83 FR 32306 - Human Gene Therapy for Hemophilia; Draft Guidance for Industry; Availability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 | 32306-32307 | |
| FR Document | 2018-14875 |
[Federal Register Volume 83, Number 134 (Thursday, July 12, 2018)] [Notices] [Pages 32306-32307] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-14875] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA 2018-D-2238] Human Gene Therapy for Hemophilia; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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(FDA or Agency) is announcing the availability of a draft document entitled ``Human Gene Therapy for Hemophilia; Draft Guidance for Industry.'' The draft guidance document provides recommendations to stakeholders developing human gene therapy (GT) products for the treatment of hemophilia. The draft guidance provides recommendations on the clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. The draft guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia. DATES: Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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. 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 2018-D-2238 for ``Human Gene Therapy for Hemophilia; 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- [[Page 32307]] 800-835-4709 or 240-402-8010. See the SUPPLEMENTARY INFORMATION section for electronic access to the draft guidance document. FOR FURTHER INFORMATION CONTACT: Jessica Walker Udechukwu, 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 ``Human Gene Therapy for Hemophilia; Draft Guidance for Industry.'' The draft guidance document provides recommendations to stakeholders developing GT products for the treatment of hemophilia. The draft guidance provides recommendations on the clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. The draft guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia. Hemophilia therapy in the United States has progressed from replacement therapies for on-demand treatment of bleeding to prophylaxis to reduce the frequency of bleeding. GT products for hemophilia are being developed as single-dose treatments that may provide long-term expression of the missing or abnormal coagulation factor in the patient at steady levels to reduce or eliminate the need for exogenous factor replacement. Elsewhere in this issue of the Federal Register, FDA is announcing the availability of two other human gene therapy draft guidance documents entitled ``Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry'' and ``Human Gene Therapy for Rare Diseases; 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 ``Human Gene Therapy for Hemophilia.'' 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 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 58 have been approved under OMB control number 0910-0119; 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 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; the collections of information in the guidance entitled ``Expedited Programs for Serious Conditions--Drugs and Biologics'' have been approved under OMB control number 0910-0765; and the collections of information in the guidance entitled ``Formal Meetings Between the FDA and Sponsors or Applicants'' have been approved under OMB control number 0910-0429. 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. 2018-14875 Filed 7-11-18; 8:45 am] BILLING CODE 4164-01-P
![32306 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices
Application No. Drug Applicant
NDA 050807 ......... Epirubicin HCl for Injection, 50 mg/vial, 200 mg/vial .............................. Hospira, Inc.
Therefore, approval of the Agency considers your comment on this a.m. and 4 p.m., Monday through
applications listed in the table, and all draft guidance before it begins work on Friday.
amendments and supplements thereto, the final version of the guidance. • Confidential Submissions—To
is hereby withdrawn as of August 13, ADDRESSES: You may submit comments submit a comment with confidential
2018. Introduction or delivery for on any guidance at any time as follows: information that you do not wish to be
introduction into interstate commerce of made publicly available, submit your
products without approved new drug Electronic Submissions comments only as a written/paper
applications violates section 301(a) and Submit electronic comments in the submission. You should submit two
(d) of the Federal Food, Drug, and following way: copies, total. One copy will include the
Cosmetic Act (21 U.S.C. 331(a) and (d)). • Federal eRulemaking Portal: information you claim to be confidential
Drug products that are listed in the table https://www.regulations.gov. Follow the with a heading or cover note that states
that are in inventory on August 13, 2018 instructions for submitting comments. ‘‘THIS DOCUMENT CONTAINS
may continue to be dispensed until the Comments submitted electronically, CONFIDENTIAL INFORMATION.’’ The
inventories have been depleted or the Agency will review this copy, including
including attachments, to https://
drug products have reached their the claimed confidential information, in
www.regulations.gov will be posted to
expiration dates or otherwise become its consideration of comments. The
