infection | Korea's MFDS cleared IND application for pivotal phase II/III trial in people with mild to moderate symptoms of infection; top-line data from phase II portion expected by year-end 2020 | 9/17/20 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 | U.K. MHRA requested meeting Sept. 9, 2020, to discuss company’s request for fast track approval to treat patients with mild to moderate symptoms; on the suggestion of the MHRA, Cytodyn will submit its current phase III CD12 study for severe to critical COVID-19 patients in the U.K. to the Urgent Public Health Research scheme to receive possible financial support from the trial sites and government | 9/2/20 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab (PRO-140) | CCR5 antagonist | HIV | FDA advised the company it has scheduled a type A meeting Sept. 8, 2020, following receipt of written responses concerning the BLA seeking approval for leronlimab as a combination therapy for highly treatment-experienced HIV patients; agency clarified items it need primarily related to dosage levels | 9/2/20 | Infection |
| Dompé Farmaceutici SpA, of Milan, Italy | Reparixin | Inhibits interleukin-8 | COVID-19 | Brazil’s regulatory agency, Anvisa, cleared a phase II/III trial in severe COVID-19 patients | 9/2/20 | Infection |
| Equillium Inc., of La Jolla, Calif. | Itolizumab (EQ-001) | Monoclonal antibody targeting CD6-ALCAM pathway | COVID-19 | Completed pre-IND meeting with FDA under Coronavirus Treatment Acceleration Program; with FDA feedback, company aims to finalize protocol and submit IND for phase III study during fourth quarter of 2020 | 9/15/20 | Infection |
| FSD Pharma Inc., of Toronto | FSD-201 (ultramicronized palmitoylethanolamide) | Cannabinoid CB2 modulator | COVID-19 infection | FDA authorized phase II study, expected to begin in October 2020, in 352 people hospitalized with documented infection | 9/28/20 | Infection |
| Inmune Bio Inc., of La Jolla, Calif. | Quellor | Selective soluble TNF inhibitor | Immune-mediated complications in COVID-19 | FDA accepted the IND for a phase II trial | 9/1/20 | Infection |
| Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | INO-4800 | DNA-based vaccine | COVID-19 prophylaxis | FDA issued a partial clinical hold for the planned phase II/III study | 9/28/20 | Infection |
| Iterum Therapeutics plc, of Dublin | Sulopenem etzadroxil/probenecid bilayer tablet | Antibiotic | Uncomplicated urinary tract infections with a quinolone-resistant pathogen | Based on discussions at a pre-NDA meeting, company plans to file an NDA in the fourth quarter of 2020 | 9/30/20 | Infection |
| Kiadis Pharma NV, of Amsterdam | K-NK-ID101 | Kiadis-NK cells | Post-exposure pre-emptive therapy for COVID-19 | FDA approved the IND | 9/14/20 | Infection |
| Mateon Therapeutics Inc., of Agoura Hills, Calif. | Artemisinin | Inhibits TGF-beta | COVID-19 | Protocol for study cleared for patient enrollment in India | 9/14/20 | Infection |
| Mateon Therapeutics Inc., of Agoura Hills, Calif. | Artemisinin | Purified from Artemisia annua; inhibits TGF-? activity | COVID-19 | Global study based on ARTI-19 protocol cleared for patient enrollment in India; study being conducted in India as part of co-development agreement with Windlas Biotech Pvt Ltd. | 9/15/20 | Infection |
| Mesoblast Ltd., of Melbourne, Australia | Remestemcel-L | Mesenchymal stem cell-based therapy | COVID-19 | Received ethics approval to include Australian hospitals in phase III study in ventilator-dependent COVID-19 patients with acute respiratory distress syndrome | 9/2/20 | Infection |
| Organicell Regenerative Medicine Inc., of Miami | Zofin | Perinatal-derived microRNAs | COVID-19 | FDA granted expanded access | 9/29/20 | Infection |
| Otsuka Novel Products GmbH, of Munich | Deltyba (delamanid) | Inhibits the synthesis of mycolic acid | Pulmonary multidrug-resistant tuberculosis | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the extension of the indication to include adolescents and children with a body weight of at least 30 kg | 9/21/20 | Infection |
| Peptilogics Inc., of Pittsburgh | PLG-0206 | Broad-spectrum antibiotic peptide | Prosthetic joint infections | FDA granted orphan status | 9/9/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | Opaganib (Yeliva, ABC-294640) | Sphingosine kinase-2 inhibitor | Severe COVID-19 pneumonia | The Brazilian Health Regulatory Agency (ANVISA) approved its ongoing global phase II/III study in hospitalized patients | 9/22/20 | Infection |
| Reven Holdings Inc., of Golden, Colo. | Rejuveinix | Anti-inflammatory/antioxidant | COVID-19 | Received positive feedback from the FDA on its pre-IND submission | 9/14/20 | Infection |
| Sanofi SA, of Paris | Menquadfi | Meningococcal (groups A, C, Y, W) conjugate vaccine | Prevention of invasive meningococcal disease | EMA’s Committee for Medicinal Products for Human Use recommended granting marketing authorization for active immunization of individuals 12 months and older against invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, W and Y | 9/18/20 | Infection |
| Seres Therapeutics Inc., of Cambridge, Mass. | SER-109 | Firmicute bacteria in spore form | Recurrent C. difficile infection | FDA reaffirmed prior guidance of efficacy requirements to support a BLA and guidance that at least 300 patients will be required for the safety database | 9/11/20 | Infection |
| Shionogi & Co. Ltd., of Osaka, Japan | Fetroja (cefiderocol) | Cephalosporin antibiotic | Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia | FDA approved the supplemental NDA | 9/28/20 | Infection |
| Sinovac Biotech Ltd., of Beijing | Coronavac | Adjuvant inactivated SARS-CoV-2 vaccine | COVID-19 infection | China NMPA authorized phase I/II trial in children and adolescents ages 3 to 17 | 9/23/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | STI-1499 (COVI-GUARD) | COVID-19 Spike glycoprotein inhibitor | COVID-19 infection | FDA cleared IND for phase I trial in people hospitalized with infection | 9/16/20 | Infection |
| Stemedica Cell Technologies Inc., of San Diego | itMSC | Intravenous allogeneic mesenchymal stem cells | Moderate to severe lung injury due to COVID-19 | FDA approved the IND for a phase II study | 9/28/20 | Infection |
| Vaxart Inc., of South San Francisco | COVID-19 vaccine | Oral vaccine | COVID-19 prophylaxis | FDA cleared the IND for a phase I study | 9/14/20 | Infection |
| Cura Foundation, of New York | hCT-MSC | Human cord tissue mesenchymal stromal cells | Multisystem inflammatory syndrome in children | FDA approved the IND; study to begin in September | 9/16/20 | Inflammatory |
| Khondrion BV, of Nijmegen, the Netherlands | Sonlicromanol (KH-176) | Redox modulator with anti-inflammatory properties | MELAS syndrome | FDA issued rare pediatric disease designation | 9/28/20 | Musculoskeletal |
| Mereo Biopharma Group plc, of London | Setrusumab | Sclerostin inhibitor | Osteogenesis imperfecta | FDA granted rare pediatric disease designation | 9/24/20 | Musculoskeletal |
| Nippon Shinyaku Co. Ltd., of Kyoto, Japan | Viltepso (viltolarsen) | Morpholino antisense oligonucleotide | Duchenne muscular dystrophy | FDA issued a priority review voucher for the approval of a rare pediatric disease product application | 9/3/20 | Musculoskeletal |
| Reneo Pharmaceuticals Inc., of San Diego | REN-001 | Selective PPAR delta agonist | LCHAD deficiency and MELAS syndrome | EMA’s Committee for Orphan Medicinal Products granted orphan designation | 9/9/20 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | SRP-9001 | DMD gene stimulator | Duchenne muscular dystrophy | Completed type C meeting with Office of Tissues and Advanced Therapies at FDA's CBER seeking concurrence on initiation of next trial using commercial process material, including additional potency assay for material release | 9/9/20 | Musculoskeletal |
| Abbvie Inc., of North Chicago | Elezanumab (ABT-555) | Monoclonal antibody targeting repulsive guidance molecule A | Spinal cord injury | FDA granted orphan drug designation | 9/28/20 | Neurology/Psychiatric |
| Aeon Biopharma Inc., of Newport Beach, Calif. | ABP-450 (prabotulinumtoxinA) | Botulinum toxin complex | Cervical dystonia | FDA accepted IND to begin phase II testing | 9/2/20 | Neurology/Psychiatric |
| American Cryostem Corp., of Eatontown, N.J. | Atcell | Autologous adipose-derived mesenchymal stem cell therapy | Post-concussion syndrome | FDA cleared IND for phase I trial of retired athletes and military service members with confirmed diagnosis from 3 or more concussion or mild traumatic brain injuries; study expected to begin in fourth quarter of 2020 | 9/23/20 | Neurology/Psychiatric |
| Amicus Therapeutics Inc., Cranbury, N.J. | AT-GTX-501 | Ceroid lipofuscinosis protein gene stimulator | CLN6 Batten disease | EMA granted PRIME designation | 9/24/20 | Neurology/Psychiatric |
| Aquestive Therapeutics Inc., of Warren, N.J. | Libervant (diazepam) | Bucal formulation of the benzodiazepine, diazepam | Seizure clusters | FDA issued a CRL for the NDA due to certain weight groups having a lower drug exposure level than desired and issues with protocol deviations in blood draws in 1 of the studies | 9/25/20 | Neurology/Psychiatric |
| Athena Bioscience LLC, of Athens, Ga. | Qdolo (tramadol) | Opioid agonist | Severe pain | FDA approved the drug | 9/8/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Fycompa (perampanel) | Anti-epileptic drug | Seizures | EMA’s Committee for Medicinal Products for Human Use recommended positive opinion to expand use to include adjunctive treatment for partial-onset seizures with or without secondarily generalized seizures in patients 4 and older; and primary generalized tonic-clonic seizures in patients 7 and older with idiopathic generalized epilepsy | 9/18/20 | Neurology/Psychiatric |
| GW Pharmaceuticals plc, of London | Epidyolex (cannabidiol) | Dual cannabinoid CB1/CB2 modulator | Lennox-Gastaut syndrome; Dravet syndrome | Australian TGA approved to treat seizures associated with indications in people 2 and older | 9/23/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | HTX-011 | Combination of local anesthetic bupivacaine and nonsteroidal anti-inflammatory drug meloxicam | Postoperative pain | Following a type A meeting with the FDA where the agency agreed that the proposed specification change was acceptable, company plans to resubmit its NDA in the fourth quarter of 2020 | 9/8/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | Zynrelef (HTX-011) | Anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory drug meloxicam | Somatic postoperative pain from small- to medium-sized surgical wounds | European Commission granted marketing authorization to the drug | 9/28/20 | Neurology/Psychiatric |
| Hyloris Pharmaceuticals SA, of Liège, Belgium, and Aft Pharmaceuticals Ltd., of Auckland, New Zealand | Maxigesic IV | Non-opioid analgesic | Pain | Drug was approved in Belgium, Sweden, Hungary, Romania, Croatia, Lithuania, Slovakia and Malta | 9/7/20 | Neurology/Psychiatric |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | ION-373 | Antisense therapeutic designed to stop mutated gene from producing excess GFAP | Alexander disease | FDA granted orphan designation | 9/30/20 | Neurology/Psychiatric |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Mavrilimumab | Monoclonal antibody targeting GM-CSFR? | Giant cell arteritis | FDA granted orphan designation | 9/15/20 | Neurology/Psychiatric |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Synthetic analogue of molecule derived from IGF-1 | Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome | Submitted orphan designation applications to the EMA for each indication | 9/29/20 | Neurology/Psychiatric |
| Novartis AG, of Basel, Switzerland | AVXS-101 intrathecal | SMN1 gene stimulator |
Spinal muscular atrophy | FDA recommended pivotal confirmatory study to supplement existing Strong data to treat older individuals with SMA | 9/23/20 | Neurology/Psychiatric |
| Pacira Pharmaceuticals Inc., of Parsippany, N.J. | Exparel (bupivacaine) | Bupivacaine liposome injectable suspension | Postoperative pain | EMA’s Committee for Medicinal Products for Human Use adopted positive opinion | 9/18/20 | Neurology/Psychiatric |
| Painreform Ltd., of Herzliya, Israel | PRF-110 (ropivacaine gel, extended-release) | Sodium channel inhibitor | Postoperative pain | FDA cleared IND for pivotal phase III trials | 9/29/20 | Neurology/Psychiatric |
| Roche Holding AG, of Basel, Switzerland | Risdiplam | SMN2 gene modulator | Spinal muscular atrophy | U.K.’s MHRA issued positive opinion on early access to medicines scientific opinion for patients, 2 months and older with type 1 or type 2 SMA not suitable for authorized treatments | 9/17/20 | Neurology/Psychiatric |
| SK Biopharmaceuticals Co. Ltd., of Pangyo, Korea | Cenobamate | Inhibits voltage-gated sodium currents | Partial-onset seizures in adults | Korean Ministry of Food and Drug Safety accepted the IND for a phase III study | 9/11/20 | Neurology/Psychiatric |
| Supernus Pharmaceuticals Inc., of Rockville, Md. | Apomorphine infusion pump (SPN-830) | Dopamine agonist | Parkinson’s disease | Submitted NDA to the FDA | 9/14/20 | Neurology/Psychiatric |
| Zosano Pharma Corp., of Fremont, Calif. | Qtrypta (zolmitriptan transdermal microneedle system) | 5-HT 1b receptor agonist; 5-HT 1d receptor agonist | Acute migraine | Received discipline review letter (DRL) from FDA in connection with 505(b)(2) NDA, describing 2 concerns with respect to clinical pharmacology section, specifically high plasma concentrations of zolmitriptan observed in 5 study subjects from 2 pharmacokinetic studies and differences in zolmitriptan exposures observed between subjects receiving different lots in clinical trials; approval by the PDUFA date of Oct. 20, 2020, is not expected given the DRL | 9/30/20 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | CBD transdermal gel | Fragile X syndrome | FDA said it will meet with company via teleconference in fourth quarter of 2020 to discuss pivotal Connect-FX data and regulatory path forward | 9/15/20 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Rhopressa (netarsudil ophthalmic solution) | Rho kinase inhibitor | Open-angle glaucoma or ocular hypertension | FDA approved sterile fill production facility in Athlone, Ireland, for production of Rhopressa for commercial distribution in U.S. | 9/15/20 | Ocular |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | AR-15512 | TRPM8 agonist eye drop | Dry eye disease | FDA accepted its IND application, and the company expects to initiate a phase IIb study in the fourth quarter of 2020 | 9/29/20 | Ocular |
| Emmetrope Ophthalmics LLC, of Menlo Park, Calif. | EO-2002 | Non-surgical, magnetic cell-based therapy | Corneal edema | FDA accepted the IND application | 9/22/20 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq (lenadogene nolparvovec) | Recombinant adeno-associated viral vector, serotype 2, containing cDNA encoding human wild-type mitochondrial NADH dehydrogenase 4 protein | Leber hereditary optic neuropathy | Submitted MAA to the EMA seeking approval to treat patients with vision loss due to LHON caused by mutation in the ND4 mitochondrial gene | 9/15/20 | Ocular |
| Inceptua AB, of Luxembourg | Jetrea (ocriplasmin) | Proteolytic enzyme | Symptomatic vitreomacular adhesion or vitreomacular traction | European Commission approved the transfer of the marketing authorization form Oxurion NV to Inceptua | 9/28/20 | Ocular |
| Neurophth Therapeutics Inc., of Newark, Del. | NR-082 (rAAV2-ND4, NFS-01) | AAV ophthalmic gene therapy | Leber’s hereditary optic neuropathy | FDA granted orphan designation for LHON associated with ND4 mutation | 9/24/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France, and Ocumension Therapeutics Ltd., of Shanghai | Zerviate | Cetirizine ophthalmic solution | Ocular itching associated with allergic conjunctivitis | Received IND approval from China's Center for Drug Evaluation | 9/22/20 | Ocular |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab) | Antibody fragment targeting VEGF-A | Age-related macular degeneration | EMA's Committee for Medicinal Products for Human Use approved an update to the summary of product characteristics to include additional information regarding retinal vasculitis and retinal vascular occlusion | 9/14/20 | Ocular |
| Novartis AG, of Basel, Switzerland | Beovu (brolucizumab) | Humanized single-chain antibody fragment | Wet age-related macular degeneration | EMA’s Committee for Medicinal Products for Human Use approved update to summary of product characteristics to include additional information regarding retinal vasculitis and retinal vascular occlusion | 9/15/20 | Ocular |
| Ocular Therapeutix Inc., of Bedford, Mass. | Resure Sealant | Hydrogel ophthalmic wound medical device | Clear corneal incisions, in order to prevent postoperative fluid egress following cataract surgery | Received a letter from the FDA closing out the warning letter it received on October 18, 2018; the violation cited in warning letter has been addressed | 9/8/20 | Ocular |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Maralixibat | Apical sodium dependent bile acid transporter inhibitor | Cholestatic pruritus in patients with Alagille syndrome | Submitted first portion of its rolling NDA to the FDA | 9/1/20 | Other/Miscellaneous |
| Prometic Biotherapeutics Inc., subsidiary of Liminal Biosciences Inc., of Cambridge, U.K. | Ryplazim | Plasminogen | Congenital plasminogen deficiency | Resubmitted a BLA to the FDA | 9/8/20 | Other/Miscellaneous |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabidiol gel | 22q11.2 deletion syndrome | FDA granted orphan designation | 9/17/20 | Other/Miscellaneous |
| Athersys Inc., of Cleveland, Ohio | Multistem | Multipotent adult progenitor cell therapy | Acute respiratory distress syndrome | FDA granted regenerative medicine advanced therapy designation | 9/23/20 | Respiratory |
| Glaxosmithkline plc, of London | Trelegy Ellipta (fluticasone furoate + umeclidinium + vilanterol) | Beta 2 adrenoceptor agonist; muscarinic receptor antagonist | Asthma | FDA approved sNDA to treat indication in adults 18 and older | 9/9/20 | Respiratory |
| Marinomed Biotech AG, of Vienna | Carragelose | Decongestant nasal spray | Viral infections of the respiratory tract | Applied for approval in a first group of EU countries | 9/8/20 | Respiratory |
| Neurorx Inc., of Radnor, Pa. | RLF-100 (aviptadil) | Vasoactive intestinal polypeptide | Prevention of respiratory failure in COVID-19 infection | Emergency use authorization submitted to FDA using same protocol as expanded access authorized in July 2020 | 9/23/20 | Respiratory |
| Spirovant Sciences Inc., of Philadelphia | SPIRO-2101 | Inhaled AAV gene therapy | Cystic fibrosis | FDA granted orphan and rare pediatric disease designations | 9/25/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Trikafta (ivacaftor/tezacaftor/elexacaftor) | CFTR modulator/stimulator | Cystic fibrosis | FDA issued a rare pediatric disease product priority review voucher | 9/14/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Trikafta (ivacaftor/tezacaftor/elexacaftor) | CFTR modulator/stimulator | Cystic fibrosis | EMA validated the type II variation marketing authorization for patients 12 and older with at least 1 copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene | 9/14/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) | CFTR modulator/stimulator | Cystic fibrosis | FDA accepted supplemental NDAs for the drugs to cover patients with additional rare CFTR mutations; applications would also allow certain patients eligible for Kalydeco to be eligible for Symdeko or Trikafta and certain patients eligible for Symdeko to be eligible for Trikafta; PDUFA action date is Dec. 30, 2020 | 9/1/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | CFTR stimulator | Cystic fibrosis | EMA’s Committee for Medicinal Products for Human use adopted a positive opinion for use in children as young as 4 months who have R117H CFTR mutation or 1 of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R; also recommended for approval in combination with tezacaftor in children as young as 6 years who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have 1 of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A?G, S945L, S977F, R1070W, D1152H, 2789+5G?A, 3272 26A?G, and 3849+10kbC?T | 9/18/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | CFTR stimulator | Cystic fibrosis | Approved by FDA for use in children, ages 4 months to less than 6 months, who have at least 1 mutation in the CFTR gene that is responsive to Kalydeco based on clinical and/or in vitro assay data | 9/25/20 | Respiratory |
| Windtree Therapeutics Inc., of Warrington, Pa. | Lyo lucinactant | KL4 surfactant | COVID-19-associated lung injury and acute respiratory distress syndrome | FDA accepted the IND application for its phase II trial | 9/29/20 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 | Genetically targeted therapy | Alcohol use disorder | Submitted a formal request to the FDA in support of company’s position that AD-04 should be considered eligible for an FDA expedited review program | 9/25/20 | Toxicity and Intoxication |
| Abbvie Inc., of North Chicago, and Calibr, the translational arm of The Scripps Research Institute | CLBR-001 + SWI-019 | CAR T-cell therapy | B-cell malignancies | FDA granted fast track designation | 10/1/20 | Cancer |
| Adicet Bio Inc., of Menlo Park, Calif. | ADI-001 | Allogeneic gamma delta T-cell therapy | Non-Hodgkin’s lymphoma | FDA cleared IND application for phase I trial | 10/22/20 | Cancer |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Tibsovo (ivosidenib) | Isocitrate dehydrogenase-1 inhibitor | Relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-1 mutation | Withdrew MAA after EMA's Committee for Medicinal Products for Human Use indicated data from the phase I study aren't sufficient to support approval; continuing two phase III studies | 10/16/20 | Cancer |
| Allarity Therapeutics A/S, of Horsholm, Denmark | Dovitinib | A pan-tyrosine kinase inhibitor | Renal cell carcinoma | The third-party contract manufacturer of the registration batch of the drug is experiencing delays, partly due to COVID-19; Allarity is now expecting to file the NDA in 2021 | 10/23/20 | Cancer |
| Apexigen Inc., of San Carlos, Calif. | APX-005M | Monoclonal antibody targeting CD40 | Esophageal and gastroesophageal junction cancer and pancreatic cancer | FDA granted orphan drug designations | 10/15/20 | Cancer |
| Aprea Therapeutics Inc., of Boston | APR-548 | Apoptosis p53 stimulating protein stimulator | Myelodysplastic syndrome | FDA accepted IND application for phase I trial | 10/7/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-115 | MDM2-p53 inhibitor | Acute myeloid leukemia | FDA granted orphan drug designation | 10/8/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-1252 | Bcl-2/Bcl-xL inhibitor | Small-cell lung cancer | FDA granted orphan drug designation | 10/8/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | Olverembatinib (HQP-1351) | Third-generation BCR-ABL inhibitor | T315I-mutant chronic phase chronic myeloid leukemia and accelerated phase CML | China’s NMPA said Center for Drug Evaluation granted priority review to NDA submitted by subsidiary Guangzhou Healthquest Pharma Co. Ltd | 10/21/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-115 | MDM2-p53 inhibitor | Advanced liposarcoma or other advanced solid tumors | Received approval from China’s Center for Drug Evaluation of the NMPA for a phase Ib/II study in combination with PD-1/PD-L1 inhibitors | 10/27/20 | Cancer |
| Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1501 | Methionine aminopeptidase-1 inhibitor | Non-muscle invasive bladder cancer | Australian regulatory authorities cleared phase I safety, tolerability and pharmacokinetics trial | 10/28/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Tagrisso (osimertinib) | EGFR antagonist | Early stage EGFR-mutated lung cancer | FDA accepted its sNDA and granted priority review | 10/20/20 | Cancer |
| Bayer AG, of Leverkusen, Germany | Nubeqa (darolutamide) | An oral, nonsteroidal androgen receptor inhibitor | Hormone-relapsed prostate cancer | U.K.’s National Institute for Health and Care Excellence recommended Nubeqa with androgen deprivation therapy as an option for treating hormone-relapsed prostate cancer in adults at high-risk of developing metastatic disease | 10/23/20 | Cancer |
| Bayer AG, of Leverkusen, Germany | Foundationone CDx | Companion diagnostic | Cancer | Approved by FDA to help identify NTRK gene fusion-positive patients for whom treatment with Vitrakvi (larotrectinib) may be appropriate | 10/23/20 | Cancer |
| Bioinvent International AB, of Lund, Sweden | BI-1808 | Type II TNF receptor modulator | Solid tumors and cutaneous T-cell lymphoma | Received regulatory authority approval of CTA in Denmark for a phase I/IIa study testing monotherapy and combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 10/26/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | Anti-PD-1 antibody and anti-CLTA4 antibody | Unresectable malignant pleural mesothelioma | Approved by the FDA for first-line treatment of adults | 10/5/20 | Cancer |
| Bristol Myers Squibb Co., of New York, and Exelixis Inc. of Alameda, Calif. | Opdivo (nivolumab) and Cabometyx (cabozantinib) | Anti-PD-1 monoclonal antibody and tyrosine kinase inhibitor | Advanced renal cell carcinoma | FDA accepted the supplemental BLA and NDA for use of the 2 drugs in combination and granted a priority review; PDUFA action date of Feb. 20, 2021 | 10/19/20 | Cancer |
| Bristol-Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | Anti-PD-1 antibody | Melanoma | EMA's CHMP issued positive opinion for 2- and 4-week dosing in adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection | 10/16/20 | Cancer |
| Carsgen Therapeutics Co. Ltd., of Shanghai | CT-041 | Anti-claudin18.2 autologous CAR T-cell product | Gastric and gastroesophageal junction adenocarcinoma | FDA granted orphan designation | 10/5/20 | Cancer |
| Curtana Pharmaceuticals Inc., of Austin, Texas | CT-179 | Olig2 inhibitor | Medulloblastoma | FDA granted rare pediatric disease designation | 10/6/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. | Enhertu (fam-trastuzumab deruxtecan-nxki) | Antibody-drug conjugate targeting HER2 | HER2-positive metastatic gastric or gastroesophageal junction adenocarcinoma | FDA accepted the supplemental BLA for a priority review; PDUFA target action date is in the first quarter of 2021 | 10/28/20 | Cancer |
| Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) and Opdivo (nivolumab) | Tyrosine kinase inhibitor and anti-PD-1 monoclonal antibody | Unresectable, advanced or metastatic renal cell carcinoma | Announced that Takeda Pharmaceutical Co. Ltd. and Ono Pharmaceutical Co. Ltd. have submitted a supplemental application to Japan's MHLW for manufacturing and marketing approval | 10/27/20 | Cancer |
| Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan | FP-001 (Camcevi, leuprorelin mesylate) | GNRH receptor agonist | Metastatic prostate cancer | FDA accepted 505(b)(2) NDA for candidate, ready-to-use 6-month depot formulation, setting PDUFA date of 5/27/21 | 10/8/20 | Cancer |
