Biologics

Guidance on Potency Assurance for Cell/Gene Therapies

FDA has issued final guidance recommending a comprehensive, risk-based approach to assuring the potency of cellular and gene therapy (CGT) products throughout development and commercial manufacturing, replacing its January 2011 guidance focused on potency testing.

The guidance, Potency Assurance for Cellular and Gene Therapy Products, recommends that manufacturers integrate potency testing with manufacturing process design, material controls, in-process testing and quality risk management to ensure that each released lot achieves its intended therapeutic effect.

FDA said potency assays remain essential but should be part of a broader control strategy rather than the sole means of demonstrating potency.

The document applies to investigational and licensed cellular and gene therapies regulated as biological products under Section 351 of the Public Health Service Act. It addresses potency assurance throughout the product lifecycle, including products undergoing rapid clinical development.

For investigational products, FDA recommends progressively strengthening potency assurance as development advances and manufacturers gain additional knowledge of product characteristics and manufacturing processes. The agency cautioned that inadequate potency controls could lead to clinical holds if they expose trial participants to unreasonable risks or prevent studies from generating to support an approval.

For licensed products, FDA reiterated that each lot must undergo potency testing, although alternative approaches may be permitted when they provide assurance equivalent to or greater than the applicable testing requirements.

The guidance recommends identifying potency-related critical quality attributes, evaluating manufacturing variables that could affect those attributes, and establishing appropriate controls and acceptance criteria.

FDA also recommends that lot-release testing for most CGT products include at least one phase-appropriate biological assay measuring activity related to the product's intended therapeutic effect.

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