Although the advisory committee was uncomfortable with the external control, US FDA was reassuring over the approach – which eventually secured a ‘yes’ vote from the panel on palovarotene for FOP.
Ipsen SA was facing an uphill battle last month at the Endocrinologic & Metabolism Drugs Advisory Committee's review of palovarotene for a rare bone tissue condition.
Ipsen didn’t follow FDA’s advice and conduct a placebo-controlled trial, and the study ended up failing.
The company is seeking approval of the drug for treatment of heterotopic ossification (HO) in adults and children with fibrodysplasia...
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.