| Genentech Inc., of South San Francisco, a unit of Basel, Switzerland-based Roche Holding | Venclexta (venetoclax) | B-cell lymphoma-2 inhibitor | Newly diagnosed acute myeloid leukemia | FDA granted full approval for use of the drug plus azacitidine, or decitabine or low-dose cytarabine in adults 75 or older or adults with co-morbidities that preclude use of intensive induction chemotherapy | 10/16/20 | Cancer |
| Genprex Inc., of Austin, Texas | GPX-001 | TUSC2 gene stimulator | Non-small-cell lung cancer | FDA conditionally accepted request for proprietary name Reqorsa | 10/29/20 | Cancer |
| Hillstream Biopharma Inc., of Bridgewater, N.J. | HSB-1216 (Salinomycin Quatramer) | Stem cell inhibitor | Small-cell lung cancer | Completed pre-IND review citing written response from FDA on proposal for phase I study in in adults with relapsed tumors or who have metastatic disease with progression on or after platinum-based chemotherapy | 10/21/20 | Cancer |
| Huya Bioscience International LLC, of San Diego | HBI-8000 | Targets class I histone deacetylase | Relapsed or refractory adult T-cell leukemia-lymphoma | Submitted a regulatory application to the Japanese Pharmaceuticals and Medical Devices Agency | 10/6/20 | Cancer |
| Immunitybio Inc., of Culver City, Calif. | hAd5-COVID-19 | Vaccine that targets the nucleocapsid and spike proteins | COVID-19 prophylaxis | FDA authorized the IND for a 35-patient phase I study | 10/15/20 | Cancer |
| Immunogen Inc., of Waltham, Mass. | IMGN-632 | CD123-targeted antibody-drug conjugate | Relapsed or refractory blastic plasmacytoid dendritic cell neoplasm | FDA granted breakthrough therapy designation | 10/5/20 | Cancer |
| Immunomedics Inc., of Morris Plains, N.J. | Trodelvy (sacituzumab govitecan-hziy) | Antibody-drug conjugate targeting Trop-2 | Glioblastoma | FDA granted orphan drug designation | 10/12/20 | Cancer |
| Innovent Biologics Inc., of Suzhou, China, and Eli Lilly & Co., of Indianapolis | Halpryza (rituximab injection) | Recombinant human/murine chimeric monoclonal antibody | Diffuse large B cell lymphoma; follicular lymphoma; chronic lymphocytic leukemia | NMPA approved to treat indications in China | 10/9/20 | Cancer |
| Iovance Biotherapeutics Inc., of San Carlos, Calif. | Lifileucel | Tumor-infiltrating lymphocyte therapy | Metastatic melanoma | Company and the FDA reached agreement on the duration of follow-up for its pivotal cohort 4 to support the planned BLA submission; as part of a recent type B meeting, the company and FDA have not been able to agree on the required potency assays to fully define its TIL therapy, which is required as part of a BLA submission, and the company anticipates a BLA submission to occur in 2021 | 10/6/20 | Cancer |
| Kite Pharma Inc., of Santa Monica, Calif. (Gilead Sciences Inc.) | KTE-X19 | B-lymphocyte antigen CD19 modulator | Mantle cell lymphoma | EMA's CHMP issued positive opinion for treatment for 3L treatment of adults with r/r mantle cell lymphoma | 10/16/20 | Cancer |
| Medicenna Therapeutics Corp., of Toronto | MDN-A55 | IL-4 receptor modulator | Glioblastoma | Said FDA advised it to proceed with open-label hybrid control design for phase III trial in recurrent GBM patients with no mutation in 1DH1/1DH2 genes; FDA also expressed willingness to consider interim analysis of trial if certain criteria met | 10/16/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Relapsed or refractory classical Hodgkin lymphoma | FDA approved the expanded label for adults after front-line therapy and for children with refractory disease or disease that relapsed after 2 or more lines of therapy | 10/15/20 | Cancer |
| Mereo Biopharma Group plc, of London | Etigilimab | Monoclonal antibody targeting TIGIT (T-cell immunoreceptor with Ig and ITIM domains) | Various tumor types | FDA cleared the IND for a phase Ib/II study; plans to start the 100-patient study testing etigilimab plus an anti-PD-1 therapy in the fourth quarter | 10/19/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Designed to destroy tumors when activated by radiotherapy | Lung cancer | Safe to proceed notification received by FDA for phase I trial | 10/13/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Designed to destroy tumors when activated by radiotherapy | Esophageal cancer | Safe to proceed notification received by FDA for phase I trial | 10/13/20 | Cancer |
| Nuvation Bio Inc., of New York | NUV-422 | CDK2/4/6 inhibitor | High-grade gliomas | FDA accepted its IND application, and the company plans to initiate a phase I/II study by the first quarter of 2021 | 10/13/20 | Cancer |
| Oncopeptides AB, of Stockholm | Melflufen (melphalan flufenamide) | Peptide-drug conjugate targeting aminopeptidases | Relapsed refractory multiple myeloma | Company informed the EMA that it plans to submit an application for a conditional marketing authorization based on the phase II Horizon study | 10/12/20 | Cancer |
| Oncopeptides AB, of Stockholm | OPD-5 | Peptide-drug conjugate targeting aminopeptidases | Relapsed refractory multiple myeloma | Submitted an IND to the FDA for a phase I study testing the safety and tolerability of OPD-5 as a myeloablative regimen followed by autologous stem cell transplantation | 10/19/20 | Cancer |
| Oncternal Therapeutics Inc., of San Diego | TK-216 | ETS transcription factor inhibitor | Ewing sarcoma | FDA granted rare pediatric disease designation | 10/6/20 | Cancer |
| OS Therapies Inc., of Cambridge, Md. | OST-HER2 | Attenuated Listeria monocytogenes expressing a tLLO-chimeric HER2 fusion protein | Recurrent osteosarcoma | FDA cleared the IND for a phase II study; plans to start the study in early 2021 | 10/15/20 | Cancer |
| Oxular Ltd., of Oxford, U.K. | OXU-003 | Undisclosed | Retinoblastoma | FDA granted rare pediatric disease designation and orphan drug designation | 10/12/20 | Cancer |
| Pepromene Bio Inc., of Irvine, Calif. | PMB-101 | BAFF-R-targeting CAR T-cell therapy | B-cell acute lymphoblastic leukemia | IND submitted to FDA for phase I trial at City of Hope in people with relapsed/refractory disease who are ineligible for or failed CD19-targeted immunotherapy | 10/28/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cypcaps | Genetically engineered human cells encapsulated using the Cell-in-a-Box technology | Locally advanced inoperable pancreatic cancer | FDA placed the IND application on hold; the initial notification didn't give a reason for the decision | 10/2/20 | Cancer |
| PMV Pharmaceuticals Inc., of Cranbury, N.J. | PC-14586 | Structural corrector of the p53 Y220C mutant protein | Advanced cancer in patients that have tumors with a p53 Y220C mutation | FDA granted fast track designation | 10/13/20 | Cancer |
| Prestige Biopharma Ltd., of Singapore | PBP-1510 | Monoclonal antibody targeting pancreatic adenocarcinoma up-regulated factor | Pancreatic cancer | Received positive EMA opinion on orphan designation | 10/13/20 | Cancer |
| Prestige Biopharma Ltd., of Singapore | PBP-1510 | Monoclonal antibody targeting pancreatic adenocarcinoma up-regulated factor | Pancreatic cancer | EMA Orphan Drug Commission granted a positive opinion for an orphan drug designation | 10/19/20 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | CPI-613 (devimistat) | 2 oxoglutarate dehydrogenase inhibitor; pyruvate dehydrogenase inhibitor/kinase stimulator | Soft tissue sarcoma | FDA granted orphan drug designation | 10/29/20 | Cancer |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Libtayo (cemiplimab-rwlc) | PD-1 inhibitor | Non-small-cell lung cancer | FDA accepted sBLA to treat first-line locally advanced or metastatic disease with ?50% PD-L1 expression and granted priority review, setting Feb. 28, 2021, PDUFA date | 10/29/20 | Cancer |
| Sesen Bio Inc., of Cambridge, Mass. | Vicineum | Locally administered fusion protein | BCG-unresponsive non-muscle invasive bladder cancer | Completed pre-submission meeting with EMA, addressing product-specific, legal, regulatory and scientific topics; agency also provided guidance on various administrative topics to clarify regulatory path forward; company intends to complete all necessary pre-submission activities for MAA by end of 2020 | 10/26/20 | Cancer |
| Teneobio Inc., of Newark, Calif. | TNB-486 | Bispecific T-cell engaging antibody | B-cell non-Hodgkin lymphoma | FDA cleared the IND for phase I studies | 10/1/20 | Cancer |
| TG Therapeutics Inc., of New York | Ublituximab and umbralisib | Glycoengineered anti-CD20 monoclonal antibody and dual inhibitor of PI3K-delta and CK1-epsilon | Chronic lymphocytic leukemia | FDA granted fast track designation | 10/21/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | TRC-102 (methoxyamine) | Small-molecule inhibitor of DNA base excision repair pathway | Malignant glioma | FDA granted orphan designation for malignant glioma, including glioblastoma | 10/26/20 | Cancer |
| Y-Mabs Therapeutics Inc., of New York | Nivatrotamab (huGD2-BsAb) | Humanized bispecific anti-GD2 antibody | Neuroblastoma | FDA granted orphan drug and rare pediatric disease designations | 10/7/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | 177Lu-omburtamab-DTPA | Antibody radiolabeled with lutetium-177 | Medulloblastoma | FDA cleared the IND for a phase I/II trial expected to start screening patients during the fourth quarter of 2020 | 10/14/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | 177Lu-omburtamab-DTPA | Omburtamab antibody radiolabeled with lutetium-177 | B7-H3+ CNS and leptomeningeal metastasis from tumors | FDA cleared the IND for testing in adults; phase I/II study expected to open for screening during fourth quarter of 2020 | 10/26/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Omburtamab | Monoclonal antibody targeting B7-H3 | Pediatric CNS/leptomeningeal metastasis from neuroblastoma | Received a refusal to file letter from FDA regarding BLA; upon preliminary review, FDA determined that certain parts of the chemistry, manufacturing and control module and the clinical module of the BLA require further detail, and no additional nonclinical data have been requested or are required | 10/6/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Forxiga (dapagliflozin) | SGLT2 inhibitor | Symptomatic chronic heart failure with reduced ejection fraction | EMA's Committee for Medicinal Products for Human Use recommended approval of the drug | 10/19/20 | Cardiovascular |
| Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson | Xarelto (rivaroxaban) | Factor Xa antagonist | Peripheral artery disease | Submitted supplemental NDA to FDA to expand use in patients with PAD | 10/26/20 | Cardiovascular |
| Nevakar Inc., of Bridgewater, N.J. | Ephedrine sulfate | Alpha/beta adrenoceptor agonist | Hypotension | FDA approved injection formulation in ready-to-use 50-mg/10 ml single use vial | 10/16/20 | Cardiovascular |
| Rhythm AI Ltd., of London | STAR Mapping System | Identifies the drivers of disease for cardiac ablation procedure | Persistent atrial fibrillation | Received approval from the U.K. Medicines and Healthcare Products Regulatory Agency to proceed with the Rocstar study | 10/8/20 | Cardiovascular |
| Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Moderate to severe atopic dermatitis | Submitted marketing applications to the FDA and EMA | 10/19/20 | Dermatologic |
| Aegle Therapeutics Corp., of Boston | AGLE-102 | Extracellular vesicle therapy | Dystrophic epidermolysis bullosa | FDA granted fast track status | 10/1/20 | Dermatologic |
| Aegle Therapeutics Corp., of Boston | AGLE-102 | Mesenchymal stem cell-derived extracellular vesicles that deliver proteins, genetic material and regenerative healing factors | Dystrophic epidermolysis bullosa | FDA granted rare pediatric disease designation | 10/6/20 | Dermatologic |
| Akari Therapeutics plc, of New York | Nomacopan | C5 complement inhibitor | Bullous pemphigoid | Completed scientific advice meeting with EMA in advance of starting a phase III pivotal study; agency agreed to company’s proposal for placebo-controlled trial with primary endpoint of disease remission on minimal corticosteroids | 10/1/20 | Dermatologic |
| Apstem Therapeutics Inc., of Fremont, Calif. | AP-Skin-01 | Allogeneic adult pluripotent stem cells | Diabetic ulcers | Completed initial targeted engagement for regulatory advice meeting with FDA's CBER Office of Tissues and Advanced Therapies to discuss preclinical development plan toward IND filing | 10/28/20 | Dermatologic |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Berotralstat | Oral antagonist of plasma kallikrein | Hereditary angioedema | U.K.'s MHRA granted a positive scientific opinion through the early access to medicines scheme | 10/30/20 | Dermatologic |
| Forte Biosciences Inc., of Torrance, Calif. | FB-401 | Topically applied live biotherapeutic consisting of strains of commensal Roseomonas mucosa | Atopic dermatitis | FDA granted fast track designation | 10/26/20 | Dermatologic |
| Leo Pharma A/S, of Ballerup, Denmark | Enstilar (calcipotriene + betamethasone dipropionate) | Vitamin D analogue + corticosteroid | Plaque psoriasis | FDA approved sNDA for long-term treatment in adults | 10/22/20 | Dermatologic |
| Pfizer Inc., of New York | Abrocitinib | Janus kinase 1 inhibitor | Moderate to severe atopic dermatitis | FDA accepted for filing and granted priority review to the NDA, and the EMA has accepted the MAA | 10/27/20 | Dermatologic |
| Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | IL-13 receptor antagonist; IL-4 receptor antagonist | Atopic dermatitis | EMA's CHMP issued positive opinion recommending extension of current EU approval to include children aged 6 to 11 years with severe atopic dermatitis who are candidates for systemic therapy | 10/16/20 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Lumasiran | Hydroxyacid oxidase 1 modulator | Hyperoxaluria | EMA's CHMP issued positive opinion for treatment of primary hyperoxaluria type 1; if approved, will be marketed as Oxlumo | 10/16/20 | Endocrine/Metabolic |
| Ascendis Pharma A/S, of Copenhagen | Transcon PTH | Parathyroid hormone prodrug | Hypoparathyroidism | European Commission granted orphan designation | 10/23/20 | Endocrine/Metabolic |
| Astrazeneca plc, of Cambridge, U.K. | Forxiga (dapagliflozin) | SGLT2 inhibitor | Type 2 diabetes | China’s National Medical Products Administration updated the label to include data from the Declare-Timi study that showed the drug reduced the composite endpoint of hospitalization for heart failure or cardiovascular death compared to placebo | 10/28/20 | Endocrine/Metabolic |
| Avrobio Inc., of Cambridge, Mass. | AVR?RD?01 | GLA gene stimulator | Fabry disease | European Commission granted orphan drug designation | 10/29/20 | Endocrine/Metabolic |
| Avrobio Inc., of Cambridge, Mass. | AVR-RD-01 | Gene therapy expressing alpha-galactosidase A | Fabry disease | EC granted orphan drug designation | 10/29/20 | Endocrine/Metabolic |
| Axovant Gene Therapies Ltd., of New York | AXO-AAV-GM1 | GLB1 gene stimulator | GM1 gangliosidosis | FDA granted rare pediatric disease designation | 10/9/20 | Endocrine/Metabolic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | BMN-307 | Gene therapy expressing phenylalanine hydroxylase | Phenylketonuria | FDA granted fast track designation | 10/2/20 | Endocrine/Metabolic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Palynziq (pegvaliase-pqpz) | Phenylalanine hydroxylase stimulator | Phenylketonuria | FDA approved sBLA to increase maximum allowable dose to 60 mg to treat adults | 10/7/20 | Endocrine/Metabolic |
| Bluebird Bio Inc., of Cambridge, Mass. | Elivaldogene autotemcel (Lenti-D) | Gene therapy expressing ABCD1 | Cerebral adrenoleukodystrophy | EMA accepted the marketing authorization application | 10/2/20 | Endocrine/Metabolic |
| Clinuvel Pharmaceuticals Ltd., of Melbourne | Scenesse (afamelanotide) | A melanocortin-1 receptor agonist | Erythropoietic protoporphyria | Australia’s Therapeutic Goods Administration (TGA) approved Scenesse for the prevention of phototoxicity in adults with erythropoietic protoporphyria | 10/30/20 | Endocrine/Metabolic |
| Cyclo Therapeutics Inc., of Gainesville, Fla. | Trappsol Cyclo | Cholesterol binding agent; Lipid metabolism modulator | Niemann-Pick disease type C | Received notification from FDA that enrollment can proceed in phase III pivotal trial | 10/21/20 | Endocrine/Metabolic |
| Diurnal Group plc, of London, and Effrx Pharmaceuticals SA, of Freienbach, Switzerland | Alkindi (hydrocortisone granules in capsules for opening) | Steroid | Adrenocortical insufficiency | Effrx submitted marketing application to Swissmedic | 10/21/20 | Endocrine/Metabolic |
| Inventiva, of Daix, France | Odiparcil | Orally available small molecule | Mucopolysaccharidoses type VI | FDA granted fast track designation | 10/20/20 | Endocrine/Metabolic |
| Novartis AG, of Basel, Switzerland | Leqvio (inclisiran) | PCSK9 gene inhibitor | Hypercholesterolemia | EMA's CHMP issued positive opinion for treatment of adults with hypercholesterolemia or mixed dyslipidemia | 10/16/20 | Endocrine/Metabolic |
| Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral GLP1-analog | Diabetes mellitus | Submitted phase III protocols to FDA, covering two studies in type 2 diabetes with about 1,125 participants; expecting to start enrollment in Q4 | 10/16/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBGM-01 | AAV-based gene therapy | GM1 gangliosidosis | European Commission granted orphan designation | 10/26/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBKR-03 | AAV-delivered gene therapy expressing GALC | Krabbe disease | FDA granted orphan drug and rare pediatric disease designations | 10/28/20 | Endocrine/Metabolic |
| Prevail Therapeutics Inc., of New York | PR-001 | Gene therapy expressing GBA1 | Neuronopathic Gaucher disease | FDA granted fast track designation | 10/27/20 | Endocrine/Metabolic |
| Saniona AB, of Copenhagen | Tesomet (tesofensine + metoprolol) | Beta 1 adrenoceptor antagonist | Prader-Willi syndrome (PWS); hypothalamic obesity (HO) | In written feedback on pre-IND submissions, FDA recommended supportive phase IIb study in PWS prior to initiating phase III and supported 505(b)(2) pathway for HO with supportive phase IIb study followed by phase III | 10/9/20 | Endocrine/Metabolic |
| Sanofi SA, of Paris | Avalglucosidase alfa | Enzyme replacement | Pompe disease | EMA accepted the marketing authorization application | 10/2/20 | Endocrine/Metabolic |
| Selecta Biosciences Inc., of Watertown, Mass., and Asklepios Biopharmaceutical Inc., of Research Triangle Park, N.C. | MMA-101 | Adeno-associated virus gene therapy | Isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations | FDA granted rare pediatric disease designation | 10/20/20 | Endocrine/Metabolic |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Hydrochloric acid binder | Metabolic acidosis | During end-of-review conference, conducted as type A meeting, with FDA's Division of Cardiology and Nephrology following CRL for NDA under accelerated approval to treat people with chronic kidney disease (CKD), agency indicated it will require evidence of study drug's effect on CKD progression from near-term interim analysis of Valor-CKD trial for accelerated approval and is unlikely to rely solely on serum bicarbonate data to determine efficacy, as requested by company, which will await formal meeting minutes to determine next regulatory steps | 10/29/20 | Endocrine/Metabolic |
| Versantis AG, of Zurich, Switzerland | VS-01 | Liposomal-based detoxification therapy | Urea cycle disorders | FDA granted rare pediatric disease designation | 10/19/20 | Endocrine/Metabolic |
| Akero Therapeutics Inc., of South San Francisco | Efruxifermin | FGF receptor agonist; FGF-21 ligand | Non-alcoholic steatohepatitis | EMA granted PRIME designation | 10/16/20 | Gastrointestinal |
| Astellas Pharma Inc., of Tokyo | ASP-5354 | Imaging for intraoperative ureter visualization | Abdominopelvic surgeries | FDA granted fast track designation | 10/28/20 | Gastrointestinal |
| Gannex Pharma Co. Ltd., of Shanghai, a unit of Ascletis Pharma Inc. | ASC-42 | Farnesoid X receptor agonist | Nonalcoholic steatohepatitis indication | FDA approved the IND | 10/11/20 | Gastrointestinal |
| Inventiva SA, of Daix, France | Lanifibranor | Activates the 3 peroxisome proliferator?activated receptor isoforms | Non-alcoholic steatohepatitis | Received FDA breakthrough therapy designation | 10/13/20 | Gastrointestinal |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Semaglutide | GLP-1 receptor agonist | Non-alcoholic steatohepatitis | FDA granted breakthrough therapy designation | 10/30/20 | Gastrointestinal |
| Optibiotix Health plc, of Heslington, U.K., and Seed Health Inc., of Venice, Calif. | DS-01 | Multispecies, multistrain probiotic | Irritable bowel syndrome | FDA authorized the IND for a phase II study | 10/2/20 | Gastrointestinal |
| Aeglea Biotherapeutics Inc., of Austin, Texas | ACN-00177 | Engineered human enzyme therapy designed to lower total level of homocysteine in plasma | Homocystinuria | FDA granted orphan designation | 10/26/20 | Genitourinary/Sexual Function |
| Amag Pharmaceuticals Inc., of Waltham, Mass. | Makena | Hydroxyprogesterone caproate | Preterm birth | FDA sent a letter proposing to withdraw approval of Makena; company has 15 days to request a hearing | 10/5/20 | Genitourinary/Sexual Function |
| Astrazeneca plc, of Cambridge, U.K. | Farxiga (dapagliflozin) | SGLT2 inhibitor | Chronic kidney disease | FDA granted breakthrough therapy designation | 10/2/20 | Genitourinary/Sexual Function |
| Bayer AG, of Leverkusen, Germany | Mirena (levonorgestrel-releasing intrauterine system) | Progesterone receptor agonist | Pregnancy prevention | Submitted supplemental NDA to FDA seeking to extend duration of use for up to 7 years, based on results of phase III extension trial | 10/14/20 | Genitourinary/Sexual Function |
| Ovoca Bio plc, of Dublin | BP-101 | Synthetic peptide | Decreased sexual desire in women, including hypoactive sexual desire disorder | Russian Ministry of Heath didn't grant the marketing authorization due to issues with the chemistry, manufacturing and controls and labeling sections of the application | 10/2/20 | Genitourinary/Sexual Function |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab-cwvz) | C5 complement inhibitor | Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome | FDA approved a 100-mg/mL formulation, which will cut infusion times by approximately 60% | 10/12/20 | Hematologic |
| CTI Biopharma Corp., of Seattle | Pacritinib | JAK2, FLT3, IRAK1 and CSF1R kinase inhibitor | Myelofibrosis with severe thrombocytopenia | Initiated a rolling submission of an NDA | 10/13/20 | Hematologic |
| Edigene Inc., of Beijing | ET-01 | CRISPR/Cas9 gene-editing hematopoietic stem cell therapy | Transfusion-dependent beta-thalassemia | China’s Center for Drug Evaluation of the NMPA accepted for review the IND application | 10/27/20 | Hematologic |
| Glycomimetics Inc., of Rockville, Md. | Rivipansel | Glycomimetic candidate that binds to E-, P- and L-selectin | Sickle cell disease | FDA granted rare pediatric disease designation for patients 18 and younger | 10/6/20 | Hematologic |
| Novartis AG, of Basel, Switzerland | Adakveo (crizanlizumab) | Binds to P-selectin | Sickle cell disease | EC approved the drug for the prevention of recurrent vaso-occlusive crises | 10/30/20 | Hematologic |
| Octapharma AG, of Lachen, Switzerland | Nuwiq (recombinant factor VIII) | Cell line-derived recombinant FVIII | Hemophilia A | FDA approved updated prescribing information to include immunogenicity data from the Nuprotect study in previously untreated patients | 10/19/20 | Hematologic |
| Orca Bio Inc., of Menlo Park, Calif. | Orca-T | Allogeneic, high-precision cell therapy | Hematopoietic stem cell transplant | FDA granted regenerative medicine advanced therapy designation for use in patients with blood cancers who are eligible for hematopoietic stem cell transplant (HSCT); drug also received FDA orphan designation for enhancing cell engraftment in patients who quality for HSCT | 10/14/20 | Hematologic |
| Protagonist Therapeutics Inc., of Newark, Calif. | PTG-300 | Injectable synthetic peptide mimetic of hepcidin | Polycythemia vera | EMA granted orphan designation | 10/21/20 | Hematologic |
| Spectrum Pharmaceuticals Inc., of Henderson, Nev. | Rolontis (eflapegrastim) | G-CSF analogue | Neutropenia in patients on myelosuppressive cancer drugs | FDA informed company it was unable to conduct inspection of manufacturing plan during the current BLA review cycle due to COVID-19-related travel restrictions; agency is deferring action until an inspection can be completed | 10/26/20 | Hematologic |
| Aimmune Therapeutics Inc., of Brisbane, Calif. (Nestle SA) | Palforzia | Peanut (Arachis hypogaea) allergen powder-dnfp | Peanut hypersensitivity | EMA's CHMP issued positive opinion for the treatment of peanut allergy in peanut allergic patients age 4-17 years | 10/16/20 | Immune |
| Insignis Therapeutics Inc., of North Haven, Conn. | IN-001 | Oral epinephrine prodrug | Anaphylaxis | FDA cleared IND for phase I study | 10/29/20 | Immune |
| Janssen-Cilag International NV, of Beerse, Belgium | Tremfya (guselkumab) | Interleukin 23A inhibitor | Psoriatic arthritis | EMA's CHMP issued positive opinion for the treatment of adults with active psoriatic arthritis | 10/16/20 | Immune |
| Kezar Life Sciences Inc., of South San Francisco | KZR-616 | Immunoproteasome inhibitor | Polymyositis and dermatomyositis | FDA granted orphan drug designations for both indications | 10/23/20 | Immune |
| Mesoblast Ltd., of Melbourne, Australia | Remestemcel-L | Mesenchymal stem cell-based therapy | Acute graft-vs.-host disease | Requested type A meeting with FDA, expected in November 2020, to discuss potential accelerated approval of BLA to treat steroid-refractory disease in children under 12 with additional study in people 12 and older as post-approval requirement | 10/29/20 | Immune |
| Mesoblast Ltd., of New York | Remestemcel-L | Allogeneic culture-expanded mesenchymal stem cells | Pediatric steroid-refractory acute graft-vs.-host disease | FDA issued a complete response letter and requested the company run at least 1 additional randomized, controlled study | 10/1/20 | Immune |
| Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Small-molecule phosphoinositide 3-kinase delta inhibitor | Activated phosphoinositide 3-kinase delta syndrome | European Commission granted orphan designation | 10/21/20 | Immune |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLX-PAD cells | Allogeneic mesenchymal-like cells | Steroid-refractory chronic graft-vs.-host disease | Received clearance from the safety committee of an investigator-initiated phase I/II study to move forward with patient enrollment for cohort II | 10/13/20 | Immune |
| X4 Pharmaceuticals Inc., of Boston | Mavorixafor | Antagonist of CXCR4 | WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome | FDA granted fast track designation | 10/8/20 | Immune |
| Adaptive Phage Therapeutics Inc., of Gaithersburg, Md. | Phagebank | Bacteriophage-based therapy | Prosthetic joint infections | FDA granted orphan drug designation | 10/22/20 | Infection |
