REGULATORY UPDATE: FDA Issues Recommendations for Cellular and Gene Therapy Products

October 06, 2021

The Food and Drug Administration released two guidance documents in September to assist in the manufacture of cellular or gene therapy products.

The guidance “Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations” finalizes recommendations in the draft guidance of the same title dated January 2020. The guidance provides FDA’s current thinking on determining sameness of human gene therapy products under FDA’s orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity. The agency intends for this guidance to assist with orphan-drug designation and orphan-drug exclusivity and the development of gene therapies for rare diseases by focusing specifically on factors that FDA generally intends to consider when determining sameness for gene therapy products.

FDA also issued the draft guidance “Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial, which includes recommendations for studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial for a single disease. FDA acknowledged that “multiple versions of a product can be studied together in a single clinical trial” and clarifies that “each version is a distinct product that is generally submitted to FDA in a separate investigational new drug application (IND)” and the objectives of early-phase clinical studies is to guide which version(s) of the product to pursue for further development in later-phase studies. This draft guidance includes recommendations for studies that evaluate multiple versions of a cellular or gene therapy product, including how to organize and structure the INDs, submit new information and report adverse events.

AABB is evaluating the draft guidance and soliciting comments for a submission and, to ensure a timely process, encourages all interested individuals to submit comments by Nov. 15 to As noted in the Federal Register notice, interested parties may submit electronic or written comments on the draft guidance directly to FDA by Dec. 29.