The latest disappointment in amyotrophic lateral sclerosis research brought crushing news for patients and physicians, forcing the community to consider just what to do with an approved medicine that doesn’t appear to work.
Amylyx Pharmaceuticals’ announcement Friday that its drug, marketed as Relyvrio, failed to beat placebo in a pivotal study marks “a sad day for us, but we have to believe the data,” said Mary Catherine Collet, an ALS advocate. “These are hard scientific facts that we have to face, and I appreciate that they’re not trying to spin us like some cheerleading practice.”
The question now is whether Relyvrio, approved in 2022, should be removed from the market entirely — and whether the Food and Drug Administration made a mistake by authorizing it in the first place. After years of pressure from patient advocates, the agency has taken a more flexible approach to medicines for devastating, incurable diseases like ALS. But the case of Relyvrio, which made $381 million in sales and $49 million in profit for Amylyx last year, has underlined questions about the limits of regulatory leniency.