Novartis CAR-T AdComm Was Smooth Sailing, But Will That Be An Anomaly?
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.
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Lengthy safety registries, extensive premarket testing for replication competent retrovirus, and manufacturing submission advice likely; pending CBER documents informed by review and approval of Novartis' Kymriah, Kite's Yescarta, and Spark's Luxturna.
Kite Pharma may have been pipped to the post to be first with a CAR-T product at the US FDA, but it has sneaked in the first EU filing courtesy of PRIME for axicabtagene ciloleucel.
FDA officials attempt to clear up questions on regulatory expectations for qualifying raw materials for cell and gene therapy products. FDA officials advise manufacturers to conduct risk assessments, use the highest-grade materials they can find and avoid using animal-derived raw materials. Industry officials describe complexities in making these products.