In March, the Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 that a single Phase 2 trial did not establish the conclusion that the drug is effective in treating ALS.
Amylyx is now continuing to study its safety and efficacy in a Phase 3 trial. The trial also showed that the drug was generally well-tolerated, but there was a greater frequency of gastrointestinal events in the group getting the medication. Frates family kicks off Ice Bucket Challenge month in Boston (WBZ-TV) The study was funded in part by a grant from the ALS Ice Bucket Challenge, the viral social media campaign that started in 2014 involving people dumping buckets of ice water over themselves to raise awareness and money around ALS. In November, Amylyx submitted a drug application to the FDA for the medication, then called AMX0035, as an oral ALS treatment, seeking approval based on a Phase 2 trial that included 137 people with ALS who received either the drug or a placebo for 24 weeks. And so, there's a vast unmet need in this disease area, which FDA has acknowledged," said Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania.Īhead of the FDA decision, Lynch told CNN that she would be "shocked" if the drug did not get approved because the FDA's Peripheral and Central Nervous System Drugs Advisory Committee made a shift in its opinion of the drug at a meeting this month, voting 7-2 in favor of approval. FDA has approved a couple of treatments, but they are minimally effective and certainly not a cure. "ALS is a horrific disease: rapidly fatal and really debilitating during the period from initial symptoms to death. It's a neurodegenerative disease that causes muscles to weaken, eventually affecting the ability to speak, swallow, move and breathe. Patients and some advocacy groups had urged the FDA to approve the drug, as there are limited treatments available for ALS, and the agency granted priority review in December.ĪLS, also known as Lou Gehrig's disease, affects as many as 30,000 people in the United States.
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