The Food and Drug Administration is convening a meeting of outside experts today to review clinical data on an experimental treatment for ALS from Amylyx Pharmaceuticals. The stakes for this all-day meeting are high, and deliberations could be contentious.
Patients with ALS, their caregivers, and advocacy groups have been applying enormous pressure on the FDA to approve the Amylyx drug, called AMX0035, which they see as a vitally necessary treatment against a neurodegenerative disease that is almost universally fatal.
But the FDA has significant reservations about the clinical trial conducted by Amylyx. In a review of AMX0035 posted on Monday, FDA officials said the results provided only a modest benefit for ALS patients and “may not be sufficiently persuasive” to support approval. Agency reviewers also noted that ALS is a serious, fatal disease with few treatment options, necessitating “regulatory flexibility” when it comes to assessing clinical data.
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