[Federal Register Volume 87, Number 204 (Monday, October 24, 2022)]
[Notices]
[Pages 64234-64236]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-23057]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2020-D-2101]
Human Gene Therapy for Neurodegenerative Diseases; 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 entitled ``Human Gene Therapy for
Neurodegenerative Diseases; Guidance for Industry.'' Neurodegenerative
diseases are a heterogeneous group of disorders characterized by
progressive degeneration of the structure and function of the central
nervous system or peripheral nervous system. The guidance document
provides recommendations to sponsors developing human gene therapy (GT)
products for neurodegenerative diseases affecting adult and pediatric
patients. The guidance focuses on considerations for product
development, preclinical testing, and clinical trial design. The
guidance announced in this notice finalizes the draft guidance of the
same title dated January 2021.
DATES: The announcement of the guidance is published in the Federal
Register on October 24, 2022.
[[Page 64235]]
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:
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-2020-D-2101 for ``Human Gene Therapy for Neurodegenerative
Diseases; Guidance for Industry.'' 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 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. 3128, Silver Spring, MD 20993-0002. Send
one self-addressed adhesive label to assist the office in processing
your requests. The guidance may also be obtained by mail by calling
CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY
INFORMATION section for electronic access to the guidance document.
FOR FURTHER INFORMATION CONTACT: Phillip Kurs, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a document entitled ``Human
Gene Therapy for Neurodegenerative Diseases; Guidance for Industry.''
Neurodegenerative diseases are a heterogeneous group of disorders
characterized by progressive degeneration of the structure and function
of the central nervous system or peripheral nervous system. These
diseases vary in etiology, prevalence, diagnosis, and management, and
include genetic as well as age-related diseases. The guidance document
provides recommendations to sponsors developing a GT product for
neurodegenerative diseases affecting adult and pediatric patients. The
guidance focuses on considerations for product development, preclinical
testing, and clinical trial design.
In the Federal Register of January 6, 2021 (86 FR 549), FDA
announced the availability of the draft guidance of the same title
dated January 2021. FDA received several comments on the draft guidance
and those comments were considered as the guidance was finalized.
Changes to the guidance include clarifications to the recommendations
regarding use of tumorigenic cell lines, comparability studies, and
crossover designs for clinical trials. In addition, editorial changes
were made to improve clarity. The guidance announced in this notice
finalizes the draft guidance dated January 2021.
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 ``Human Gene Therapy for Neurodegenerative
Diseases.'' 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 part 50 have been
[[Page 64236]]
approved under OMB control number 0910-0130; 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 601
have been approved under OMB control number 0910-0338; and the
collections of information in the guidance entitled ``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 guidance at
https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or
https://www.regulations.gov.
Dated: October 18, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-23057 Filed 10-21-22; 8:45 am]
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