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

 
• By Sue Sutter

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 with the CENTAUR trial, including a modest p-value for...

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