Even in a rare disease with no approved treatments, the agency shows once again that without clear efficacy data, FDA has few options for approval.
Regulatory flexibility afforded to rare disease products may be able to help push them closer to US Food and Drug Administration approval than those for common diseases, but Y-mAbs Therapeutics Inc. seems to have discovered the policy has a breaking point.
Y-mAbs’ I-omburtamab only reached the application assessment and advisory committee stages seemingly because the product is intended for a rare...
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.
