83 FR 32307 - Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 134 (July 12, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 134 (July 12, 2018)
| Page Range | 32307-32308 | |
| FR Document | 2018-14866 |
[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)] [Notices] [Pages 32307-32308] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-14866] ----------------------------------------------------------------------- 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Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry.'' The draft guidance document provides sponsors of a human gene therapy IND with recommendations regarding CMC information required to assure product safety, identity, quality,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 The draft guidance applies to human gene therapy products and to combination products that contain a human gene therapy in combination with a drug or device. The draft guidance, when finalized, is intended to supersede the document entitled ``Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and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 DATES: Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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: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. 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 [[Page 32308]] 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 (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. 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.thefederalregister.org/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
![Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 32307
800–835–4709 or 240–402–8010. See II. Paperwork Reduction Act of 1995 Applications (INDs); Draft Guidance for
the SUPPLEMENTARY INFORMATION section This draft guidance refers to Industry.’’ The draft guidance document
for electronic access to the draft previously approved collections of provides sponsors of a human gene
guidance document. information subject to review by the therapy IND with recommendations
Office of Management and Budget regarding CMC information required to
FOR FURTHER INFORMATION CONTACT:
(OMB) under the Paperwork Reduction assure product safety, identity, quality,
Jessica Walker Udechukwu, Center for purity, and strength (including potency)
Biologics Evaluation and Research, Act of 1995 (44 U.S.C. 3501–3520). The
collections of information in 21 CFR of the investigational product. The draft
Food and Drug Administration, 10903 guidance applies to human gene therapy
New Hampshire Ave., Bldg. 71, Rm. part 58 have been approved under OMB
control number 0910–0119; the products and to combination products
7301, Silver Spring, MD 20993–0002, that contain a human gene therapy in
240–402–7911. collections of information in 21 CFR
part 211 have been approved under combination with a drug or device.
SUPPLEMENTARY INFORMATION: OMB control number 0910–0139; the The draft guidance, when finalized, is
collections of information in 21 CFR intended to supersede the document
I. Background entitled ‘‘Guidance for FDA Reviewers
part 312 have been approved under
FDA is announcing the availability of OMB control number 0910–0014; the and Sponsors: Content and Review of
a draft document entitled ‘‘Human Gene collections of information in 21 CFR Chemistry, Manufacturing, and Control
part 601 have been approved under (CMC) Information for Human Gene
Therapy for Hemophilia; Draft Guidance
OMB control number 0910–0338; the Therapy Investigational New Drug
for Industry.’’ The draft guidance
collections of information in the Applications (INDs),’’ dated April 2008
document provides recommendations to
guidance entitled ‘‘Expedited Programs (April 2008 guidance).
stakeholders developing GT products
for the treatment of hemophilia. The for Serious Conditions—Drugs and DATES: Submit either electronic or
draft guidance provides Biologics’’ have been approved under written comments on the draft guidance
recommendations on the clinical trial OMB control number 0910–0765; and by October 10, 2018 to ensure that the
design and related development of the collections of information in the Agency considers your comment on this
coagulation factor VIII (hemophilia A) guidance entitled ‘‘Formal Meetings draft guidance before it begins work on
and IX (hemophilia B) activity assays, Between the FDA and Sponsors or the final version of the guidance.
including how to address discrepancies Applicants’’ have been approved under ADDRESSES: You may submit comments
in factor VIII and factor IX activity OMB control number 0910–0429. on any guidance at any time as follows:
assays. The draft guidance also includes III. Electronic Access Electronic Submissions
recommendations regarding preclinical
considerations to support development Persons with access to the internet Submit electronic comments in the
of GT products for the treatment of may obtain the draft guidance at either following way:
hemophilia. Hemophilia therapy in the https://www.fda.gov/BiologicsBlood • Federal eRulemaking Portal:
United States has progressed from Vaccines/GuidanceCompliance https://www.regulations.gov. Follow the
replacement therapies for on-demand RegulatoryInformation/Guidances/ instructions for submitting comments.
treatment of bleeding to prophylaxis to default.htm or https:// Comments submitted electronically,
reduce the frequency of bleeding. GT www.regulations.gov. including attachments, to https://
products for hemophilia are being Dated: July 5, 2018. www.regulations.gov will be posted to
developed as single-dose treatments that Leslie Kux. the docket unchanged. Since your
may provide long-term expression of the Associate Commissioner for Policy.
