The U.S. Food and Drug Administration today issued draft guidance to help developers bring promising cell and gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.
When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge — including chemistry, manufacturing and controls (CMC) data, nonclinical study results and clinical information — to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.
“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.”
What the Guidance Proposes
The draft guidance supports development of a wide range of cell and gene therapy products, including those that use genome editing. Sponsors may leverage prior knowledge across chemistry, manufacturing and controls, nonclinical study results and clinical information to reduce redundant testing. In all cases, a scientific rationale must demonstrate the applicability of the data being leveraged to the specific product and development context.
Complementary FDA Actions
For sponsors developing genome editing therapies, the guidance complements the agency’s Plausible Mechanism Framework, providing scientific tools and data-sharing strategies to efficiently establish the evidentiary foundation this approach requires. It also works in tandem with the FDA’s recently issued draft guidance on safety assessment of genome editing using next-generation sequencing, which recommends methods for evaluating off-target editing risks.
“By outlining how sponsors can intelligently build upon existing nonclinical, clinical, and manufacturing knowledge, we can meaningfully streamline development programs and lower the cost barriers that have historically slowed access to these potentially life-changing treatments,” said Vijay Kumar M.D., Acting Director of the Office of Therapeutic Products in CBER.
Encouraging Early Engagement
The FDA encourages sponsors to engage early in product development, even before submitting an IND application, through INTERACT and pre-IND meetings to discuss their specific development strategies. The draft guidance is available for public comment for 90 days following publication in the Federal Register.
Source: FDA Press Release
