The much-awaited and much-debated drug for Lou Gehrig’s disease won U.S. approval Thursday, despite the questionable data presented by makers.
In a long-sought victory for patients, the Food and Drug Administration approved the ALS drug from Amylyx Pharmaceuticals based on results from one small, mid-stage study in which patients with the disease appeared to progress more slowly and survive several months longer.
Typically, the FDA requires two large studies or one study with “very persuasive” survival results for approval. “This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” FDA’s neurology drug director Dr. Billy Dunn said in a statement.
Named Relyvrio, the drug is the third U.S.-approved medicine for amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking, and swallowing. About 20,000 people in the U.S. are living with the disease.
The approval of the drug has now become a key point in the debate about the FDA, including how flexible it should be when reviewing drugs for deadly diseases and how much weight it should give to appeals from patients and other outside voices.
“I think it demonstrates the FDA’s ability to be facile and I think it demonstrates a lot of tenacity on the part of ALS patients and advocates,” said Dr. Catherine Lomen-Hoerth, an ALS specialist at the University of California San Francisco. “The company really tried to do everything possible to get this potentially promising drug out to patients.”