Company’s prediction that Duchenne muscular dystrophy patients likely would not be able to access SRP-9001 until four months after accelerated approval eased some US FDA panelists’ concerns about interference with the ongoing confirmatory trial.
Sarepta Therapeutics, Inc.’s prediction of a four-month lag in commercial access to its gene therapy SRP-9001 (delandistrogene moxeparvovec) helped sway a US Food and Drug Administration panel vote in favor of accelerated approval for the Duchenne muscular dystrophy treatment.
At their 12 May meeting, some members of the Cellular, Tissue and Gene Therapies Advisory Committee raised ethical and practical...
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.
