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Amylyx’s ALS Drug Should Wait For Phase III Results, US FDA Panel Says

Executive Summary

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.

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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.

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