Concerns about the clinical trials, the manufacturing, and the preclusive nature of Sarepta's gene therapy for Duchenne muscular dystrophy raise questions ahead of the upcoming US FDA advisory committee meeting.
US Food and Drug Administration reviewers are unsure whether Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy gene therapy candidate is effective, raising questions about the product’s ability to gain accelerated approval.
And even though the agency has granted accelerated approval to several DMD drugs despite concerns about the validity of their...
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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.
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.
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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