FDA Makes Public Information on Requirements for Cell and Gene Therapies

The FDA has announced that it is “sharing information about the agency’s flexible approach to overseeing chemistry, manufacturing, and control (CMC) requirements for cell and gene therapies (CGT).”

The hope is that sharing this information will be “helpful in expediting product development and will help guide the FDA’s evaluation of development strategies in preparation for a Biologics License Application (BLA) submission.” The FDA’s Center for Biologics Evaluation and Research (CBER) has approved near 50 CGTs over the last decade.

Historically, CBER has had “similar CMC expectations across products, including small-batch products such as CGTs. CGTs are inherently complex biologic products, often individualized for patients, and may need sophisticated manufacturing under particular time constraints. CBER has leveraged its growing experience with CGT products to identify and implement regulatory flexibilities allowed under FDA’s regulations that accommodate the unique characteristics of these innovative therapies, while maintaining rigorous quality standards through appropriate control measures.”

This step aims to “remove barriers and perceived misconceptions that stand in the way of expedited product development. These flexibilities will enable progress while not compromising or undermining the FDA’s ability to assure safety, purity and potency of a product, or weaken the FDA’s dependency on understanding the benefits and risks of both the specific therapy and the disease context.”