In wide-ranging meeting, majority of panelists believe volanesorsen's surrogate marker will correlate to clinical outcomes such as reduced pancreatitis.
The US FDA's Endocrinologic and Metabolic Drugs Advisory Committee showed a degree of faith in the surrogate endpoint of percent change in fasting triglyceride (TG) at three months for Akcea Therapeutics Inc.'s Waylivra (volanesorsen) with a 12 to 8 vote in favor of approval for the treatment of familial chylomicronemia syndrome (FCS).
Panelists who voted "yes" during the May 10 meeting generally felt that the surrogate would correlate to clinical outcomes of...
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.
