Amylyx’s ALS Pledge Vs. FDA’s Obscure Withdrawal Authority: Which Holds More Power?
Office of Neuroscience Director Billy Dunn touted FDA’s ability to withdraw approval for AMX0035 if subsequent data indicate a lack of substantial evidence of effectiveness. However, this provision appears to have been infrequently used and involves a potentially time-consuming hearing.
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Amidst bipartisan congressional pressure to approve ALS drugs, Billy Dunn, head of FDA’s neuroscience division, told advisory committee members that the law ‘demands’ flexibility in review of Amylyx’s AMX0035, and he came prepared with the citations.
In switching their votes from the first panel review in March, advisory committee members cited supportive additional analyses of a survival benefit, framing of the issues in the context of unmet need and regulatory flexibility, and the company’s statement that it would withdraw AMX0035 if the Phase III PHOENIX trial does not succeed.