Agency questions whether post hoc analyses and use of natural history subjects as an external control group in a Phase III study support effectiveness of palovarotene; cites apparent increased incidence of flare-up events as a key safety issue.
The US Food and Drug Administration expressed uncertainty about whether a Phase III study of Ipsen SA’s palovarotene demonstrates efficacy for treatment of patients with fibrodysplasia ossificans progressiva since the study failed its prespecified primary efficacy analysis and the new drug application relies on post hoc analyses to support a showing of efficacy.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee is to consider Ipsen’s application at its 28 June meeting. In a...
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.
