[Federal Register Volume 85, Number 20 (Thursday, January 30, 2020)]
[Notices]
[Pages 5447-5448]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01701]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2008-D-0205]


Chemistry, Manufacturing, and Control Information for Human Gene 
Therapy Investigational New Drug Applications; 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 ``Chemistry, 
Manufacturing, and Control (CMC) Information for Human Gene Therapy 
Investigational New Drug Applications (INDs); Guidance for Industry.'' 
The guidance document provides sponsors of human gene therapy INDs with 
recommendations regarding CMC information to be submitted in an IND. 
The guidance document informs sponsors how to provide sufficient CMC 
information required to assure product safety, identity, quality, 
purity, and strength (including potency) of the investigational 
product. The guidance applies to human gene therapy products and to 
combination products that contain a human gene therapy in combination 
with a drug or device.
    The guidance announced in this notice finalizes the draft guidance 
of the same title dated July 2018 and supersedes the document entitled 
``Guidance for FDA Reviewers and Sponsors: Content and Review of 
Chemistry, Manufacturing, and Control (CMC) Information for Human Gene 
Therapy Investigational New Drug Applications (INDs),'' dated April 
2008 (April 2008 guidance).

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-2008-D-0205 for ``Chemistry, Manufacturing, and Control Information 
for Human Gene Therapy Investigational New Drug Applications; 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

[[Page 5448]]

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: Sana F. Hussain, 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 
``Chemistry, Manufacturing, and Control (CMC) Information for Human 
Gene Therapy Investigational New Drug Applications (INDs); Guidance for 
Industry.'' The guidance document provides sponsors of human gene 
therapy INDs with recommendations regarding CMC information to be 
submitted in an IND. The guidance document informs sponsors how to 
provide sufficient CMC information required to assure product safety, 
identity, quality, purity, and strength (including potency) of the 
investigational product (21 CFR 312.23(a)(7)(i)). The guidance applies 
to human gene therapy products and to combination products that contain 
a human gene therapy in combination with a drug or device.
    The field of gene therapy has progressed rapidly since FDA issued 
the April 2008 guidance. Therefore, FDA is updating the guidance to 
provide current FDA recommendations regarding the CMC content of a gene 
therapy IND. In addition, the guidance is organized to follow the 
structure of the FDA guidance on the Common Technical Document.
    In the Federal Register of July 12, 2018 (83 FR 32307), FDA 
announced the availability of the draft guidance of the same title. FDA 
received numerous comments on the draft guidance and those comments 
were considered as the guidance was finalized. In addition, editorial 
changes were made to improve clarity. The guidance announced in this 
notice finalizes the draft guidance dated July 2018. The guidance also 
supersedes the April 2008 guidance.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of two other final guidances. In a separate document, 
FDA is announcing the availability of a document entitled ``Long Term 
Follow-Up After Administration of Human Gene Therapy Products; Guidance 
for Industry'' and the availability of a document entitled ``Testing of 
Retroviral Vector-Based Human Gene Therapy Products for Replication 
Competent Retrovirus During Product Manufacture and Patient Follow-up; 
Guidance for Industry.''
    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 ``Chemistry, Manufacturing, and Control 
Information for Human Gene Therapy Investigational New Drug 
Applications.'' 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 211 have been approved under 
OMB control number 0910-0139; the collections of information in 21 CFR 
part 312 and Form FDA 1571 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 21 CFR part 1271 have been approved under OMB control 
number 0910-0543.

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. 2020-01701 Filed 1-29-20; 8:45 am]
 BILLING CODE 4164-01-P