Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators; Draft Guidance for Industry; Availability, 69653-69655 [2021-26454]
Download as PDF
Federal Register / Vol. 86, No. 233 / Wednesday, December 8, 2021 / Notices
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copies of the draft guidance to the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access to the draft guidance document.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:
jspears on DSK121TN23PROD with NOTICES1
I. Background
FDA is announcing the availability of
a draft guidance for sponsorinvestigators entitled ‘‘IND Submissions
for Individualized Antisense
Oligonucleotide Drug Products for
Severely Debilitating or LifeThreatening Diseases: Clinical
Recommendations.’’ FDA is publishing
this draft guidance to provide sponsorinvestigators (hereafter referred to as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This draft guidance describes clinical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products referred to in this draft
guidance belong to a well-characterized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
VerDate Sep<11>2014
16:53 Dec 07, 2021
Jkt 256001
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
PO 00000
Frm 00043
Fmt 4703
Sfmt 4703
69653
approved under OMB control number
0910–0130.
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2021–D–1139]
Investigational New Drug Application
Submissions for Individualized
Antisense Oligonucleotide Drug
Products for Severely Debilitating or
Life-Threatening Diseases: Chemistry,
Manufacturing, and Controls
Recommendations, Guidance for
Sponsor-Investigators; Draft Guidance
for Industry; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘IND
Submissions for Individualized
Antisense Oligonucleotide Drug
Products for Severely Debilitating or
Life-Threatening Diseases: Chemistry,
Manufacturing, and Controls
Recommendations.’’ This draft guidance
provides FDA’s recommendations on
the chemistry, manufacturing, and
controls (CMC) information needed to
support an investigational new drug
application (IND) submitted by a
sponsor-investigator developing an
individualized antisense
oligonucleotide (ASO) drug product for
a severely debilitating or life-threatening
(SDLT) disease caused by a unique
genetic variant affecting a small number
of individuals (typically one or two).
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.
SUMMARY:
E:\FR\FM\08DEN1.SGM
08DEN1
69654
Federal Register / Vol. 86, No. 233 / Wednesday, December 8, 2021 / Notices
You may submit comments
on any guidance at any time as follows:
ADDRESSES:
jspears on DSK121TN23PROD with NOTICES1
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
instructions for submitting comments.
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–
2021–D–1139 for ‘‘IND Submissions for
Individualized Antisense
Oligonucleotide Drug Products for
Severely Debilitating or LifeThreatening Diseases: Chemistry,
Manufacturing, and Controls
Recommendations.’’ 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
VerDate Sep<11>2014
16:53 Dec 07, 2021
Jkt 256001
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-201509-18/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:
Carla R. Lankford, Center for Drug
Evaluation and Research (HFD–123),
Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 75, Rm.
6656, Silver Spring, MD 20993–0002,
301–796–5203.
SUPPLEMENTARY INFORMATION:
PO 00000
Frm 00044
Fmt 4703
Sfmt 4703
I. Background
FDA is announcing the availability of
a draft guidance for industry entitled
‘‘IND Submissions for Individualized
Antisense Oligonucleotide Drug
Products for Severely Debilitating or
Life-Threatening Diseases: Chemistry,
Manufacturing, and Controls
Recommendations.’’ The purpose of this
draft guidance is to provide FDA’s
current thinking on the recommended
CMC data and information to support an
IND submitted by a sponsor-investigator
developing an ASO drug product for a
small number of individuals (typically
one or two) with an SDLT disease. This
draft guidance also explains FDA’s
recommendations around compliance
with current good manufacturing
practice, including the applicability of
21 CFR part 211, to support
investigational use of an ASO drug
product in a small number of
individuals with an SDLT 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: Chemistry,
Manufacturing, and Controls
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
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 parts 312 and
314 have been approved under OMB
control numbers 0910–0014 and 0910–
0001, respectively. The collections of
information in 21 CFR 211 for
manufacture of drug product is
approved under OMB control number
0910–0139. The collections of
information for oversight of clinical
investigations and safety reporting have
been approved under OMB control
number 0910–0733.
