FDA guidance to help accelerate cell and gene therapies

FDA issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.

According to a press release from the FDA, the guidance will outline how sponsors can use publicly available information and establish platform knowledge, including chemistry, mmanufacturing and controls data (CMC), nonclinical study results, and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.

Karim Mikhail, B. Pharm, MS, Acting Director of the Center for Biologics Evaluation and Research (CBER), commented, "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.”

“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."

The FDA’s press release noted that the guidance supports development of an array of cell and gene therapy products, including those using genome editing, and is part of a broader set of complementary FDA actions in this area.

Sponsor guidance

Regarding the development of genome editing therapies, the draft guidance complements the agency's Plausible Mechanism Framework, which facilitates the development of individualized therapy for specific genetic, cellular, or molecular disease-causing abnormalities and provides the scientific tools and data-sharing strategies to efficiently establish the evidentiary foundation this approach requires.

In addition, the guideline works with another draft guidance titled Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing, which, according to the press release, recommends methods to evaluate off-target editing risks. This new draft guidance explains how sponsors can use existing knowledge to streamline regulatory submissions across multiple stages of product development. Together with the FDA’s other recent actions, developers of cell and gene therapies have a clear, science-based path for building on existing knowledge and experience while maintaining the standards for patient safety.

According to Vijay Kumar, MD, acting director of the Office of Therapeutic Products in CBER, “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.”

He continued, “For patients with serious or rare diseases, time matters. We encourage developers to engage with this guidance because their perspectives are essential to shaping a regulatory framework that works for everyone, and most importantly, for the patients who are counting on us."

Sponsor assistance

Denise Gavin, PhD, director of the Office of Therapeutic Products’ Office of Gene Therapy – CMC, pointed out that sponsors can have close guidance available to them to implement the guideline and build on previous knowledge.

The FDA noted, “In all cases, sponsors should provide a scientific rationale demonstrating the applicability of the data being leveraged to their specific product and development context. The FDA encourages sponsors to engage early in product development, even before submitting an Investigational New Drug (IND) application, for example, through Initial Targeted Engagement for Regulatory Advice on CBER/CDER Products (INTERACT) and pre-IND meetings, to discuss their specific development strategies.”

The public can comment on the draft guidance, and this must be submitted within 90 days of publication in the Federal Register at Regulations.gov. The agency will review and consider comments received before the guidance is finalized.