the docket unchanged. Because your
violative, whichever occurs first. second copy, which will have the
comment will be made public, you are
claimed confidential information
Dated: July 9, 2018. solely responsible for ensuring that your
redacted/blacked out, will be available
Leslie Kux, comment does not include any
for public viewing and posted on
Associate Commissioner for Policy. confidential information that you or a
https://www.regulations.gov. Submit
third party may not wish to be posted,
[FR Doc. 2018–14935 Filed 7–11–18; 8:45 am] both copies to the Dockets Management
such as medical information, your or
BILLING CODE 4164–01–P Staff. If you do not wish your name and
anyone else’s Social Security number, or contact information to be made publicly
confidential business information, such available, you can provide this
DEPARTMENT OF HEALTH AND as a manufacturing process. Please note information on the cover sheet and not
HUMAN SERVICES that if you include your name, contact in the body of your comments, and you
information, or other information that must identify this information as
Food and Drug Administration identifies you in the body of your ‘‘confidential.’’ Any information marked
comments, that information will be as ‘‘confidential’’ will not be disclosed
[Docket No. FDA 2018–D–2238]
posted on https://www.regulations.gov. except in accordance with 21 CFR 10.20
Human Gene Therapy for Hemophilia; • If you want to submit a comment and other applicable disclosure law. For
Draft Guidance for Industry; with confidential information that you more information about FDA’s posting
Availability do not wish to be made available to the of comments to public dockets, see 80
public, submit the comment as a FR 56469, September 18, 2015, or access
AGENCY: Food and Drug Administration, written/paper submission and in the the information at: https://www.gpo.gov/
HHS. manner detailed (see ‘‘Written/Paper fdsys/pkg/FR-2015-09-18/pdf/2015-
ACTION: Notice of availability. Submissions’’ and ‘‘Instructions’’). 23389.pdf.
Written/Paper Submissions Docket: For access to the docket to
SUMMARY: The Food and Drug
read background documents or the
Administration (FDA or Agency) is Submit written/paper submissions as electronic and written/paper comments
announcing the availability of a draft follows: received, go to https://
document entitled ‘‘Human Gene • Mail/Hand Delivery/Courier (for www.regulations.gov and insert the
Therapy for Hemophilia; Draft Guidance Written/Paper Submissions): Dockets docket number, found in brackets in the
for Industry.’’ The draft guidance Management Staff (HFA–305), Food and heading of this document, into the
document provides recommendations to Drug Administration, 5630 Fishers ‘‘Search’’ box and follow the prompts
stakeholders developing human gene Lane, Rm. 1061, Rockville, MD 20852. and/or go to the Dockets Management
therapy (GT) products for the treatment • For written/paper comments Staff, 5630 Fishers Lane, Rm. 1061,
of hemophilia. The draft guidance submitted to the Dockets Management Rockville, MD 20852.
provides recommendations on the Staff, FDA will post your comment as You may submit comments on any
clinical trial design and related well as any attachments, except for guidance at any time (see 21 CFR
development of coagulation factor VIII information submitted, marked, and 10.115(g)(5)).
(hemophilia A) and IX (hemophilia B) identified as confidential, if submitted Submit written requests for single
activity assays, including how to as detailed in ‘‘Instructions.’’ copies of the draft guidance to the Office
address discrepancies in factor VIII and Instructions: All submissions received of Communication, Outreach and
factor IX activity assays. The draft must include the Docket No. FDA 2018– Development, Center for Biologics
guidance also includes D–2238 for ‘‘Human Gene Therapy for Evaluation and Research (CBER), Food
amozie on DSK3GDR082PROD with NOTICES1
recommendations regarding preclinical Hemophilia; Draft Guidance for and Drug Administration, 10903 New
considerations to support development Industry.’’ Received comments will be Hampshire Ave., Bldg. 71, Rm. 3128,
of GT products for the treatment of placed in the docket and, except for Silver Spring, MD 20993–0002. Send
hemophilia. those submitted as ‘‘Confidential one self-addressed adhesive label to
DATES: Submit either electronic or Submissions,’’ publicly viewable at assist the office in processing your
written comments on the draft guidance https://www.regulations.gov or at the requests. The draft guidance may also be
by October 10, 2018 to ensure that the Dockets Management Staff between 9 obtained by mail by calling CBER at 1–
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![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
VerDate Sep<11>2014 17:21 Jul 11, 2018 Jkt 244001 PO 00000 Frm 00048 Fmt 4703 Sfmt 4703 E:\FR\FM\12JYN1.SGM 12JYN1](https://thefederalregister.org/doc/83_FR_32440/page/2.svg)
Document Created: 2018-11-06 10:22:42
Document Modified: 2018-11-06 10:22:42
| 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 | Jessica Walker Udechukwu, 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 32306 |
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