comment will be made public, you are
missing or abnormal coagulation factor solely responsible for ensuring that your
[FR Doc. 2018–14875 Filed 7–11–18; 8:45 am]
in the patient at steady levels to reduce comment does not include any
BILLING CODE 4164–01–P
or eliminate the need for exogenous confidential information that you or a
factor replacement. third party may not wish to be posted,
such as medical information, your or
Elsewhere in this issue of the Federal DEPARTMENT OF HEALTH AND
anyone else’s Social Security number, or
Register, FDA is announcing the HUMAN SERVICES
confidential business information, such
availability of two other human gene as a manufacturing process. Please note
therapy draft guidance documents Food and Drug Administration
that if you include your name, contact
entitled ‘‘Human Gene Therapy for [Docket No. FDA–2008–D–0205] information, or other information that
Retinal Disorders; Draft Guidance for identifies you in the body of your
Industry’’ and ‘‘Human Gene Therapy Chemistry, Manufacturing, and Control
comments, that information will be
for Rare Diseases; Draft Guidance for Information for Human Gene Therapy
posted on https://www.regulations.gov.
Industry.’’ Investigational New Drug Applications;
• If you want to submit a comment
This draft guidance is being issued Draft Guidance for Industry;
with confidential information that you
consistent with FDA’s good guidance Availability
do not wish to be made available to the
practices regulation (21 CFR 10.115). AGENCY: Food and Drug Administration, public, submit the comment as a
The draft guidance, when finalized, will HHS. written/paper submission and in the
represent the current thinking of FDA ACTION: Notice of availability. manner detailed (see ‘‘Written/Paper
on ‘‘Human Gene Therapy for Submissions’’ and ‘‘Instructions’’).
amozie on DSK3GDR082PROD with NOTICES1
Hemophilia.’’ It does not establish any SUMMARY: The Food and Drug
rights for any person and is not binding Administration (FDA or Agency) is Written/Paper Submissions
on FDA or the public. You can use an announcing the availability of a draft Submit written/paper submissions as
alternative approach if it satisfies the guidance for industry entitled follows:
requirements of the applicable statutes ‘‘Chemistry, Manufacturing, and Control • Mail/Hand Delivery/Courier (for
and regulations. This guidance is not (CMC) Information for Human Gene Written/Paper Submissions): Dockets
subject to Executive Order 12866. Therapy Investigational New Drug Management Staff (HFA–305), Food and
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![32308 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices
Drug Administration, 5630 Fishers www.regulations.gov and insert the The draft guidance, when finalized, is
Lane, Rm. 1061, Rockville, MD 20852. docket number, found in brackets in the intended to supersede the April 2008
• For written/paper comments heading of this document, into the guidance. Elsewhere in this issue of the
submitted to the Dockets Management ‘‘Search’’ box and follow the prompts Federal Register, FDA is announcing
Staff, FDA will post your comment as and/or go to the Dockets Management the availability of two other draft
well as any attachments, except for Staff, 5630 Fishers Lane, Rm. 1061, guidances. In a separate document, FDA
information submitted, marked, and Rockville, MD 20852. is announcing the availability of a draft
identified as confidential if submitted as You may submit comments on any document entitled ‘‘Long Term Follow-
detailed in ‘‘Instructions.’’ guidance at any time (see 21 CFR Up After Administration of Human
Instructions: All submissions received 10.115(g)(5)). Gene Therapy Products; Draft Guidance
must include the Docket No. FDA– Submit written requests for single for Industry’’ and the availability of a
2008–D–0205 for ‘‘Chemistry, copies of the draft guidance to the Office draft document entitled ‘‘Testing of
Manufacturing, and Control (CMC) of Communication, Outreach and Retroviral Vector-Based Human Gene
Information for Human Gene Therapy Development, Center for Biologics Therapy Products for Replication
Investigational New Drug Applications Evaluation and Research (CBER), Food Competent Retrovirus During Product
(INDs); Draft Guidance for Industry.’’ and Drug Administration, 10903 New Manufacture and Patient Follow-up;
Received comments will be placed in Hampshire Ave., Bldg. 71, Rm. 3128, Draft Guidance for Industry.’’
the docket and, except for those Silver Spring, MD 20993–0002. Send This draft guidance is being issued
submitted as ‘‘Confidential one self-addressed adhesive label to consistent with FDA’s good guidance
Submissions,’’ publicly viewable at assist the office in processing your practices regulation (21 CFR 10.115).
