“Today’s action reflects the FDA’s commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options,” said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research (CBER). “By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on. Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible.”
This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area.
For sponsors developing genome editing therapies, it complements the agency’s Plausible Mechanism Framework, providing the scientific tools and data-sharing strategies that allow sponsors to efficiently establish the evidentiary.It also works in tandem with the FDA’s recently issued draft guidance, Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing, which recommends methods for evaluating off-target editing risks. This new draft guidance explains how sponsors can use existing public and platform knowledge to streamline regulatory submissions across multiple stages of product development.
About FDA Cell and Gene Therapy Guidance
The FDA issued this draft guidance to provide a clear framework for developers seeking to leverage existing scientific evidence and regulatory experience during product development. Rather than requiring sponsors to recreate data that already exists, the guidance explains how relevant public information, platform knowledge, manufacturing experience, and previous research findings may be used to support regulatory submissions.
The FDA cell and gene therapy guidance supports the development of a broad range of cell and gene therapy products, including advanced genome editing therapies. It complements several recent FDA initiatives designed to improve regulatory efficiency while ensuring patient safety.