E:\FR\FM\08DEN1.SGM
08DEN1
69655
Federal Register / Vol. 86, No. 233 / Wednesday, December 8, 2021 / Notices
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
regulatory-information/search-fdaguidance-documents, or https://
www.regulations.gov.
Dated: December 1, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021–26454 Filed 12–7–21; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Resources and Services
Administration
Agency Information Collection
Activities: Proposed Collection: Public
Comment Request; Environmental
Information and Documentation, OMB
No. 0915–0324, Extension
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services.
ACTION: Notice.
AGENCY:
In compliance with the
requirement for opportunity for public
comment on proposed data collection
projects of the Paperwork Reduction Act
of 1995, HRSA announces plans to
submit an Information Collection
Request (ICR), described below, to the
SUMMARY:
Office of Management and Budget
(OMB). Prior to submitting the ICR to
OMB, HRSA seeks comments from the
public regarding the burden estimate,
below, or any other aspect of the ICR.
DATES: Comments on this ICR should be
received no later than February 7, 2022.
ADDRESSES: Submit your comments to
paperwork@hrsa.gov or by mail to the
HRSA Information Collection Clearance
Officer, Room 14N136B, 5600 Fishers
Lane, Rockville, MD 20857.
FOR FURTHER INFORMATION CONTACT: To
request more information on the
proposed project or to obtain a copy of
the data collection plans and draft
instruments, email paperwork@hrsa.gov
or call Samantha Miller, the acting
HRSA Information Collection Clearance
Officer at (301) 443–9094.
SUPPLEMENTARY INFORMATION: When
submitting comments or requesting
information, please include the
information collection request title for
reference.
Information Collection Request Title:
Environmental Information and
Documentation (EID), OMB No. 0915–
0324, Extension.
Abstract: HRSA is requesting
approval of an extension for the EID
checklist which consists of information
that the agency is required to obtain to
comply with the National
Environmental Policy Act of 1969
(NEPA). NEPA establishes the federal
government’s national policy for
protection of the environment. HRSA
has developed the EID for applicants of
funding that would potentially impact
the environment and to ensure that their
decision-making processes are
consistent with NEPA.
Need and Proposed Use of the
Information: Applicants must provide
information and assurance of
compliance with NEPA on the EID
checklist. This information is reviewed
in the Pre-Award stage (and/or prior to
the implementation of the project). The
information is reviewed in the PostAward stage for project changes and the
information is reviewed before the
implementation of the project changes.
Likely Respondents: HRSA applicants
applying for federal loan guarantees,
federal construction grants, and
cooperative agreements.
Burden Statement: Burden in this
context means the time expended by
persons to generate, maintain, retain,
disclose or provide the information
requested. This includes the time
needed to review instructions; to
develop, acquire, install and utilize
technology and systems for the purpose
of collecting, validating and verifying
information, processing and
maintaining information, and disclosing
and providing information; to train
personnel and to be able to respond to
a collection of information; to search
data sources; to complete and review
the collection of information; and to
transmit or otherwise disclose the
information. The total annual burden
hours estimated for this ICR are
summarized in the table below.
TOTAL ESTIMATED ANNUALIZED BURDEN HOURS
Number of
respondents
jspears on DSK121TN23PROD with NOTICES1
Form name
Number of
responses per
respondent
Total
responses
Average
burden per
response
(in hours)
Total burden
hours
NEPA EID Checklist ............................................................
1,500
1
1,500
1
1,500
Total ..............................................................................
1,500
........................
1,500
........................
1,500
HRSA specifically requests comments
on (1) the necessity and utility of the
proposed information collection for the
proper performance of the agency’s
functions, (2) the accuracy of the
estimated burden, (3) ways to enhance
the quality, utility, and clarity of the
information to be collected, and (4) the
use of automated collection techniques
or other forms of information
technology to minimize the information
collection burden.
