Notice2021-26453
Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations; Draft Guidance for Sponsor-Investigators; Availability
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Published
December 8, 2021
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The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for sponsor-investigators entitled "IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations." FDA is publishing this draft guidance to provide sponsor-investigators (hereafter referred to as sponsors) who are interested in developing individualized antisense oligonucleotide (ASO) drug products for a rapidly progressive, severely debilitating, or life-threatening (SDLT) genetic disease (caused by a unique genetic variant or variants), with clinical recommendations for submission of investigational new drug applications (INDs). These recommendations specifically address the following clinical considerations: Ethical and human subject protection, diagnostic and genetic, dosing, administration, safety, and assessment of clinical response to treatment.
Full Text
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<title>Federal Register, Volume 86 Issue 233 (Wednesday, December 8, 2021)</title>
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[Federal Register Volume 86, Number 233 (Wednesday, December 8, 2021)]
[Notices]
[Pages 69652-69653]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2021-26453]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-1140]
Investigational New Drug Application Submissions for
Individualized Antisense Oligonucleotide Drug Products for Severely
Debilitating or Life-Threatening Diseases: Clinical Recommendations;
Draft Guidance for Sponsor-Investigators; Availability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the availability of a draft guidance for sponsor-investigators entitled
``IND Submissions for Individualized Antisense Oligonucleotide Drug
Products for Severely Debilitating or Life-Threatening Diseases:
Clinical Recommendations.'' FDA is publishing this draft guidance to
provide sponsor-investigators (hereafter referred to as sponsors) who
are interested in developing individualized antisense oligonucleotide
(ASO) drug products for a rapidly progressive, severely debilitating,
or life-threatening (SDLT) genetic disease (caused by a unique genetic
variant or variants), with clinical recommendations for submission of
investigational new drug applications (INDs). These recommendations
specifically address the following clinical considerations: Ethical and
human subject protection, diagnostic and genetic, dosing,
administration, safety, and assessment of clinical response to
treatment.
DATES: Submit either electronic or written comments on the draft
guidance by February 7, 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:
<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-2021-D-1140 for ``IND Submissions for Individualized Antisense
Oligonucleotide Drug Products for Severely Debilitating or Life-
Threatening Diseases: Clinical Recommendations.'' 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
[[Page 69653]]
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: Hobart Rogers, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Avenue, Bldg. 51, Rm 3114, Silver Spring, MD 20903-0002, 301-
796-2213.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for sponsor-
investigators entitled ``IND Submissions for Individualized Antisense
Oligonucleotide Drug Products for Severely Debilitating or Life-
Threatening Diseases: Clinical Recommendations.'' FDA is publishing
this draft guidance to provide sponsor-investigators (hereafter
referred to as sponsors) who are interested in developing
individualized ASO drug products for a rapidly progressive SDLT genetic
disease (caused by a unique genetic variant or variants), with clinical
recommendations for submission of INDs. These recommendations
specifically address the following clinical considerations: Ethical and
human subject protection, diagnostic and genetic, dosing,
administration, safety, and assessment of clinical response to
treatment.
This draft guidance describes clinical considerations and, when
applicable, important information to include in IND submissions for
such ASO drug products for a small number of participants (typically
one to two) with SDTL diseases. In general, ASO drug products referred
to in this draft guidance belong to a well-characterized chemical class
and for which there is considerable nonclinical and clinical experience
that is publicly available or to which the sponsor has a right of
reference. The draft guidance discusses considerations and information
to submit in an IND regarding: (1) Confirmation of the participant's
genetic diagnosis and genetic variant(s) targeted by the ASO drug
product, (2) the requirements and procedures for informed consent of
the participant, (3) appropriate and safe dosing and administration
procedures that are detailed and supported by relevant nonclinical
evidence, (4) the nature and schedule of the specific safety
assessments (adverse events and laboratory testing) to be conducted,
and (5) methods for continuous clinical monitoring (e.g., via clinical
outcome assessments, pharmacodynamic biomarkers) of the participant to
evaluate and document their clinical response(s) and to allow for an
informed benefit-risk determination. This draft guidance is expected to
facilitate the preparation of adequate and complete IND submissions for
investigational ASO drug products for participants with SDLT diseases
targeted by the specified ASO drug product.
This draft guidance represents one guidance in a series of
guidances that FDA intends to publish to advise and help sponsors
planning to use individualized ASO drug products for SDLT diseases
caused by unique genetic variant(s) and for whom there are no
alternative therapies available to treat their disease.
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 ``IND
Submissions for Individualized Antisense Oligonucleotide Drug Products
for Severely Debilitating or Life-Threatening Diseases: Clinical
Recommendations.'' 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 new 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 part 312 have been approved under OMB control
number 0910-0014. The collections of information in 21 CFR parts 50 and
56 for obtaining informed consent for prospective patients have been
approved under OMB control number 0910-0130.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at either <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: December 1, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021-26453 Filed 12-7-21; 8:45 am]
BILLING CODE 4164-01-P
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