THURSDAY, March 31, 2022 (HealthDay News) — In a close vote, an advisory panel for the U.S. Food and Drug Administration decided not to recommend the approval of an experimental drug for amyotrophic lateral sclerosis (ALS).
The panel’s decision had been closely watched, with patient advocacy groups lobbying hard for fast-track approval. However, on Wednesday, the panel ruled 6-4 that data from a single, midstage study sponsored by Amylyx Pharmaceuticals were not enough to prove the company’s drug makes a meaningful difference in patients’ lives.
“I think it would be a disservice to the patients and their families to approve a treatment that is of uncertain benefit,” Kenneth Fischbeck, M.D., of the U.S. National Institutes of Health, said during the panel meeting, according to the Associated Press. “It gets in the way of developing truly effective treatments if it turns out not to be effective.”
Fischbeck and his fellow panelists said they hoped results of a larger study, now underway, would provide more evidence on the drug, known for now as AMX0035. Even panelists who voted in favor of the drug said the decision was not an easy one. “I went back and forth during the day, but ultimately I agreed with the [drugmaker’s] primary analysis,” Dean Follman, a biostatistician with the NIH, told the AP.
Importantly, the vote is not binding, and the FDA has until the end of June to make the final call on whether to grant approval of the drug. The panel vote could be seen as confirmation of the agency’s own negative review published days prior to the panel meeting. It criticized the submitted Amylyx study for its small size, missing data, and questionable statistical analysis.
Still, the FDA has stressed the need for “regulatory flexibility” when weighing drugs for fatal diseases like ALS. And the fact that the panel vote was close could tip the agency toward an approval, given the shortage of treatment options for ALS.
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