Maria G. Button,
Director, Executive Secretariat.
BILLING CODE 4165–15–P
16:53 Dec 07, 2021
Jkt 256001
ACTION:
Notice.
In compliance with the
requirement for opportunity for public
comment on proposed data collection
projects of the Paperwork Reduction Act
of 1995, HRSA announces plans to
submit an Information Collection
Request (ICR), described below, to the
Office of Management and Budget
(OMB). Prior to submitting the ICR to
OMB, HRSA seeks comments from the
public regarding the burden estimate,
below, or any other aspect of the ICR.
SUMMARY:
Health Resources and Services
Administration
Agency Information Collection
Activities: Proposed Collection: Public
Comment Request; Rural Communities
Opioid Response Program
Performance Measures, OMB No.
0906–0044, Revision
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services.
AGENCY:
[FR Doc. 2021–26560 Filed 12–7–21; 8:45 am]
VerDate Sep<11>2014
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
PO 00000
Frm 00045
Fmt 4703
Sfmt 4703
Comments on this ICR should be
received no later than February 7, 2022.
DATES:
E:\FR\FM\08DEN1.SGM
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Agencies
[Federal Register Volume 86, Number 233 (Wednesday, December 8, 2021)]
[Notices]
[Pages 69653-69655]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-26454]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-D-1139]
Investigational New Drug Application Submissions for
Individualized Antisense Oligonucleotide Drug Products for Severely
Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing,
and Controls Recommendations, Guidance for Sponsor-Investigators; Draft
Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft guidance for industry entitled ``IND
Submissions for Individualized Antisense Oligonucleotide Drug Products
for Severely Debilitating or Life-Threatening Diseases: Chemistry,
Manufacturing, and Controls Recommendations.'' This draft guidance
provides FDA's recommendations on the chemistry, manufacturing, and
controls (CMC) information needed to support an investigational new
drug application (IND) submitted by a sponsor-investigator developing
an individualized antisense oligonucleotide (ASO) drug product for a
severely debilitating or life-threatening (SDLT) disease caused by a
unique genetic variant affecting a small number of individuals
(typically one or two).
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.
[[Page 69654]]
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. 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-2021-D-1139 for ``IND Submissions for Individualized Antisense
Oligonucleotide Drug Products for Severely Debilitating or Life-
Threatening Diseases: Chemistry, Manufacturing, and Controls
Recommendations.'' 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: Carla R. Lankford, Center for Drug
Evaluation and Research (HFD-123), Food and Drug Administration, 10903
New Hampshire Ave., Bldg. 75, Rm. 6656, Silver Spring, MD 20993-0002,
301-796-5203.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``IND Submissions for Individualized Antisense Oligonucleotide
Drug Products for Severely Debilitating or Life-Threatening Diseases:
Chemistry, Manufacturing, and Controls Recommendations.'' The purpose
of this draft guidance is to provide FDA's current thinking on the
recommended CMC data and information to support an IND submitted by a
sponsor-investigator developing an ASO drug product for a small number
of individuals (typically one or two) with an SDLT disease. This draft
guidance also explains FDA's recommendations around compliance with
current good manufacturing practice, including the applicability of 21
CFR part 211, to support investigational use of an ASO drug product in
a small number of individuals with an SDLT 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: Chemistry,
Manufacturing, and Controls 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 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 parts 312 and 314 have been approved under OMB
control numbers 0910-0014 and 0910-0001, respectively. The collections
of information in 21 CFR 211 for manufacture of drug product is
approved under OMB control number 0910-0139. The collections of
information for oversight of clinical investigations and safety
reporting have been approved under OMB control number 0910-0733.
[[Page 69655]]
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, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or https://www.regulations.gov.
Dated: December 1, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021-26454 Filed 12-7-21; 8:45 am]
BILLING CODE 4164-01-P