CVM GFI #251 - Heritable Intentional Genomic Alterations in Animals of Food-Producing Species for Use as Models of Disease January 2025

GUIDANCE DOCUMENT

Submit Comments by 03/10/2025

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All written comments should be identified with this document's docket number: FDA-2024-D-4778

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• Docket Number: FDA-2024-D-4778
• Issued by: Center for Veterinary Medicine

Intentional genomic alterations (IGAs) in animals are made using modern molecular technologies, which include rDNA technologies and genome editing technologies such as CRISPR.  Some IGAs are in animals of food producing species (such as pigs and rabbits) that serve as models of human or animal disease for use in research and development of therapeutic products.

Guidance for Industry (GFI) #187A, “Heritable Intentional Genomic Alterations in Animals: Risk-Based Approach,” describes FDA’s general, risk-based approach to regulating IGAs in animals. GFI #187A generally discusses FDA’s approach to regulation of certain IGAs in animals for which the agency may not expect submission of an approval application. 

GFI #251 provides information that is more specific to FDA’s policy regarding heritable IGAs in animals of food-producing species, such as swine and rabbits, that are intended to be marketed for use as models of human or animal disease in biomedical research under contained and controlled conditions (IGAs in animal models of disease).  This research may be basic research of general applicability (e.g., understanding the underlying pathophysiology of a disease or disease processes) or it may be research or pre-clinical testing for a particular medical product that may support an application for product approval (e.g., preclinical trials of safety or effectiveness in altered animal models closely resembling human disease).  The guidance describes the conditions under which we generally may not expect developers of IGAs in animal models of disease to submit an application to FDA’s Center for Veterinary Medicine (CVM) or to get our approval before marketing their animals following CVM’s prior review of risk factor data.

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