In a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.
PTC Therapeutics, Inc. sees a year full of regulatory milestones ahead in 2024 following its biologics license application for Upstaza, which could become the first gene therapy delivered directly into deep gray matter of the brain.
PTC announced the submission of the Upstaza BLA for the rare genetic central nervous system disorder aromatic L-amino acid decarboxylase...
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.
