Amylyx’s ALS Drug Brings Questions On Efficacy Data Robustness To US FDA Panel

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.

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Amylyx relied on a 'questionable linearity assumption' in its primary endpoint analysis for the CENTAUR study, FDA said. • Source: Alamy

Issues with the design and statistical robustness of results from Amylyx Pharmaceuticals, Inc.’s Phase II trial of AMX0035 raise questions as to whether a single study can serve as substantial evidence of effectiveness for the amyotrophic lateral sclerosis drug’s approval, US Food and Drug Administration reviewers said.

In a briefing document released ahead of a 30 March advisory committee meeting, FDA reviewers cited a host of concerns...

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