Maker of much-debated ALS drug says it may stop selling it after study showed it didn't work
The maker of a much-debated drug for Lou Gehrig's disease said it failed to help patients with the fatal disease
WASHINGTON — The maker of a much-debated drug for Lou Gehrig’s disease said Friday its therapy failed to help patients in a large follow-up study, but stopped short of committing to follow through on a prior pledge to pull the drug from the U.S. market.
The Food and Drug Administration approved Amylyx Pharmaceuticals’ Relyvrio in September 2022, following a years-long advocacy campaign by patients with amyotrophic lateral sclerosis, or ALS, a fatal muscle-wasting disease.
Amylyx said Friday it would discuss its plans for Relyvrio with patients and the FDA, which “may include voluntarily withdrawing” the drug. Executives said in a statement they were “surprised and disappointed” by the results and hoped to announce their plans in the next two months.
The latest company study showed that the drug did not slow the disease compared with a dummy treatment. The drug also failed to show improvement on any secondary measures, such as muscle strength.
Amylyx’s medication is part of a string of drugs for deadly, degenerative diseases that have won FDA approval in recent years despite questionable proof that they work.
The 2022 approval was mainly based on results from one small, mid-stage study that was criticized by some of the agency’s own internal scientists. An outside committee of experts also voted against the drug initially, before being swayed to back it at a follow-up meeting requested by patients. At the time, Amylyx noted it was continuing a larger follow-up study of more than 600 patients that would provide further data on the drug.
In a highly unusual move, company executives at the meeting told FDA
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