[Federal Register Volume 90, Number 4 (Tuesday, January 7, 2025)]
[Notices]
[Pages 1144-1145]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-31530]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-D-4778]


Heritable Intentional Genomic Alterations in Animals of Food-
Producing Species for Use as Models of Disease; Draft Guidance for 
Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA, Agency, or we) is 
announcing the availability of a draft guidance for industry (GFI) #251 
entitled ``Heritable Intentional Genomic Alterations in Animals of 
Food-Producing Species for Use as Models of Disease.'' This draft 
guidance, when finalized, will set forth FDA's policy regarding 
heritable intentional genomic alterations (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. The draft guidance 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.

DATES: Submit either electronic or written comments on the draft 
guidance by March 10, 2025 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand Delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2024-D-4778 for ``Heritable Intentional Genomic Alterations in 
Animals of Food-Producing Species for Use as Models of Disease.'' 
Received comments will be placed in the docket and, except

[[Page 1145]]

for those submitted as ``Confidential Submissions,'' publicly viewable 
at https://www.regulations.gov or at the Dockets Management Staff 
between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. If you do not wish 
your name and contact information to be made publicly available, you 
can provide this information on the cover sheet and not in the body of 
your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852, 240-402-7500.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of the guidance to the 
Policy and Regulations Staff (HFV-6), Center for Veterinary Medicine, 
Food and Drug Administration, 7500 Standish Pl., Rockville, MD 20855. 
Send one self-addressed adhesive label to assist that office in 
processing your requests. See the SUPPLEMENTARY INFORMATION section for 
electronic access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Adam Moyer, Center for Veterinary 
Medicine, Food and Drug Administration, 7500 Standish Pl., Rockville, 
MD 20855, 301-796-2319, [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
#251 entitled ``Heritable Intentional Genomic Alterations in Animals of 
Food-Producing Species for Use as Models of Disease.'' This guidance, 
when finalized, will set forth FDA's policy regarding 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).
    In the Federal Register of May 2, 2024 (89 FR 35834), we announced 
the availability of final GFI #187A entitled ``Heritable Intentional 
Genomic Alterations in Animals: Risk-Based Approach.'' GFI #187A 
clarifies that heritable IGAs in animals are subject to FDA oversight 
and are regulated according to our risk-based regulatory approach. GFI 
#187A indicates that while, in general, FDA approval of IGAs in animals 
is required, under certain conditions we do not expect developers of 
IGAs in animals to submit an application or get our approval before 
marketing their product following our prior review of risk factor data. 
Among these IGAs are those that GFI #187A describes as Category 2, for 
which we may not expect developers to submit an application for 
approval if, after analyzing data submitted about a product's risks, we 
find we understand the product's risks for the specified intended use; 
any identified risks, including their potential severity and likelihood 
of occurring, are appropriately mitigated; and we have no further 
questions for which we would need to see additional data.
    In this draft GFI #251, we address those circumstances under which 
we may not expect developers to submit an application for approval of 
an IGA in an animal model of disease if, after looking at data 
submitted about that product's risk, we determine that it appropriately 
fits in Category 2. FDA believes that IGAs in animal models of disease 
are likely to fit in Category 2 in part because the animals are 
unlikely to enter the food supply or to escape and establish themselves 
in the environment. For these reasons, based on case-by-case evaluation 
of data and information as described in draft GFI #251, we may 
determine that IGAs in an animal model of disease fits in Category 2, 
and we do not expect developers of these IGAs to submit an application 
for approval to us prior to marketing.
    This level 1 draft guidance is being issued consistent with FDA's 
good guidance practices regulation (21 CFR 10.115). The draft guidance, 
when finalized, will represent the current thinking of FDA on 
``Heritable Intentional Genomic Alterations in Animals of Food-
Producing Species for Use as Models of Disease.'' It does not establish 
any rights for any person and is not binding on FDA or the public. You 
can use an alternative approach if it satisfies the requirements of the 
applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. The 
previously approved collections of information are subject to review by 
the Office of Management and Budget (OMB) under the Paperwork Reduction 
Act of 1995 (PRA) (44 U.S.C. 3501-3521). The collections of information 
in 21 CFR part 514 have been approved under OMB control number 0910-
0032; the collections of information in 21 CFR part 511 have been 
approved under OMB control number 0910-0117; and the collections of 
information in 21 CFR part 58 have been approved under OMB control 
number 0910-0119.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at https://www.fda.gov/animal-veterinary/guidance-regulations/guidance-industry, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.

    Dated: December 27, 2024.
Kimberlee Trzeciak,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2024-31530 Filed 1-6-25; 8:45 am]
BILLING CODE 4164-01-P