
[Federal Register Volume 80, Number 198 (Wednesday, October 14, 2015)]
[Notices]
[Pages 61822-61824]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-26108]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-3399]


Recommendations for Microbial Vectors Used for Gene Therapy; 
Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft document entitled ``Recommendations for 
Microbial Vectors Used for Gene Therapy; Draft Guidance for Industry.'' 
The draft guidance provides investigational new drug application (IND) 
sponsors, with recommendations concerning IND submissions for microbial 
vectors used for gene therapy (MVGTs) in early-phase clinical trials. 
MVGTs meet the regulatory definition of

[[Page 61823]]

``biological product'', when such products are applicable to the 
prevention, treatment, or cure of a disease or condition of human 
beings. The draft guidance focuses on the chemistry, manufacturing, and 
control (CMC) information that sponsors should submit in an IND for 
MVGTs and provides an overview of preclinical and clinical 
considerations for these products. The draft guidance, when finalized, 
will supplement the guidance 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: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by December 14, 2015.

ADDRESSES: You may submit comments as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to http://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 http://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): Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Division of 
Dockets Management, 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-2015-D-3399 for ``Recommendations for Microbial Vectors Used for 
Gene Therapy; Draft Guidance for Industry.'' Received comments will be 
placed in the docket and, except for those submitted as ``Confidential 
Submissions'' publicly viewable at http://www.regulations.gov or at the 
Division of Dockets Management 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 http://www.regulations.gov. 
Submit both copies to the Division of Dockets Management. 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: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to http://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 Division of Dockets Management, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.
    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: Paul E. Levine, Jr., 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, 
``Recommendations for Microbial Vectors Used for Gene Therapy; Draft 
Guidance for Industry.'' The draft guidance provides IND sponsors, with 
recommendations concerning IND submissions for MVGTs in early-phase 
clinical trials. MVGTs meet the definition of ``biological product'' in 
section 351(i) of the Public Health Service Act (42 U.S.C. 262), when 
such products are applicable to the prevention, treatment, or cure of a 
disease or condition of human beings. MVGTs include bacterial vectors 
such as Salmonella, Listeria, or E. coli genetically modified to 
express human tumor antigens, cytokines, growth factors, enzymes, 
therapeutic proteins, or nucleotides. MVGTs may also be generated by 
the modification (deletion, truncation, or point mutation) of 
chromosomal or episomal genes and by the insertion of foreign genetic 
material into the chromosome, or into naturally occurring episomes; or 
by the introduction of one or more plasmids. The MVGTs may consist of 
microbes that are either live, replication restricted (division under 
specific growth conditions), capable of limited or no cell divisions, 
or killed, or a combination of these forms. The guidance focuses on the 
CMC information that sponsors should submit in an IND for MVGTs and 
provides an overview of preclinical and clinical considerations for 
these products.

[[Page 61824]]

    In the Federal Register of April 10, 2008 (73 FR 19511), FDA 
announced the availability of the April 2008 Guidance. In that 
guidance, FDA provided sponsors of a human gene therapy IND, including 
those with combination products that contain a human gene therapy 
biological product with a drug or device as part of the final product, 
with recommendations on CMC information that is to be included in an 
original IND. That guidance also provided instruction to FDA CMC 
reviewers about the information to record and assess as part of an IND 
review. The draft guidance, when finalized, will supplement the April 
2008 Guidance.
    The draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent FDA's current thinking on recommendations for 
MVGTs. It does not establish any rights for or on any person and does 
not operate to bind FDA or the public. An alternative approach may be 
used if such approach satisfies the requirement of the applicable 
statutes and regulations.

II. Paperwork Reduction Act of 1995

    The 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 parts 211, 610, and 312 have been 
approved under OMB control numbers 0910-0139 and 0910-0114, 
respectively.

III. Electronic Access

    Persons with access to the Internet may obtain the draft guidance 
at either http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or http://www.regulations.gov.

    Dated: October 7, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-26108 Filed 10-13-15; 8:45 am]
BILLING CODE 4164-01-P


