CAR-T Therapies: Failure To Meet Release Specifications May Not Bar Patient Use
Some patients in Novartis' pediatric leukemia pivotal trial for tisagenlecleucel (CTL019) received out-of-spec product following the sponsor's consultation with US FDA, and the same thing might occur in the commercial setting.
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Other CAR-T sponsors can only hope for the same kind of US FDA advisory committee success that Novartis enjoyed with tisagenlecleucel, an outcome that reflected the sponsor's experience and preparation, a lack of major show-stopping safety or product quality concerns, and robust efficacy in a narrow pediatric population.
US advisory committee's favorable views of Novartis' tisagenlecleucel-T suggest road map for other CAR-T developers to follow; with an Oct. 3 user fee deadline, pediatric leukemia agent is on path to becoming first chimeric antigen receptor T-cell therapy in US.
Rather than wait for regulatory approval prior to commercial-scale manufacturing, Novartis is proving out its global commercial manufacturing process for a CAR-T cell therapy in a Phase II clinical trial as questions remain on how to translate the manufacturing process from the research laboratory to the factory.