[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Draft Guidance for Industry; Availability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Microbial Vectors Used for Gene Therapy; Draft Guidance for Industry.''
The draft guidance provides investigational new drug application (IND)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 The draft guidance focuses on the chemistry, manufacturing, and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 The draft guidance, when finalized,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draft guidance before it begins work on the final version of the
guidance, submit either electronic or written comments on the draft
guidance by December 14, 2015.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]
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