Thoughts After Amylyx AMX0035 Committee: How Special Is ALS At FDA?
An advisory committee rejection of the proposed ALS therapy AMX0035 amounts to a vote in favor of US FDA’s approach to the review. It also amounts to an implicit rejection of the idea that ALS merits unique approaches given the devastating nature of the illness.
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Clinical trial diversity, FDA hiring authority, and inactive ingredient disclosure provisions all get slight modifications. The bill glides through committee, but not without some complaints about agency performance.
Six of 10 advisory committee members say Phase II CENTAUR trial cannot stand on its own to support approval at this time. However, patients and advocates say thousands will die if they have to wait for the Phase III PHOENIX trial to complete in 2024 before the drug becomes widely available.
Citing numerous concerns about design of the Phase II CENTAUR trial and statistical robustness of the results, agency reviewers question whether a single study provides substantial evidence of efficacy for approval of AMX0035.