[Federal Register Volume 87, Number 122 (Monday, June 27, 2022)]
[Notices]
[Pages 38161-38163]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-13606]


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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Oligonucleotide Therapeutics; Draft Guidance for Industry; Availability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 or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Clinical 
Pharmacology Considerations for the Development of Oligonucleotide 
Therapeutics.'' This draft guidance describes FDA's recommendations 
regarding clinical pharmacology considerations during the development 
of oligonucleotide therapeutics, 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. The intent of 
this guidance is to assist industry in the conduct of these studies.

DATES: Submit either electronic or written comments on the draft 
guidance by September 26, 2022 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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.

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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-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 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 draft 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 draft guidance document.

FOR FURTHER INFORMATION CONTACT: Anuradha Ramamoorthy, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 3118, Silver Spring, MD 20903, 
[email protected], 240-402-6426.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft 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 draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). This draft guidance, 
when finalized, will represent 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. Therefore, 
clearance by the Office of Management and Budget (OMB) under the 
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not 
required for this guidance. The previously approved collections of 
information are subject to review by OMB under the PRA. The collections 
of information in 21 CFR 312 for investigational new drug applications 
and 21 CFR part 314 for new drug applications and abbreviated new drug 
applications have been approved under OMB control numbers 0910-0014 and 
0910-0001. The collections of information 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 draft guidance 
at either https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs or https://www.regulations.gov.


[[Page 38163]]


    Dated: June 21, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-13606 Filed 6-24-22; 8:45 am]
BILLING CODE 4164-01-P