For Immediate Release:

The U.S. Food and Drug Administration today announced 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 agency’s more flexible approach has been, and is expected to continue to 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.  

“Regulatory flexibility must be tailored for cell and gene therapies,” said FDA Commissioner Marty Makary, M.D., M.P.H. “These are common-sense reforms that will address the unique characteristics of cell and gene therapies and foster more innovation.”

Over the last decade, the FDA’s Center for Biologics Evaluation and Research (CBER) has approved close to 50 CGTs. The transformative potential of these therapies has captured the imagination of the patient community and ignited product development.

“There has been tremendous enthusiasm amongst product developers resulting in an explosive growth of cell and gene therapy submissions, many of which target serious or life-threatening conditions with an unmet medical need,” Vinay Prasad, M.D., M.P.H., Chief Medical and Scientific Officer and Director of the FDA’s Center for Biologics Evaluation and Research. “CBER is eager for stakeholders to know that our effectiveness at exercising greater regulatory flexibility around chemistry, manufacturing and control requirements furthers innovative product development.”  

CBER has historically 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. While there is a long history of making concerted efforts to help sponsors meet standards to assure product safety, purity and potency, the application of flexibilities has not always been fully clear to stakeholders.  

“CBER is proactively communicating about regulatory flexibilities that were previously applied case-by-case to select CGT therapies. By communicating these approaches broadly, we aim to expedite product development across the CGT field,” said Vijay Kumar M.D., Acting Director, Office of Therapeutic Products in the FDA’s Center for Biologics Evaluation and Research. “It is vital that every sponsor, no matter the CBER reviewer team they engage with, understand what types of regulatory flexibility may be scientifically acceptable.”

Given the rapid scientific developments witnessed during the decade, it is a high priority for both the agency and the administration 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.  

In June, the FDA hosted a Cell and Gene Therapy Roundtable, bringing together leading experts to discuss advancing the field of cell and gene therapies for patients and innovators.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.