FDA issues new potency assurance guidance for cell and gene therapies

The U.S. Food and Drug Administration (FDA) has released a final guidance document titled “Potency Assurance for Cellular and Gene Therapy Products.” This new regulation replaces a previous version that had been in effect since 2011.

The guidance requires manufacturers to implement a comprehensive, science-based, and risk-based approach to ensure that every batch of cellular and gene therapy meets expected therapeutic standards. This process, referred to as a potency assurance strategy, must be applied throughout the entire product lifecycle, from initial development through post-market monitoring.

Karim Mikhail, director of the Center for Biologics Evaluation and Research (CBER), emphasized the importance of the update for patients facing life-threatening conditions, such as rare diseases and cancers. The goal is to ensure that these advanced therapies function as intended before they are administered in clinical settings.