Notice2024-13271
Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics; Guidance for Industry; Availability
Primary source
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Published
June 17, 2024
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics," which provides recommendations for the development of oligonucleotide therapeutics. Specifically, this guidance addresses FDA's current thinking regarding clinical pharmacology considerations and recommendations for oligonucleotide therapeutic development programs, including characterizing the potential for QT interval prolongation, performing immunogenicity risk assessment, characterizing the impact of hepatic and renal impairment, and assessing the potential for drug-drug interactions. This guidance finalizes the draft guidance of the same name issued on June 27, 2022.
Full Text
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<title>Federal Register, Volume 89 Issue 117 (Monday, June 17, 2024)</title>
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[Federal Register Volume 89, Number 117 (Monday, June 17, 2024)]
[Notices]
[Pages 51354-51355]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-13271]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2022-D-0235]
Clinical Pharmacology Considerations for the Development of
Oligonucleotide Therapeutics; 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 for industry entitled ``Clinical
Pharmacology Considerations for the Development of Oligonucleotide
Therapeutics,'' which provides recommendations for the development of
oligonucleotide therapeutics. Specifically, this guidance addresses
FDA's current thinking regarding clinical pharmacology considerations
and recommendations for oligonucleotide therapeutic development
programs, including characterizing the potential for QT interval
prolongation, performing immunogenicity risk assessment, characterizing
the impact of hepatic and renal impairment, and assessing the potential
for drug-drug interactions. This guidance finalizes the draft guidance
of the same name issued on June 27, 2022.
DATES: The announcement of the guidance is published in the Federal
Register on June 17, 2024.
ADDRESSES: You may submit either electronic or written comments on
Agency guidances 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 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-2022-D-0235 for ``Clinical Pharmacology Considerations for the
Development of Oligonucleotide Therapeutics.'' 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
[[Page 51355]]
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 this guidance to the
Division of Drug Information, Center for Drug Evaluation and Research,
Food and Drug Administration, 10001 New Hampshire Ave., Hillandale
Building, 4th Floor, Silver Spring, MD 20993-0002. Send one self-
addressed adhesive label to assist that office in processing your
requests. See the SUPPLEMENTARY INFORMATION section for electronic
access to the guidance document.
FOR FURTHER INFORMATION CONTACT: Anuradha Ramamoorthy, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20903, 240-402-6426,
<a href="/cdn-cgi/l/email-protection#dc9db2a9aebdb8b4bdf28ebdb1bdb1b3b3aea8b4a59cbab8bdf2b4b4aff2bbb3aa"><span class="__cf_email__" data-cfemail="9edff0ebecfffaf6ffb0ccfff3fff3f1f1eceaf6e7def8faffb0f6f6edb0f9f1e8">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a guidance for industry
entitled ``Clinical Pharmacology Considerations for the Development of
Oligonucleotide Therapeutics.'' Oligonucleotide therapeutics are an
emerging therapeutic modality with increasing numbers of drugs in
development. While antisense and siRNA oligonucleotide therapeutics
have been approved in recent years to treat rare diseases, many
oligonucleotide therapeutics are in development to treat common chronic
diseases. This guidance provides recommendations to assist industry in
the development of oligonucleotide therapeutics. Specifically, this
guidance represents FDA's recommendations for certain pharmacokinetic
and pharmacodynamic investigations including characterizing QT interval
prolongation potential, performing immunogenicity risk assessment,
characterizing the impact of hepatic and renal impairment, and
assessing the potential for drug-drug interactions during
oligonucleotide therapeutic development. This guidance provides
recommendations on when these assessments may be appropriate and what
types of assessments can help address these issues.
This guidance finalizes the draft guidance of the same name issued
on June 27, 2022 (87 FR 38161). FDA considered comments received on the
draft guidance as the guidance was finalized. Changes from the draft to
the final guidance include: (1) updates to terms used in the guidance
to provide clarity, (2) additional references to FDA guidance that have
been published since publication of the draft guidance, and (3)
editorial changes to improve clarity.
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 ``Clinical Pharmacology Considerations for
the Development of Oligonucleotide Therapeutics.'' 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 312 for investigational new drug applications have been
approved under OMB control number 0910-0014. The collections of
information in 21 CFR part 314 for new drug applications have been
approved under OMB control number 0910-0001. The collections of
information in 21 CFR part 601 for 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/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</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>.
Dated: June 12, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-13271 Filed 6-14-24; 8:45 am]
BILLING CODE 4164-01-P
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