The first treatment for the rare neurodegenerative disorder Friedreich’s ataxia relied on a single placebo-controlled efficacy trial.
Reata Pharmaceuticals, Inc. notched its first approval from its pipeline of drug candidates that activate the transcription factor Nrf2 when the US FDA approved Skyclarys (omaveloxolone) on 28 February 2023 as the first treatment for the Friedreich’s ataxia, an ultra-rare neurodegenerative disorder that is typically diagnosed in adolescence.
Reata emphasized clarity on FDA expectations for confirmatory evidence during the clinical development of omaveloxolone, which the company planned to...
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.
