FDA issues final potency guidance for cell and gene therapy makers
The FDA issued final guidance telling cell and gene therapy makers how to plan and test for potency. It replaces the agency's 2011 potency guidance. It is not an approval of any treatment.
The U.S. Food and Drug Administration on Monday issued final guidance for makers of cellular and gene therapy products on how to build a plan to make sure each product meets potency standards.
The guidance recommends what the FDA calls a Potency Assurance Strategy. Manufacturers would test and verify, before a product reaches a patient, that every batch meets a defined standard for doing what the product is meant to do therapeutically.
The final guidance replaces the FDA's 2011 potency testing guidance for these products. The FDA said it covers the full lifecycle, from early-phase investigational submissions through approval and post-market monitoring. Topics include controls for raw material variability, testing during manufacturing, and lab tests that confirm each batch is biologically active.
It also addresses products with short shelf lives, viral vector gene therapies and patient-specific therapies.
The guidance is a set of recommendations for manufacturers. It is not an approval of any treatment and does not show that any therapy works. The FDA said it will host a webinar on Dec. 10 to help stakeholders understand and apply it.