ALS advocacy groups have lobbied vigorously to convince the FDA to reconsider, especially after the agency controversial Last year, Aduhelm approved the Alzheimer’s drug despite doubts about whether it would work. Soon after, FDA officials began asking Amylyx to apply for approval using existing data.
In March, a committee of independent advisors to the FDA voted by a narrow margin that the treatment has not yet been shown to be effective, a conclusion also reached by FDA’s own reviewer. The agency then allowed Amylyx to submit more data and took the unusual step of scheduling a second independent advisory committee meeting for September 7. In a report presented there, dealer reviewers said it also considers the new data insufficient.
But Dr. Billy Dunn, director of the FDA’s office of neuroscience, told the advisory committee that “although some may reasonably argue that substantial evidence does not currently exist” to justify Justifying approval, the agency should exercise “the broadest flexibility” by considering the severity of the disease and the scarcity of available treatments.
Dr. Dunn posed a question to the company: If the treatment received approval now and was proven ineffective in a Phase 3 trial, would Amylyx voluntarily withdraw from the market? , save the agency a lengthy recall process? Mr. Klee said the company would.
That commitment by Amylyx, plus emotional testimony from patients and doctors, convinced more advisory committee members at the September meeting, where the vote in favor of approval was seven to two.
One of those who voted against the approval, Dr Kenneth Fischbeck, a distinguished investigator with the National Institutes of Health, said the company could offer the therapy free of charge to patients in the meantime. better evidence, but “I don’t think it meets the standard of proof to allow them to sell drugs. ”
Mark Weston, a member of the ALS advisory committee, said he was disappointed that the new information the company presented was not more robust. “I was hoping for something more,” he said. But Mr Weston, who earlier in the meeting named ALS patients who have died since the hearing in March, said he voted yes because “I can’t separate my thoughts on that from my own.” my thoughts on unmet need.”