The latest drug development news and highlights from our US FDA Performance Tracker.
Here's your US review and submission news in brief: The Vyondys 53 (golodirsen) saga took a surprising turn last week, as its sponsor Sarepta Therapeutics Inc.scored an approval for the Duchenne muscular dystrophy (DMD) drug from the US Food and Drug Administration after quietly appealing the complete response letter (CRL) from four months earlier.
The Center for Drug Evaluation and Research (CDER) has now approved 42 novel agents in 2019.
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.
Stay up to date on regulatory guidelines from around the world with the Pink Sheet's Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
