Insights on the FDA’s 2024 Guidance: Considerations for the Development of CAR-T Cell Products
Authors:
Dr Dmitry Zamoryakhin
Principal Medical Consultant
Introduction
Eight CAR-T products have been approved in the US thus far, and CAR-T products account for approximately 55% of all Gene Therapy INDs. A combination of factors, including manufacturing challenges, clinical development timelines and differences in regional regulatory requirements, complicate the development of CAR-T products.
FDA has recently published a guidance for industry providing considerations for the development of CAR-T cell products, with a focus on ex vivo modified products for oncology indications, including solid tumours and hematologic malignancies. The recommendations in this document are based on FDA’s experience with the development of CAR-T cell products. The guidance should be read in conjunction with FDA’s existing guidance documents on cell and gene therapies, such as the guidance for industry on expected CMC documentation for INDs, preclinical assessment, comparability assessments, early-phase clinical trials and long-term follow-up. This white paper summarizes and interprets FDA’s January 2024 guidance for ex vivo–manufactured CAR-T cell products. The guidance is nonbinding and reflects FDA’s current thinking; sponsors may use alternative approaches if they meet applicable regulations…