FDA Approves ALS Drug Whose Study Was Partly Funded By Ice Bucket Challenge

A new treatment for amyotrophic lateral sclerosis, or ALS, has been approved by the US Food and Drug Administration. CNN reports: The FDA announced approval of Relyvrio, developed by Amylyx Pharmaceuticals, on Thursday. The oral medication works as a standalone therapy or when added to other treatments, according to the company, and it has been shown to slow disease progression. 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. 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. 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. 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. Amylyx is now continuing to study its safety and efficacy in a Phase 3 trial. 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. One key difference between the FDA advisory committee's March and September meetings is that in the later meeting, Amylyx indicated that if the drug was approved but its Phase 3 trial results fail to confirm the drug's benefits, the company would consider withdrawing the drug from the market, Lynch said. She added, however, that the company didn't say specifically what it would view as a failure. "So at the vote, the advisory committee members switched, and most of them said, 'Yes, we are now convinced that this product should be approved.' And when they were asked why they changed their minds, some of them said, 'Well, the company said they would withdraw,'" she said. "And they were also convinced by patients' testimonies that they very much want to try this drug." But overall, the FDA's approval was based on Phase 2 trial data, which, Lynch said, may send a message to other pharmaceutical companies that they don't need robust Phase 3 trial data to get products on the market. Although people with ALS want access to this promising drug, there are concerns that such a message could open the door more broadly to the approval of medications that have not been proved to work, says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania. "The FDA could later withdraw those products if needed, she said, but doing so without voluntary company agreement is 'a huge pain' and often requires a very lengthy process," reports CNN.

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