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The final guidance provides specific recommendations for CMC, pharmacology, toxicology, and clinical study design for CAR-T cell products.
FDA published final guidance on Jan. 30, 2024 that provides consideration for the development of chimeric antigen receptor (CAR) T-cell products. According to FDA, “[CAR T-cell] products are human gene therapy products in which the T cell specificity is genetically modified to enable recognition of a desired target antigen for therapeutic purposes.”
The guidance provides sponsors with information to help develop CAR T-cell products including specific recommendations regarding chemistry, manufacturing, and controls (CMC), pharmacology and toxicology, and clinical study design. Considerations for autologous or allogeneic CAR T-cell products are addressed as well as analytical comparability studies.
The provided recommendations are also applicable to other genetically modified lymphocyte products including CAR natural killer cells and T-cell receptor-modified T cells. FDA states that, to get considerations specific to these, highly specialized sponsors should communicate with the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) before submitting an investigational new drug application.
Specifically, the guidance discusses CAR-T cell design and development including CAR construct, vectors, cellular starting material, and fresh or cryopreserved final products. Vector manufacturing and testing as well as collection, handling, and testing of cellular starting material are addressed. Managing manufacturing changes and assessing comparability during the CAR T-cell product lifecycle and single-site or multi-site CAR T cell manufacturing are also discussed in the guidance document.