| Algernon Pharmaceuticals Inc., of Vancouver, British Columbia | NP-120 (ifenprodil) | N-methyl-D-aspartate receptor antagonist | COVID-19 | External data and safety monitoring board approved the continuation of the study | 10/30/20 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | AB-201 (rNAPc2) | Tissue factor inhibitor | COVID-19 infection | FDA accepted IND application for phase IIb/III sequential Aspen-COVID-19 trial in about 100 people hospitalized with infection, expected to begin in fourth quarter of 2020 | 10/7/20 | Infection |
| Arch Biopartners Inc., of Toronto | Metablok | LSALT peptide | Moderate to severe COVID-19 infection | Turkey Ministry of Health cleared site activation in phase II trial targeting acute lung and acute kidney injury in infected individuals | 10/7/20 | Infection |
| Armata Pharmaceuticals Inc., of Marina Del Rey, Calif. | AP-PA02 | Bacteriophage | Pseudomonas aeruginosa infections | FDA cleared the IND for Swarm-P.a., a phase Ib/IIa single ascending dose and multiple ascending dose study | 10/15/20 | Infection |
| Astrazeneca plc, of Cambridge, U.K. | ChAd0x1-S | Vaccine | COVID-19 prophylaxis | The Therapeutic Goods Administration in Australia granted a provisional determination making it eligible to apply for provisional registration | 10/9/20 | Infection |
| Astrazeneca plc, of Dublin | AZD-1222 | Vaccine | COVID-19 | FDA authorized restart of clinical trials | 10/23/20 | Infection |
| Biontech SE, of Mainz, Germany, and Pfizer Inc., of New York | BNT-162b2 | mRNA-based vaccine | COVID-19 prophylaxis | Started rolling submission of the marketing application with the EMA | 10/6/20 | Infection |
| Celltrion Group, of Incheon, South Korea | CT-P59 | Monoclonal antibody targeting SARS-CoV-2 | COVID-19 prophylaxis in people who had contact with SARS-CoV-2-infected patients | Korean Ministry of Food and Drug Safety approved the IND for a phase III study | 10/8/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | Monoclonal antibody targeting the CCR5 receptor | HIV | U.K.'s MHRA cleared it to file a BLA for 1 injection per week as a combination HIV therapy | 10/27/20 | Infection |
| Direct Biologics LLC, of Austin, Texas | Exoflo | Extracellular vesicle product isolated from human bone marrow mesenchymal stem or stromal cells | COVID-19-associated acute respiratory distress syndrome | FDA granted expanded access for use as treatment for patients with severe COVID-19 | 10/13/20 | Infection |
| Dr. Reddy’s Laboratories Ltd., of Hyderabad, India | Sputnik V | Adenovirus-based vaccine | COVID-19 prophylaxis | Drug Control General of India granted approval to conduct an adaptive phase II/III study | 10/17/20 | Infection |
| Edesa Biotech Inc., of Toronto | EB-05 | Monoclonal antibody targeting Toll-like receptor 4 | Hospitalized COVID-19 | FDA approved starting the phase II portion of the phase II/III study; site initiation process has begun | 10/19/20 | Infection |
| Eli Lilly and Co., of Indianapolis | LY-CoV555 | COVID-19 Spike glycoprotein modulator | Mild to moderate COVID-19 infection | Initial request for emergency use authorization submitted to FDA to use as monotherapy in higher-risk people recently diagnosed with infection | 10/7/20 | Infection |
| Enlivex Therapeutics Ltd., of Nes Ziona, Israel | Allocetra | Autologous cell-based therapy | COVID-19 | Israeli Ministry of Health authorized initiation of proposed investigator-initiated phase II trial in severe and critical COVID-19 patients | 10/13/20 | Infection |
| Equillium Inc., of La Jolla, Calif. | Itolizumab | Targets the CD6-ALCAM pathway | Hospitalized COVID-19 with acute respiratory distress syndrome | Received a study may proceed letter from the FDA for an 800-patient phase III study; primary endpoint is to evaluate the benefit of itolizumab for patients hospitalized with COVID-19; secondary endpoints includes mortality benefit and other measures of clinical improvement | 10/29/20 | Infection |
| Evgen Pharma plc, of Cheshire, U.K. | SFX-01 | Up-regulates Nrf2 pathway | Acute respiratory distress syndrome in patients with suspected COVID-19 | All necessary regulatory approvals have been received for the phase II/III STAR trial and recruitment is expected to commence around the end of the month | 10/23/20 | Infection |
| F2G Ltd., of Manchester, U.K. | Olorofim | Dihydroorotate dehydrogenase inhibitor | Central nervous system coccidioidomycosis | FDA granted breakthrough therapy designation for refractory or otherwise untreatable disease with standard of care | 10/22/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Antiviral | COVID-19 | FDA reissued the emergency use authorization with a clarification that an alternate care site that meets certain criteria is considered an "inpatient hospital setting" as described in the scope of the authorization | 10/16/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Antiviral | COVID-19 | FDA approved for treating patients requiring hospitalization | 10/22/20 | Infection |
| Innovation Pharmaceuticals Inc., of Wakefield, Mass. | Brilacidin | Defensin mimetic | COVID-19 | Submitted a request for a pre-IND meeting to discuss plans for a 120-patient phase II study | 10/2/20 | Infection |
| Insmed Inc., of Bridgewater, N.J. | Arikayce (amikacin) | 30S ribosomal protein inhibitor | Nontuberculous mycobacterial lung infections caused by Mycobacterium avium complex | European Commission granted marketing authorization for the drug | 10/28/20 | Infection |
| Janssen-Cilag International NV, of Beerse, Belgium and Viiv Healthcare Ltd., of London | Rekambys (rilpivirine) and Vocabria (cabotegravir) | Antiretroviral medicines | HIV-1 infection | EMA's CHMP issued positive opinion for use of the long-acting drugs together for maintenance treatment of HIV-1 infection | 10/16/20 | Infection |
| Matinas Biopharma Holdings Inc., of Bedminster, N.J. | MAT-2203 | Encochleated formulation of the antifungal amphotericin B | HIV-infected patients with cryptococcal meningitis | Data and safety monitoring board of the Enact study completed review of data from the first cohort and recommended starting the second cohort of 40 active-treatment patients | 10/19/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | mRNA vaccine candidate | COVID-19 | Initiated rolling submission to Health Canada; in separate news, received written confirmation from EMA that vaccine candidate is eligible for submission of marketing authorization application under agency’s centralized procedure | 10/14/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | mRNA vaccine candidate | COVID-19 | U.K.'s MHRA has started the rolling review process | 10/27/20 | Infection |
| Nektar Therapeutics Inc., of San Francisco | Bempegaldesleukin | CD122-preferential IL-2 pathway agonist | Mild COVID-19 | FDA cleared the IND for a phase Ib study that is scheduled to begin enrollment in early November; study will assess the change over time in absolute lymphocyte counts and measurements of clinical progression based upon the WHO Clinical Progression Scale | 10/27/20 | Infection |
| Nitric Oxide Innovations LLC, of Austin, Texas | NOviricid | Sodium nitrite dosing in oral lozenge | COVID-19 | FDA cleared IND for phase IIb/IIIa outpatient study in African Americans diagnosed with COVID-19 | 10/26/20 | Infection |
| Oncosec Medical Inc., of Pennington, N.J. | Corvax12 | COVID-19 Spike glycoprotein modulator + IL-12 gene stimulator | COVID-19 infection | FDA cleared IND application for phase I trial in up to 36 volunteers | 10/29/20 | Infection |
| Oragenics Inc., of Tampa, Fla. | Terra CoV-2 | SARS-CoV-2 vaccine | COVID-19 prophylaxis | Received feedback to its type B pre-IND meeting request that FDA is in broad agreement with the planned approach to clinical development, and the company said it expects to file an IND by the end of the first quarter of 2021 and to start enrollment in a phase I study early in the second quarter of 2021 | 10/6/20 | Infection |
| Pluristem Therapeutics Inc., of Haifa, Israel | PLXPAD | Stem cell therapy | COVID-19 | Received approval from Israel Ministry of Health to expand enrollment in Israel | 10/7/20 | Infection |
| Qualigen Therapeutics Inc., of Carlsbad, Calif. | AS-1411 | Nucleolin-targeting DNA aptamer | COVID-19 | Received written feedback from a type B pre-IND meeting with the FDA; plans to start a phase IIa study in the first half of 2021 | 10/28/20 | Infection |
| Recce Pharma Ltd., of Sydney, Australia | Recce-327 | Broad-spectrum antibiotic | Infected burn wounds | Human Research Ethics Committee approved the start of a phase I/II study in Australia; study will initially enroll up to 30 patients, testing daily and 3-times-per-week treatment regimens | 10/19/20 | Infection |
| Recce Pharmaceuticals Ltd., of Sydney, Australia | RECCE-327 | Spray-on formulation of bactericidal permeability protein inhibitor | Sepsis | Received Human Research Ethics Committee approval to start a phase I/II | 10/16/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | RHB-204 | Fixed-dose oral capsule of clarithromycin, rifabutin and clofazimine | Nontuberculous mycobacteria disease | FDA granted orphan designation | 10/14/20 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGN-COV2 | 2 monoclonal antibodies targeting SARS-CoV-2 | COVID-19 | Submitted a request to the FDA for an emergency use authorization | 10/7/20 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGN-COV2 | Antibody cocktail targeting SARS-CoV2 | COVID-19 | Independent data monitoring committee recommended stopping enrollment of patients requiring high-flow oxygen or mechanical ventilation based on unfavorable risk/benefit profile; enrollment of patients requiring either no or low-flow oxygen can continue, as will outpatient study | 10/30/20 | Infection |
| Regeneron Pharmaceuticals, of Tarrytown, N.Y. | Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) | Monoclonal antibodies targeting Zaire ebolavirus | Ebola virus infection | FDA approved the drug | 10/14/20 | Infection |
| Revelation Biosciences Inc., of Menlo Park, Calif. | REVTx?99 | Intranasal formulation of a synthetic version of monophosphoryl lipid A | Healthy volunteers (eventually COVID-19) | Received acknowledgment by the Therapeutic Goods Administration in Australia to conduct the phase I study of up to 32 participants | 10/7/20 | Infection |
| Reven Holdings Inc. (aka Reven Pharmaceuticals), of Golden, Colo. | Rejuveinix | Antioxidant and anti-inflammatory combination | COVID-19 infection | FDA approved IND for phase II study in people hospitalized with mild to moderate disease or hypoxemic respiratory failure | 10/22/20 | Infection |
| Russian Direct Investment Fund, of Moscow | Sputnik V | Coronavirus vaccine | COVID-19 | Submitted applications to the World Health Organization for accelerated registration (emergency use listing) and prequalification | 10/27/20 | Infection |
| Russian Direct Investment Fund, of Moscow, and União Química Farmacêutica Nacional SA, of Embu-Guaçu, Brazil | Sputnik V | COVID-19 vaccine | COVID-19 prophylaxis | Completed pre-submission of preliminary documents to Brazil’s National Health Surveillance Agency | 10/30/20 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Oral ibrexafungerp | 1,3 beta glucan synthase inhibitor | Vulvovaginal candidiasis | Submitted NDA to FDA | 10/14/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | Abivertinib | EGFR gene inhibitor; BTK tyrosine kinase inhibitor | COVID-19 | Brazilian regulatory ANVISA cleared start of phase II study in mild, moderate and severe COVID-19 patients; about 400 patients will be enrolled | 10/14/20 | Infection |
| Taigen Biotechnology Co. Ltd., of Taipei, Taiwan | TG-1000 | Pan-influenza antiviral | Influenza | Filed an IND with the FDA | 10/5/20 | Infection |
| Taiwan Liposome Co. Ltd. (TLC), of Taipei, Taiwan | TLC-19 | Hydroxychloroquine liposome inhalation suspension | COVID-19 infection | Australian Human Research Ethics Committee and Taiwan FDA cleared initiation of phase I trial in healthy volunteers | 10/6/20 | Infection |
| Tevogen Bio Inc., of Metuchen, N.J. | Antigen-specific T-cell technology | Antigen-specific T-cell technology | COVID-19 | Submitted IND to the FDA to test in hospitalized COVID-19 patients | 10/14/20 | Infection |
| Valneva SE, of Saint-Herblain, France | VLA-1553 | Live attenuated viral vaccine | Chikungunya virus infection | EMA granted PRIME designation | 10/16/20 | Infection |
| Xortx Therapeutics Inc., of Calgary, Alberta | XRx-101 | Xanthine oxidase inhibitor | Acute kidney injury in COVID-19 | Received feedback from pre-IND submission to the FDA on plan to develop the drug | 10/8/20 | Infection |
| Antisense Therapeutics Ltd., of Melbourne, Australia | ATL-1102 | Inhibitor of CD49d expression on certain immune cells | Duchenne muscular dystrophy | FDA granted rare pediatric disease designation | 10/2/20 | Musculoskeletal |
| Italfarmaco Group, of Milan | Givinostat | Histone deacetylase inhibitor | Duchenne muscular dystrophy | FDA granted rare pediatric disease designation | 10/9/20 | Musculoskeletal |
| Kyowa Kirin Co. Ltd., of Tokyo | Crysvita (burosumab) | Monoclonal antibody targeting fibroblast growth factor 23 | X-linked hypophosphatemia | European Commission approved the new indication, which includes all adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults | 10/2/20 | Musculoskeletal |
| Pfizer Inc., of New York | PF-06939926 | Gene therapy | Duchenne muscular dystrophy | FDA granted fast track designation | 10/1/20 | Musculoskeletal |
| Solid Biosciences Inc., of Cambridge, Mass. | SGT-001 | DMD gene stimulator | Duchenne muscular dystrophy | FDA lifted clinical hold on Ignite DMD phase I/II trial; dosing expected to resume in first quarter of 2021 | 10/1/20 | Musculoskeletal |
| Zhittya Genesis Medicine Inc., of Las Vegas | Human FGF-1 | Fibroblast growth factor | Amyotrophic lateral sclerosis | Mexican regulatory authorities gave permission to start a study | 10/15/20 | Musculoskeletal |
| Amacathera Inc., of Toronto | AMT-143 | Long-acting anesthetic formulation | Postsurgical pain | Received approval from Health Canada to proceed to a phase I trial | 10/20/20 | Neurology/Psychiatric |
| Aprinoia Therapeutics Inc., of Taipei, Taiwan | 18F-APN-1607 | Imaging tracer targeting abnormal tau protein aggregates | Mild cognitive impairment and different stages of Alzheimer's disease | China's National Medical Products Administration approved the start of a phase III study testing the ability of the tracer to distinguish between patients with MCI and AD from healthy subjects | 10/28/20 | Neurology/Psychiatric |
| Atai Life Sciences AG, of Berlin | N-acetylcysteine | Precursor of glutathione | Acute mild traumatic brain injury | Completed its pilot study intended to show nose-to-brain delivery, and was granted IND clearance by the FDA to continue phase I trial development | 10/27/20 | Neurology/Psychiatric |
| Avenue Therapeutics Inc., of New York | I.V. tramadol | Analgesic | Acute pain | FDA issued a complete response letter because it determined the drug wasn't safe for the intended population due to the potential for opioid stacking | 10/12/20 | Neurology/Psychiatric |
| Axovant Gene Therapies Ltd., of New York | AXO-AAV-GM2 | Gene therapy | Tay-Sachs disease and Sandhoff disease | FDA granted rare pediatric disease designation | 10/13/20 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Aducanumab | Monoclonal antibody targeting amyloid beta plaques | Alzheimer’s disease | EMA accepted the MAA | 10/30/20 | Neurology/Psychiatric |
| Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. | Nurtec ODT (rimegepant) | CGRP receptor antagonist in an orally disintegrating tablet | Migraine | FDA accepted for review the supplemental NDA seeking use for preventive treatment of migraine; PDUFA date is expected in second quarter of 2021 | 10/14/20 | Neurology/Psychiatric |
| Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 | Orally dissolving sublingual thin film formulation of dexmedetomidine | Agitation associated with delirium | FDA cleared IND; phase II study set to start within next several months | 10/26/20 | Neurology/Psychiatric |
| Cerecin Inc., of Singapore | Tricaprilin | Medium chain triglyceride | Infantile spasms | FDA granted rare pediatric disease designation | 10/8/20 | Neurology/Psychiatric |
| Cerecin Inc., of Singapore | Tricaprilin | Medium chain triglyceride | Infantile spasms | FDA granted orphan drug designation | 10/29/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Fycompa (perampanel) | AMPA receptor antagonist | Partial seizure | China's NMPA approved sNDA for partial-onset seizures and pediatric indication for partial onset seizures in patients with epilepsy 4 years or older | 10/16/20 | Neurology/Psychiatric |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | ET-101 (topiramate oral solution) | GABA receptor agonist; NMDA receptor antagonist | Seizure disorder; migraine | NDA submitted to FDA as monotherapy to treat partial-onset or primary general tonic-clonic seizures in people 2 and older, as adjunctive therapy to treat partial-onset seizures in those 2 and older and as preventative treatment of migraine in those 12 and older | 10/6/20 | Neurology/Psychiatric |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | Zonisamide | Blocks T-type calcium channels | Partial seizures in patients with epilepsy | FDA accepted the NDA; PDUFA target action date is May 29, 2021 | 10/15/20 | Neurology/Psychiatric |
| Gemvax & Kael Co. Ltd., of Seoul, South Korea | GV-1001 (tertomotide) | Telomerase modulator | Alzheimer's disease | Announced the decision to apply for an expanded access IND based on a recent analysis of results from a phase II trial | 10/6/20 | Neurology/Psychiatric |
| Harmony Bioscience Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Cataplexy | FDA approved use for treating cataplexy in adults with narcolepsy; agency approved drug in 2019 for use in excessive daytime sleepiness in narcolepsy patients | 10/14/20 | Neurology/Psychiatric |
| Intellia Therapeutics Inc., of Cambridge, Mass. | NTLA-2001 | CRISPR-associated endonuclease Cas9 modulator; TTR gene modulator | Transthyretin amyloidosis | U.K. MHRA cleared CTA for phase I study in hereditary disease with polyneuropathy, expected to begin dosing by year-end 2020 | 10/19/20 | Neurology/Psychiatric |
| Irlab Therapeutics AB, of Stockholm | Mesdopetam (IRL-790) | Dopamine D3 receptor antagonist | Parkinson’s disease | FDA accepted IND for inclusion of U.S. patients in planned phase IIb/III study | 10/1/20 | Neurology/Psychiatric |
| Neurelis Inc., of San Diego | Valtoco (diazepam nasal spray) | Benzodiazepine | Intermittent, stereotypic episodes of frequent seizure activity | FDA granted an extended expiration for the drug | 10/19/20 | Neurology/Psychiatric |
| Neurocrine Biosciences Inc., of San Diego, and Xenon Pharmaceuticals Inc., of Burnaby, British Columbia | NBI-921352 | Nav1.6 sodium channel inhibitor | SCN8A developmental and epileptic encephalopathy syndrome | FDA requested nonclinical data to support proposed dosing in the pediatric study; companies plan to respond with a goal of starting the phase II study in 2021 | 10/8/20 | Neurology/Psychiatric |
| Nexus Pharmaceuticals Inc., of Linconshire, Ill. | Succinylcholine | Generic of the ultra-short-acting, depolarizing, skeletal muscle relaxant Quelicin | Adjunct to general anesthesia, to facilitate tracheal intubation and to provide skeletal muscle relaxation during surgery or mechanical ventilation | FDA approved the generic drug | 10/29/20 | Neurology/Psychiatric |
| Orchard Therapeutics Ltd., of London | Libmeldy (OTL-200) | ARSA gene stimulator | Metachromatic leukodystrophy | EMA's CHMP issued positive opinion for the treatment of MLD, characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in certain children | 10/16/20 | Neurology/Psychiatric |
| OWP Pharmaceuticals Inc., of Naperville, Ill. | Quetiapine fumarate (liquid/oral suspension) | 5-HT 2 receptor antagonist; dopamine D2 receptor antagonist | Schizophrenia; bipolar disorder | FDA approved IND application for formulation advancing on 505(b)(2) pathway | 10/7/20 | Neurology/Psychiatric |
| PTC Therapeutics Inc., of South Plainfield, N.J., and Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | SMN2-directed RNA splicing modifier | Spinal muscular atrophy | Approved in Brazil by National Health Surveillance Agency (ANVISA); NDA filed in Japan | 10/21/20 | Neurology/Psychiatric |
| PTC Therapeutics Inc., of South Plainfield, N.J., and Roche Holding AG, of Basel, Switzerland | Evrysdi (risdiplam) | Small-molecule SMN2-directed RNA splicing modifier | Spinal muscular atrophy | Partner Chugai Pharmaceutical Co. Ltd., a member of the Roche Group, of Basel, Switzerland, filed an NDA in Japan | 10/21/20 | Neurology/Psychiatric |
| Taysha Gene Therapies Inc., of Dallas | TSHA-102 | AAV9-based gene therapy | Rett syndrome | Received FDA rare pediatric disease designation and orphan designation | 10/14/20 | Neurology/Psychiatric |
| Taysha Gene Therapies Inc., of Dallas | TSHA-104 | AAV9-based gene therapy | SURF1-associated Leigh syndrome | FDA granted rare pediatric disease and orphan drug designations | 10/27/20 | Neurology/Psychiatric |
| Trevena Inc., of Chesterbrook, Pa. | Olinvyk (oliceridine) | Opioid | Acute pain | DEA classified the drug as a scheduled II controlled substance | 10/30/20 | Neurology/Psychiatric |
| Voyager Therapeutics Inc., of Cambridge, Mass. | VY-HTT01 | Gene therapy | Huntington’s disease | Was notified that the IND was placed on clinical hold pending the resolution of certain chemistry, manufacturing and controls matters; the company expects to receive specific feedback on these matters within 30 days and plans to work closely with the agency to resolve them | 10/13/20 | Neurology/Psychiatric |
| Xenon Pharmaceuticals Inc., of Burnaby, British Columbia | XEN-496 | Kv7 potassium channel modulator | KCNQ2 developmental and epileptic encephalopathy | Received a positive opinion from the EMA's Committee for Orphan Medicinal Products on the request for orphan medicinal product designation | 10/8/20 | Neurology/Psychiatric |
| Xeris Pharmaceuticals Inc., of Chicago | XP-0863 (diazepam non-aqueous injection) | Benzodiazepine | Acute repetitive seizures | FDA granted fast track designation | 10/20/20 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (fenfluramine) | Anticonvulsant agent | Dravet syndrome | EMA's CHMP issued positive opinion for the treatment of seizures associated with Dravet syndrome, as an add-on therapy to other antiepileptic medicines for patients 2 years of age and older | 10/16/20 | Neurology/Psychiatric |
| Zosano Pharma Corp., of Fremont, Calif. | Qtrypta (zolmitriptan transdermal microneedle system) | 5-HT 1b receptor agonist; 5-HT 1d receptor agonist | Migraine | Received complete response letter from FDA, citing inconsistent zolmitriptan exposure levels observed across clinical pharmacology studies | 10/20/20 | Neurology/Psychiatric |
| Eyevensys SAS, of Paris | EYS-611 | DNA plasmid that encodes for human transferrin protein | Retinitis pigmentosa | FDA granted orphan designation | 10/5/20 | Ocular |
| Kala Pharmaceuticals Inc., of Watertown, Mass. | Eysuvis (loteprednol etabonate) | Corticosteroid | Dry eye disease | Approved by FDA for short-term treatment of DED | 10/27/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France, and Ocumension Therapeutics Ltd., of Shanghai | NCX-470 | Second-generation nitric oxide-donating bimatoprost analogue | Open-angle glaucoma or ocular hypertension | Received approval from China’s Center for Drug Evaluation of the NMPA to carry out the Chinese part of the ongoing Mont Blanc trial, the first phase III trial testing the drug in lowering of intraocular pressure in open-angle glaucoma or ocular hypertension | 10/26/20 | Ocular |
| Revision Therapeutics Inc., of Ridgewood, N.J. | REV-0100 | Binds and clears lipofuscin | Stargardt disease | FDA granted orphan drug and rare pediatric disease designations | 10/28/20 | Ocular |
| Samsung Bioepis Co. Ltd., of Incheon, Korea, and Biogen Inc., of Cambridge, Mass. | SB-11 | Biosimilar of ranibizumab | Retinal vascular disorders | EMA accepted for review the MAA | 10/6/20 | Ocular |
| Santen Inc., of Emeryville, Calif. | Cyclosporine topical ophthalmic emulsion 0.1% | Cyclosporine topical ophthalmic emulsion 0.1% | Severe vernal keratoconjunctivitis | FDA accepted NDA and set PDUFA date of June 26, 2021 | 10/26/20 | Ocular |
| Vanda Pharmaceuticals Inc., of Washington | VSJ-110 | Cystic fibrosis transmembrane conductance regulator activator | Allergic conjunctivitis | FDA has approved the IND application | 10/27/20 | Ocular |
| Polarean Imaging plc, of Durham, N.C. | Hyperpolarized 129 Xenon | Drug-device diagnostic | Lung disease | NDA submitted to FDA, requesting priority review, for use to evaluate pulmonary function and visualize lung using MRI | 10/7/20 | Other/Miscellaneous |
| Astrazeneca plc, of Cambridge, U.K. | Trixeo Aerosphere (formoterol fumarate/glycopyrronium bromide/budesonide) | Long-acting muscarinic antagonist, long-acting beta2-agonist, inhaled corticosteroid | Moderate to severe chronic obstructive pulmonary disease | EMA's Committee for Medicinal Products for Human Use recommended approval of the drug for patients not adequately treated by a combination of an inhaled corticosteroid and a long-acting beta2-agonist (LABA), or a combination of a LABA and a long-acting muscarinic antagonist | 10/19/20 | Respiratory |
| Pharmaxis Ltd., of Sydney, and Chiesi USA Inc., affiliate of Chiesi Farmaceutici SpA, of Parma, Italy | Bronchitol (mannitol inhalation powder) | Mucolytic agent | Cystic fibrosis | FDA approved for maintenance therapy to improve pulmonary function in people 18 and older | 10/30/20 | Respiratory |
| Adial Pharmaceuticals Inc., of Charlottesville, Va. | AD-04 | Genetically targeted therapeutic | Alcohol use disorder | FDA reactivated IND and lifted clinical hold | 10/21/20 | Toxicity and Intoxication |
| Amphastar Pharmaceuticals Inc., of Rancho Cucamonga, Calif. | Atropine Sulfate | Antimuscarinic | Bradycardia, reduce salivation and bronchial secretions before surgery, and antidote for overdose of cholinergic drugs or mushroom poisoning | FDA approved the abbreviated NDA for the drug that's been sold as a grandfather exception for the past 40 years | 10/6/20 | Toxicity and Intoxication |
| Eton Pharmaceuticals Inc., of Deer Park, Ill. | Dehydrated alcohol | Ethanol | Methanol poisoning | FDA accepted the NDA; PDUFA target action date is May 27, 2021 | 10/8/20 | Toxicity and Intoxication |
| ADC Therapeutics SA, of Lausanne, Switzerland | Loncastuximab tesirine | Antibody-drug conjugate targeting CD19 | Relapsed or refractory diffuse large B-cell lymphoma | FDA accepted the BLA with a priority review; PDUFA target action date is May 21, 2021 | 11/20/20 | Cancer |
| Agilent Technologies Inc., of Santa Clara, Calif. | PD-L1 IHC 22C3 Pharmdx | Companion diagnostic | Triple-negative breast cancer | Approved by FDA for use to identify patients for treatment with Keytruda (pembrolizumab, Merck & Co. Inc.) | 11/13/20 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Kyprolis (carfilzomib) | Proteasome inhibitor; ubiquitin inhibitor | Multiple myeloma (MM) | EMA's CHMP recommended update to existing indication supporting Kyprolis treatment with daratumumab and dexamethasone (dex), with lenalidomide and dex, or with dex alone for adults with MM who've received at least one prior therapy | 11/13/20 | Cancer |
