The EMA is standing firm on its refusal to recommend approval of the amyotrophic lateral sclerosis (ALS) treatment Albrioza in Europe after re-examining Amylyx Pharmaceuticals Inc.’s marketing authorization application and remaining unconvinced that the main study demonstrated the drug effectively slows disease progression.

“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.

Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA dug in its heels about withdrawing the drug from the U.S. market.