FDA Approves ALS Treatment Despite Questions About Effectiveness

Author: Yuvi September 30, 2022

FDA Approves ALS Treatment Despite Questions About Effectiveness

ALS advocacy groups campaigned vehemently to persuade the FDA to reconsider, especially after the agency’s controversial approval last year of the Alzheimer’s drug Aduhelm despite doubts about whether it worked. Soon after, FDA officials began suggesting that Amylyx submit an application for approval using existing data.

In March, a committee of independent advisers to the FDA voted by a narrow margin that the treatment had not yet been shown to be effective, a conclusion also reached by the FDA’s own reviewers. The agency then allowed Amylyx to submit more data and took the unusual step of scheduling a second independent advisory committee meeting on Sept. 7. In a report presented there, agency reviewers said they also considered the new data insufficient.

But Dr. Billy Dunn, director of the FDA’s office of neuroscience, told the advisory committee that “although some might reasonably argue that substantial evidence does not currently exist” to justify approval, the agency should exercise “the broadest flexibility” by also considering the seriousness of the disease and the dearth of available treatments.

Dr. Dunn posed a question to the company: If the treatment received approval now and was shown to be ineffective in the Phase 3 trial, would Amylyx voluntarily withdraw it from the market, saving the agency a lengthy recall process? Mr. Klee said the company would.

That commitment by Amylyx, plus emotional testimony from patients and doctors, persuaded more advisory committee members at the September meeting, where the vote favoring approval was seven to two.

One of those who voted against approval, Dr. Kenneth Fischbeck, a distinguished investigator for the National Institutes of Health, said the company could provide the therapy to patients for free while waiting for better evidence, but “I don’t think it’s met the standard of evidence to allow them to sell the drug ”

Mark Weston, a member of the advisory committee who has ALS, said he was disappointed that the new information the company presented wasn’t stronger. “I was hoping for something more,” he said. But Mr. Weston, who earlier in the meeting named ALS patients who had died since the March hearing, said he voted for approval because “I can’t decouple my thoughts about that from my thoughts about the unmet need.”

30 September, 2022, 2:37 am

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