Guidance from the FDA on Developing CAR-T Cell Products
The final guidance offers detailed recommendations for CMC, pharmacology, toxicology, and clinical study design for CAR-T cell products.
The FDA released a finalized guidance on Jan. 30, 2024, which offers insights into the creation of chimeric antigen receptor (CAR) T-cell products. The FDA states that CAR T-cell products are a type of human gene therapy. These products involve genetically modifying T cells to recognize a specific target antigen for therapeutic purposes.
This guidance offers sponsors valuable information to assist in the development of CAR T-cell products. It includes specific recommendations on various aspects such as chemistry, manufacturing, and controls (CMC), pharmacology and toxicology, and clinical study design. We also discuss considerations for autologous or allogeneic CAR T-cell products, along with analytical comparability studies.
The recommendations provided can be applied to other genetically modified lymphocyte products, such as CAR natural killer cells and T-cell receptor-modified T cells. If sponsors have specific considerations regarding these highly specialized topics, the FDA advises reaching out to the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) before submitting an investigational new drug application.
The guidance provides detailed information on CAR-T cell design and development, covering aspects such as CAR construct, vectors, cellular starting material, and fresh or cryopreserved final products. The text covers vector manufacturing and testing, as well as the collection, handling, and testing of cellular starting material. The guidance document also covers the management of manufacturing changes and the evaluation of comparability throughout the CAR T-cell product lifecycle, including both single-site and multi-site CAR T cell manufacturing.
Join us for a public webinar hosted by the Office of Therapeutic Products in FDA's Center for Biologics Evaluation and Research. The webinar will cover important guidance and take place on Thursday, March 7, 2024 at 1:00 PM ET.
Source: FDA
