What ‘Confirmatory Evidence’ Does US FDA Need When Sponsors Want To Rely On A Single Trial?

Amylyx’s Relyvrio survival data, drawn from a post hoc exploratory survival analysis of randomized patients, and the primary ALS functional endpoint ‘capture distinct concepts,’ FDA states. Use of long-term survival data from the same trial as confirmatory evidence was unusual, but not unique.

Outside the box
Relying on post hoc overall survival data as confirmatory evidence suggests some outside the box thinking by US FDA. • Source: Shutterstock

The US Food and Drug Administration’s approval of Amylyx Pharmaceuticals, Inc.’s amyotrophic lateral sclerosis drug Relyvrio (sodium phenylbutyrate/taurursodiol) has brought new focus to when the agency will accept confirmatory evidence to support approval on the basis of a single adequate and well-controlled trial.

In approving Relyvrio (previously known as AMX0035), the agency said data on an overall survival benefit can serve as confirmatory...

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