The US FDA’s Genetic Metabolic Diseases Advisory Committee will comprise three regular voting members and 13 temporary members, including some adcomm veterans, when it reviews Zevra Denmark’s arimoclomol for Niemann-Pick disease type C.
The first meeting of the US Food and Drug Administration’s Genetic Metabolic Diseases Advisory Committee will be heavy on temporary voting members, several of whom are veterans of the adcomm process.
Thirteen of 16 voting members at the 2 August meeting on Zevra’s Denmark A/S’ arimoclomol are temporary members, including the...
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.
