FDA advisors vote 6-4 against Amylyx's ALS drug, further dimming its hope for regulatory success
In a marathon hearing Wednesday, outside FDA advisors recommended to the agency that a single randomized study did not provide conclusive proof over whether an experimental ALS drug is effective.
Panelists voted 6-4 in the negative, substantiating the agency’s concerns about the robustness of data in a Phase II study run by Amylyx Pharmaceuticals — by the slimmest possible margin. The drug, known as AMX0035, produced a statistically significant reduction on a functional rating scale, but the effect proved unconvincing to the committee.
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