FDA Issues Guidance on CAR-T Cell Product Development
The final guidance provides specific recommendations for CMC, pharmacology, toxicology, and clinical study design for CAR-T cell products.
Update March 12, 2024: The agency's webinar, hosted on March 7, 2024, is now available on FDA's website.
Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com
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.
The Office of Therapeutic Products in FDA’s Center for Biologics Evaluation and Research is hosting a public webinar on the guidance on Thursday, March 7, 2024 at 1:00 PM ET. More information can be found on FDA’s website.
Source: FDA
Guidance on Quality Culture Standards
June 3rd 2025Matt Cushing, VP of Quality and Science, Nelson Labs, and Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, a Nelson Labs Company, discuss PDA/ANSI Standard 06-2025: Assessment of Quality Culture Guidance Documents, Models, and Tools, which was published in February 2025.
Ushering in Industry 4.0 with the IDMO Model to Solve the CGT Manufacturing Bottleneck
May 28th 2025Global scale-up requires developing replicable processes that work the same no matter where they are performed. This can be accomplished with smart factories that utilize fully automated manufacturing platforms.
