The FDA has issued final guidance outlining how cell and gene therapy (CGT) manufacturers should establish and maintain product potency throughout development, production, and commercialization, replacing its 2011 guidance on potency testing.
Published on November 2, Potency Assurance for Cellular and Gene Therapy Productsrecommends a comprehensive, science- and risk-based strategy to ensure that each manufactured batch meets predefined potency standards before administration to patients.
Unlike an approach centered primarily on testing the finished product, the updated framework emphasizes integrating potency assurance throughout the product lifecycle, from early investigational new drug (IND) development to biologics license application approval and post-approval monitoring.
The FDA recommends that manufacturers establish a Potency Assurance Strategy combining controls over raw materials, manufacturing processes, and laboratory testing. Together, these measures are intended to manage variability and demonstrate that each batch possesses the biological activity necessary to achieve its intended therapeutic effect.
The guidance also addresses challenges specific to CGT manufacturing, including short product shelf lives, viral vector-based gene therapies, and patient-specific treatments. Such products can present particular difficulties for potency assessment because of their biological complexity, manufacturing variability, and, in some cases, limited opportunities for testing before administration.
The recommendations are intended to provide manufacturers with flexibility in selecting appropriate controls and analytical methods based on product characteristics and associated risks, rather than prescribing a single testing approach for all CGT products.
“Patients who receive cellular and gene therapies are often battling serious or life-threatening conditions, such as cancers and rare diseases, and they deserve the assurance that the product administered to them will perform as intended,” said Karim Mikhail, director of the FDA's Center for Biologics Evaluation and Research, in a November 2 news release.
For developers, the guidance places greater emphasis on demonstrating potency through a coordinated manufacturing and quality-control strategy rather than relying solely on an individual release assay. This could influence how companies design manufacturing processes, establish analytical methods, and document product consistency as therapies advance toward commercialization.
The final guidance represents the FDA's current recommendations for meeting existing potency requirements, rather than introducing a new approval pathway. Its publication provides manufacturers with an updated framework for addressing one of the central quality challenges in CGT development: demonstrating that complex biological products consistently retain their intended therapeutic activity.
The FDA will host a webinar on December 10 to discuss implementation of the guidance.
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