[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5444-5445]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01702]
[[Page 5444]]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-2238]
Human Gene Therapy for Hemophilia; 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
Hemophilia; Guidance for Industry.'' The guidance document provides
recommendations to stakeholders developing human gene therapy (GT)
products for the treatment of hemophilia. The guidance provides
recommendations on the clinical trial design and related development of
coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity
assays, including how to address discrepancies in factor VIII and
factor IX activity assays. The guidance also includes recommendations
regarding preclinical considerations to support development of GT
products for the treatment of hemophilia. The guidance announced in
this notice finalizes the draft guidance of the same title dated July
2018.
DATES: The announcement of the guidance is published in the Federal
Register on January 30, 2020.
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-2018-D-2238 for ``Human Gene Therapy for Hemophilia; 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.
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.
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: Jessica Walker Udechukwu, 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 Hemophilia; Guidance for Industry.'' The guidance
document provides recommendations to stakeholders developing GT
products for the treatment of hemophilia. The guidance provides
recommendations on the clinical trial design and related development of
coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity
assays, including how to address discrepancies in factor VIII and
factor IX activity assays. The guidance also includes recommendations
regarding preclinical considerations to support development of GT
products for the treatment of hemophilia.
Hemophilia therapy in the United States has progressed from
replacement
[[Page 5445]]
therapies for on-demand treatment of bleeding to prophylaxis to reduce
the frequency of bleeding. GT products for the treatment of hemophilia
are being developed as single-dose treatments that may provide long-
term expression of the deficient coagulation factor at steady levels to
reduce or eliminate the need for exogenous factor replacement.
Elsewhere in this issue of the Federal Register, FDA is announcing
the availability of two other human gene therapy guidance documents
entitled ``Human Gene Therapy for Retinal Disorders; Guidance for
Industry'' and ``Human Gene Therapy for Rare Diseases; Guidance for
Industry.''
In the Federal Register of July 12, 2018 (83 FR 32306), FDA
announced the availability of the draft guidance of the same title. FDA
received several comments on the draft guidance, and those comments
were considered as the guidance was finalized. The guidance announced
in this notice finalizes the draft guidance dated July 2018.
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 Hemophilia.'' 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
This guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are subject to review by the Office of Management and Budget (OMB)
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3521). The
collections of information in 21 CFR part 58 have been approved under
OMB control number 0910-0119; the collections of information in 21 CFR
part 211 have been approved under OMB control number 0910-0139; 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; 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; and the collections of
information in the guidance entitled ``Formal Meetings Between the FDA
and Sponsors or Applicants'' have been approved under OMB control
number 0910-0429.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
either https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances or
https://www.regulations.gov.
Dated: January 27, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01702 Filed 1-29-20; 8:45 am]
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