ALS drug wins FDA approval despite questionable data
U.S. health officials have approved a much-debated drug to treat the deadly illness known as Lou Gehrig's disease
WASHINGTON (AP) — A much-debated drug for Lou Gehrig’s disease won U.S. approval Thursday, a long-sought victory for patients that is likely to renew questions about the scientific rigor behind government reviews of experimental medicines.
Amylyx Pharmaceuticals said the Food and Drug Administration approved the drug for adults with amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing. It is only the third U.S.-approved medicine for ALS.
The FDA based the approval on one small, mid-stage study in which patients appeared to progress more slowly against the debilitating disease. Typically, the FDA requires two large studies or one study with “very persuasive” results for approval.
The FDA's review has become a flashpoint in broader debates about the regulatory agency, 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.
