In approving Amylyx Pharmaceuticals, Inc.’s Relyvrio (formerly AMX0035) for amyotrophic lateral sclerosis on 29 September, the US Food and Drug Administration decided it did not first need to see the results of the ongoing Phase III PHOENIX trial.
Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’
The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.
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