| Angiocrine Bioscience Inc., of San Diego | AB-205 | Human engineered cord endothelial cells | Organ vascular niche injuries in patients with Hodgkin lymphoma and non-Hodgkin lymphoma undergoing high-dose therapy and autologous hematopoietic stem cell transplantation | FDA granted regenerative medicine advanced therapy designation | 11/11/20 | Cancer |
| Anixa Biosciences Inc., of San Jose, Calif. | Prophylactic breast cancer vaccine | Prophylactic breast cancer vaccine | Breast cancer | Filed an IND with the FDA to begin phase I testing | 11/23/20 | Cancer |
| Antengene Corp. Ltd., of Shanghai | ATG-016 (eltanexor) | Selective inhibitor of nuclear export | Intermediate- and higher-risk myelodysplastic syndrome after failure of hypomethylating agent-based therapy | China's National Medical Products Administration approved the start of a phase I/II study testing the pharmacokinetics, safety and efficacy of ATG-016 monotherapy | 11/25/20 | Cancer |
| Apollomics Inc., of Foster City, Calif. | APL-102 | Oral multikinase inhibitor | Advanced solid tumors | China’s NMPA cleared IND for phase I study | 11/12/20 | Cancer |
| Aprea Therapeutics Inc., of Boston | Eprenetapopt | Restores wild-type p53 conformation and function | TP53-mutant acute myeloid leukemia | FDA granted fast track designation | 11/30/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 inhibitor | Waldenström macroglobulinemia | China’s NMPA cleared phase Ib/II trial testing drug as single agent or in combination with ibrutinib/rituximab | 11/23/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-2575 | Bcl-2 inhibitor | Multiple myeloma | China’s NMPA cleared start of phase Ib/II trial testing drug as single agent or in combination with lenalidomide/dexamethasone | 11/23/20 | Cancer |
| Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1202 | Methionine aminopeptidases II inhibitor | Non-muscle invasive bladder cancer | China's NMPA cleared CTA for phase III trial as single oral agent for first-line treatment | 11/5/20 | Cancer |
| Astellas Pharma Inc., of Tokyo | Xtanri (enzalutamide) | Androgen receptor signaling inhibitor | Non-metastatic castration-resistant prostate cancer with high risk of metastasis | China's National Medical Products Administration approved the drug | 11/6/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K, and Merck & Co Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Metastatic castration-resistant prostate cancer | European Commission approved to treat disease in people with BRCA1/2 mutations | 11/5/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Calquence (acalabrutinib) | Next-generation BTK inhibitor | Chronic lymphocytic leukemia | Approved in EU for adults | 11/9/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Merck & Co Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Ovarian cancer | European Commission approved, in combination with VEGF inhibitor Avastin (bevacizumab, Roche AG), for first-line maintenance treatment of HRD-positive advanced disease | 11/5/20 | Cancer |
| Astrazeneca plc, of Wilmington, Del. | Imfinzi (durvalumab) | Antibody targeting PD-L1 | Unresectable Stage III non-small-cell lung cancer after chemoradiation therapy and previously treated advanced bladder cancer | FDA approved dosing option of 1,500 mg fixed dose every 4 weeks | 11/20/20 | Cancer |
| Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-1706 | Biosimilar to Avastin (bevacizumab); VEGF-targeting antibody | Metastatic carcinoma of the colon or rectum, metastatic breast cancer, non–small-cell lung cancer, advanced or metastatic renal cell cancer, epithelial ovarian, fallopian tube and primary peritoneal cancer, and cervical cancer | Submitted MAA to EMA seeking license for all approved indications in EU member states, Iceland, Norway and Liechtenstein | 11/25/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) and Yervoy (ipilimumab) | Monoclonal antibodies targeting PD-1 and CTLA-4 | First-line metastatic non-small-cell lung cancer | FDA approved use of the drugs in combination with two cycles of platinum-based chemotherapy | 11/6/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Lisocabtagene maraleucel | B-lymphocyte antigen CD19 modulator | Large B-cell lymphoma | FDA said it could not complete its review of BLA to treat adults with relapsed/refractory disease prior to Nov. 16, 2020, PDUFA date; agency was unable to inspect third-party manufacturing facility in Texas during review cycle due to COVID-19-related travel restrictions; new PDUFA date not set | 11/16/20 | Cancer |
| Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | Anti-PD-1 monoclonal antibody | Esophageal squamous cell carcinoma | Approved by European Commission for treating adults with unresectable advanced, recurrent or metastatic disease after prior fluoropyrimidine- and platinum-based combination chemotherapy | 11/24/20 | Cancer |
| Cellectis SA, of New York | UCART-CS1 | Gene-edited allogeneic CAR T cells | Relapsed or refractory myeloid myeloma | FDA lifted clinical hold on phase I MELANI-01 trial | 11/17/20 | Cancer |
| CNS Pharmaceuticals Inc., of Houston | Berubicin | Topoisomerase II inhibitor | Glioblastoma | IND filed with FDA for phase II trial in adults who failed first-line therapy; trial expected to begin in first quarter of 2021 | 11/17/20 | Cancer |
| Day One Biopharmaceuticals LLC, of South San Francisco | DAY-101 | Pan-RAF inhibitor | Relapsed low-grade glioma | FDA granted breakthrough therapy designation for treating pediatric patients with advanced low-grade glioma harboring RAF alteration | 11/19/20 | Cancer |
| Day One Biopharmaceuticals LLC, of South San Francisco | DAY-101 | Pan-RAF inhibitor | Malignant glioma | FDA granted orphan designation | 11/19/20 | Cancer |
| Engene Inc., of Boston | EG-70 | Nonviral gene therapy | Non-muscle invasive bladder cancer | FDA cleared the IND for a phase I/II trial that is expected to begin in early 2021 | 11/2/20 | Cancer |
| Eusa Pharma Ltd., of Hemel Hempstead, U.K., and Beigene Ltd., of Beijing, China | Qarziba (dinutuximab beta) | Targeted immunotherapy | High-risk neuroblastoma | BLA accepted by China’s NMPA and granted priority review | 11/9/20 | Cancer |
| Exelixis Inc., of Alameda, Calif., and Takeda Pharmaceutical Co. Ltd., of Tokyo | Cabometyx (cabozantinib) | Kinase inhibitor | Unresectable hepatocellular carcinoma that has progressed after prior systemic therapy | Japan's Ministry of Health, Labour and Welfare approved the drug | 11/27/20 | Cancer |
| F-star Therapeutics Ltd., of Cambridge, U.K. | FS-222 | Bispecific antibody targeting CD137 and PD-L1 | Cancer | Regulators in Spain authorized CTA for phase I trial | 11/24/20 | Cancer |
| Halozyme Therapeutics Inc., of San Diego, and Janssen Pharmaceutical Cos., part of New Brunswick, N.J.-based Johnson & Johnson | Darzalex Faspro (daratumumab and hyaluronidase-fihj) | ADP ribosyl cyclase-1 inhibitor | Relapsed/refractory multiple myeloma | Janssen submitted regulatory applications to the FDA and EMA seeking approval for use in combination with pomalidomide and dexamethasone to treat patients who have received at least 1 prior line of therapy | 11/12/20 | Cancer |
| Hillstream Biopharma Inc., of Bridgewater, N.J. | HSB-888 | Cancer stem cell inhibitor | Osteosarcoma | FDA granted rare pediatric disease and orphan drug designations | 11/23/20 | Cancer |
| Immune-Onc Therapeutics Inc., of Palo Alto, Calif. | IO-202 | Antagonist antibody | Acute myeloid leukemia | FDA granted orphan designation | 11/10/20 | Cancer |
| Imugene Ltd., of Sydney | PD1-Vaxx | PD-1 inhibitor | Non-small-cell lung cancer | FDA cleared IND for phase I trial | 11/2/20 | Cancer |
| Innate Pharma SA, of Paris | Lacutamab | Anti-KIR3DL2 humanized cytotoxicity-inducing antibody | Sézary syndrome | EMA granted PRIME designation for 3L treatment of relapsed or refractory Sézary syndrome patients | 11/13/20 | Cancer |
| Ipsen SA, of Paris | Onivyde (irinotecan sucrosofate) | Topoisomerase I inhibitor | Small-cell lung cancer | FDA granted fast track designation | 11/30/20 | Cancer |
| Istari Oncology Inc., of Durham, N.C. | PVS-RIPO | Live-attenuated serotype 1 poliovirus vaccine | Advanced melanoma | FDA accepted IND for phase II Luminos-102 trial examining therapy alone or in combination with PD-1 inhibitor in treatment-refractory disease, expected to begin in first quarter of 2021; interim analysis expected when 20 participants have been randomized and treated for 3 months; efficacy measures include objective response rates by RECIST criteria, durability of responses and overall survival over 24 months | 11/16/20 | Cancer |
| Jounce Therapeutics Inc., of Cambridge, Mass. | JTX-8064 | Anti-leukocyte immunoglobulin-like receptor B2 | Solid tumors | Received a study may proceed letter from FDA to begin phase I Innate study | 11/16/20 | Cancer |
| Lianbio Co. Ltd., of Shanghai and Princeton, N.J. | Infigratinib | Oral FGFR 1-3 inhibitor | Cholangiocarcinoma | China’s NMPA cleared the start of phase III Proof trial testing drug as first-line treatment in patients with locally advanced or metastatic unresectable disease with FGFR2 gene fusions | 11/3/20 | Cancer |
| Menari Group, of Florence, Italy | Elzonris (tagraxofusp) | IL-3 receptor modulator; CDw123 modulator | Blastic plasmacytoid dendritic cell neoplasm (BPDCN) | Following re-examination, EMA's CHMP recommended approval for a restricted indication in patients who had not yet received any treatment for BPDCN | 11/13/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | PD-1-inhibiting antibody | Triple-negative breast cancer | Approved by FDA for use in patients with locally recurrent unresectable or metastatic disease whose tumors express PL-L1 as determined by an FDA-approved test | 11/13/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide nanoparticles | Recurrent or metastatic head and neck squamous cell carcinoma with limited PD-L1 expression or refractory to PD-1 blockade | FDA issued a safe to proceed notification for the study testing NBTXR-3 activated by radiation plus Keytruda (pembrolizumab, Merck & Co. Inc.) in up to 60 patients; primary endpoint is tumor response | 11/17/20 | Cancer |
| Nanobiotix SA, of Paris | NBTXR-3 | Hafnium oxide nanoparticles | Inoperable locoregional recurrent head and neck squamous cell carcinoma | FDA issued a safe to proceed notification for the study testing NBTXR-3 activated by radiation plus Keytruda (pembrolizumab, Merck & Co. Inc.) in up to 80 patients; primary endpoint is progression-free survival | 11/17/20 | Cancer |
| Nanomab Technology Ltd., of London | NM-01 | PD-L1 diagnostic | Cancer | U.K.’s MHRA cleared the CTA for the phase II Pelican study to assess PD-L1 expression using 99mTc-NM-01 as a SPECT/CT radiotracer and its correlation to PD-L1 expression results based on routine immunohistochemical testing in biopsy diagnostic specimens | 11/3/20 | Cancer |
| Neoimmunetech Inc., of Rockville, Md. | NT-17 (efineptakin alfa) | Long-acting human interleukin-7 | Non-small-cell lung cancer | FDA cleared the IND for phase II study testing combination with Tecentriq (atezolizumab, Roche Holding AG) in patients with previously untreated, PD-L1-expressing, locally advanced or metastatic disease | 11/16/20 | Cancer |
| Onconova Therapeutics Inc., of Newtown, Pa. | ON-123300 | Multikinase inhibitor | Relapsed/refractory advanced cancer | Filed IND with FDA to start phase I trial to include patients with HR+/HER2-negative metastatic breast cancer with resistance to approved second-generation CDK4/6 inhibitors | 11/23/20 | Cancer |
| Pharmacyte Biotech Inc., of Laguna Hills, Calif. | Cell-in-a-Box | Live-cell encapsulation technology | Cancer | Received clinical hold letter from FDA regarding IND, previously placed on hold Oct. 2, 2020 | 11/4/20 | Cancer |
| Poseida Therapeutics Inc., of San Diego | P-PSMA-101 | PSMA-targeted autologous CAR T-cell therapy | Prostate cancer | FDA lifted clinical hold on phase I study, expected to resume immediately, in people with metastatic castration-resistant disease | 11/2/20 | Cancer |
| PTC Therapeutics Inc., of South Plainfield, N.J. | PTC-596 | Small-molecule tubulin binding agent | Leiomyosarcoma | FDA granted orphan designation and fast track designation | 11/18/20 | Cancer |
| PTC Therapeutics Inc., of South Plainfield, N.J. | PTC-596 | Small-molecule tubulin binding agent | Diffuse intrinsic pontine glioma | FDA granted rare pediatric disease designation and orphan designation | 11/18/20 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | CPI-613 (devimistat) | Targets mitochondrial tricarboxylic acid cycle | Metastatic pancreatic cancer | FDA granted fast track designation | 11/10/20 | Cancer |
| Rhovac AB, of Lund, Sweden | RV-001 | RhoC GTPase modulator | Prostate cancer | FDA granted fast track designation | 11/25/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | PD-L1 inhibitor | Hepatocellular carcinoma | European Commission approved, in combination with Avastin (bevacizumab, Roche), to treat adults with advanced or unresectable disease who did not receive prior systemic therapy | 11/2/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland | Phesgo (pertuzumab + trastuzumab) | Erbb2 tyrosine kinase receptor inhibitor | Metastatic breast cancer (MAA); breast tumor | EMA's CHMP recommended approval of MAA | 11/13/20 | Cancer |
| Samsung Bioepis Co. Ltd., of Incheon, Korea | Onbevzi (bevacizumab) | Anti-VEGF antibody | Multiple cancers | EMA's CHMP recommended approval of MAA | 11/13/20 | Cancer |
| Shanghai Junshi Biosciences Co. Ltd., of Shanghai | JS-006 | TIGIT receptor inhibitor | Solid tumors | China NMPA accepted IND | 11/30/20 | Cancer |
| Sirtex Medical Ltd., of Woburn, Mass. | SIR-Spheres Y-90 resin microspheres | Prescription device for selective internal radiation therapy | Colorectal cancer | Notified that China’s NMPA accepted NDA seeking approval for use in treating patients with unresectable disease in combination with fluorouridine adjuvant chemotherapy | 11/25/20 | Cancer |
| Surface Oncology Inc., of Cambridge, Mass. | SRF-388 | Monoclonal antibody targeting IL-27 | Hepatocellular carcinoma after standard therapies | FDA granted fast track designation | 11/11/20 | Cancer |
| Theralase Technologies Inc., of Toronto | Theralase | Photodynamic therapy | BCG-unresponsive non-muscle invasive bladder cancer | FDA granted fast track designation for phase II study | 11/23/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego, Alphamab Oncology Co. Ltd., of Suzhou, China, and 3D Medicines Co. Ltd., of Chengdu, China | Envafolimab (KN-035) | Single-domain antibody against PD-L1 | Microsatellite instability-high/deficient mismatch repair cancer | Alphamab and 3D Medicines submitted NDA to China’s NMPA | 11/16/20 | Cancer |
| XNK Therapeutics AB, of Stockholm | Undisclosed | NK cell-based immunotherapy | Multiple myeloma | FDA granted orphan designation | 11/4/20 | Cancer |
| Y-mabs Therapeutics Inc., of New York | Danyelza (naxitamab-gqgk) | Monoclonal antibody targeting ganglioside GD2 | Relapsed or refractory high-risk neuroblastoma in the bone or bone marrow | FDA approved the drug for use in patients who have demonstrated a partial response, minor response or stable disease to prior therapy | 11/25/20 | Cancer |
| Zymeworks Inc, of Vancouver, British Columbia | Zanidatamab | Bispecific antibody targeting 2 non-overlapping epitopes of HER2 | Previously treated HER2 gene-amplified biliary tract cancer | FDA granted breakthrough designation | 11/30/20 | Cancer |
| Zymeworks Inc., of Vancouver, British Columbia | Zanidatamab | HER2-targeted bispecific antibody | Gastric cancer | European Commission granted orphan drug designation | 11/19/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Forxiga (dapagliflozin) | Sodium glucose transporter-2 inhibitor | Heart failure with reduced ejection fraction | European Commission approved to treat symptomatic chronic disease in adults with and without type 2 diabetes | 11/5/20 | Cardiovascular |
| Astrazeneca plc, of Cambridge, U.K. | Forxiga (dapagliflozin) | SGLT2 inhibitor | Chronic heart failure | Japan's Ministry of Health, Labour and Welfare approved the drug | 11/30/20 | Cardiovascular |
| Bayer AG, of Leverkusen, Germany | Xarelto (rivaroxaban) | Factor Xa antagonist | Venous thromboembolism | EMA's CHMP recommended new strength and new indication: treatment of VTE and prevention of VTE recurrence in term neonates, infants and toddlers, children, and adolescents aged less than 18 years after at least 5 days of initial parenteral anticoagulation treatment | 11/13/20 | Cardiovascular |
| Boehringer Ingelheim GmbH, of Ingelheim, Germany | Pradaxa (dabigatran etexilate) | Factor IIa antagonist; protease-activated receptor-1 antagonist | Venous thromboembolic events | EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for treating VTE and prevention of recurrent VTE in pediatric patients, from birth to less than 18 years | 11/16/20 | Cardiovascular |
| Boehringer Ingelheim International GmbH, of Ingelheim, Germany | Pradaxa (dabigatran etexilate) | Factor IIa antagonist; Protease-activated receptor-1 antagonist | Venous thromboembolism | EMA's CHMP recommended new form an strength together with new indication for treatment of VTE and prevention of recurrent VTE in pediatric patients from birth to less than 18 years of age | 11/13/20 | Cardiovascular |
| Clinigen Group plc, of Burton Upon Kent, U.K. | Totect | Dexrazoxane hydrochloride | Cardiomyopathy associated with doxorubicin | FDA granted approval to broaden indication to include reducing incidence and severity of cardiomyopathy associated with doxorubicin administration in women with metastatic breast cancer who have received a cumulative doxorubicin dose of 300 mg/m² and who will continue to receive doxorubicin therapy to maintain tumor control | 11/10/20 | Cardiovascular |
| Dicerna Pharmaceuticals Inc., of Lexington, Mass., and Eli Lilly and Co., of Indianapolis | LY-3561774 | RNAi drug | Cardiometabolic disease | FDA accepted IND filed by Lilly to start phase I trial; study expected to begin by year-end | 11/16/20 | Cardiovascular |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Rilonacept | Recombinant dimeric fusion protein blocking interleukin-2-alpha and interleukin-1-beta signaling | Recurrent pericarditis | FDA accepted supplemental BLA and granted priority review; PDUFA date of March 21, 2021 | 11/23/20 | Cardiovascular |
| Liquidia Technologies Inc., of Research Triangle Park, N.C. | LIQ-861 (treprostinil, dry powder formulation) | Vasodilator | Pulmonary arterial hypertension | FDA issued complete response letter, identifying need for additional information and clarification on chemistry, manufacturing and controls data pertaining to drug product and device biocompatibility; FDA also reconfirmed the need to conduct on-site pre-approval inspections of 2 of Liquidia’s U.S. manufacturing facilities before application can be approved | 11/25/20 | Cardiovascular |
| Omeros Corp., of Seattle | Narsoplimab | Mannan-binding lectin serine protease-2 inhibitor | Hematopoietic stem cell transplant-associated thrombotic microangiopathy | Completed rolling BLA to FDA | 11/18/20 | Cardiovascular |
| Plx Pharma Inc., of Sparta, N.J. | Vazalore 325 mg and Vazalore 81 mg | Liquid-filled aspirin capsule | Vascular disease | FDA confirmed receipt of 2 CMC supplemental NDAs, setting estimated completion date for reviews for end of February 2021 | 11/17/20 | Cardiovascular |
| Amnio Technology LLC, of Phoenix | Palingen Flow | Amniotic tissue allograft | Chronic ulcers of lower extremities | FDA cleared IND for phase II trial in venous stasis or diabetic etiologies | 11/4/20 | Dermatologic |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Vixarelimab | Monoclonal antibody targeting oncostatin M receptor beta | Prurigo nodularis | FDA granted breakthrough therapy designation | 11/16/20 | Dermatologic |
| Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda | Vixarelimab | Oncostatin M receptor subunit beta inhibitor | Pruritus | FDA granted breakthrough therapy designation for disease associated with prurigo nodularis | 11/16/20 | Dermatologic |
| Polarityte Inc., of Salt Lake City | Skinte | Autologous cell and tissue-based therapy | Diabetic foot ulcers | FDA provided written response following type B pre-IND meeting, including feedback and recommendations on manufacturing, preclinical studies, data submitted in briefing package and additional studies to support IND submission, expected in second half of 2021 | 11/2/20 | Dermatologic |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Dupixent (dupilumab) | Monoclonal antibody targeting interleukin-4 and interleukin-13 | Severe atopic dermatitis | European Commission extended the marketing authorization to include children 6 to 11 years of age | 11/30/20 | Dermatologic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Oxlumo (lumasiran) | RNAi targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | European Commission granted marketing authorization | 11/19/20 | Endocrine/Metabolic |
| Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Oxlumo (lumasiran) | RNAi targeting hydroxyacid oxidase 1 | Primary hyperoxaluria type 1 | Approved by FDA | 11/24/20 | Endocrine/Metabolic |
| Asklepios Biopharmaceutical, Inc., of Research Triangle Park, N.C., and Selecta Biosciences Inc., of Watertown, Mass. | MMA-101 | Gene therapy | Isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations | FDA granted orphan drug designation | 11/19/20 | Endocrine/Metabolic |
| Janssen Pharmaceutical Cos. of Johnson & Johnson, of New Brunswick, N.J., and Genmab A/S, of Copenhagen | Darzalex (daratumumab) | ADP ribosyl cyclase-1 inhibitor | Light chain amyloidosis | Type II variation application submitted to EMA to expand use of subcutaneous formulation in indication | 11/5/20 | Endocrine/Metabolic |
| Lipocine Inc., of Salt Lake City | Tlando | Testosterone replacement therapy | Hypogonadism | FDA informed the company that it plans to make a decision on the NDA on or about the week of Nov. 30, 2020 | 11/6/20 | Endocrine/Metabolic |
| Logicbio Therapeutics Inc., of Lexington, Mass. | LB-001 | MUT gene stimulator | Methylmalonic acidemia | FDA granted fast track designation | 11/4/20 | Endocrine/Metabolic |
| Orphazyme A/S, of Copenhagen, Denmark | Arimoclomol | Chaperonin stimulator; Hsp70 stimulator | Niemann-Pick disease type C | Submitted MAA to EMA | 11/9/20 | Endocrine/Metabolic |
| Polaryx Therapeutics Inc., of Paramus, N.J. | PLX-300 | Small molecule that activates PPAR? | GM2 gangliosidosis | FDA granted both rare pediatric disease and orphan drug designations | 11/25/20 | Endocrine/Metabolic |
| Protalix Biotherapeutics Inc., of Carmiel, Israel, and Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici SpA, of Parma, Italy | Pegunigalsidase alfa (PRX–102) | Chemically modified stabilized version of alpha-galactosidase-A | Fabry disease | FDA extended the PDUFA target action date by 3 months to April 27, 2021 | 11/27/20 | Endocrine/Metabolic |
| Provention Bio Inc., of Red Bank, N.J. | Teplizumab | CD3 antagonist | Type 1 diabetes | Completed submission of rolling BLA to FDA to delay or prevent T1D in at-risk individuals | 11/2/20 | Endocrine/Metabolic |
| Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Melanocortin-4 receptor agonist | Obesity due to pro-opiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency | FDA approved the drug | 11/27/20 | Endocrine/Metabolic |
| Sanofi SA, of Paris | Avalglucosidase alfa | Long-term enzyme replacement therapy | Pompe disease | FDA accepted for review the BLA; PDUFA date is May 18, 2021 | 11/18/20 | Endocrine/Metabolic |
| Azurrx Biopharma Inc., of Delray Beach, Fla. | MS-1819 | Lipase modulator | Exocrine pancreatic insufficiency | Protocol amendment expected to be submitted to FDA for phase IIb Option 2 trial in people with cystic fibrosis, requesting addition of study arm using immediate-release capsules to compare data with existing arm using delayed-release capsules and select optimal delivery method; trial on target to report top-line data in first quarter of 2021 | 11/17/20 | Gastrointestinal |
| Exegi Pharma LLC, of Rockville, Md. | EXE-346 | Live biotherapeutic containing fixed-proportion of 8 strains of live probiotic bacteria | Pouchitis | FDA granted orphan designation for prevention of disease relapse in pouchitis | 11/13/20 | Gastrointestinal |
| Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium | Filgotinib | JAK1 inhibitor | Ulcerative colitis | EMA validated the MAA | 11/2/20 | Gastrointestinal |
| Inventiva SA, of Daix, France | Lanifibranor | Activates the 3 peroxisome proliferator?activated receptor isoforms | Nonalcoholic steatohepatitis | Completed end-of-phase-II meeting with the FDA | 11/10/20 | Gastrointestinal |
| Mirum Pharmaceuticals Inc., of Foster City, Calif. | Maralixibat | Apical sodium-dependent bile acid transporter inhibitor | Progressive familial intrahepatic cholestasis type 2 | EMA accepted the MAA | 11/30/20 | Gastrointestinal |
| Sebela Pharmaceuticals Inc., of Braintree, Mass. | Sutab | Sulfate-based tablet | Bowel prep for colonoscopies | FDA approved the drug | 11/10/20 | Gastrointestinal |
| Vivet Therapeutics SAS, of Paris, and Pfizer Inc., of New York | VTX-801 | Gene therapy | Wilson disease | FDA cleared IND for phase I/II Gateway study, expected to begin in early 2021 | 11/18/20 | Gastrointestinal |
| Astellas Pharma Inc., of Tokyo, and Fibrogen Inc., of San Francisco | Evrenzo (roxadustat) | HIF-PH inhibitor | Anemia in chronic kidney disease | Japan's Ministry of Health, Labour and Welfare approved the drug | 11/27/20 | Genitourinary/Sexual Function |
| Bayer AG, of Leverkusen, Germany | Finerenone | Nonsteroidal, selective mineralocorticoid receptor antagonist | Chronic kidney disease and type 2 diabetes | Submitted NDA to FDA and MAA to EMA | 11/9/20 | Genitourinary/Sexual Function |
| Obseva SA, of Geneva | Yselty (linzagolix 100 mg and 200 mg; OBE-2109) | Oral, once daily, GnRH receptor antagonist | Uterine fibroids | Submitted MAA to EMA seeking approval for use in management of heavy menstrual bleeding associated with uterine fibroids | 11/24/20 | Genitourinary/Sexual Function |
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Mitapivat | Pyruvate kinase R activator | Sickle cell disease (SCD) | FDA granted orphan status in SCD | 11/12/20 | Hematologic |
| Alexion Pharmaceuticals Inc., of Boston | Ultomiris (ravulizumab) | Long-acting C5 inhibitor | Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome | European Commission approved a 100 mg/mL intravenous formulation | 11/20/20 | Hematologic |
| Apellis Pharmaceuticals Inc., of Waltham, Mass. | Pegcetacoplan | Targeted C3 therapy | Paroxysmal nocturnal hemoglobinuria | FDA accepted and granted priority review designation for NDA; PDUFA date is May 14, 2021 | 11/17/20 | Hematologic |
| Aruvant Sciences Ltd., of New York, part of the Roivant Sciences group | ARU-1801 | Lentiviral gene therapy | Sickle cell disease | EMA granted orphan designation | 11/16/20 | Hematologic |
| Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Roctavian (valoctocogene roxaparvovec) | F8 gene stimulator | Factor VIII deficiency | Subsidiary Biomarin International Ltd. withdrew MAA for treatment of severe hemophilia A; received FDA CRL in August | 11/13/20 | Hematologic |
| Bluebird Bio Inc., of Cambridge, Mass. | Lentiglobin (betibeglogene autotemcel) | HBB gene stimulator | Sickle cell disease | FDA requested use of drug product manufactured from SCD patient cells in addition to healthy donors as well as commercial lentiviral vector to show comparability, expecting to delay BLA submission to late 2022 | 11/4/20 | Hematologic |
| CSL Behring, unit of CSL Ltd., of Melbourne, Australia | CSL-889 | Hemopexin modulator | Sickle cell disease | EU and FDA granted orphan drug designation | 11/2/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | FDA revised prescribing information; updated label includes statement that treatment benefit was observed irrespective of hydroxyurea usage and a new step-by-step instruction for use section | 11/3/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | Submitted temporary license application to the National Health Regulatory Authority in the Kingdom of Bahrain | 11/10/20 | Hematologic |
| Forma Therapeutics Inc., of Watertown, Mass. | FT-4202 | Selective red blood cell pyruvate kinase-R activator | Sickle cell disease | European Commission granted orphan designation | 11/10/20 | Hematologic |
| Rigel Pharmaceuticals Inc., of South San Francisco, and Medison Pharma Ltd., of Petach Tikva, Israel | Tavalisse (fostamatinib disodium hexahydrate) | Syk tyrosine kinase inhibitor | Thrombocytopenia | Approved by Health Canada for use in adults with chronic immune thrombocytopenia who have had insufficient response to other treatments | 11/23/20 | Hematologic |
| Sanofi SA, of Paris | Rilzabrutinib | Oral BTK inhibitor | Immune thrombocytopenia | FDA granted fast track status | 11/18/20 | Hematologic |
| Alvotech hf, of Reykjavik, Iceland, and Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | AVT-02 | Biosimilar of Humira (adalimumab) | Rheumatoid arthritis, plaque psoriasis, Crohn's disease, ulcerative colitis and others | Submitted BLA to the FDA | 11/19/20 | Immune |
| Ars Pharmaceuticals, of San Diego, and Recordati SpA, of Milan | Neffy (epinephrine) | Adrenaline nasal spray | Severe allergic reactions, including anaphylaxis | EMA accepted the MAA | 11/30/20 | Immune |
| Bioventus Inc., of Durham, N.C. | PTP-001 | Placental tissue particulate comprising amnion, chorion and umbilical cord from full-term, healthy births and provided sterile in micronized form | Osteoarthritis of the knee | FDA cleared IND for phase I study | 11/18/20 | Immune |
| DBV Technologies SA, of Montrouge, France | Viaskin Peanut (DBV-712) | Peanut allergy desensitization vaccine | Peanut hypersensitivity | EMA validated MAA to treat children 4 to 11 years old | 11/2/20 | Immune |
| Immupharma plc., of London | Lupuzor | Modulates activation of autoreactive T cells | Systemic lupus erythematosus | FDA scheduled a type A meeting for 12/4/20 to discuss the design of a phase III study and the potential for a conditional approval before the phase III study is completed | 11/20/20 | Immune |
| Innocare Pharma Ltd., of Beijing | Orelabrutinib | BTK inhibitor | Multiple sclerosis | FDA cleared IND for phase II trial in 160 people with relapsing-remitting disease | 11/2/20 | Immune |
| Innovent Biologics Inc., of San Francisco, and Suzhou, China | Sulinno (adalimumab biosimilar) | Anti-TNF-alpha monoclonal antibody | Polyarticular juvenile idiopathic arthritis | Approved by China’s NMPA | 11/23/20 | Immune |
| Janssen Pharmaceutical Cos., of Beerse, Belgium, part of Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting IL-23 | Active psoriatic arthritis | Approved by European Commission for use in adults who have had an inadequate response or who have been intolerant to a prior disease-modifying antirheumatic drug therapy | 11/25/20 | Immune |
| Kadmon Inc., of New York | Belumosudil (KD-025) | Rho-associated coiled-coil kinase 2 inhibitor | Chronic graft-vs.-host disease | FDA accepted the NDA with a priority review; PDUFA target action date is May 30, 2021 | 11/30/20 | Immune |
| Mustang Bio Inc., of Worcester, Mass. | MB-107 and MB-207 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | European Commission issued positive opinion on orphan designation, which applies to MB-107 for the treatment of newly diagnosed infants between 2 months and 2 years of age and to MB-207 in patients who have been previously treated with hematopoietic stem cell transplantation and for whom retreatment is indicated | 11/24/20 | Immune |
| Swedish Orphan Biovitrum AB, of Stockholm | Gamifant (emapalumab) | Interferon gamma ligand inhibitor | Hemophagocytic lymphohistiocytosis | EMA's CHMP re-examined initial negative opinion confirmed its refusal to recommend granting MAA | 11/13/20 | Immune |
| Vifor Fresenius Medical Care Renal Pharma Ltd., of St. Gallen, Switzerland, and Chemocentryx Inc., of Mountain View, Calif. | Avacopan | Complement 5a receptor inhibitor | ANCA-associated vasculitis | EMA validated the MAA | 11/3/20 | Immune |
| Adaptive Phage Therapeutics Inc., of Gaithersburg, Md. | Phagebank | Antibacterial | Pneumonia or bacteremia/septicemia | FDA cleared the expanded access IND for treating pneumonia or bacteremia/septicemia due to Acinetobacter baumannii, Pseudomonas aeruginosa or Staphylococcus aureus in COVID-19 patients | 11/18/20 | Infection |
| Altimmune Inc., of Gaithersburg, Md. | Adcovid | Intranasal COVID-19 vaccine | COVID-19 | Submitted IND to FDA to start phase I trial of single-dose candidate | 11/25/20 | Infection |
| Appili Therapeutics Inc., of Halifax, Nova Scotia | Avigan tablets (favipiravir) | Broad-spectrum antiviral | COVID-19 | Health Canada cleared start of phase III post-exposure prophylaxis study | 11/24/20 | Infection |
| Arca Biopharma Inc., of Westminster, Colo. | AB-201 | Selective inhibitor of tissue factor | COVID-19 | FDA granted fast track designation | 11/23/20 | Infection |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland, and Pfizer Inc., of New York | Cresemba (isavuconazole) | Antifungal | Invasive aspergillosis | MAA submitted by Pfizer was accepted for review by China’s NMPA | 11/25/20 | Infection |
| Basilea Pharmaceutica Ltd., of Basel, Switzerland, and Shenzhen China Resources Gosun Pharmaceuticals Co. Ltd., of Shenzhen, China | Zevtera (ceftobiprole) | Cephalosporin antibiotic | Community- and hospital-acquired pneumonia | China's National Medical Products Administration approved the drug | 11/6/20 | Infection |
| Bavarian Nordic A/S, of Copenhagen, Denmark | Imvamune | Non-replicating smallpox vaccine | Smallpox | Health Canada expanded approval to include additional indications, specifically monkeypox and related orthopoxvirus infections and disease in adults 18 and older determined to be at high risk for exposure | 11/12/20 | Infection |
| Betterlife Pharma Inc., of Vancouver, British Columbia | AP-003 | Inhalation-based interferon alpha 2b formula | COVID-19 | Meeting with FDA provided guidance for IND filing to start clinical trials in U.S. | 11/10/20 | Infection |
| Cormedix Inc., of Berkeley Heights, N.J. | Defencath | Antibacterial and antifungal solution | Catheter-related bloodstream infections | FDA decided to cancel Antimicrobial Drug Advisory Committee meeting tentatively scheduled for Jan. 14, 2021, to discuss the NDA seeking use of the product in hemodialysis patients; PDUFA date remains Feb. 28, 2021 | 11/18/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Leronlimab (PRO-140) | CCR5 antagonist | COVID-19 infection | Protocol filed with FDA to conduct phase II trial in 102 people without detectable virus but with so-called long-hauler symptoms | 11/17/20 | Infection |
| Cytodyn Inc., of Vancouver, Washington | Leronlimab (PRO-140) | CCR5 antagonist | HIV | Health Canada cleared firm to file BLA as combination therapy for multidrug-resistant HIV | 11/16/20 | Infection |
| Eli Lilly and Co., of Indianapolis | Bamlanivimab (LY-CoV555) | Antibody | COVID-19 | FDA granted emergency use authorization for treatment of mild to moderate COVID-19 in adults and pediatric patients, 12 and older, with a positive COVID-19 test, who are at risk for progressing to severe COVID-19 and/or hospitalization | 11/9/20 | Infection |
| Eli Lilly and Co., of Indianapolis | Baricitinib | Monoclonal antibody targeting SARS-CoV-2 | COVID-19 patients who require supplemental oxygen, invasive mechanical ventilation or extracorporeal membrane oxygenation | FDA issued an emergency use authorization | 11/19/20 | Infection |
| Eli Lilly and Co., of Indianapolis | Bamlanivimab (LY-CoV555) | Antibody | COVID-19 | Health Canada granted authorization under Interim Order Respecting the Importation, Sale and Advertising of Drugs for Use in Relation to COVID-19 for use as a treatment for adults and pediatric patients 12 or older with mild to moderate COVID-19 who weigh at least 40 kg and are at high risk of progressing to severe COVID-19 illness and/or hospitalization | 11/20/20 | Infection |
| Enlivex Therapeutics Ltd., of Nes Ziona, Israel | Allocetra | Autologous cell-based therapy | Sepsis | Israeli Ministry of Health authorized the start of a 160-patient phase IIb study; co-primary endpoints are change from baseline in Sequential Organ Failure Assessment score over a period of 28 days and number and severity of adverse events and serious adverse events | 11/3/20 | Infection |
| Genentech, of South San Francisco, part of the Roche Group | Xofluza (baloxavir marboxil) | Endonuclease inhibitor | Influenza | FDA expanded approved indication to include post-exposure prevention of flu in patients 12 and older after contact with an individual who has the flu | 11/23/20 | Infection |
| Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Antiviral | COVID-19 | The WHO Guideline Development Group panel advises against use of the drug due to lack of evidence that it has an effect on mortality, the need for mechanical ventilation or time to clinical improvement | 11/19/20 | Infection |
| Innovation Pharmaceuticals Inc., of Wakefield, Mass. | Brilacidin | Dual PDE3/PDE4 inhibitor | COVID-19 infection | FDA provided written feedback on development plan for phase II trial in people hospitalized with infection, completing pre-IND process | 11/2/20 | Infection |
| Innovation Pharmaceuticals Inc., of Wakefield, Mass. | Brilacidin | Defensin mimetic | COVID-19 | Overseas CTA submitted to governing health agency for phase II study, expected to begin in 2020 | 11/16/20 | Infection |
| Innovation Pharmaceuticals Inc., of Wakefield, Mass. | Brilacidin | Defensin mimetic | Hospitalized COVID-19 | Filed documentation with regulatory agencies, including an IND with the FDA, for conduct of a multinational phase II study | 11/30/20 | Infection |
| Inovio, of Plymouth Meeting, Pa. | INO-4800 | Nucleic-acid based vaccine | COVID-19 prophylaxis | FDA cleared the phase II/III Innovate study comparing 2 dose levels of the vaccine to placebo to determine the best dose(s) to study in the phase III portion | 11/16/20 | Infection |
| Iterum Therapeutics plc., of Dublin | Oral sulopenem | Penem anti-infective | Uncomplicated urinary tract infections in patients with a quinolone non-susceptible pathogen | Submitted an NDA to the FDA | 11/30/20 | Infection |
| Janssen Cilag Pty. Ltd., part of New Brunswick, N.J.-based Johnson & Johnson | Ad26.COV.S | Vaccine | COVID-19 | Australia’s TGT granted provisional determination; the company is now eligible to apply for provisional registration for the vaccine in the Australian Register of Therapeutic Goods | 11/16/20 | Infection |
| Lupin Pharmaceuticals Inc., of Mumbai, India | Solosec (secnidazole) | Antibacterial | Trichomoniasis | FDA accepted supplemental NDA seeking approval for use in adults and adolescents; PDUFA date of June 30, 2021 | 11/16/20 | Infection |
| Merck & Co. Inc., of Kenilworth, N.J. | V-114 | 15-valent pneumococcal conjugate vaccine | Pneumococcal disease | Submitted applications to FDA and EMA seeking approval for use in adults 18 and older | 11/23/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | COVID-19 Spike glycoprotein modulator | COVID-19 infection | Swissmedic started a rolling review | 11/13/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | mRNA vaccine | COVID-19 | EMA’s Committee for Medicinal Products for Human Use started a rolling review following confirmation of eligibility for submission on Oct. 14, 2020 | 11/17/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1273 | COVID-19 spike glycoprotein modulator | COVID-19 infection | FDA scheduled Vaccines and Related Biological Products advisory committee meeting on Dec. 17, 2020, to discuss request for emergency use authorization | 11/30/20 | Infection |
| Mycovia Pharmaceuticals Inc., of Durham, N.C. | Oteseconazole (VT-1161) | Lanosterol-14 demethylase inhibitor; oral antifungal | Recurrent vulvovaginal candidiasis | Completed pre-NDA meeting with FDA | 11/12/20 | Infection |
| Novavax Inc., of Gaithersburg, Md. | NVX-CoV2373 | Vaccine | COVID-19 | FDA granted fast track designation | 11/9/20 | Infection |
| Pfizer Inc., of New York and Biontech SE, of Mainz, Germany | BNT-162b2 | mRNA-based vaccine targeting SARS-CoV-2 | COVID-19 prophylaxis | Submitted application for EUA to the FDA on 11/20/20; rolling submissions already initiated in Australia, Canada, Europe, Japan and the U.K. | 11/20/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | Vaccine | COVID-19 | FDA scheduled meeting of Vaccines and Related Biological Products Advisory Committee on Dec. 10, 2020, to discuss the request for emergency use authorization | 11/23/20 | Infection |
| Polypid Ltd., of Petah Tikva, Israel | D-Plex | Prolonged and constant release of broad-spectrum antibiotic doxycycline | Surgical site infections | FDA granted breakthrough therapy designation for prevention of SSIs in patients undergoing elective colorectal surgery | 11/24/20 | Infection |
| Redhill Biopharma Ltd., of Tel Aviv, Israel | RHB-107 (upamostat) | Antiviral | COVID-19 | FDA cleared the IND for phase II/III study in patients with symptomatic COVID-19 who do not require hospitalization | 11/17/20 | Infection |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGN-CoV2 (casirivimab and imdevimab) | COVID-19 Spike glycoprotein inhibitor | COVID-19 | FDA issued emergency use authorization for combination to treat mild to moderate COVID-19 in adults and pediatric patients 12 and older weighing at least 40 kg, with positive results of direct SARS-CoV-2 viral testing and who are at high risk of progressing to severe COVID-19 | 11/21/20 | Infection |
| Roche Holding AG, of Basel, Switzerland and Shionogi & Co. Ltd., of Osaka, Japan | Xofluza (baloxavir marboxil) | Endonuclease inhibitor | Influenza virus infection | EMA's CHMP recommended approval of MAA for the treatment of uncomplicated influenza in patients aged 12 years and above | 11/13/20 | Infection |
| Sanofi SA, of Paris | Sutimlimab | Monoclonal antibody | Hemolysis in adults with cold agglutinin disease | FDA issued a complete response letter regarding BLA, referring to deficiencies identified during a pre-license inspection of a third-party facility responsible for manufacturing; no clinical or safety deficiencies noted | 11/14/20 | Infection |
| Sanofi SA, of Paris | Supemtek | Quadrivalent (4-strain) recombinant influenza vaccine | Influenza | Approved by European Commission for prevention of influenza in adults 18 and older | 11/18/20 | Infection |
| Sanofi SA, of Paris | Menquadfi (quadrivalent meningococcal tetanus toxoid conjugate vaccine) | Protein subunit vaccine | Invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, W and Y | Approved by European Commission for active immunization of individuals from the age of 12 months and older | 11/23/20 | Infection |
| Senhwa Biosciences Inc., of Taipei | Silmitasertib | Casein kinase 2 inhibitor | Moderate COVID-19 | FDA issued a study may proceed letter for a 20-patient investigator-initiated phase II study | 11/6/20 | Infection |
| Senhwa Biosciences Inc., of Taipei, Taiwan | Silmitasertib | Casein kinase II inhibitor | COVID-19 infection | IND submitted to FDA for phase II trial in individuals with severe infection | 11/2/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | COVI-AMG (STI-2020) | COVID-19 Spike glycoprotein inhibitor | COVID-19 | Filed IND to test in healthy volunteers and in patients with mild symptoms | 11/9/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | Covi-Drops (STI-2099) | Neutralizing antibody against SARS-CoV-2 | Mild COVID-19 | Filed an IND with the FDA | 11/11/20 | Infection |
| Taigen Biotechnology Co. Ltd., of Taipei, Taiwan | TG-1000 | Pan-influenza antiviral | Influenza A and B infection | FDA approved IND for phase I trial | 11/1/20 | Infection |
| Valeo Pharma Inc., of Montreal | Amikacin | Antibiotic | Infection | Received notice of compliance from Health Canada granting marketing authorization | 11/12/20 | Infection |
| Vasomune Therapeutics Inc., of Toronto | AV-001 | Tie2 tyrosine kinase receptor agonist | COVID-19 | FDA allowed the IND for clinical testing in hospitalized patients diagnosed with moderate to severe COVID-19 | 11/12/20 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | 3-antigen prophylactic hepatitis B vaccine | 3-antigen prophylactic hepatitis B vaccine | Prevention of hepatitis B virus | Submitted MAA to EMA seeking approval for prevention of infection caused by all known subtypes of HBV in adults | 11/23/20 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Penn. | VP-102 (cantharidin, topical film-forming solution) | Protein phosphatase 2A inhibitor | Molluscum contagiosum | Received final meeting nots from FDA type A meeting to discuss resubmission of NDA; company reaffirmed expectations to resubmit NDA in first quarter of 2021 | 11/17/20 | Infection |
| Viatris Inc., of Pittsburgh | Pediatric dolutegravir tablets for oral suspension, 10 mg | HIV-1 integrase inhibitor | HIV/AIDS | Received tentative approval from FDA for formulation to help expand access to children living with HIV/AIDS in low- and middle-income countries; NDA granted under the U.S. President's Emergency Plan for AIDS Relief, which permits products that are not approved for marketing in the U.S. because of patent protection or other marketing restrictions to be distributed in other countries where needed | 11/23/20 | Infection |
| Viiv Healthcare Ltd., of London | Tivicay (dolutegravir) | HIV-1 integrase inhibitor | HIV-1 infection | EMA's CHMP recommended new form an strength together with new indication: combination with other anti-retroviral medicinal products for the treatment HIV infected adults, adolescents and children of at least 4 weeks of age or older and weighing at least 3 kg | 11/13/20 | Infection |
| Viiv Healthcare Ltd., of London | Cabotegravir | HIV integrase inhibitor | HIV pre-exposure prophylaxis | FDA granted breakthrough therapy designation for long-acting injectable cabotegravir | 11/17/20 | Infection |
| Liminal Biosciences Inc., of Laval, Quebec | Ryplazim (plasminogen) | Plasmin stimulator | Congenital plasminogen deficiency | FDA extended review period for BLA by 3 months, setting new PDUFA date of June 5, 2021 | 11/8/20 | Inflammatory |
| Amo Pharma Ltd., of London | AMO-02 | Glycogen synthase kinase-3 beta inhibitor | Congenital myotonic dystrophy | FDA granted rare pediatric disease designation | 11/9/20 | Musculoskeletal |
| Beigene Co., Ltd., of Beijing and Cambridge, Mass., and Amgen Inc., of Thousand Oaks, Calif. | Xgeva (denosumab) | Antibody targeting RANKL | Prevention of skeletal-related events in patients with bone metastases from solid tumors and in patients with multiple myeloma | China National Medical Products Administration approved the drug | 11/20/20 | Musculoskeletal |
| Biomarin Pharmaceutical Inc., of San Rafael | Vosoritide | C-type natriuretic peptide | Achondroplasia | FDA accepted the NDA and assigned a PDUFA action date of Aug. 20, 2021; agency doesn't plan to hold an advisory committee meeting | 11/2/20 | Musculoskeletal |
| Brainever SAS, of Paris | BREN-02 | Recombinant human homeoprotein Engrailed 1 | Amyotrophic lateral sclerosis | FDA granted orphan designation | 11/23/20 | Musculoskeletal |
| Sarepta Therapeutics Inc., of Cambridge, Mass. | SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) | Gene therapy expressing micro-dystrophin | Duchenne muscular dystrophy | After a type C meeting with the FDA, company plans to start the SRP-9001-103 study in 10 patients using the commercial process material later this year | 11/5/20 | Musculoskeletal |
| Seelos Therapeutics Inc., of New York | SLS-005 | Transcription factor EB stimulator | Amyotrophic lateral sclerosis | FDA granted orphan drug designation | 11/19/20 | Musculoskeletal |
| Alkermes plc, of Dublin | ALKS-3831 (olanzapine + samidorphan) | Atypical antipsychotic + opioid receptor modulator | Schizophrenia; bipolar I disorder | FDA issued complete response letter, citing need to resolve conditions related to tablet coating process at manufacturing facility | 11/17/20 | Neurology/Psychiatric |
| Alzecure Pharma AB, of Stockholm | ACD-856 | NGF receptor modulator; TrkA receptor modulator; TrkB receptor modulator; brain-derived neurotrophic factor ligand modulator | Alzheimer’s disease | Received approval from regulatory authorities in Sweden to start phase I trial | 11/12/20 | Neurology/Psychiatric |
| Aquestive Therapeutics Inc., of Warren, N.J. | Libervant (diazepam) | Buccal formulation of diazepam, a benzodiazepine | Seizure clusters | At a type A meeting, the FDA confirmed that the issues identified in the complete response letter may be addressed by utilizing modeling and simulations; FDA recommended a follow-up meeting to review the final modeling and simulations, which the company plans to schedule | 11/18/20 | Neurology/Psychiatric |
| Astrazeneca plc, of Cambridge, U.K. | Brilinta (ticagrelor) | P2Y12 receptor antagonist | Acute ischemic stroke | FDA approved the drug to reduce the risk of stroke | 11/6/20 | Neurology/Psychiatric |
| Axovant Gene Therapies Ltd., of New York | AXO-AAV-GM2 | Gene therapy | Tay-Sachs disease and Sandhoff disease | FDA lifted clinical hold and cleared IND to initiate registrational study | 11/9/20 | Neurology/Psychiatric |
| Bioxcel Inc., of New Haven, Conn. | BXCL-501 | Sublingual thin film formulation of dexmedetomidine | Agitation in patients with schizophrenia and bipolar disorders | After completing the pre-NDA meeting with the FDA, the company believes its regulatory data package will be sufficient for submission of the NDA; plans to complete rolling submission in the first quarter of 2021 | 11/11/20 | Neurology/Psychiatric |
| Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores the normal shape and function of altered filamin A | Alzheimer's disease | World Health Organization advised the U.S. Adopted Names Council to modify the chemical name of the company’s lead drug candidate to simufilam due to a potential trademark conflict with a drug marketed in the Far East | 11/27/20 | Neurology/Psychiatric |
| Eisai Co. Ltd., of Tokyo | Fycompa (perampanel) | Selective, noncompetitive AMPA receptor antagonist | Partial-onset seizures | Approved by European Commission for pediatric patients, extending use as adjunctive therapy for POS, with or without secondary generalization, by expanding approved age range from 12 and older to 4 and older | 11/16/20 | Neurology/Psychiatric |
| Heron Therapeutics Inc., of San Diego | HTX-011 (bupivacaine + meloxicam) | Cyclooxygenase 2 inhibitor; sodium channel inhibitor | Pain | Resubmitted NDA following June CRL; class 2 resubmission | 11/13/20 | Neurology/Psychiatric |
| Impel Neuropharma Inc., of Seattle | INP-104 (dihydroergotamine mesylate) | 5-HT 1d receptor agonist; 5-HT 1f receptor agonist; 5-HT 1b receptor agonist | Migraine | Submitted NDA to FDA for use in acute treatment of migraine with or without aura in adults | 11/9/20 | Neurology/Psychiatric |
| Janssen Pharmaceutical Cos. of Johnson & Johnson, of New Brunswick, N.J. | Paliperidone palmitate 6-month | 5-HT receptor antagonist; dopamine D2 receptor antagonist | Schizophrenia | Supplemental NDA submitted to FDA for twice-yearly dosing in adults | 11/2/20 | Neurology/Psychiatric |
| Mindmed Inc., of New York | Microdoses of LSD | Psychedelic | Attention deficit hyperactivity disorder | Received positive response on its protocol design for phase IIa trial in adults from Swiss and Dutch health authorities | 11/16/20 | Neurology/Psychiatric |
| Mindmed Inc., of New York | Microdoses of LSD | Psychedelic | Anxiety disorders | Submitted pre-IND briefing package to FDA for potential phase IIb trial | 11/16/20 | Neurology/Psychiatric |
| Neuraly Inc., of Gaithersburg, Md. | NLY-01 (exenatide pegylated, subcutaneous) | GLP-1R agonist | Alzheimer's disease | FDA cleared IND for phase IIb trial in 500+ participants with mild cognitive impairment due to AD | 11/2/20 | Neurology/Psychiatric |
| Neurelis Inc., of San Diego | NRL-4 | Nasal formulation; unspecified drug target | Acute agitation | Concluded pre-IND meeting with FDA | 11/23/20 | Neurology/Psychiatric |
| Neuronasal Inc., of Wexford, Pa. | NAC | N-acetylcysteine | Acute mild traumatic brain injury | Granted IND clearance by FDA to continue plans for a phase I study testing nose-to-brain delivery of NAC in partnership with Atai Life Sciences | 11/12/20 | Neurology/Psychiatric |
| Orchard Therapeutics Ltd., of London | OTL-200 | ARSA gene stimulator | Metachromatic leukodystrophy | FDA cleared the IND | 11/19/20 | Neurology/Psychiatric |
| Oryzon Genomics SA, of Madrid, Spain | Vafidemstat | Oral, CNS optimized LSD1 inhibitor | Borderline personality disorder | Received approval from Spanish Drug Agency (AEMPS) for CTA to start phase IIb trial | 11/10/20 | Neurology/Psychiatric |
| Praxis Precision Medicines Inc., of Cambridge, Mass. | PRAX-114 | GABA A receptor modulator | Major depressive disorder | Received a response from the FDA on its IND submission seeking to start a phase II/III trial, with the agency placing the IND on full clinical hold; Praxis expects to receive final comments from FDA within 30 days | 11/10/20 | Neurology/Psychiatric |
| Praxis Precision Medicines Inc., of Cambridge, Mass. | PRAX-114 | GABAA receptor PAM | Major depressive disorder | Received comments from FDA on full clinical hold of IND submission; agency proposed company conduct further toxicological investigation of the effect of PRAX-114 and its metabolites on fertility, reproduction and embryofetal development to support the planned trial; company believes results of ongoing standard fertility and reproductive studies, expected to be completed in first quarter of 2021, together with available toxicology package will satisfy the FDA request | 11/17/20 | Neurology/Psychiatric |
| Prevail Therapeutics Inc., of New York | PR-006 | AAV9 gene therapy expressing GRN | Frontotemporal dementia | European Commission granted orphan designation | 11/30/20 | Neurology/Psychiatric |
