Advisory committee discussion on the adequacy of Vertex’s analyses, using in silico and cellular methods, of off-target alterations with the gene therapy for sickle cell disease could help guide nonclinical assessment of future products made with CRISPR/Cas9-mediated gene editing.
A US Food and Drug Administration advisory committee review of Vertex Pharmaceuticals Incorporated’s exa-cell gene therapy for sickle cell disease will help inform how potential off-target effects should be assessed for future products produced by genome editing.
On 31 October, the Cellular, Tissue and Gene Therapies Advisory Committee will discuss Vertex’s exagamglogene autotemcel (exa-cel) for treatment 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.
