Weighing Vertex’s evaluation of theoretical risks of off-target editing with the gene therapy’s strong clinical results in sickle cell disease, advisory committee says in silico and cellular analyses were sufficient but suggests closer monitoring of potential on- and off-target effects postapproval.
Vertex Pharmaceuticals Incorporated’s nonclinical analyses for potential off-target effects of its sickle cell disease gene therapy exa-cel are sufficient, particularly given the robust clinical efficacy seen in a disease with high unmet need, a US Food and Drug Administration advisory committee said on 31 October.
However, the Cellular, Tissue and Gene Therapies Advisory Committee said long-term follow-up of patients receiving the treatment, which is made...
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.
