FDA advisers to discuss ALS drug at Wednesday meeting

David Lim and Katherine Ellen Foley, Politico, March 29, 2022

A highly anticipated meeting of FDA’s advisory committee on neurological drugs is convening Wednesday to discuss a drug that aims to treat amyotrophic lateral sclerosis, or ALS, from Amylyx, Katherine reports.

Background: Amylyx has completed Phase II clinical testing of its drug, for now called AMX0035. The drug slowed disease progression, though data showing the drug helped patients live longer wasn’t statistically significant. But a longer-term continuation of the study showed the drug extended patients’ lives by about six months.


Doubting the data: Committee members will vote on whether the drug is effective at treating ALS. In presentation materials released Monday, FDA said the findings from Amylyx’s Phase II study “do not translate to robust support for a drug effect in patients with ALS.” The agency’s presentation cites issues with the clinical trial design, including questions over how Amylyx counted deaths during the trial, trouble randomizing patients and potential accidental unblinding because of the drug’s bitter taste.

A repeat of Aduhelm? FDA’s neurological drug advisory committee hasn’t met since it discussed the controversial Alzheimer’s drug Aduhelm, which it voted against approving. Both Aduhelm and AMX0035 are intended for fatal neurological diseases that have limited treatment options. FDA told both drugs’ manufacturers they needed more data before approval. And both drugs have strong patient advocates behind them, who have pressed both FDA and Congress to give patients access to the drugs.

“Did the FDA schedule an advisory committee meeting to placate patient advocates and other political pressure in the hope that the advisory committee would vote against it? Or is the FDA really considering approving it?” said Diana Zuckerman, the president of the National Center for Health Research, a nonprofit organization. She noted that a negative vote from the independent panel could give cover to the agency if it ultimately does not approve the drug.


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