US agency’s advisory committees will consider whether ongoing trial of exon 45- and 53-skipping compounds for Duchenne muscular dystrophy should allow use of in-dwelling ports to aid infusion.
Eight months after the controversial approval of Sarepta Therapeutics Inc.’s Exondys 51 (eteplirsen), the US FDA is convening its external advisors to weigh changes in an ongoing trial involving two of the company’s investigational Duchenne muscular dystrophy (DMD) treatments.
On May 18, FDA’s Pediatric Advisory Committee and Pediatric Ethics Subcommittee will meet to consider whether in-dwelling drug ports should...
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.
