US FDA Releases Guidance for Heritable Intentional Genomic Alterations in Animals

In May 2024, the U.S. Food and Drug Administration (FDA) Center for Veterinary Medicine (CVM) released Guidance for Industry (GFI) #187A “Heritable Intentional Genomic Alterations in Animals: Risk Based Approach” and draft GFI #187B “Heritable Intentional Genomic Alterations in Animals: The Approval Process” to clarify requirements and recommendations for developers of intentional genomic alterations (IGAs) in animals.

FDA defined IGAs in animals as “changes to an animal's genomic DNA produced using modern molecular technologies, which may include random or targeted DNA sequence changes including nucleotide insertions, substitutions, or deletions.” IGAs in animals have been used in various applications for improving human and animal health.

GFI #187A describes a risk-based regulatory approach to oversee IGAs in animals. The approach categorizes products into three levels: Category 1 products, which do not require consultation before marketing; Category 2 products, which may not need an approval application if risks are understood and mitigated; and Category 3 products, which require FDA review and approval based on data requirements proportionate to the associated risk.

The draft of GFI #187B provides technical guidance for obtaining FDA approval for IGAs in animals. It applies to IGAs categorized as Category 2 or 3 in GFI #187A. The FDA CVM intends to finalize GFI #187B in the future after consultations with stakeholders.

For more information, read the final guidance document of GFI #187A and the draft guidance document of GFI #187B on the FDA website.