Notice2025-18653
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry; Availability
Primary source
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Published
September 25, 2025
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft document entitled "Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry." The draft guidance document provides sponsors engaged in the development of regenerative medicine therapies for serious or life-threatening diseases or conditions with FDA's recommendations on the expedited development and review of these therapies. This draft guidance, when finalized, will supersede the final guidance of the same title dated February 2019.
Full Text
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<title>Federal Register, Volume 90 Issue 184 (Thursday, September 25, 2025)</title>
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[Federal Register Volume 90, Number 184 (Thursday, September 25, 2025)]
[Notices]
[Pages 46225-46227]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2025-18653]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2017-D-6159]
Expedited Programs for Regenerative Medicine Therapies for
Serious Conditions; Draft Guidance for Industry; Availability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 document entitled ``Expedited
Programs for Regenerative Medicine Therapies for Serious Conditions;
Draft Guidance for Industry.'' The draft guidance document provides
sponsors engaged in the development of regenerative medicine therapies
for serious or life-threatening diseases or conditions with FDA's
recommendations on the expedited development and review of these
therapies. This draft guidance, when finalized, will supersede the
final guidance of the same title dated February 2019.
DATES: Submit either electronic or written comments on the draft
guidance by November 24, 2025 to ensure that the Agency considers your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:
<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
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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-2017-D-6159 for ``Expedited Programs for Regenerative Medicine
Therapies for Serious Conditions; Draft Guidance for Industry.''
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 draft guidance to
the Office of Communication, Outreach and Development, 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 draft guidance may also be obtained by
mail by calling CBER at 1-800-835-4709 or 240-402-8010 or emailing
<a href="/cdn-cgi/l/email-protection#83eaede7f6f0f7f1faade1eaecefece4eae0f0c3e5e7e2adebebf0ade4ecf5"><span class="__cf_email__" data-cfemail="0a63646e7f797e78732468636566656d6369794a6c6e6b24626279246d657c">[email protected]</span></a>. See the SUPPLEMENTARY INFORMATION
section for electronic access to the draft guidance document.
FOR FURTHER INFORMATION CONTACT: Andrew C. Harvan, Center for Biologics
Evaluation and Research, Food and Drug Administration, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance entitled
``Expedited Programs for Regenerative Medicine Therapies for Serious
Conditions; Draft Guidance for Industry.'' The draft guidance document
describes the expedited programs available to sponsors of regenerative
medicine therapies for serious or life-threatening diseases or
conditions (referred to in the guidance as ``serious conditions''),
including those products designated as regenerative advanced therapies
(which FDA refers to as ``regenerative medicine advanced therapy''
(RMAT) designation); provides information about the provisions in the
21st Century Cures Act (Pub. L. 114-225) regarding the use of the
accelerated approval pathway for regenerative medicine therapies that
have been granted designation as an RMAT; describes how CBER will
encourage flexibility in clinical trial design to facilitate the
development of data to demonstrate the safety and effectiveness of
regenerative medicine therapies that are being developed to address
unmet needs in patients with serious conditions; provides additional
thinking on post-approval requirements and on approaches and processes
relating to chemistry, manufacturing, and controls; and describes the
opportunities for sponsors of regenerative medicine therapies to
interact with CBER review staff.
FDA is issuing this draft guidance in accordance with a commitment
outlined in the reauthorization of the Prescription Drug User Fee Act
(PDUFA VII) under the 2022 FDA User Fee Reauthorization Act.
This draft guidance, when finalized, will supersede the guidance
entitled, ``Expedited Programs for Regenerative Medicine Therapies for
Serious Conditions; Guidance for Industry'' dated February 2019.
This 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 ``Expedited
Programs for Regenerative Medicine Therapies for Serious Conditions.''
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.
As we develop final guidance on this topic, FDA will consider
comments on costs or cost savings the guidance may generate, relevant
for Executive Order 14192.
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 312 have been approved under OMB control number 0910-
0014; the collections of information in 21 CFR part 314 and the
collections of information regarding formal meetings described in the
draft guidance, ``Formal Meetings Between the FDA and Sponsors or
Applicants of PDUFA Products,'' have been approved under OMB control
number 0910-0001; the collections of information in 21 CFR
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part 601 have been approved under OMB control number 0910-0338; the
collections of information for expedited programs in ``Guidance for
Industry: Expedited Programs for Serious Conditions--Drugs and
Biologics'' have been approved under OMB control number 0910-0765.
III. Electronic Access
Persons with access to the internet may obtain the draft 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. 2025-18653 Filed 9-24-25; 8:45 am]
BILLING CODE 4164-01-P
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