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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Agency considered Amylyx’s plan to submit unblinded efficacy data from the ongoing Phase III trial during the ALS drug's NDA review but ultimately decided against it, citing concerns about study integrity and the small amount of data likely to be available.
Makena Sponsor Covis Seeks To Use FDA Officials’ Words, Actions On Accelerated Approval Against CDER
Company cites comments by Oncology Center of Excellence Director Richard Pazdur and Office of Neuroscience Director Billy Dunn on reasons why confirmatory trials fail and the need for regulatory flexibility. Ironically, both senior officials have publicly extracted commitments from companies to withdraw products if postmarketing trials do not verify clinical benefit.
The FDA gave full approval based on a Phase II study, without the need for Phase III confirmatory data, for Amylyx’s ALS drug Relyvrio but the $158,000 WAC could spark a debate over pricing.