Multi-luminance mobility testing was developed by firm to measure functional vision across a range of lighting levels in patients with RPE65 mutations; advisory committee also will consider minimum patient age and clinical stage of disease appropriate for subretinal injections with voretigene.
A US FDA advisory committee will opine on use of a novel clinical outcome measure, appropriate patient population and long-term safety and efficacy for Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec), a gene therapy for vision loss, at the Oct. 12 review of the product.
The Cellular, Tissue and Gene Therapies Advisory Committee will review Spark's biologics license application (BLA) for voretigene (AAV2-hRPE65v2) in patients...
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.