https://www.regulations.gov or at the requests. The draft guidance may also be The draft guidance, when finalized, will
Dockets Management Staff between 9 obtained by mail by calling CBER at 1– represent the current thinking of FDA
a.m. and 4 p.m., Monday through 800–835–4709 or 240–402–8010. See on CMC information for human gene
Friday. the SUPPLEMENTARY INFORMATION section therapy INDs. It does not establish any
• Confidential Submissions—To for electronic access to the draft rights for any person and is not binding
submit a comment with confidential guidance document. on FDA or the public. You can use an
information that you do not wish to be FOR FURTHER INFORMATION CONTACT: alternative approach if it satisfies the
made publicly available, submit your Gretchen Opper, Center for Biologics requirements of the applicable statutes
comments only as a written/paper Evaluation and Research, Food and and regulations. This guidance is not
submission. You should submit two Drug Administration, 10903 New subject to Executive Order 12866.
copies, total. One copy will include the Hampshire Ave., Bldg. 71, Rm. 7301,
information you claim to be confidential Silver Spring, MD 20993–0002, 240– II. Paperwork Reduction Act of 1995
with a heading or cover note that states 402–7911. This draft guidance refers to
‘‘THIS DOCUMENT CONTAINS SUPPLEMENTARY INFORMATION: previously approved collections of
CONFIDENTIAL INFORMATION.’’ The information found in FDA regulations.
Agency will review this copy, including I. Background
These collections of information are
the claimed confidential information, in FDA is announcing the availability of subject to review by the Office of
its consideration of comments. The a draft document entitled ‘‘Chemistry, Management and Budget (OMB) under
second copy, which will have the Manufacturing, and Control (CMC) the Paperwork Reduction Act of 1995
claimed confidential information Information for Human Gene Therapy (44 U.S.C. 3501–3520). The collections
redacted/blacked out, will be available Investigational New Drug Applications of information in 21 CFR part 211 have
for public viewing and posted on (INDs); Draft Guidance for Industry.’’ been approved under OMB control
https://www.regulations.gov. Submit The draft guidance provides sponsors of number 0910–0139; the collections of
both copies to the Dockets Management a human gene therapy IND with information in 21 CFR part 312 and
Staff. If you do not wish your name and recommendations regarding CMC Form FDA 1571 have been approved
contact information to be made publicly information required to assure product under OMB control number 0910–0014;
available, you can provide this safety, identity, quality, purity, and and the collections of information in 21
information on the cover sheet and not strength (including potency) of the CFR part 1271 have been approved
in the body of your comments, and you investigational product (21 CFR under OMB control number 0910–0543.
must identify this information as 312.23(a)(7)(i)). The draft guidance
‘‘confidential.’’ Any information marked applies to human gene therapy products III. Electronic Access
as ‘‘confidential’’ will not be disclosed and to combination products that Persons with access to the internet
except in accordance with 21 CFR 10.20 contain a human gene therapy in may obtain the draft guidance at either
and other applicable disclosure law. For combination with a drug or device. The https://www.fda.gov/BiologicsBlood
more information about FDA’s posting field of gene therapy has progressed Vaccines/GuidanceCompliance
of comments to public dockets, see 80 rapidly since FDA issued the April 2008 RegulatoryInformation/Guidances/
FR 56469, September 18, 2015, or access guidance. Therefore, FDA is updating default.htm or https://
the information at: https://www.gpo.gov/ the guidance to provide current FDA www.regulations.gov.
fdsys/pkg/FR-2015-09-18/pdf/2015- recommendations regarding the CMC
23389.pdf. Dated: July 5, 2018.
content of a gene therapy IND. In
amozie on DSK3GDR082PROD with NOTICES1
Docket: For access to the docket to addition, the draft guidance is organized Leslie Kux,
read background documents or the to follow the structure of the FDA Associate Commissioner for Policy.
electronic and written/paper comments guidance on the Common Technical [FR Doc. 2018–14866 Filed 7–11–18; 8:45 am]
received, go to https:// Document. BILLING CODE 4164–01–P
VerDate Sep<11>2014 17:21 Jul 11, 2018 Jkt 244001 PO 00000 Frm 00049 Fmt 4703 Sfmt 4703 E:\FR\FM\12JYN1.SGM 12JYN1](https://thefederalregister.org/doc/83_FR_32441/page/2.svg)
Document Created: 2018-11-06 10:22:41
Document Modified: 2018-11-06 10:22:41
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of availability. | |
| 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. | |
| 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. | |
| FR Citation | 83 FR 32307 |
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