May 1, 2024 - FDA Clarifies Approach to Genomic Alterations in Animals

Today, the U.S. Food and Drug Administration took important steps to advance the FDA's regulatory process for intentional genomic alterations (IGAs) in animals. The updated guidance documents underscore the agency's commitment to further modernize its approach to evaluating and supporting the development of innovative animal and veterinary products to increase regulatory flexibility, predictability and efficiency. In addition, the agency established a memorandum of understanding (MOU) with the U.S. Department of Agriculture (USDA) to clarify roles and responsibilities for regulation of IGAs in animals.

"We recognize that innovations in animal biotechnology offer tremendous opportunities for advancing human and animal health, and that we as an agency need to keep our regulatory approach current with the evolution of the science," said Tracey Forfa, director of the FDA's Center for Veterinary Medicine. "These updated guidance documents demonstrate our commitment to facilitating innovation while ensuring product safety. These technologies hold great promise for many uses and public and animal health benefits, such as animal disease resistance, control of zoonotic disease transmission, improved animal husbandry, and increased food production and quality."

IGAs in animals can be made using modern molecular technologies, which may include random or targeted DNA sequence changes, including nucleotide insertions, substitutions, or deletions, or other technologies that introduce specific changes to the genome of the animal. IGAs in animals have many different intended uses, including applications in human health (e.g., reduced allergenicity; "biopharm" animals that produce substances, generally in their milk or eggs, for use in the production of human therapeutics; animals used to model human disease), in improved animal health, well-being, and husbandry practices (e.g., disease resistance, heat tolerance), and in enhanced production and food quality (e.g., faster growth, feed efficiency, nutritional benefits).

The two documents released today, final Guidance for Industry (GFI) #187A, "Heritable Intentional Genomic Alterations in Animals: Risk-Based Approach," and draft revised GFI #187B, "Heritable Intentional Genomic Alterations in Animals: The Approval Process," are intended to help industry understand the existing statutory and regulatory requirements as they apply to IGAs in animals and to inform the public about the FDA's process for regulating them.

The guidance documents align with the goals of the FDA's Animal and Veterinary Innovation Agenda (AVIA), which aims to foster development of safe, innovative products and implement smart, risk-based approaches to regulating modern animal and veterinary products, while protecting human, animal and environmental health.

The FDA first issued final guidance on the regulation of certain IGAs in animals in 2009. In January 2017, due in part to evolution in the science of IGA technologies, the agency clarified the scope of its guidance to include animals with intentionally altered genomic DNA developed through use of genome editing technologies, in updated draft GFI #187, "Regulation of Intentionally Altered Genomic DNA in Animals."

Final GFI #187A outlines the FDA's overarching policy for regulating all IGAs in animals and describes the agency's risk-based regulatory approach to the oversight of heritable IGAs. This final GFI also describes how the FDA determines when it is appropriate to exercise enforcement discretion over certain products. The types of products that may be appropriate for enforcement discretion have expanded since the 2017 draft to include IGAs in food-producing animals, such as SLICK cattle, in circumstances including where they have been altered to be equivalent to animals of the same species that already exist and have a history of safe use. In a complementary document, Draft GFI #187B, we describe how the FDA approval process applies to heritable IGAs in animals.

The FDA is committed to collaborating with its regulatory partners across the federal government to ensure that approved IGAs are safe for the animal, safe for people, and that the product does what it claims to do. On April 18, 2024, the FDA and USDA signed an MOU that applies to the FDA's activities involving IGAs in animals that are also subject to certain USDA authorities. The MOU establishes policies and procedures to enhance the exchange of information between the agencies, describes the regulatory roles of the agencies, and promotes coordination of regulatory responsibilities in a manner that will enable an efficient, seamless regulatory process. It does not change or add regulatory requirements for developers. Additional details can be found here.

The FDA encourages developers of IGAs in animals to approach the agency early in their research and development phase to discuss the specific risk profile of the proposed product and the appropriate pathway for commercialization. For products that qualify, the agency recommends participation in the Veterinary Innovation Program (VIP), which is intended to assist developers of innovative veterinary products, including certain IGAs in animals, by providing intensive technical and programmatic assistance throughout the approval process in order to make it as efficient as possible.

The FDA will continue to work with its domestic and global partners as the agency implements an appropriate, risk-based regulatory approach based on sound science that facilitates the development of safe innovative products and ensures consumer confidence. This action also aligns with the goals of Executive Order 14081, Advancing Biotechnology and Biomanufacturing Innovation for a Sustainable, Safe, and Secure American Bioeconomy.

The agency is requesting public comment on GFI #187B for 90 days starting on May 2, 2024. To ensure the FDA considers comments before it begins work on the final version of GFI #187B, members of the public should submit comments by July 31, 2024.

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