[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)]
[Notices]
[Pages 32307-32308]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-14866]


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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Therapy Investigational New Drug Applications; Draft Guidance for 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the availability of a draft guidance for industry entitled ``Chemistry, 
Manufacturing, and Control (CMC) Information for Human Gene Therapy 
Investigational New Drug Applications (INDs); Draft Guidance for 
Industry.'' The draft guidance document provides sponsors of a human 
gene therapy IND with recommendations regarding CMC information 
required to assure product safety, identity, quality, purity, and 
strength (including potency) of the investigational product. The draft 
guidance applies to human gene therapy products and to combination 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    The draft guidance, when finalized, is intended to supersede the 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DATES: Submit either electronic or written comments on the draft 
guidance by October 10, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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: 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. Since 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.
    <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

[[Page 32308]]

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-2008-D-0205 for ``Chemistry, Manufacturing, and Control (CMC) 
Information for Human Gene Therapy Investigational New Drug 
Applications (INDs); Draft 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.
    <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 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.gpo.gov/fdsys/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 draft 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 draft 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 draft 
guidance document.

FOR FURTHER INFORMATION CONTACT: Gretchen Opper, 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 draft document entitled 
``Chemistry, Manufacturing, and Control (CMC) Information for Human 
Gene Therapy Investigational New Drug Applications (INDs); Draft 
Guidance for Industry.'' The draft guidance provides sponsors of a 
human gene therapy IND with recommendations regarding 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 draft 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 draft 
guidance is organized to follow the structure of the FDA guidance on 
the Common Technical Document.
    The draft guidance, when finalized, is intended to supersede the 
April 2008 guidance. Elsewhere in this issue of the Federal Register, 
FDA is announcing the availability of two other draft guidances. In a 
separate document, FDA is announcing the availability of a draft 
document entitled ``Long Term Follow-Up After Administration of Human 
Gene Therapy Products; Draft Guidance for Industry'' and the 
availability of a draft document entitled ``Testing of Retroviral 
Vector-Based Human Gene Therapy Products for Replication Competent 
Retrovirus During Product Manufacture and Patient Follow-up; Draft 
Guidance for Industry.''
    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 CMC 
information for human gene therapy INDs. 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. This guidance is not subject to 
Executive Order 12866.

II. Paperwork Reduction Act of 1995

    This draft 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. 3501-3520). 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; 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 draft guidance 
at either https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: July 5, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-14866 Filed 7-11-18; 8:45 am]
 BILLING CODE 4164-01-P