Potency Assurance for Cellular and Gene Therapy Products; Guidance for Industry; Availability

Issuing agencies

Abstract

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance titled "Potency Assurance for Cellular and Gene Therapy Products." The guidance document provides recommendations to help assure the potency of human cellular therapy or gene therapy (CGT) products at all stages of the product lifecycle. FDA is recommending a comprehensive approach to potency assurance of CGT products that is grounded in quality risk management. For investigational products, we describe how to progressively implement a strategy for potency assurance during product development and provide additional considerations to help assure the potency of products that are undergoing rapid clinical development. For licensed products, we describe requirements for potency assurance, including testing required for lot release assays. The guidance announced in this notice finalizes the draft guidance of the same title issued on December 28, 2023.

Full Text

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<title>Federal Register, Volume 91 Issue 211 (Tuesday, November 3, 2026)</title>
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[Federal Register Volume 91, Number 211 (Tuesday, November 3, 2026)]
[Notices]
[Pages 69259-69261]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-22427]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-D-4299]


Potency Assurance for Cellular and Gene Therapy Products; 
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 or Agency) is announcing 
the availability of a final guidance titled ``Potency Assurance for 
Cellular and Gene Therapy Products.'' The guidance document provides 
recommendations to help assure the potency of human cellular therapy or 
gene therapy (CGT) products at all stages of the product lifecycle. FDA 
is recommending a comprehensive approach to potency assurance of CGT 
products that is grounded in quality risk management. For 
investigational products, we describe how to progressively implement a 
strategy for potency assurance during product development and provide 
additional considerations to help assure the potency of products that 
are undergoing rapid clinical development. For licensed products, we 
describe requirements for potency assurance, including testing required 
for lot release assays. The guidance announced in this notice finalizes 
the draft guidance of the same title issued on December 28, 2023.

DATES: The announcement of the guidance is published in the Federal 
Register on November 3, 2026.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances at any time as follows:

[[Page 69260]]

Electronic Submissions

    Submit electronic comments in the following way:
    <bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
    <bullet> 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:
    <bullet> 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.
    <bullet> 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-2023-D-4299 for ``Potency Assurance for Cellular and Gene Therapy 
Products.'' Received comments will be placed in the docket and, except 
for those submitted as ``Confidential Submissions,'' publicly viewable 
at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets Management Staff 
between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.
    <bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>. 
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 
Center for Biologics Evaluation and Research (CBER), Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3103, Silver 
Spring, MD 20993-0002. Send one self-addressed adhesive label to assist 
the office in processing your requests. The guidance may also be 
obtained by mail by calling CBER at 1-800-835-4709 or 240-402-8010. See 
the SUPPLEMENTARY INFORMATION section for electronic access to the 
guidance document.

FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 240-402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a document titled ``Potency 
Assurance for Cellular and Gene Therapy Products.'' This guidance 
provides recommendations to help assure the potency of human CGT 
products that are regulated as biological products under section 351 of 
the Public Health Service Act (42 U.S.C. 262).
    This final guidance provides recommendations for developing a 
science- and risk-based strategy to help assure the potency of human 
CGT products. A potency assurance strategy is a multifaceted approach 
that reduces risks to the potency of a product through: (1) 
manufacturing process design, (2) manufacturing process control, (3) 
material control, (4) in-process testing, and (5) potency lot release 
assays. The goal of a potency assurance strategy is to ensure that 
every lot of a product released will have the specific ability or 
capacity to achieve the intended therapeutic effect.
    The guidance emphasizes that potency assays and their corresponding 
acceptance criteria should be designed to make meaningful contributions 
to potency assurance by reducing risks to product potency. We provide 
illustrative examples of approaches to potency assay development that 
are grounded in quality risk management. Due to the diversity of CGT 
products and the product-specific nature of potency assays, the 
recommendations in this final guidance regarding the selection and 
design of potency assays are necessarily general.
    In the Federal Register of December 28, 2023 (88 FR 89707), FDA 
announced the availability of the draft guidance of the same title 
dated December 28, 2023. FDA received several comments on the draft 
guidance and those comments were considered as the guidance was 
finalized. A summary of changes includes: advice on integration of 
potency assurance into existing quality systems, expanded risk 
assessment guidance, advice on regulatory engagement pathways, and 
additional practical examples with refined terminology throughout. In 
addition, editorial changes were made to improve clarity. The guidance 
announced in this notice finalizes the draft guidance dated December 
28, 2023.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Potency Assurance for Cellular and Gene 
Therapy Products.'' 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

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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 parts 210 and 211 pertaining to manufacturing, processing, packing, 
and holding of finished pharmaceuticals and biological products for 
human use have been approved under OMB control number 0910-0139. The 
collections of information in 21 CFR 312.23(a)(7)(i) and (ii) 
pertaining to submission of chemistry, manufacturing, and control 
information sufficient to assure the proper identification, quality, 
purity, and strength of an investigational drug in an IND application, 
and 21 CFR 312.47(a) pertaining to meetings between FDA and sponsors, 
including pre-IND meetings, have been approved under OMB control number 
0910-0014. The collections of information in 21 CFR 600.14(b) 
pertaining to biological product deviation reporting is included in OMB 
control number 0910-0458. The collections of information in 21 CFR part 
601 pertaining to biologics license applications have been approved 
under OMB control number 0910-0338.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
<a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or 
<a href="https://www.regulations.gov">https://www.regulations.gov</a>.

Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-22427 Filed 11-2-26; 8:45 am]
BILLING CODE 4164-01-P


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