Advisory committee will consider how approval of SRP-9001 now might affect completion of the 52-week endpoint in EMBARK, a fully enrolled trial with two-thirds of the subjects coming from the US. Sarepta says all study subjects will have guaranteed access to the DMD gene therapy by end of September, making the risk of dropout ‘extremely low.’
The potential impact of an accelerated approval for Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy gene therapy SRP-9001 (delandistrogene moxeparvovec) on completion of the randomized, Phase III EMBARK trial is among the key issues on which the US Food and Drug Administration seeks advisory committee input.
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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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