Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 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Therapy for Hemophilia; Draft Guidance 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 The draft guidance 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draft guidance provides 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 The draft guidance also includes 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therapy draft guidance documents 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Retinal Disorders; Draft Guidance for 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for Rare Diseases; Draft Guidance for 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This draft guidance is being issued 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The draft guidance, when finalized, will 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/BiologicsBlood

Vaccines/GuidanceCompliance

RegulatoryInformation/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 

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 


amozie on DSK3GDR082PROD with NOTICES1
32308 Federal Register / Vol. 83, No. 134 / Thursday, July 12, 2018 / Notices 

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.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 FollowUp 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/BiologicsBlood

Vaccines/GuidanceCompliance

RegulatoryInformation/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 


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