With sympathy for patients, FDA advisers find Amylyx data beyond limits of flexibility

In explaining their 6-4 vote against effectiveness of Amylyx’s ALS therapy, nearly all members of FDA’s Peripheral and Central Nervous System Drug advisory committee stated they struggled with the decision.

All agreed the results of the company’s Phase II CENTAUR study held promise, and that the need for regulatory flexibility was exceptionally acute given the 100% fatality rate of amyotrophic lateral sclerosis. However, deeming the data sufficient to support approval seemed to most committee members to bend FDA’s statutory requirements beyond the breaking point...