FDA finalizes CAR-T and gene therapy guidance, offers support for accelerated approvals

The FDA on Monday finalized two guidance documents to help sponsors developing therapies in two crowded areas: gene therapies using genome editing and ex vivo-manufactured CAR-T cell products.

The 19-page final version of the genome editing guidance is clear about the agency’s stance on the use of the accelerated approval pathway and now includes a statement expressing the agency’s support, as FDA officials like Peter Marks and others have previously signaled:

FDA is supportive of the use of accelerated approval for GE [genome editing] products and encourages sponsors to discuss the potential eligibility of a GE product for such program, including the proposed surrogate endpoints or intermediate clinical endpoints, early in development of the clinical trial.

Other changes between the draft from March 2022 and today’s final version include clarifying the recommendations for single-use genome editing components (for example, in the manufacture of a master cell bank, “the extent of information and release testing for the GE components may be reduced”), expectations for potency assays, and considerations for nonclinical studies with respect to the potential for off-target toxicity.

The 36-page final guidance on CAR-T therapy development, meanwhile, offers specific recommendations on chemistry, manufacturing, and controls, pharmacology and toxicology, and clinical study design.

Changes from the March 2022 draft include clarifying the scope of the guidance, adding a new focus on cancer indications, recommendations for CAR-T cells manufactured using cellular starting material from patients who have received CAR-T cells previously, details on potency for CAR-T cells that express multiple transgene elements, stability studies, and clinical monitoring.

While the guidance specifically focuses on CAR-Ts, the FDA also notes that some of the information and recommendations may be applicable to other genetically modified lymphocyte products, such as CAR-NK cells or T-cell receptor modified T cells.

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