The FDA has issued final guidance outlining a science- and risk-based approach to potency assurance for cell and gene therapy (CGT) products, replacing its previous 2011 recommendations and setting out expectations across the product lifecycle.
The US Food and Drug Administration (FDA) has issued final guidance to help manufacturers of cellular and gene therapies establish comprehensive strategies for ensuring product potency, from early development through commercialisation and post-market monitoring.
The guidance, Potency Assurance for Cellular and Gene Therapy Products, supersedes the FDA’s 2011 guidance on potency testing for CGT products. It provides recommendations for implementing a comprehensive, science- and risk-based strategy to ensure that products meet potency standards.
The recommendations cover controls for raw material variability, testing during manufacturing and laboratory assays to confirm that each batch is biologically active in a way that matches its intended therapeutic effect. The guidance also addresses specific challenges associated with CGT manufacturing, including products with short shelf lives, viral vector-based gene therapies and patient-specific treatments.
The FDA recommends integrating potency considerations at every stage of product development and commercialisation, from early-phase investigational new drug (IND) submissions through biologics licence application (BLA) approval and post-market verification.
The guidance provides manufacturers with a clear, predictable roadmap for CGT development, supporting faster access to safe and effective therapies for patients, many of whom have limited treatment options. Karim Mikhail, Director of the FDA’s Center for Biologics Evaluation and Research, emphasised that patients “deserve the assurance that the product administered to them will perform as intended”, adding that innovation must be matched by the highest standards of product quality and patient safety.
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