FDA Releases Draft Guidance on Potency Assurance for Cellular and Gene Therapy Products

The Food and Drug Administration released a new draft guidance for industry, “Potency Assurance for Cellular and Gene Therapy Products,” on Dec. 28. In the draft guidance, the agency provides recommendations to develop a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product at all stages of the product lifecycle.

For investigational products, FDA describes how to progressively implement a strategy for potency assurance during product development and provides considerations for assuring the potency of products undergoing rapid clinical development. For licensed products, the agency describes requirements for potency assurance, including testing required for lot release. The guidance also provides examples of approaches to potency assay development that are grounded in quality risk management.

FDA stated that due to the diversity of CGT products and the product-specific nature of potency assays, the recommendations in the guidance document regarding the selection and design of potency assays are “necessarily general.” The agency may issue additional guidance documents that provide further advice about potency assays for specific classes of CGT products.

The agency is accepting comments on the draft guidance through March 27. Submission instructions are available in the Federal Register notice.