Patient testimony to the contrary, advisory committee members were not persuaded that PTC Therapeutics' ataluren, which failed its primary endpoint in two trials, is effective in treating nonsense mutation Duchenne muscular dystrophy.
Compelling personal testimony about the clinical benefits experienced by Duchenne muscular dystrophy (DMD) patients taking PTC Therapeutics Inc.'s Translarna (ataluren) was not enough to overcome less-than-compelling randomized clinical trial data, a US FDA advisory committee concluded Sept. 28.
Ten of 11 members of the Peripheral and Central Nervous System Drugs Advisory Committee voted that while it is possible...
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.
