Senators write US FDA commissioner seeking ‘fulsome public input’ at panel review of DMD gene therapy. The open public hearing is scheduled to run an average amount for a neurodegenerative advisory committee – but less than half the time of the OPH at the last Sarepta meeting.
In addition to carrying the weight of future development in Duchenne muscular dystrophy and potentially airing internal FDA review dynamics, the upcoming advisory committee for Sarepta Therapeutics, Inc.’s DMD gene therapy could also serve as an exhibit on the potential impact of public comments.
Sarepta’s SRP-9001 (delandistrogene moxeparvovec) will go before the US FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee on 12 May,...
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.
