Genome Editing in Animals: FDA Preps for New Flexible Regulatory Approach

Regulatory NewsRegulatory News | 30 October 2018 |  By 

With the use of CRISPR becoming almost ubiquitous and gene drives on the horizon in the US, the US Food and Drug Administration (FDA) said Tuesday that it is looking to take a risk-based approach to regulating the gene editing of animals.

As part of that approach, the agency is establishing a new pilot program, known as the Veterinary Innovation Program (VIP), to provide assistance, both technical and programmatic, for developers seeking FDA approval of intentionally altered genomic DNA in animals and animal cells, tissues and cell- or tissue-based products that provide a benefit to human health, animal health, animal well-being or food production.

“The goal of the VIP is to facilitate advancements in the development of innovative animal products by providing greater certainty in the regulatory process, encouraging development and research, and supporting an efficient and predictable pathway to approval for certain innovative animal products,” FDA said in a new report.

In 2019, the agency also intends to publish guidance clarifying that risk-based categories will include: “an FDA decision not to enforce approval requirements with no prior review, an FDA decision not to enforce approval requirements following a review of data that address specific risk questions, and an FDA decision to review for approval with data requirements proportionate to the risk associated with the particular product.”

This approach includes flexibility to transfer products across these categories based on specific conditions and as FDA gains familiarity with different product risk profiles. However, FDA is still prohibited by Congress from accepting any investigational new drug application for “research in which a human embryo is intentionally created or modified to include a heritable genetic modification.”

In another draft guidance, FDA intends to clarify its regulatory approach for categories of intentionally genetically altered animals used in research and plans to outline, based on risk, when FDA intends to exercise enforcement discretion or when it intends to enforce the requirement for an approved new animal drug application.

An additional draft guidance for industry will establish an alternative type of file as a repository for information exchanges with the FDA’s Center for Veterinary Medicine (CVM) for products that are in early development stages or that are developed for research purposes only and may never progress to a marketable product.

In addition, the FDA’s CVM intends to hold a public webinar on 3 December to discuss the science behind genome editing in animals, the promising uses of this technology in animals and the potential risks, and information about CVM’s risk-based approach to the regulation of intentional genomic alterations to animals.

And to enhance transparency, FDA also said it will list on its website the specific animals or categories of animals with intentional genomic alterations for which the agency has decided to exercise enforcement discretion with regard to premarket approval requirements.


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