The oral medication can be taken as a standalone therapy or with other treatments, according to the company, and it has been shown to slow disease progression.
But there's still some uncertainty about the drug's efficacy: Amylyx's submission for approval is based on data from a small Phase 2 trial, and the FDA's own advisory committee initially voted this spring that the data didn't show that the drug was effective, before changing its opinion this month.
"Amylyx' goal is that every person who is eligible for Relyvrio will have access as quickly and efficiently as possible as we know people with ALS and their families have no time to wait," they said.
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.
"In terms of establishing the conclusion that it's effective, we were asked to look for substantial evidence with persuasiveness and robustness and I think this one trial doesn't quite meet that bar," Dr. Kenneth Fischbeck, one of the committee members and an investigator at the National Institutes of Health, said at the March meeting. Fischbeck added that he has cared for ALS patients.
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.
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