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.
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...
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
New marketing authorization applications filed with the European Medicines Agency's human medicines committee (the CHMP) for review under the EU's centralized procedure. Updated monthly.
Proposal to have panelists rate the strength of efficacy and safety evidence on a numeric scale also draws support in written comments on ways to optimize the advisory committee process.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
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Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
