FDA is critical of data on Amylyx ALS drug, but cites ‘regulatory flexibility’

Results from a single clinical trial of an experimental drug for ALS developed by Amylyx Pharmaceuticals “may not be sufficiently persuasive” to support approval, according to a review of the drug posted Monday by officials with the Food and Drug Administration.

Reviewers in the FDA’s Office of Neuroscience criticized the data, which showed the drug demonstrated a modest slowing of disease progression but no survival benefit, as well as the conduct of the study. But the agency, in its review, noted that it can exercise “regulatory flexibility” to approve drugs for serious diseases with unmet medical needs.