| Reata Pharmaceuticals Inc., of Plano, Texas | Omaveloxolone | Oral, once-daily activator of Nrf2 | Friedreich’s ataxia | FDA completed internal review of baseline-controlled study results and concluded results do not strengthen the results of part 2 of the Moxie study; FDA proposed some additional exploratory analyses using patients randomized to placebo during the Moxie part 2 study, but stated that the potential for those analyses to strengthen the study results was questionable due to the small number of patients available for analysis; company plans to submit to FDA analyses and request meeting to discuss development program; company also is considering next steps, including whether to conduct a second pivotal study | 11/24/20 | Neurology/Psychiatric |
| Sedana Medical AB, of Stockholm | Sedaconda (isoflurane) | General anesthetic | Sedation | Submitted marketing application to German Medicines Agency BfArM and a number of other agencies through the decentralized procedure | 11/27/20 | Neurology/Psychiatric |
| Sedor Pharmaceuticals LLC, of Paoli, Pa. | Sesquient (fosphenytoin sodium for injection) | Captisol-enabled Fosphenytoin | Status epilepticus in adult and pediatric patients | FDA approved | 11/6/20 | Neurology/Psychiatric |
| Supernus Pharmaceuticals Inc., of Rockville, Md. | SPN-812 (viloxazine hydrochloride) | Psychomodulator; norepinephrine uptake inhibitor | Attention-deficit hyperactivity disorder | FDA issued a complete response letter to NDA seeking approval for use in pediatric patients, ages 6-17; primary issue cited relates to company’s in-house laboratory that conducts analytical testing, which recently moved to a new location; no clinical safety or efficacy issues were identified | 11/9/20 | Neurology/Psychiatric |
| Supernus Pharmaceuticals Inc., of Rockville, Md. | SPN-830 (apomorphine infusion pump) | Dopamine D2 receptor agonist | Parkinson’s disease | Received a refuse-to-file letter from FDA regarding NDA seeking approval for continuous treatment of motor fluctuations in Parkinson’s disease; agency determined the NDA was not sufficiently complete to permit a substantive review; company plans to seek guidance from FDA, including type A meeting | 11/9/20 | Neurology/Psychiatric |
| UCB SA, of Brussels, Belgium | Xyrem (sodium oxybate) | GABA B receptor agonist | Narcolepsy; cataplexy | EMA's CHMP recommended extending indication to include treatment of narcolepsy with cataplexy in adult patients, adolescents and children from the age of 7 years | 11/13/20 | Neurology/Psychiatric |
| UCB SA, of Brussels, Belgium | Vimpat (lacosamide) | Dihydropyrimidinase related protein 2 modulator; sodium channel modulator | Primary generalized tonic-clonic seizures | FDA approved Vimpat CV for use as adjunctive therapy in patients 4 and older and Vimpat injection for intravenous use in children 4 and older | 11/17/20 | Neurology/Psychiatric |
| Aerie Pharmaceuticals Inc., of Durham, N.C. | Roclanda (latanoprost + netarsudil mesylate) | Rho associated protein kinase inhibitor; PGF2 alpha agonist | Glaucoma; ocular hypertension | EMA's CHMP recommended approval of MAA for reduction of elevated intraocular pressure in adults with primary open-angle glaucoma or ocular hypertension not helped by monotherapy; EC decision expected early next year | 11/13/20 | Ocular |
| Gensight Biologics SA, of Paris | Lumevoq (endogene nolparvovec) | Gene therapy using a mitochondrial targeting sequence | Leber hereditary optic neuropathy | EMA validated the MAA | 11/3/20 | Ocular |
| Gyroscope Therapeutics Ltd., of London | GT-005 | Complement factor stimulator | Geographic atrophy in patients with variants in their complement genes | FDA granted fast track designation | 11/11/20 | Ocular |
| Samsung Bioepis Co. Ltd., of Incheon, South Korea, and Biogen Inc., of Cambridge, Mass. | SB-11 | Biosimilar of Lucentis (ranibizumab); anti-VEGF therapy | Retinal vascular disorders | FDA accepted for review BLA | 11/18/20 | Ocular |
| Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 | Designed to reduce neuronal death and dysfunction | Wolfram syndrome | FDA granted orphan designation | 11/16/20 | Other/Miscellaneous |
| Artelo Biosciences Inc., of La Jolla, Calif. | ART-27.13 | Synthetic cannabinoid | Cancer anorexia and weight loss | Received clinical trial authorization in the U.K. for Cares phase I/II study; enrollment expected to initiate before year-end | 11/16/20 | Other/Miscellaneous |
| Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Zokinvy (lonafarnib) | Blocks the farnesylation of progerin | Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies | Approved by FDA; agency also issued rare pediatric disease priority review voucher | 11/20/20 | Other/Miscellaneous |
| Neonmind Biosciences Inc., a subsidiary of Better Plant Sciences Inc., of Vancouver, British Columbia | Psilocybin | 5-HT receptor agonist | Weight loss | Received Health Canada approval to advance its preclinical trial on psilocybin-based research | 11/12/20 | Other/Miscellaneous |
| Otonomy Inc., of San Diego | Otividex | Sustained-exposure formulation of dexamethasone | Ménière’s disease | FDA confirmed use of the negative binomial model for analysis of the endpoint for the ongoing phase III study | 11/30/20 | Other/Miscellaneous |
| Revance Therapeutics Inc., of Newark, Calif. | DaxibotulinumtoxinA for injection | Botulinum toxin A stimulator | Moderate to severe glabellar lines | FDA deferred decision on BLA, reiterating that inspection of manufacturing facility is required for BLA approval process; inspection was unable to be completed due COVID-19-related travel restrictions; company confirmed FDA communication was not a complete response letter | 11/25/20 | Other/Miscellaneous |
| Chiesi Farmaceutici SpA, of Parma, Italy | Trimbow (beclometasone / formoterol / glycopyrronium bromide) | Beta 2 adrenoceptor agonist; muscarinic receptor antagonist; glucocorticoid receptor agonist | Asthma | EMA's CHMP recommended new strength and new indication: maintenance treatment of asthma | 11/13/20 | Respiratory |
| Insmed Inc., of Bridgewater, N.J. | Brensocatib | Oral, reversible inhibitor of DPP1 | Non-cystic fibrosis bronchiectasis | EMA granted priority medicines (PRIME) designation | 11/13/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | CFTR modulator | Cystic fibrosis | European Commission approved label extension to treat infants 4 months and older who weigh at least 5 kg and have R117H mutation or certain gating mutations in CFTR gene | 11/5/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Symkevi (tezacaftor/ivacaftor) plus Kalydeco (ivacaftor) | CFTR corrector/CFTR regulator | Cystic fibrosis | European Commission approved the label extension to include patients ages 6 and older who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or 1 copy of the F508del mutation and 1 copy of 1 of 14 mutations in the CFTR gene that result in residual CFTR activity | 11/27/20 | Respiratory |
| Agenus Inc., of Lexington, Mass. | Balstilimab | PD-1 inhibitor | Recurrent/metastatic cervical cancer | Due to 2 newly identified late responses in clinical trial, completion of BLA filing planned for first half of 2021; Agenus also is working with FDA to clarify diagnostic requirements for PD-L1 testing | 12/3/20 | Cancer |
| Aim Immunotech Inc., of Ocala, Fla. | Ampligen (rintatolimod) | TLR-3 agonist; interferon type I receptor agonist; ribonuclease stimulator; 2,5-oligoadenylate synthetase stimulator | Pancreatic cancer | FDA granted orphan designation | 12/21/20 | Cancer |
| Alligator Bioscience AB, of Lund, Sweden | Mitazalimab | CD40-targeting antibody | Pancreatic cancer | IND approved | 12/4/20 | Cancer |
| Alligator Bioscience AB, of Lund, Sweden | Mitazalimab | Monoclonal antibody targeting CD40 | Metastatic pancreatic cancer | Submitted a CTA to the relevant European regulatory authorities for the 66-patient Optimize phase II study testing mitazalimab in combination with the chemotherapy mFolfirinox; study expected to start in the first half of 2021 | 12/28/20 | Cancer |
| Allogene Therapeutics Inc., of South San Francisco | ALLO-316 | CD70 antigen modulator | Renal cell carcinoma | FDA cleared IND; phase I study set to start in 2021 | 12/6/20 | Cancer |
| Allogene Therapeutics Inc., of South San Francisco | ALLO-715 | BCMA AlloCAR T therapy | Relapsed/refractory multiple myeloma | FDA cleared IND for study testing combination with nirogacestat (Springworks Therapeutics Inc.); enrollment in that cohort is expected to begin in first quarter of 2021 | 12/23/20 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Sotorasib | KRASG12C inhibitor | Non-small-cell lung cancer | FDA granted breakthrough therapy designation | 12/8/20 | Cancer |
| Amgen Inc., of Thousand Oaks, Calif. | Sotorasib | KRAS-G12C inhibitor | Non-small-cell lung cancer | MAA submitted to EMA to treat adults with previously treated KRAS G12C-mutated locally advanced or metastatic disease | 12/22/20 | Cancer |
| Anixa Biosciences Inc., of San Jose, Calif. | Breast cancer vaccine | Breast cancer vaccine | Triple-negative breast cancer | FDA cleared IND | 12/21/20 | Cancer |
| Antengene Corp. Ltd., of Shanghai and Hong Kong | Xpovio (selinexor, ATG-010) | Nuclear export protein exportin 1 inhibitor | Multiple myeloma and diffuse large B-cell lymphoma | Submitted NDAs to Health Sciences Authority of Singapore and to Australia’s TGA for 3 indications: in adults with relapsed/refractory MM who have received at least 4 prior therapies and whose disease is refractory to at least 2 proteasome inhibitors, at least 2 immunomodulatory agents and an anti-CD38 monoclonal antibody; in combination with bortezomib and dexamethasone for the treatment of patients with MM who have received at least 1 prior line of therapy; and for the treatment of adults with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy | 12/3/20 | Cancer |
| Antengene Corp. Ltd., of Shanghai and Hong Kong | ATG-010 (selinexor) | Nuclear export protein exportin 1 inhibitor | Relapsed/refractory diffuse large B-cell lymphoma | China’s NMPA accepted IND for a phase II/III trial testing combination with R-GDP | 12/6/20 | Cancer |
| Antengene Ltd., of Shanghai | ATG-010 (selinexor) | Selective inhibitor of nuclear export | Relapsed/refractory multiple myeloma | China's NMPA accepted the IND for a 150-patient phase III study testing selinexor in combination with bortezomib and dexamethasone | 12/11/20 | Cancer |
| Artiva Biotherapeutics Inc., of San Diego | AB-101 | Optimized and cryopreserved off-the-shelf NK cell therapy | Relapsed/refractory B-cell non-Hodgkin lymphoma | FDA cleared IND for a phase I/II study testing drug alone and in combination with rituximab in patients who have progressed beyond 2 or more prior lines of therapy | 12/7/20 | Cancer |
| Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon TLR7/8 Agonist | Long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors 7 and 8 | Cancer | Filed NDA with FDA for this product candidate which allows for intratumoral, sustained release of resiquimod over several weeks from a single administration with minimal systemic exposure | 12/31/20 | Cancer |
| Ascentage Pharma Group International, of Suzhou, China | APG-115 | MDM2-p53 inhibitor | Relapsed/refractory T-cell prolymphocytic leukemia | China’s NMPA cleared a phase IIa study testing drug as single agent or in combination with Bcl-2 inhibitor APG-2575 | 12/1/20 | Cancer |
| Astrazeneca plc, Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. | Lynparza (olaparib) | PARP inhibitor | Ovarian, prostate and pancreatic cancer | Japanese Ministry of Health, Labor and Welfare approved the 3 indications | 12/28/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K. | Tagrisso (osimertinib) | Protein tyrosine kinase inhibitor; epidermal growth factor receptor antagonist | Non-small-cell lung cancer | FDA approved for adjuvant therapy after tumor resection in patients with NSCLC whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations; review was conducted under Project Orbis | 12/18/20 | Cancer |
| Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co Ltd., of Tokyo | Trastuzumab deruxtecan | Antibody-drug conjugate targeting HER2 | Unresectable or metastatic HER2-positive breast cancer | EMA's Committee for Medicinal Products for Human Use recommended conditional approval of the drug for patients who have received 2 or more prior anti-HER2 based regimens | 12/14/20 | Cancer |
| Astrazeneca plc, of Wilmington, Del. | Imfinzi (durvalumab) | Antibody targeting PD-L1 | Locally advanced, unresectable non-small-cell lung cancer in adults whose tumors express PD-L1 on at least 1% of tumor cells | Recommended for marketing authorization by the EMA's Committee for Medicinal Products for Human Use for a less-frequent, fixed-dose use | 12/15/20 | Cancer |
| Avacta Group plc, of Cambridge, U.K. | AVA-6000 | Prodrug of the chemotherapy doxorubicin | Locally advanced or metastatic selected solid tumors | Submitted a CTA in the U.K. for a phase I study | 12/23/20 | Cancer |
| Avelas Biosciences Inc., of San Diego | Pegloprastide (AVB-620) | Fluorescent marker | Breast cancer surgery | FDA granted breakthrough therapy designation | 12/14/20 | Cancer |
| Beigene Ltd., of Beijing | Blincyto (blinatumomab) | CD3 modulator; B-lymphocyte antigen CD19 modulator | Relapsed or refractory B-cell precursor acute lymphoblastic leukemia | Approval granted in China | 12/8/20 | Cancer |
| Bellicum Pharmaceuticals Inc., of Houston | BPX-601 | GoCAR-T candidate | Metastatic pancreatic or prostate cancers | FDA placed clinical hold on patient enrollment and dosing in ongoing phase I/II trial in previously treated patients, following a death of pancreatic cancer patient; clinical investigator and Bellicum classified death as unrelated to BPX-601 and rimiducid | 12/7/20 | Cancer |
| Biocure Technology Corp., of Vancouver, British Columbia, Canada, and Pharos Vaccine Inc., of Seongnam, South Korea | CD-19 | CAR-T therapy | Relapse and refractory B-cell precursor acute lymphoblastic leukemia | Submitted IND application to the Korean Ministry of Food and Drug Safety to proceed with phase I trial | 12/30/20 | Cancer |
| Bioinvent International AB, of Lund, Sweden, and Transgene SA, of Strasbourg, France | BT-001 | Oncolytic vaccinia virus | Solid tumors | Received CTA approval in Belgium for a phase I/IIa study | 12/21/20 | Cancer |
| Bridgebio Pharma Inc. and QED Therapeutics Inc., both of Palo Alto, Calif. | Infigratinib | FGFR1-3 inhibitor | Cholangiocarcinoma | FDA accepted NDA application, granting priority review under Real-Time Oncology Review pilot program | 12/1/20 | Cancer |
| CASI Pharmaceuticals Inc., of Rockville, Md., and Juventas Cell Therapy Ltd., of Beijing | CNCT-19 | CAR T targeting CD19 | Relapsed/refractory B-cell acute lymphoblastic leukemia | Chinese Center for Drug Evaluation granted breakthrough therapy designation | 12/23/20 | Cancer |
| Cerecor Inc., of Rockville, Md. | CERC-007 | Monoclonal antibody targeting IL-18 | Relapsed or refractory multiple myeloma | IND application accepted by FDA | 12/8/20 | Cancer |
| Clovis Oncology Inc., of Boulder, Colo. | FAP-2286 | Peptide-radionuclide conjugate targeting fibroblast activation protein | Multiple tumor types | Submitted two INDs to the FDA, one for lutetium-177 labeled FAP-2286 as a therapeutic and one for gallium-68 labeled FAP-2286 as a diagnostic to detect fibroblast activation protein-positive tumors | 12/29/20 | Cancer |
| Daiichi Sankyo Co. Ltd., of Tokyo | Trastuzumab deruxtecan | Antibody-drug conjugate targeting HER2 | Unresectable or metastatic HER2 positive breast cancer | Committee for Medicinal Products for Human Use of the EMA recommended conditional approval the drug for patients who have received two or more prior anti-HER2 based regimens | 12/11/20 | Cancer |
| EMD Serono, part of Merck KGaA, of Darmstadt, Germany, and Pfizer Inc., of New York | Bavencio (avelumab) | Monoclonal antibody targeting PD-L1 | First-line maintenance of locally advanced or metastatic urothelial carcinoma | EMA's Committee for Medicinal Products for Human Use recommended approval of the drug for patients who are progression-free following platinum-based chemotherapy | 12/11/20 | Cancer |
| Engene Inc., of Boston | EG-70 | Non-viral gene therapy | Bladder cancer | Awarded fast-track status | 12/4/20 | Cancer |
| Engene Inc., of Boston | EG-70 | Nonviral gene therapy | Bacille Calmette-Guerin-unresponsive non-muscle invasive bladder cancer | FDA granted fast track designation | 12/3/20 | Cancer |
| Genentech, of South San Francisco, a member of the Roche Group | Gavreto (pralsetinib) | Metastatic RET fusion-positive non-small-cell lung cancer | RET-mutant medullary thyroid cancer | Approved by FDA for treatment of adult and pediatric patients 12 and older with advanced or metastatic disease who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory | 12/1/20 | Cancer |
| Gilead Sciences Inc., of Foster City, Calif. | Trodelvy (sacituzumab govitecan-hziy) | Trop-2-directed antibody/topoisomerase inhibitor conjugate | Breast cancer | Based on data from phase III Ascent trial, sBLA submitted to FDA for full approval to treat adults with metastatic triple negative disease who received at least 2 prior therapies | 12/10/20 | Cancer |
| Hutchison China Meditech Ltd., of Hong Kong | Surufatinib | Vascular endothelial growth factor receptor and fibroblast growth factor receptor tyrosine kinase inhibitor | Pancreatic and non-pancreatic neuroendocrine tumors | Started rolling submission of the NDA to the FDA; plans to complete the submission in the first half of 2021 | 12/28/20 | Cancer |
| Hutchison China Meditech Ltd., of Hong Kong | Surufatinib (Sulanda in China) | Vascular endothelial growth factor receptor and fibroblast growth factor receptor tyrosine kinase inhibitor | Non-pancreatic neuroendocrine tumors | Approved by National Medical Products Administration of China | 12/30/20 | Cancer |
| Immunicum AB, of Stockholm | Ilixadencel | Cell-based, off-the-shelf immune primer | Gastrointestinal stromal tumors | FDA granted fast track designation | 12/7/20 | Cancer |
| Immunicum AB, of Stockholm | Ilixadencel | Cell-based, off-the-shelf immune primer | Hepatocellular carcinoma (HCC) | FDA granted orphan drug designation based on data from a phase I/II trial in patients with unresectable and/or metastatic HCC | 12/31/20 | Cancer |
| Innocare Pharma Ltd., of Beijing | Orelabrutinib | BTK inhibitor | Relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma and relapsed/refractory mantle cell lymphoma | China National Medical Products Administration approved the 2 indications | 12/28/20 | Cancer |
| Innovent Biologics Inc., of Suzhou, China | Byvsda | Biosimilar to the anti-VEGF monoclonal antibody bevacizumab | Recurrent glioblastoma | China National Medical Products Administration approved the expanded indication | 12/28/20 | Cancer |
| IO Biotech, of Copenhagen | IO-102 and IO-103 | Indoleamine 2,3-dioxygenase derived peptide and PD-L1-derived peptide | Unresectable/metastatic melanoma | FDA granted breakthrough therapy designation in combination with anti-PD-1 monoclonal antibody | 12/15/20 | Cancer |
| ITM Isotopen Technologien München AG, of Garching, Germany | Gegant | Germanium-68/Gallium-68 generator | Various cancers | FDA issued the drug master file | 12/8/20 | Cancer |
| Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Amivantamab | Epidermal growth factor receptor and mesenchymal epithelial transition factor bispecific antibody | Metastatic non-small-cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations after failure of platinum-based chemotherapy | Submitted an MMA to the EMA | 12/28/20 | Cancer |
| Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson, and Genmab A/S, of Copenhagen | Amivantamab | Fully human EGFR and mesenchymal epithelial transition factor bispecific antibody | Non-small-cell lung cancer | Submitted BLA to FDA seeking use in treating patients with metastatic disease with EGFR exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy | 12/3/20 | Cancer |
| Janssen Pharmaceutical Cos., of Raritan, N.J., part of Johnson & Johnson, and Legend Biotech Corp., of Somerset, N.J. | Ciltacabtagene autoleucel | BCMA-directed CAR T therapy | Relapsed and/or refractory multiple myeloma | Initiated rolling BLA to FDA | 12/21/20 | Cancer |
| Jazz Pharmaceuticals plc, of Dublin | JZP-458 | Recombinant Erwinia asparaginase | Acute lymphoblastic leukemia or lymphoblastic lymphoma | Initiated submission of BLA seeking approval for use as a component in multi-agent chemotherapeutic regimen for treating adult and pediatric patients who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginase | 12/21/20 | Cancer |
| Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | Exportin 1 inhibitor | Multiple myeloma (MM) | FDA approved for use in combination with bortezomib and dexamethasone for second line treatment of adults with MM | 12/18/20 | Cancer |
| Kite, of Santa Monica, a Gilead Co. | Tecartus (KTE-X19) | Autologous, anti-CD19-transduced CD3+ cells | Relapsed or refractory mantle cell lymphoma | European Commission granted conditional marketing authorization for use in adults after 2 or more lines of systemic therapy including a BTK inhibitor | 12/16/20 | Cancer |
| Kronos Bio Inc., of San Mateo, Calif. | KB-0742 | Selective, oral inhibitor of CDK9 | Advanced solid tumors | FDA cleared IND for phase I/II study, set to start in first quarter of 2021 | 12/7/20 | Cancer |
| Lantheus Holdings Inc., of North Billerica, Mass., | Pyl (18F-DCFPyL) | Prostate-specific membrane antigen-targeted positron emission tomography imaging agent | Prostate cancer | FDA accepted the NDA for a priority review with a PDUFA target action date of May 28, 2021; agency doesn't plan to hold an advisory committee meeting | 12/9/20 | Cancer |
| Legend Biotech Corp., of Somerset, N.J. | LB-1901 | Autologous CAR T targeting CD4 | Relapsed or refractory T-cell lymphoma | FDA cleared the IND for a phase I study designed to test the safety and tolerability of LB-1901 and to determine the recommended phase II dose | 12/14/20 | Cancer |
| Macrogenics Inc., of Rockville, Md. | Margetuximab | ERBB-2 tyrosine kinase receptor inhibitor | Metastatic breast cancer | During mid-cycle communication, FDA disclosed that it is no longer planning to hold advisory committee meeting to discuss BLA, reconfirming Dec. 18, 2020, PDUFA date | 12/16/20 | Cancer |
| Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | First line metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion on the expanded indication | 12/11/20 | Cancer |
| Moleculin Biotech Inc., of Houston | WP-1066 | JAK inhibitor; C-myc binding protein inhibitor; STAT-3 inhibitor; hypoxia inducible factor-1 alpha inhibitor | Solid tumors | FDA granted rare pediatric disease designations to treat diffuse intrinsic pontine glioma, medulloblastoma and atypical teratoid rhabdoid tumor | 12/1/20 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | DNA intercalator; anticancer | Soft tissue sarcoma lung metastases | FDA cleared IND for phase Ib/II study in patients with soft tissue sarcoma that has metastasized to the lungs after first-line therapy | 12/17/20 | Cancer |
| Moleculin Biotech Inc., of Houston | Annamycin | Next-generation anthracycline | Soft tissue sarcomas | FDA granted orphan drug designation | 12/29/20 | Cancer |
| Myovant Sciences Ltd., of Basel, Switzerland | Orgovyx (relugolix) | Oral GnRH receptor antagonist | Advanced prostate cancer | Approved by FDA under priority review | 12/18/20 | Cancer |
| Nascent Biotech Inc., of San Diego | Pritumumab | Vimentin modulator | Brain cancer | FDA removed a partial clinical hold, allowing it to begin phase I trials | 12/15/20 | Cancer |
| Neoleukin Therapeutics Inc., of Seattle | NL-201 | IL-2 and IL-15 mimetic | Relapsed or refractory solid tumors | Submitted IND to the FDA and a clinical trial notification in Australia for a phase I study of up to 120 patients | 12/10/20 | Cancer |
| Oblato Inc., of Princeton, N.J., a unit of Gtreebnt Co. Ltd. | OKN-007 | Sulfatase inhibitor | Diffuse intrinsic pontine glioma | FDA gave advice in a meeting on the design of a phase I/II study, including patient population, starting dose, approach for dose escalation and criteria for evaluating disease response | 12/10/20 | Cancer |
| Onconova Therapeutics Inc., of Newtown, Pa. | ON-123300 | Multikinase CDK4/6 inhibitor | Metastatic breast cancer | FDA cleared IND for phase I trial in relapsed/refractory advanced disease, including people with HR+/HER2- metastatic disease with resistance to approved second-generation CDK4/6 inhibitors | 12/21/20 | Cancer |
| Pfizer Inc., of New York | Lorbrena (lorlatinib) | Anaplastic lymphoma kinase inhibitor | Anaplastic lymphoma kinase-positive metastatic non-small-cell lung cancer | FDA accepted the supplemental NDA for a priority review; PDUFA target action date is in April 2021 | 12/28/20 | Cancer |
| Rafael Pharmaceuticals Inc., of Cranbury, N.J. | CPI-613 (devimistat) | Targets mitochondrial tricarboxylic acid cycle | Acute myeloid leukemia | FDA granted fast track designation | 12/15/20 | Cancer |
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Odronextamab | CD20 x CD3 bispecific antibody | B-cell non-Hodgkin lymphomas | FDA issued partial clinical hold, and Regeneron is pausing new enrollment in 2 trials; agency requested company amend trial protocols to further reduce incidence of ?grade 3 cytokine release syndrome during step-up dosing; protocol amendment expected to be submitted to FDA with goal of resuming enrollment in first quarter of 2021 | 12/14/20 | Cancer |
| Roche Holding AG, of Basel, Switzerland, and Halozyme Therapeutics Inc., of San Diego | Phesgo (pertuzumab/trastuzumab) | Monoclonal antibodies targeting HER2 | Early and metastatic HER2-positive breast cancer | European Commission approved the fixed-dose combination that uses Halozyme's Enhanze technology to create the subcutaneous formulation | 12/23/20 | Cancer |
| Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | HER2-positive locally advanced or metastatic breast cancer | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion recommending approval of the drug for patients who have received at least 2 prior anti-HER2 treatment regimens | 12/11/20 | Cancer |
| Sesen Bio Inc., of Cambridge, Mass. | Vicineum | Locally administered fusion protein | High-risk BCG-unresponsive non-muscle invasive bladder cancer | Submitted completed BLA to FDA, requesting priority review | 12/21/20 | Cancer |
| Sigilon Therapeutics Inc., of Cambridge, Mass. | SIG-005 | Contains human cell line genetically modified with nonviral vector designed to express human a-L-iduronidase | Mucopolysaccharidosis type I | FDA granted orphan designation | 12/17/20 | Cancer |
| Spectrum Pharmaceuticals Inc., of Henderson, Nev. | Poziotinib | EGFR tyrosine kinase inhibitor | Previously treated patients with non-small-cell lung cancer with HER2 exon 20 insertion mutations | At a pre-NDA meeting, FDA agreed to submission of an NDA based on cohort 2 of the Zenith20 phase II study | 12/22/20 | Cancer |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Iclusig (ponatinib) | Kinase inhibitor targeting BCR-ABL1 | Chronic myeloid leukemia | FDA approved sNDA to treat adults with chronic-phase disease who have resistance or intolerance to at least 2 prior kinase inhibitors | 12/18/20 | Cancer |
| Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX-592 | Targets PSMA | Advanced prostate cancer | Received clearance from TGA to begin phase I study | 12/2/20 | Cancer |
| TG Therapeutics Inc., of New York | Ublituximab | Anti-CD20 monoclonal antibody | Chronic lymphocytic leukemia | Rolling BLA submission to FDA initiated, with completion expected in first half of 2021 | 12/1/20 | Cancer |
| Tracon Pharmaceuticals Inc., of San Diego | Envafolimab | Subcutaneously administered single-domain antibody against PD-L1 | MSI-H/dMMR cancer | Chinese National Medical Products Administration accepted new drug application for review | 12/30/20 | Cancer |
| Turning Point Therapeutics Inc., of San Diego | Repotrectinib | Macrocyclic tyrosine kinase inhibitor of ROS1, TRK and ALK | ROS1-positive metastatic non-small-cell lung cancer | FDA granted breakthrough therapy designation for the treatment of patients who have not been treated with a ROS1 tyrosine kinase inhibitor | 12/8/20 | Cancer |
| Tvax Biomedical Inc., of Olathe, Kan. | Tvax Immunotherapy | Personalized T-cell based cell therapy | Glioblastoma | FDA granted fast track designation and orphan product designation | 12/9/20 | Cancer |
| V2act Therapeutics LLC, of San Diego | V2ACT | Oncolytic immunotherapy and a vaccine-enhanced adoptive cell therapy | Newly diagnosed surgically-resectable pancreatic cancer | FDA cleared an IND for a phase I/IIa study | 12/11/20 | Cancer |
| Viracta Therapeutics Inc., of San Diego | Nanatinostat and valganciclovir | HDAC 1/2/3 inhibitor | Relapsed/refractory Epstein-Barr virus-positive lymphomas | Based on outcome of end-of-phase II meeting with FDA, company intends to initiate global registration study in first half of 2021, designed to support multiple potential marketing approvals across various subtypes of EBV+ lymphoma | 12/7/20 | Cancer |
| Ziopharm Oncology Inc., of Boston | Patient-derived CD19-specific CAR T | B-lymphocyte antigen CD19 modulator; IL-15 receptor agonist | Leukemia; lymphoma | FDA cleared IND for phase I study in Taiwan by Eden Biocell Ltd., joint venture between Ziopharm and Triarm Therapeutics Ltd., in people with relapsed CD19+ disease | 12/21/20 | Cancer |
| Can-Fite Biopharma Ltd., of Petach Tikva | Cannabinoid fractions | At nanomolar concentrations binding with the A3 adenosine receptor | Cancer, inflammatory diseases and metabolic diseases associated with fat accumulation | Received approval from the Medical Cannabis Unit of Israel’s Ministry of Health to conduct preclinical studies | 12/15/20 | Cancer, Inflammatory |
| Antengene Ltd., of Shanghai | ATG-010 (selinexor) | Selective inhibitor of nuclear export | Relapsed/refractory multiple myeloma | China's NMPA accepted the IND for a 150-patient phase III study testing selinexor in combination with bortezomib and dexamethasone | 12/18/20 | Cancer; Hematologic |
| Amgen Inc., of Thousand Oaks, Calif. | Riabni (rituximab-arrx) | B-lymphocyte antigen CD20 inhibitor; biosimilar | Non-Hodgkin lymphoma; chronic lymphocytic leukemia; Wegener granulomatosis; microscopic polyangiitis | FDA approved for the treatment of adults with NHL, CLL, WG, and MPA; will be made available in the U.S. in January 2021 | 12/17/20 | Cancer; Immune |
| Acceleron Pharma Inc., of Cambridge, Mass. | Sotatercept | Ligand trap for TGF-beta superfamily to rebalance BMPR-II signaling | Pulmonary arterial hypertension | European Commission granted orphan drug designation | 12/14/20 | Cardiovascular |
| Novartis AG, of Basel, Switzerland | Entresto (sacubitril/valsartan) | Inhibits neprilysin and blocks angiotensin II type-I receptor | Heart failure with preserved ejection fraction | FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 12-1 that data presented support use of drug; FDA decision expected in first quarter of 2021 | 12/15/20 | Cardiovascular |
| Scpharmaceuticals Inc., of Burlington, Mass. | Furoscix | Furosemide solution formulated to a neutral pH | Worsening heart failure due to congestion | Received complete response letter from FDA; agency cited its need to conduct pre-approval inspections at 2 third-party manufacturing facilities that could not be conducted due to travel restrictions; FDA also raised questions related to testing, labeling and features of the combination product unrelated to the drug constituent | 12/7/20 | Cardiovascular |
| Amryt plc, of Dublin | AP-103 | Gene therapy | Dystrophic epidermolysis bullosa | FDA granted orphan drug designation | 12/23/20 | Dermatologic |
| Athenex Inc., of Buffalo, N.Y. | Klisyri | Tirbanibulin | Actinic keratosis on the face or scalp | FDA approved it | 12/14/20 | Dermatologic |
| Cara Therapeutics Inc., of Stamford, Conn. | Korsuva (difelikefalin) | KOR agonist | Moderate-to-severe pruritus in hemodialysis patients | Submitted NDA to the FDA with a request for a priority review | 12/28/20 | Dermatologic |
| Hoth Therapeutics Inc., of New York | Biolexa | Small molecule-based lotion | Atopic dermatitis | Received human research ethics committee clearance in Australia to initiate first cohort of phase Ib trial in mild to moderate disease; dosing of healthy volunteers expected in early 2021, with top-line data from cohort in first quarter of 2021 | 12/10/20 | Dermatologic |
| Hoth Therapeutics Inc., of New York | HT-001 | Topical drug | Rash and skin disorders associated with EGFR inhibitor therapy | Requested pre-IND meeting with FDA to discuss proposed drug development program; drug is being positioned for submission via the 505(b)(2) development pathway | 12/23/20 | Dermatologic |
| Sol-Gel Technologies Ltd., of Ness Ziona, Israel | Twyneo (benzoyl peroxide and tretinoin) | Retinoic acid receptor agonist | Acne vulgaris | FDA accepted for filing the NDA; PDUFA date of Aug. 1, 2021 | 12/7/20 | Dermatologic |
| Vyne Therapeutics Inc., of Bridgewater, N.J. | FCD-105 | 3% minocycline/0.3% adapalene foam | Moderate to severe acne vulgaris | Completed end-of-phase II meeting with FDA and established key elements of phase III program to support NDA; 2 identical 12-week, double-blind studies planned (Trilogy-1 and Trilogy-2), each to enroll 550 subjects, ages 9 and older, with 1 40-week, open-label study (Trilogy-3) to enroll minimum of 450 subjects from 12-week studies; program expected to start in 2021 | 12/17/20 | Dermatologic |
| Innovent Biologics Inc., of San Francisco | Sulinno (adalimumab) | Monoclonal antibody targeting TNF-alpha | Pediatric plaque psoriasis and non-infectious intermediate uveitis, posterior uveitis and panuveitis in adults | National Medical Products Administration of China approved the new indications | 12/23/20 | Dermatologic and Ocular |
| Amicus Therapeutics Inc., of Cranbury, N.J. | AT-GAA (ATB-200 + miglustat) | Enzyme replacement therapy + alpha-glucosidase stimulator | Pompe disease | Rolling BLA submission to FDA initiated to treat late onset disease | 12/1/20 | Endocrine/Metabolic |
| Amryt Pharma plc, of Dublin | Lojuxta (lomitapide) | Microsomal triglyceride transfer protein inhibitor | Homozygous familial hypercholesterolemia | Brazilian Health Regulatory Agency approved the drug as adjunct to a low-fat diet and other lipid-lowering treatments, with or without low-density lipoprotein apheresis | 12/9/20 | Endocrine/Metabolic |
| Autobahn Therapeutics Inc., of San Diego | ABX-002 | Thyroid hormone receptor beta agonist | Adrenomyeloneuropathy | Completed pre-IND meeting with FDA and gained alignment on dose selection strategy and patient inclusion criteria for phase Ia/b trial and on proposal to conduct single phase IIb/III adaptive trial to support potential registration | 12/1/20 | Endocrine/Metabolic |
| Cerecor Inc., of Rockville, Md. | CERC-803 (L-fucose) | Substrate replacement therapy | Leukocyte adhesion deficiency type II | FDA cleared IND for pivotal trial expected to begin by first half of 2021 and to report top-line data in second half of 2021 | 12/1/20 | Endocrine/Metabolic |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Esperion Therapeutics Inc., of Ann Arbor, Mich. | Nilemdo (bempedoic acid) | Inhibits ATP citrate lyase | High low-density lipoprotein cholesterol | Approved by Swissmedic | 12/16/20 | Endocrine/Metabolic |
| Daiichi Sankyo Co. Ltd., of Tokyo, and Esperion Therapeutics Inc., of Ann Arbor, Mich. | Nustendi (bempedoic acid and ezetimibe) tablet | ATP citrate lyase inhibitor plus cholesterol absorption inhibitor | High low-density lipoprotein cholesterol | Approved by Swissmedic | 12/16/20 | Endocrine/Metabolic |
| Fortress Biotech Inc., of New York | CUTX-101 | Restores copper levels | Menkes disease | The FDA granted breakthrough therapy designation | 12/15/20 | Endocrine/Metabolic |
| Hemoshear Therapeutics Inc., of Charlottesville, Va. | HST-5040 | Oral small molecule | Methylmalonic acidemia and propionic acidemia | FDA granted orphan designations | 12/16/20 | Endocrine/Metabolic |
| Hepion Pharmaceuticals Inc., of Edison, N.J. | CRV-431 | Hepatitis B structural protein inhibitor; peptidyl-prolyl cis-trans isomerase A inhibitor | Nonalcoholic steatohepatitis | Independent data safety monitoring board approved continuation of the phase IIa AMBITION trial | 12/30/20 | Endocrine/Metabolic |
| Lipocine Inc., of Salt Lake City | Tlando | Testosterone replacement therapy | Hypogonadism | FDA informed company it is continuing to work toward taking action on the NDA during week of Dec. 7, 2020; however, company cannot assure that FDA will act in that time frame | 12/7/20 | Endocrine/Metabolic |
| Lipocine Inc., of Salt Lake City | Tlando | Testosterone replacement therapy | Primary hypogonadism and hypogonadotropic hypogonadism | FDA granted tentative approval; drug will be eligible for final approval after the expiration of the exclusivity period for Jatenzo (testosterone undecanoate, Clarus Therapeutics Inc.) on March 27, 2022 | 12/8/20 | Endocrine/Metabolic |
| Neurogene Inc., of New York | Adeno-associated virus vector with engineered transgene encoding human AGA gene | Adeno-associated virus vector with engineered transgene encoding human AGA gene | Aspartylglucosaminuria | EMA granted orphan drug designation | 12/3/20 | Endocrine/Metabolic |
| Novartis AG, of Basel, Switzerland | Zolgensma (onasemnogene abeparvovec) | AAV-based gene therapy | Spinal muscular atrophy | Approved by Health Canada for treating pediatric patients with 5q SMA with bi-allelic mutations in the SMN1 gene and 3 or fewer copies of SMN2 gene; or infantile-onset SMA | 12/16/20 | Endocrine/Metabolic |
| Novartis AG, of Basel, Switzerland | Inclisiran ((KJX-839) | siRNA targeting PCSK9 | Hyperlipidemia in adults with atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia | FDA issued a complete response letter citing unresolved facility inspection-related conditions; those conditions will be conveyed to the European manufacturing facility within 10 business days from 12-18-20; the third-party facility is responsible for drug manufacturing and satisfactory resolution of the unresolved inspection-related conditions is required before the NDA may be approved; no onsite inspection was conducted but if one is needed, the FDA will define an approach for scheduling once safe travel may resume | 12/18/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Semaglutide | Glucagon-like peptide-1 analog | Chronic weight management | NDA submitted to FDA | 12/4/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Semaglutide | Glucagon-like peptide-1 analog | Chronic weight management | Submitted MAA to EMA; potential indication is for the treatment of adults with obesity or overweight with at least one weight-related comorbidity, as an adjunct to reduced-calorie diet and increased physical activity | 12/18/20 | Endocrine/Metabolic |
| Novo Nordisk A/S, of Bagsværd, Denmark | Ozempic (semaglutide) | Glucagon-like peptide-1 analogue | Type 2 diabetes | Submitted a label extension application to the EMA for the 2 mg dose | 12/29/20 | Endocrine/Metabolic |
| Orphazyme A/S, of Copenhagen | Arimoclomol | Chaperonin stimulator; Hsp70 stimulator | Niemann-Pick Disease Type C | FDA extended review period; updated PDUFA target action date is 6/17/21 | 12/27/20 | Endocrine/Metabolic |
| Passage Bio Inc., of Philadelphia | PBGM-01 | Gene therapy delivering the GLB1 gene | GM1 gangliosidosis | U.K.'s Medicines Healthcare Products Regulatory Agency approved the CTA for the phase I/II Imagine-1; global study expected to start in second quarter of 2021 | 12/11/20 | Endocrine/Metabolic |
| Polaryx Therapeutics Inc., of Paramus, N.J. | PLX-300 | Small molecule that activates PPAR? | Niemann Pick Disease Types A and B | FDA granted both rare pediatric disease and orphan drug designations, following similar grants for candidate in GM2 gangliosidosis | 12/18/20 | Endocrine/Metabolic |
| Taysha Gene Therapies Inc., of Dallas | TSHA-101 | AAV9-based gene therapy | GM2 gangliosidosis | Queen’s University in Ontario received (CTA approval from Health Canada for phase I/II study | 12/21/20 | Endocrine/Metabolic |
| Xeris Pharmaceuticals Inc., of Chicago | Ogluo (glucagon) | Peptide hormone | Severe hypoglycemia in diabetes mellitus | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion recommending approval of the drug | 12/11/20 | Endocrine/Metabolic |
| Albireo Pharma Inc., of Boston | Odevixibat | Ileal bile acid transport inhibitor | Familial intrahepatic cholestasis | Submitted an NDA to the FDA and an MAA to the EMA | 12/8/20 | Gastrointestinal |
| Bristol Myers Squibb, of New York | Zeposia (ozanimod) | Sphingosine-1-phosphate receptor modulator | Severely active ulcerative colitis | EMA validated the MAA | 12/28/20 | Gastrointestinal |
| Durect Corp., of Cupertino, Calif. | DUR-928 | Epigenetic regulator | Alcoholic hepatitis | FDA granted fast track status | 12/16/20 | Gastrointestinal |
| Gannex, a unit of Shanghai-based Ascletis Pharma Inc. | ASC-42 | Farnesoid X receptor agonist | Nonalcoholic steatohepatitis | FDA granted fast track designation | 12/13/20 | Gastrointestinal |
| LG Chem Life Sciences, of Seoul, Korea, and division of LG Chem, and Transthera Biosciences Co. Ltd., of Nanjing, China | TT-01025 | SSAO/VAP-1 inhibitor | Nonalcoholic steatohepatitis | FDA cleared IND for phase I trial in healthy subjects, expected to start in first quarter of 2021 | 12/1/20 | Gastrointestinal |
| Lygenesis Inc., of Pittsburgh | Allogeneic cell therapy | Allogeneic cell therapy | End stage liver disease | FDA cleared IND; phase IIa trial | 12/30/20 | Gastrointestinal |
| Merz Therapeutics Inc., of Raleigh, N.C. | Xeomin (incobotulinumtoxinA) | Neuromodulator | Chronic sialorrhea | FDA approved supplemental BLA for treating patients 2 and older; received priority review designation | 12/21/20 | Gastrointestinal |
| Prometheus Biosciences Inc., of San Diego | PRA-023 | Humanized IgG1 monoclonal antibody that blocks tumor necrosis factor-like ligand 1A | Ulcerative colitis and Crohn's disease | FDA accepted the IND application, and the company commenced dosing in phase Ia study | 12/15/20 | Gastrointestinal |
| Rafarma Pharmaceuticals Inc., of Nicosia, Cyprus, and Bebig LLC, of Moscow | Nanostructured microspheres based on yttrium-90 | Low-dose brachytherapy | Primary and metastatic liver lesions | Received an updated Russian registration certificate for the production and sale of the drug | 12/9/20 | Gastrointestinal |
| Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | TAK-721 (budesonide oral suspension) | Glucocorticoid receptor agonist | Eosinophilic esophagitis | FDA accepted the NDA and granted priority review | 12/15/20 | Gastrointestinal |
| Tetra Bio-Pharma Inc., of Ottawa, Ontario, Canada | Dronabinol soft gel capsules (PPP-002) | Cannabinoid receptor agonist | AIDS-related anorexia associated with weight loss and severe nausea, and vomiting associated with cancer chemotherapy | New drug submission filed with Health Canada | 12/30/20 | Gastrointestinal |
| Thetis Pharmaceuticals Inc., of Ridgefield, Conn. | TP-317 | Oral small molecule; Resolvin E1 | Pediatric ulcerative colitis | FDA granted orphan designation | 12/16/20 | Gastrointestinal |
| Ultragenyx Pharmaceutical Inc., of Novato, Calif. | UX-701 | Gene therapy expressing ATP7B copper transporter | Wilson disease | FDA granted orphan drug designation | 12/9/20 | Gastrointestinal |
| Aeglea Biotherapeutics Inc., of Austin, Texas | ACN-00177 | Cystathionine gamma-lyase stimulator | Homocystinuria | FDA granted rare pediatric disease designation | 12/1/20 | Genitourinary/Sexual Function |
| Amag Pharmaceuticals Inc., of Waltham, Mass. | Makena | Hydroxyprogesterone caproate | Preterm birth | Company submitted its response to the FDA's notice of opportunity for a hearing regarding the agency's proposed withdraw of the drug | 12/14/20 | Genitourinary/Sexual Function |
| Regent Pacific Group Ltd., of Hong Kong, and Wanbang Pharmaceutical Marketing and Distribution Co. Ltd., a company of Fosun Pharmaceutical, of Shanghai | Fortacin/Sendstend (lidocaine + prilocaine) | Sodium channel inhibitor | Premature ejaculation | Wanbang received approval from China NMPA to submit IND for human trial | 12/2/20 | Genitourinary/Sexual Function |
| Tricida Inc., of South San Francisco | Veverimer (TRC-101) | Hydrochloric acid binder | Metabolic acidosis in patients with chronic kidney disease | Submitted a formal dispute resolution request to the FDA over whether the magnitude of serum bicarbonate change seen in the TRCA-301 and TRCA-301E trials is reasonably likely enough to predict clinical benefit to justify an accelerated approval; a decision is expected in the first quarter of 2021 at which point the company may resubmit its NDA | 12/8/20 | Genitourinary/Sexual Function |
| Urovant Sciences, part of New York-based Sumitovant Biopharma Ltd. | Vibegron | ?3-adrenoreceptor agonist | Overactive bladder | FDA approved the drug to treat patients with symptoms of urge urinary incontinence, urgency and urinary frequency | 12/23/20 | Genitourinary/Sexual Function |
| Bristol Myers Squibb Co., of New York | Inrebic (fedratinib) | Wild type and mutationally activated janus associated kinase 2 and FMS-like tyrosine kinase 3 inhibitor | Primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis | Committee for Medicinal Products for Human Use of the EMA recommended approving the drug for patients who are Janus Associated Kinase inhibitor naïve or have been treated with ruxolitinib | 12/11/20 | Hematologic |
| Canbridge Pharmaceuticals Inc., of Beijing and Cambridge, Mass. | CAN-106 | Recombinant human MAb targeting complement C5 | Paroxysmal nocturnal hemoglobinuria | IND approved by Health Sciences Authority in Singapore | 12/18/20 | Hematologic |
| Catalyst Biosciences Inc., of South San Francisco | Marzeptacog alfa (MarzAA) | Subcutaneously administered next-generation engineered coagulation factor VIIa | Hemophilia A or B | FDA granted fast track designation for use in episodic bleeding in subjects with inhibitors | 12/2/20 | Hematologic |
| Editas Medicine Inc., of Cambridge, Mass. | EDIT-301 | CRISPR/Cas9/HBB stem cell-based gene therapy | Sickle cell disease | IND submitted to FDA for phase I/II trial | 12/9/20 | Hematologic |
| Emmaus Life Sciences Inc., of Torrance, Calif. | Endari | L-glutamine oral powder | Sickle cell disease | Swissmedic granted orphan drug status | 12/3/20 | Hematologic |
| Excellthera Inc., of Montreal | ECT-001 | Stem cell product | Allogeneic hematopoietic stem cell transplants | EMA granted priority medicines designation | 12/11/20 | Hematologic |
| Fibrogen Inc., of San Francisco | Roxadustat | Oral, small-molecule hypoxia-inducible factor prolyl hydroxylase inhibitor | Anemia of chronic kidney disease | FDA extended the review period of the NDA; the updated PDUFA action date is 3-20-21 | 12/18/20 | Hematologic |
| Graphite Bio Inc., of South San Francisco | GPH-101 | CRISPR-based gene editing of the sickle globin gene | Severe sickle cell disease | FDA cleared the IND for a phase I/II study; plans to start enrollment in early 2021 | 12/14/20 | Hematologic |
| Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson, and Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | Bruton's tyrosine kinase inhibitor | Waldenström's macroglobulinemia | FDA approved the updated label to include use of Imbruvica with rituximab based on the phase III Innovate study | 12/23/20 | Hematologic |
| Protagonist Therapeutics Inc., of Newark, Calif. | PTG-300 | Injectable synthetic peptide mimetic of natural hormone hepcidin | Polycythemia vera | FDA granted fast track designation | 12/2/20 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm | Emapalumab | Monoclonal antibody targeting interferon-gamma | Primary hemophagocytic lymphohistiocytosis with refractory, recurrent or progressive disease or intolerance with conventional therapy | MAA accepted for review in China | 12/10/20 | Hematologic |
| Swedish Orphan Biovitrum AB, of Stockholm | Doptelet (avatrombopag) | Thrombopoietin receptor agonist | Primary chronic immune thrombocytopenia | EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the approval of the drug for patients who are refractory to other treatments | 12/11/20 | Hematologic |
| Uniqure NV, of Amsterdam | Etranacogene dezaparvovec (AMT-061) | F9 gene stimulator; factor IX modulator | Hemophilia B | FDA placed program, including pivotal phase III Hope-B study, on clinical hold following safety report on possibly related serious adverse event associated with preliminary diagnosis of hepatocellular carcinoma in Hope-B participant with multiple risk factors; dosing completed in hemophilia B gene therapy studies, with no plan to enroll or treat additional people | 12/21/20 | Hematologic |
| Valeo Pharma Inc., of Montreal | Redesca and Redesca HP | Low molecular weight heparin biosimilars | Preventing deep vein thrombosis and pulmonary embolism | Health Canada issued notice of compliance | 12/9/20 | Hematologic/Respiratory |
| 9 Meters Biopharma Inc., of Raleigh, N.C. | NM-003 | Long-acting GLP-2 receptor agonist | Acute graft-vs.-host disease prophylaxis | FDA granted orphan drug designation | 12/9/20 | Immune |
| Aimmune Therapeutics Inc., of Vevey, Switzerland | Palforzia (defatted powder of Arachis hypogaea L., semen [peanuts]) | Immunomodulator | Peanut allergy | Approved by European Commission for use in patients, ages 4-17, in conjunction with peanut-avoidant diet | 12/21/20 | Immune |
| Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Orladeyo (berotralstat) | Kallikrein inhibitor | Hereditary angioedema | Approved for marketing | 12/4/20 | Immune |
| Biogen Inc., of Cambridge, Mass. | Plegridy, intramuscular (peginterferon beta-1a) | Interferon-beta ligand | Relapsing-remitting multiple sclerosis | European Commission granted marketing authorization | 12/21/20 | Immune |
| Cerecor Inc., of Rockville, Md. | CERC-007 | Monoclonal antibody targeting IL-18 | Still’s disease | FDA accepted the IND for a phase Ib study scheduled to start in the first quarter of 2021; initial data expected in the second quarter of 2021 | 12/22/20 | Immune |
| Gamida Cell Ltd., of Boston | Omidubicel | Cell therapy | Bone marrow transplant | FDA indicated at a type B meeting that the company should generate additional data to demonstrate analytical and clinical comparability from the company’s planned commercial manufacturing sites prior to requesting a pre-BLA meeting; company now plans to submit BLA in the second half of 2021 | 12/14/20 | Immune |
| Genentech, of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing and primary progressive multiple sclerosis | FDA approved a shorter 2-hour infusion time, dosed twice-yearly | 12/15/20 | Immune |
| Ose Immunotherapeutics SA, of Nantes, France | FR-104 | Monoclonal antibody CD28 antagonist | Renal transplant | French Central Ethics Committee approved initiation of phase I/II trial to be conducted as part of collaboration with University Hospital of Nantes | 12/3/20 | Immune |
| X4 Pharmaceuticals Inc., of Boston | Mavorixafor | Antagonist of CXCR4 | WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome | FDA granted rare pediatric disease designation | 12/10/20 | Immune |
| Abbvie, of North Chicago | Rinvoq (upadacitinib) | JAK inhibitor | Psoriatic arthritis and ankylosing spondylitis | EMA's Committee for Medicinal Products for Human Use recommended approval of the drug | 12/11/20 | Immune and musculoskeletal |
| Abionyx Pharma SA, of Toulouse, France | CER-001 | HDL-mimetic | Sepsis | Italian authorities cleared phase IIa study in infected individuals in ICU at high risk of developing acute kidney injury; enrollment expected to begin in first half of 2021 | 12/23/20 | Infection |
| Altimmune Inc., of Gaithersburg, Md. | AdCOVID | Intranasal SARS-VoV-2 adenovirus vector vaccine | COVID-19 infection | FDA placed clinical hold on phase I IND, requesting protocol modifications and submission of additional CMC data, to which company agreed in written response; no significant impact on development timetable expected | 12/23/20 | Infection |
| Aptorum Group Ltd., of New York | ALS-4 | Small-molecule drug | Infections caused by Staphylococcus aureus including methicillin-resistant S. aureus | Submitted CTA to Health Canada for phase I study in healthy volunteers | 12/21/20 | Infection |
| Arcturus Therapeutics Holdings Inc., of San Diego | ARCT-021 | Self-replicating mRNA-based vaccine | COVID-19 prophylaxis | Singapore Health Sciences Authority gave permission to proceed with a phase II study | 12/28/20 | Infection |
| Arix Bioscience plc., of London | Reltecimod | Peptide targeting CD28 | Necrotizing soft tissue infection | FDA accepted the NDA with a PDUFA target action date of 9/30/2021 | 12/11/20 | Infection |
| Astrazeneca plc, of Cambridge, U.K., and University of Oxford | COVID-19 vaccine Astrazeneca (formerly AZD-1222) | Vaccine | COVID-19 infection | U.K. Medicines and Healthcare products Regulatory Agency provided authorisation for emergency supply | 12/30/20 | Infection |
| Atox Bio Ltd., of Ness Ziona, Israel | Reltecimod | T-cell surface glycoprotein CD28 modulator | Necrotizing soft tissue infection | FDA accepted NDA, filed under accelerated approval, to treat to suspected organ dysfunction or failure in infected individuals, setting PDUFA date of Sept. 30, 2021 | 12/10/20 | Infection |
| Capricor Therapeutics Inc., of Los Angeles | CAP-1002 | Allogeneic cell therapy | Severe COVID-19 | Independent data and safety monitoring board recommended the Inspire study continue as designed; data expected in the first half of 2021 | 12/29/20 | Infection |
| Celltrion Inc., of Incheon, Korea | CT-P59 | Monoclonal antibody targeting SARS-CoV-2 | Mild-to-moderate COVID-19 | Submitted an application for conditional marketing authorization to the Korean Ministry of Food and Drug Safety | 12/29/20 | Infection |
| Chimerix Inc., of Durham, N.C. | Brincidofovir | Nucleotide analogue lipid-conjugate | Smallpox | FDA accepted the filing of NDA seeking approval as a medical countermeasure; agency granted priority review and set PDUFA date of April 7, 2021 | 12/7/20 | Infection |
| China National Pharmaceutical Group Co. Ltd. (Sinopharm CNBG), of Beijing | Vaccine | Inactivated vaccine for COVID-19 | COVID-19 | Chinese regulators granted approval for general use | 12/31/20 | Infection |
| Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Wrap | Liquefying gel-based wrap containing minocycline and rifampin | Post-mastectomy infections associated with the use of a tissue expander | In a written response to the company's pre-IND consultation request, the FDA indicated that bio absorption simulation studies may provide information to support development of the product; agency agreed that a large animal pharmacology study would be appropriate for drug elution studies; FDA agreed that a 28-day toxicology study appears appropriate and that microbiology support through existing data is acceptable | 12/9/20 | Infection |
| Cytodyn Inc., of Vancouver, Wash. | Vyrologix (leronlimab) | CCR5 antagonist | COVID-19 infection | FDA resumed emergency IND approval following completion of phase III trial, authorizing use by treating physician | 12/22/20 | Infection |
| Cyxone AB, of Stockholm | Rabeximod | Anti-rheumatic compound | COVID-19 infection therapy | Regulatory approval granted to begin phase II trial in Poland | 12/8/20 | Infection |
| Cyxone AB, of Stockholm | Rabeximod | Anti-rheumatic compound | COVID-19 and other indications, such as rheumatoid arthritis | Filed an IND following positive feedback from a pre-IND meeting with the FDA | 12/15/20 | Infection |
| Dr. Reddy’s Laboratories Ltd., of Hyderabad, India, Appili Therapeutics Inc., of Nova Scotia, and Global Response Aid FZCO | Reeqonus (favipiravir, also Avigan, Fujifilm Toyama Chemical Co. Ltd.) | RNA polymerase inhibitor | COVID-19 infection | Application filed by subsidiary Dr. Reddy’s Canada on behalf of the consortium for treatment of mild to moderate infection in adults under Health Canada’s interim order respecting importation, sale and advertising of drugs for use in relation to COVID-19 | 12/22/20 | Infection |
| Dynavax Technologies Corporation, of Emeryville, Calif. | Heplisav-B | Hepatitis B vaccine | Hepatitis B virus infection prophylaxis | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion on the MAA | 12/10/20 | Infection |
| Elusys Therapeutics Inc., of Parsippany, N.J. | Obiltoxaximab | Monoclonal antibody anthrax antitoxin | Inhalation anthrax | European Commission approved treatment for use in all age groups in combination with appropriate antibacterial drugs for inhalational anthrax due to Bacillus anthracis; and for post-exposure prophylaxis of inhalational anthrax when alternative therapies are not available or not appropriate | 12/2/20 | Infection |
| Gilead Sciences Canada Inc., unit of Gilead Sciences Inc., of Foster City, Calif. | Descovy (emtricitabine + tenofovir alafenamide) | Antiretroviral therapy | HIV pre-exposure prophylaxis | Health Canada granted notice of compliance to reduce risk of sexually acquired HIV-1 infection | 12/1/20 | Infection |
| Incannex Healthcare Ltd., of Melbourne, Australia | IHL-675 (hydroxychloroquine + cannabidiol fixed-dose) | Anti-inflammatory | COVID-19 infection | Requested expedited pre-IND meeting with FDA to pursue emergency use authorization to treat COVID-19-related sepsis-associated acute respiratory distress syndrome | 12/1/20 | Infection |
| Innovation Pharmaceuticals Inc., of Wakefield, Mass. | Brilacidin | Host defense protein mimetic | COVID-19 infection | FDA approved IND for phase II trial in people hospitalized with infection | 12/21/20 | Infection |
| Inotrem SA, of Paris | Nangibotide | Formulation of TREM-1 inhibitor LR-12 | COVID-19 infection | French government steering committee for therapeutic clinical trials and other research designated ongoing phase IIa study in severe forms of infection as national priority research, allowing accelerated enrollment and activation of fast-track reviews and approval processes by French regulators | 12/24/20 | Infection |
| Janssen-Cilag International NV, unit of Johnson & Johnson, of New Brunswick, N.J. | Ad26.COV2.S (JNJ-78436735) | SARS-CoV-2 recombinant adenoviral vector vaccine | COVID-19 infection | EMA initiated rolling review | 12/1/20 | Infection |
| Mesoblast Ltd., of New York and Melbourne, Australia | Remestemcel-L | Culture-expanded mesenchymal stromal cells derived from bone marrow of unrelated donor | COVID-19 | FDA granted fast track designation for treatment of acute respiratory distress syndrome due to COVID-19 infection | 12/2/20 | Infection |
| Moderna Biotech Spain SL, unit of Moderna Inc., of Cambridge, Mass. | mRNA-1273 | COVID19 spike glycoprotein modulator | COVID-19 infection | Application for conditional marketing authorization submitted to EMA, requesting accelerated assessment | 12/1/20 | Infection |
| Moderna Biotech Spain SL, unit of Moderna Inc., of Cambridge, Mass. | mRNA-1273 | COVID19 spike glycoprotein modulator | COVID-19 infection | FDA granted emergency use authorization | 12/21/20 | Infection |
| Moderna Inc., of Cambridge, Mass. | mRNA-1272 | mRNA vaccine | COVID-19 | Health Canada authorized vaccine in an interim order for immunization of people 18 and older | 12/23/20 | Infection |
| Open Orphan plc, of London, and Codagenix Inc., of Farmingdale, N.Y. | COVI-VAC | Intranasal SARS-CoV-2 vaccine | COVID-19 prophylaxis | U.K.'s MHRA approved the phase I study, which is expected to start in January 2021 | 12/14/20 | Infection |
| Pfizer Inc., of New York | 20vPnC | 20-valent pneumococcal conjugate vaccine | Streptococcus pneumoniae infection prophylaxis | FDA accepted the BLA for a priority review with a PDUFA goal action date is in June 2021 | 12/8/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | COVID-19 spike glycoprotein modulator | COVID-19 infection | Application for conditional marketing authorization submitted to EMA, which will proceed under accelerated assessment | 12/1/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | mRNA vaccine | COVID-19 | U.K. MHRA granted temporary authorization for emergency use | 12/2/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | Vaccine | COVID-19 | Health Canada granted authorization under interim order for emergency use | 12/9/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | mRNA-based vaccine | COVID-19 prophylaxis | FDA issued an emergency use authorization | 12/11/20 | Infection |
| Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | BNT-162b2 | Vaccine | COVID-19 | EMA’s Committee for Medicinal Products for Human Use issued positive opinion to recommend conditional marketing authorization to prevent COVID-19 caused by SARS-CoV-2 virus in individuals 16 and older | 12/21/20 | Infection |
| Polyphor AG, of Allschwil, Switzerland | Murepavadin, inhaled | Outer membrane protein inhibitor | Pseudomonas aeruginosa lung infection | U.K.'s MHRA cleared CTA to initiate phase I study using Eflow technology nebulizer (Pari Pharma GmbH) in healthy volunteers | 12/22/20 | Infection |
| Rhizen Pharmaceuticals SA, of La Chaux du Fonds, Switzerland | RP-7214 | Dihydroorotate dehydrogenase inhibitor | COVID-19 infection | FDA cleared IND for phase I trial in healthy volunteers | 12/1/20 | Infection |
| Ridgeback Biotherapeutics LP, of Miami | Ebanga (ansuvimab-zykl) | Monoclonal antibody targeting Zaire ebolavirus | Ebola | FDA approved the drug | 12/22/20 | Infection |
| Russian Direct Investment Fund, and the National Research Center for Epidemiology and Microbiology, both of Moscow | Sputnik V | Adenoviral-based vaccine | COVID-19 | Vaccine registered in Argentina under emergency use authorization procedure and approved by regulator ANMAT | 12/23/20 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp | Antifungal | Vulvovaginal candidiasis | FDA accepted for filing the NDA; agency granted priority review and set PDUFA date of June 1, 2021 | 12/7/20 | Infection |
| Scynexis Inc., of Jersey City, N.J. | Ibrexafungerp IV | Liposomal intravenous formulation of the antifungal agent | Healthy volunteers (eventually fungal infections) | Health authority and ethics committee in South Africa granted approval to start a phase I study; plans to start study in the first quarter of 2021 | 12/10/20 | Infection |
| Shenyang Tonglian Group Co. Ltd., of Shenyang, China | Carrimycin | Antiviral | COVID-19 | FDA cleared a phase III study that's scheduled to begin in January 2021 | 12/23/20 | Infection |
| Sinovac Biotech Ltd., of Beijing | Vaccine | 23-valent pneumococcal polysaccharide vaccine | Streptococcus pneumonia | Approved by China’s NMPA for prevention of infection in adults and children, 2 and older | 12/9/20 | Infection |
| Sorrento Therapeutics Inc., of San Diego | STI-2020 | COVID-19 spike glycoprotein inhibitor | COVID-19 | FDA accepted IND for phase I trials in healthy volunteers and outpatient COVID-19 patients with mild symptoms | 12/9/20 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | Sci-B-Vac | Hepatitis B large envelope protein modulator | Hepatitis B virus infection | BLA submitted to FDA covering 3-antigen prophylactic HBV vaccine candidate | 12/1/20 | Infection |
| VBI Vaccines Inc., of Cambridge, Mass. | 3-antigen prophylactic hepatitis B vaccine | S, pre-S1 and pre-S2 surface antigens of the hepatitis B virus | Hepatitis B virus infection prophylaxis | EMA accepted the MAA | 12/23/20 | Infection |
| Verrica Pharmaceuticals Inc., of West Chester, Pa. | VP-102 (cantharidin) | Protein phosphatase 2A inhibitor | Molluscum contagiosum | Resubmitted the NDA that was issued a complete response letter in June 2020 | 12/23/20 | Infection |
| Viiv Healthcare Ltd., of London | Vocabria (cabotegravir injection and tablets) | Inhibits HIV replication by preventing viral DNA from integrating into genetic material of human immune cells | HIV | Approved in European Union for use in combination with Rekambys (rilpivirine injection, Johnson & Johnson) for treating adults with HIV-1 infection who are virologically suppressed | 12/21/20 | Infection |
| Viiv Healthcare, of London | Rukobia (fostemsavir) | Attachment inhibitor | HIV-1 infection | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion recommending approval of the drug | 12/11/20 | Infection |
| Yiling Pharmaceutical Co. Ltd., of Shijiazhuang City, China | Lianhua qingwen capsules | Traditional Chinese medicine | COVID-19 | Approved by China’s NMPA to treat lightly and ordinarily infected patients | 12/7/20 | Infection |
| Synairgen plc, of Southampton, U.K. | SNG-001 | Interferon beta ligand | COVID-19 infection | Following discussions with the regulatory agencies, amended trial to remove lower dose arm, add second primary endpoint and assessments for long COVID-19 symptoms | 12/18/20 | Infection; Respiratory |
| Ampio Pharmaceuticals Inc., of Englewood, Colo. | Ampion | Stem cell antigen-1 inhibitor | Osteoarthritis of the knee | FDA provided feedback on proposed modifications to the AP-013 study, which was suspended due to COVID-19 pandemic | 12/29/20 | Inflammatory |
| Henlius Biotech Inc., of Shanghai | HLX-03 | Biosimilar to Humira (adalimumab, Abbvie Inc.) | Rheumatoid arthritis, ankylosing spondylitis and plaque psoriasis | Approval granted in China | 12/8/20 | Inflammatory |
| Swedish Orphan Biovitrum AB, of Stockholm | Kineret (anakinra) | Interleukin-1 receptor antagonist | Deficiency of IL-1 receptor antagonist (DIRA) | FDA approved the supplemental BLA | 12/22/20 | Inflammatory |
| Xentria Inc., of Chicago | XTMAB-16 | Intravenous TNF-alpha inhibitor | Sarcoidosis | FDA granted orphan drug designation | 12/15/20 | Inflammatory |
| Entera Bio Ltd., of Jerusalem | EB-613 | Parathyroid hormone receptor agonist | Osteoporosis | FDA cleared IND for initial U.S. trial; pivotal phase III trial expected for potential approval under 505(b)(2) regulatory pathway | 12/10/20 | Musculoskeletal |
| Merz Therapeutics, unit of Merz Pharmaceuticals GmbH, of Frankfurt | Xeomin (incobotulinumtoxinA) | Neuromuscular blocking agent | Excessive drooling | Health Canada granted notice of compliance to treat condition associated with neurological disorders in adults | 12/1/20 | Musculoskeletal |
| Novadip Biosciences SA, of Mont-Saint-Guibert, Belgium | NVD-003 | Autologous cell-based osteogenic product | Congenital pseudarthrosis of the tibia | FDA granted rare pediatric disease and orphan drug designations | 12/23/20 | Musculoskeletal |
| PTC Therapeutics Inc., of South Plainfield, N.J. | Translarna (ataluren) | Protein restoration therapy | Nonsense mutation Duchenne muscular dystrophy | Approved for marketing in Russia | 12/4/20 | Musculoskeletal |
| Alkermes plc., of Dublin | ALKS-3831 (olanzapine/samidorphan) | Atypical antipsychotic | Schizophrenia and bipolar I disorder | FDA received the resubmission of the NDA and classified it as a class 2 response of the complete response letter issued in November 2020; new PDUFA target action date is 6/1/21 | 12/29/20 | Neurology/Psychiatric |
| Alzecure Pharma AB, of Stockholm | ACD-440 | Vanilloid VR1 antagonist | Neuropathic pain | German regulators cleared start of phase Ib study testing topical drug; results expected in first half of 2021 | 12/7/20 | Neurology/Psychiatric |
| Audentes Therapeutics Inc., of San Francisco, a unit of Astellas Pharma Inc. | AT-132 | AAV8-delivered gene therapy | X-linked myotubular myopathy | FDA lifted the clinical hold on the phase I/II Aspiro study | 12/24/20 | Neurology/Psychiatric |
| Avadel Pharmaceuticals plc, of Dublin | FT-218 | Once-nightly formulation of sodium oxybate | Excessive daytime sleepiness and cataplexy in narcolepsy | Submitted NDA to FDA | 12/16/20 | Neurology/Psychiatric |
| Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Aducanumab | Beta-amyloid antagonist | Alzheimer's disease | J-NDA submitted to Japan's MHLW | 12/9/20 | Neurology/Psychiatric |
| Biogen, of Cambridge, Mass., and Eisai Co., Ltd., of Tokyo, Japan | Aducanumab | Monoclonal antibody targeting amyloid beta | Alzheimer’s disease | Submitted a Japanese NDA to the Ministry of Health, Labor and Welfare | 12/9/20 | Neurology/Psychiatric |
| Chugai Pharmaceutical Co. Ltd., of Tokyo | Enspryng (satralizumab) | IL-6 receptor antagonist | Neuromyelitis optica spectrum disorder | Taiwan Food and Drug Administration issued an import drug license for the drug | 12/9/20 | Neurology/Psychiatric |
| Diamedica Therapeutics Inc., of Minneapolis | DM-199 | Kallikrein 1 modulator | Acute ischemic stroke | Based on written responses from FDA on type B meeting, IND application for phase II/III study expected in first quarter of 2021; agency guided that adaptive trial design with primary endpoint based on modified Rankin Scale at day 90 may qualify as a single trial to support BLA submission | 12/10/20 | Neurology/Psychiatric |
| Ix Biopharma Ltd., of Singapore | Wafermine | Sublingual ketamine wafer | Pain | EMA endorsed design of pivotal phase III studies | 12/8/20 | Neurology/Psychiatric |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Spravato (esketamine) | N-methyl-D-aspartate glutamate receptor antagonist | Major depressive disorder | Committee for Medicinal Products for Human Use of the EMA recommended expanding use of the drug for psychiatric emergencies | 12/11/20 | Neurology/Psychiatric |
| Johnson & Johnson, of Whitehouse Station, N.J. | Paliperidone palmitate 6 | Atypical anti-psychotic | Schizophrenia | Submitted marketing authorization extension in EU | 12/4/20 | Neurology/Psychiatric |
| Kempharm Inc., of Celebration, Fla. | KP-415 | Serdexmethylphenidate co-formulated with immediate-release d-methylphenidate | Attention deficit hyperactivity disorder | In late-cycle communication meeting with FDA Dec. 1, agency did not raise any substantive issues related to safety and efficacy at this point in review; re-affirmed PDUFA date of March 2, 2021 | 12/2/20 | Neurology/Psychiatric |
| Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | LX-9211 | Adapter-associated kinase 1 inhibitor | Diabetic peripheral neuropathic pain | FDA granted fast track designation | 12/11/20 | Neurology/Psychiatric |
| Lumosa Therapeutics Ltd., of Taipei, Taiwan | LT-1001 (Naldebain) | Extended-release analgesic injection | Analgesic | Approved by Singapore’s Health Science Agency | 12/21/20 | Neurology/Psychiatric |
| Mindmed Inc., of New York | Lysergic acid diethylamide | Psychedelic | Anxiety disorder | Completed a pre-IND meeting; plans to open an IND for a phase IIb study in August 2021 | 12/14/20 | Neurology/Psychiatric |
| Minerva Neurosciences Inc., of Waltham, Mass. | Roluperidone | 5-HT 2a receptor antagonist; opioid receptor sigma antagonist 2 | Schizophrenia | In type C meeting minutes, FDA advised that NDA submission based on current data from phase IIb and III studies would be unlikely to be filed and would prompt review issues due to lack of 2 well-controlled trials to support efficacy claims to treat negative symptoms of disease | 12/1/20 | Neurology/Psychiatric |
| Moebius Medical Ltd., of Tel Aviv, Israel | MM-II | Non-opioid intra-articular injection | Pain in knee osteoarthritis | FDA cleared IND for phase IIb trial | 12/21/20 | Neurology/Psychiatric |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Synthetic analogue of molecule derived from IGF-1 | Angelman syndrome |
EMA issued positive opinion for orphan designation | 12/7/20 | Neurology/Psychiatric |
| Novaremed AG, of Basel, Switzerland | NRD135S.E1 | Blk tyrosine kinase stimulator; Lyn tyrosine kinase stimulator | Pain; diabetic peripheral neuropathy | FDA granted fast track status for treatment of painful diabetic peripheral neuropathy | 12/18/20 | Neurology/Psychiatric |
| Orchard Therapeutics plc, of London | Libmeldy | ARSA gene stimulator | Metachromatic leukodystrophy | European Commission granted full authorization to treat children with late infantile or early juvenile forms without clinical manifestations of disease or with early juvenile form, with early clinical manifestations of disease, ability to walk independently and before onset of cognitive decline | 12/21/20 | Neurology/Psychiatric |
| Osmotica Pharmaceuticals plc, of Bridgewater, N.J. | Arbaclofen extended release | GABA B receptor agonist | Spasticity resulting from multiple sclerosis | FDA issued CRL, stating the company didn't provide adequate justification (including in its most recent NDA amendment) for the statistical analysis of the change from baseline to day 84 in TNmAS-MAL scores comparing arbaclofen 40 mg to placebo, one of the co-primary endpoints; FDA made a number of recommendations, including conducting a new study | 12/30/20 | Neurology/Psychiatric |
| Realta Life Sciences Inc., of Norfolk, Va. | RLS-0071 (PIC1-dPEG24) | Complement C1s subcomponent inhibitor | Hypoxic-ischemic encephalopathy | EMA granted orphan designation | 12/3/20 | Neurology/Psychiatric |
| Sanofi SA, of Paris | Plavix (clopidogrel) | Inhibits ADP-induced platelet aggregation | High-risk transient ischemic attack or minor ischemic stroke | Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion on the expanded indication | 12/11/20 | Neurology/Psychiatric |
| Taysha Gene Therapies Inc., of Dallas | TSHA-103 | AAV9-based gene therapy | SLC6A1-related epilepsy | FDA granted rare pediatric disease and orphan drug designations | 12/3/20 | Neurology/Psychiatric |
| Therapeutic Solutions International Inc., of Oceanside, Calif. | Jadicells | Umbilical cord-derived mesenchymal stem cell | Chronic traumatic encephalopathy | Filed IND with FDA to initiate phase I/II study | 12/17/20 | Neurology/Psychiatric |
| Voyager Therapeutics Inc., of Cambridge, Mass., and Neurocrine Biosciences Inc., of San Diego | NBIb-1817 | Intracerebral AAV-based gene therapy | Parkinson’s disease | FDA placed a clinical hold on the Restore-1 study due to MRI abnormalities in some study participants; clinical implications are currently unknown and are being evaluated | 12/22/20 | Neurology/Psychiatric |
| Zogenix Inc., of Emeryville, Calif. | Fintepla (fenfluramine) | 5-HT 2c receptor modulator; 5-HT 1d receptor modulator; 5-HT 2a receptor modulator; opioid receptor sigma modulator 1 | Dravet syndrome | European Commission granted marketing authorization for treating seizures associated with Dravet syndrome as add-on therapy to other anti-epileptic medicines for patients 2 and older | 12/21/20 | Neurology/Psychiatric |
| Zynerba Pharmaceuticals Inc., of Devon, Pa. | Zygel | Cannabinoid CB2 receptor modulator | Fragile X syndrome | After meeting with FDA, company plans to conduct double-blind, placebo-controlled pivotal trial in patients who have highly methylated FMR1 gene to confirm positive results observed in that population of responders in CONNECT-FX trial; company will review trial design and protocol through type C meeting with FDA and expects to start pivotal study before end of 2021 | 12/17/20 | Neurology/Psychiatric |
| Algo Therapeutix SAS, of Suresnes, France | ATX-01 | Topical amitriptyline | Chemotherapy-induced peripheral neuropathy | Received regulatory approval in the Czech Republic to initiate phase I development | 12/15/20 | Neurology/Psychiatric, Cancer |
| Arctic Vision Biotechnology Co. Ltd., of Shanghai | ARVN-001 (triamcinolone acetonide) | Corticosteroid | Uveitic macular edema | China National Medical Products Administration cleared the IND for a phase III study | 12/25/20 | Ocular |
| Eyenovia Inc., of New York | Microline | Pilocarpine formulation | Presbyopia | FDA accepted IND for phase III Vision program, with Vision-1 study set to start later this month | 12/3/20 | Ocular |
| Nicox SA, of Sophia Antipolis, France | Latanoprostene bunod, 0.024% | PGF2 alpha agonist | Open-angle glaucoma; ocular hypertension | Partner Bausch + Lomb said regulators in Colombia approved clinical data package for agent, branded in country as Cliropta | 12/22/20 | Ocular |
| Ocular Therapeutix Inc., of Bedford, Mass. | Dextenza (dexamethasone, intracanalicular) 0.4 mg | Glucocorticoid receptor agonist | Allergic conjunctivitis | sNDA submitted to FDA to treat ocular itching associated with condition | 12/22/20 | Ocular |
| Oyster Point Pharma Inc., of Princeton, N.J. | OC-01 (varenicline) nasal spray | Selective cholinergic agonist | Neurotrophic keratitis | Submitted protocol to FDA to start study in adults with NK; enrollment of first patient in phase II Olympia study planned for first half of 2021 | 12/1/20 | Ocular |
| Oyster Point Pharma Inc., of Princeton, N.J. | OC-01 (varenicline) | Nasal formulation of varenicline | Xerophthalmia | Submitted a 505(b)(2) NDA to FDA | 12/18/20 | Ocular |
| Rezolute Inc., of Redwood City, Calif. | RZ-402 | Oral plasma kallikrein inhibitor | Diabetic macular edema | FDA cleared the IND for a phase I trial, set to start in the first quarter of 2021 | 12/3/20 | Ocular |
| Tarsier Pharma Ltd., of Tel Aviv, Israel | TRS-01 | Immunomodulator | Uveitis | Completed end-of-phase II meeting with FDA and received feedback for phase III trial design; pivotal study will be randomized, active control, double-masked, multicenter study in patients with noninfectious anterior uveitis, including those with uveitic glaucoma; trial expected to start in 2021 | 12/17/20 | Ocular |
| Tarsus Pharmaceuticals Inc., of Irvine, Calif | TP-03 | Non-competitive antagonist of insect and arachnid GABA-Cl channels | Demodex blepharitis | Minutes from a type C meeting with the FDA outlined requirements for an NDA filing, including nonclinical toxicology, clinical and CMC components | 12/23/20 | Ocular |
| Tearclear Corp., of Boston | Latanoprost | PGF2 alpha agonist | Glaucoma | Completed pre-IND meeting with FDA regarding development plan for preservative-free multidose delivery of proprietary formulation, including design for registration study and IND filing in mid-2021 via 505(b)(2) pathway | 12/1/20 | Ocular |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Synthetic analogue of molecule derived from IGF-1 | Phelan-McDermid syndrome | EMA issued positive opinion for orphan designation | 12/7/20 | Other/Miscellaneous |
| Neuren Pharmaceuticals Ltd., of Melbourne, Australia | NNZ-2591 | Synthetic analogue of molecule derived from IGF-1 | Pitt Hopkins syndrome | EMA issued positive opinion for orphan designation | 12/7/20 | Other/Miscellaneous |
| Novo Nordisk A/S, of Bagsvaerd, Denmark | Saxenda (liraglutide) | GLP-1 receptor agonist | Obesity | FDA approved updated label for use in treating adolescents, ages 12-17, with body weight above 60 kg and initial body mass index corresponding to 30 kg/m2 or greater for adults, as an adjunct to reduced-calorie meals and increased physical activity | 12/4/20 | Other/Miscellaneous |
| Roche Holding AG, of Basel, Switzerland | Xolair (omalizumab) | Antibody designed to target and block immunoglobulin E | Nasal polyps | FDA approved supplemental BLA for add-on maintenance treatment of nasal polyps in adult patients 18 and older with inadequate response to nasal corticosteroids | 12/1/20 | Other/Miscellaneous |
| Soleno Therapeutics Inc., of Redwood City, Calif. | DCCR | Diazoxide choline controlled release | Prader-Willi Syndrome | Minutes from a type C meeting with the FDA confirmed that data from completed and ongoing studies, together with external, natural history studies can support an NDA filing scheduled for the second half of 2021 | 12/11/20 | Other/Miscellaneous |
| Takeda Pharmaceutical Co. Ltd., of Tokyo | Takhzyro (lanadelumab) | Kallikrein blocker | Hereditary angioedema | Approval granted in China | 12/8/20 | Other/Miscellaneous |
| Vanda Pharmaceuticals Inc., of Washington | Hetlioz (tasimelteon) capsule and liquid formulations | Melatonin MT1/MT2 receptor agonist | Smith-Magenis syndrome | FDA approved for use in adults and children with nighttime sleep disturbances associated with SMS | 12/1/20 | Other/Miscellaneous |
| Astrazeneca plc, of Cambridge, U.K. | Trixeo Aerosphere (formoterol fumarate/glycopyrronium bromide/budesonide) | Long-acting beta2-agonist. long-acting muscarinic antagonist and inhaled corticosteroid | Moderate to severe chronic obstructive pulmonary disease | European Commission approved the drug | 12/14/20 | Respiratory |
| Polarean Imaging plc., of Durham, N.C. | Hyperpolarized xenon-129 gas | Imaging agent | Pulmonary | FDA accepted the NDA for standard review; PDUFA target action date is Oct. 5, 2021 | 12/23/20 | Respiratory |
| United Therapeutics Corp., of Silver Spring, Md. | Tyvaso (treprostinil) | Prostacyclin vasodilator | Idiopathic pulmonary fibrosis | FDA granted orphan designation | 12/9/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Trikafta (elexacaftor + tezacaftor + ivacaftor) | CFTR modulator/stimulator | Cystic fibrosis | FDA expanded eligibility to include people 12 and older with any of 177 additional mutations | 12/21/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Symdeko (tezacaftor + ivacaftor) | CFTR stimulator | Cystic fibrosis | FDA expanded eligibility to treat people 6 and older with any of 127 additional mutations | 12/21/20 | Respiratory |
| Vertex Pharmaceuticals Inc., of Boston | Kalydeco (ivacaftor) | CFTR stimulator | Cystic fibrosis | FDA expanded eligibility to treat people 4 months and older with any of 59 additional mutations | 12/21/20 | Respiratory |
| Kempharm Inc., of Celebration, Fla. | KP-879 (serdexmethylphenidate, extended release) | Alpha adrenoceptor agonist | Stimulant use disorder | IND filed for human trials, expected to begin in 2021 | 12/22/20 | Toxicity and Intoxication |