New FDA draft guidance on potency assurance for cellular and gene therapy products

Cell and gene therapies (CGTs) have shown impressive clinical benefits for patients worldwide, with 89 approved therapies reported globally as of second-quarter 2023.1

These therapies represent ground-breaking advancements, offering potentially curative treatments for patients who previously had few options. However, the development of these CGTs has frequently encountered challenges related to chemistry, manufacturing, and control considerations and drug manufacturers have expressed uncertainties surrounding regulatory requirements for ensuring consistent product quality.

This uncertainty is about to change, with new guidance from the US Food and Drug Administration (FDA) making it clear that demonstrating potency – the specific ability of a product to achieve intended results through laboratory tests or clinical data – is fundamental to securing approval for these innovative treatments.2