In reversal, FDA panel votes to recommend experimental ALS drug
An FDA advisory panel voted to recommend a drug for ALS, reversing a decision from earlier this year. The drug was funded in part by the Ice Bucket Challenge.
A Food and Drug Administration advisory panel voted Wednesday to recommend an experimental drug for patients with ALS, a reversal from its decision in March and a victory for advocacy groups pushing for the drug’s approval.
The 7-2 vote by the agency’s Peripheral and Central Nervous System Drugs Advisory Committee concluded an all-day meeting that at times turned emotional.
ALS, or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disease for which there is no cure. The meeting included a lengthy public comment period where caregivers and patients with ALS exhorted the FDA to approve the drug without delay.
The vote does not mean the drug is now approved; that decision still lies with the FDA, which is expected to make a final ruling by Sept. 29.
The favorable vote came despite concerns from panel members about whether an additional analysis of a single phase 2 clinical trial, from drugmaker Amylyx Pharmaceuticals, showed clear evidence the drug slows the progression of the disease.
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