80_FR_33072 80 FR 32961 - Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment; Draft Guidance for Industry; Availability

80 FR 32961 - Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 111 (June 10, 2015)

Page Range32961-32961
FR Document2015-14100

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment.'' The purpose of this draft guidance is to assist sponsors in the clinical development of drugs for the treatment of X-linked Duchenne muscular dystrophy (DMD) and related dystrophinopathies.

Federal Register, Volume 80 Issue 111 (Wednesday, June 10, 2015) 
[Federal Register Volume 80, Number 111 (Wednesday, June 10, 2015)]
[Notices]
[Page 32961]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-14100]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-1884]


Duchenne Muscular Dystrophy and Related Dystrophinopathies: 
Developing Drugs for Treatment; Draft Guidance for Industry; 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the availability of a draft guidance for industry entitled ``Duchenne 
Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for 
Treatment.'' The purpose of this draft guidance is to assist sponsors 
in the clinical development of drugs for the treatment of X-linked 
Duchenne muscular dystrophy (DMD) and related dystrophinopathies.

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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by August 10, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993-0002. Send one self-addressed adhesive label to assist that 
office in processing your requests. See the SUPPLEMENTARY INFORMATION 
section for electronic access to the draft guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.

FOR FURTHER INFORMATION CONTACT: Colleen Locicero, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 4242, Silver Spring, MD 20993-0002, 301-
796-1114.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Duchenne Muscular Dystrophy and Related Dystrophinopathies: 
Developing Drugs for Treatment.''
    DMD and other dystrophinopathies result from genetic mutations in 
the dystrophin gene that decrease levels of dystrophin and/or cause 
dysfunction of the dystrophin protein, leading to muscle degeneration, 
including cardiac and respiratory muscles, and greatly decreased life 
expectancy. There remains a high level unmet medical need for effective 
drug treatments for DMD and other dystrophinopathies. This draft 
guidance addresses FDA's current thinking regarding the clinical 
development program and clinical trial designs for drugs to support an 
indication for the treatment of dystrophinopathies. Development of this 
draft guidance was greatly facilitated by the efforts of Parent Project 
Muscular Dystrophy to coordinate a consortium of stakeholders including 
patients, parents and caregivers, clinicians, academic experts, and 
industry representatives in producing a proposed draft guidance with 
extensive background information about DMD. That stakeholder proposal 
was submitted to FDA and made available for comment through a Federal 
Register notice seeking public comment. The comments received were also 
considered in writing this draft guidance.
    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 developing 
drugs for the treatment of DMD. 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.

II. The Paperwork Reduction Act of 1995

    This guidance refers to previously approved collections of 
information that 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 312 and 314 have 
been approved under OMB control numbers 0910-0014 and 0910-0001, 
respectively.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

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

    Dated: June 4, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-14100 Filed 6-9-15; 8:45 am]
BILLING CODE 4164-01-P

Federal Register / Vol. 80, No. 111 / Wednesday, June 10, 2015 / Notices 32961 ANNUAL BURDEN ESTIMATES Number of Average Number of Total burden Instrument responses per burden hours respondents hours respondent per response State Developmental Disabilities Program Performance Report (PPR) ......... 56 1 138 7,728 Estimated Total Annual Burden Hours: .................................................... ........................ ........................ ........................ 7,728 Dated: June 3, 2015. www.regulations.gov. Submit written on developing drugs for the treatment of Kathy Greenlee, comments to the Division of Dockets DMD. It does not establish any rights for Administrator and Assistant Secretary for Management (HFA–305), Food and Drug any person and is not binding on FDA Aging. Administration, 5630 Fishers Lane, Rm. or the public. You can use an alternative [FR Doc. 2015–14051 Filed 6–9–15; 8:45 am] 1061, Rockville, MD 20852. approach if it satisfies the requirements BILLING CODE 4154–01–P FOR FURTHER INFORMATION CONTACT: of the applicable statutes and Colleen Locicero, Center for Drug regulations. Evaluation and Research, Food and DEPARTMENT OF HEALTH AND Drug Administration, 10903 New II. The Paperwork Reduction Act of HUMAN SERVICES Hampshire Ave., Bldg. 22, Rm. 4242, 1995 Silver Spring, MD 20993–0002, 301– Food and Drug Administration This guidance refers to previously 796–1114. approved collections of information that [Docket No. FDA–2015–D–1884] SUPPLEMENTARY INFORMATION: are subject to review by the Office of Duchenne Muscular Dystrophy and I. Background Management and Budget (OMB) under Related Dystrophinopathies: FDA is announcing the availability of the Paperwork Reduction Act of 1995 Developing Drugs for Treatment; Draft a draft guidance for industry entitled (44 U.S.C. 3501–3520). The collections Guidance for Industry; Availability ‘‘Duchenne Muscular Dystrophy and of information in 21 CFR parts 312 and Related Dystrophinopathies: Developing 314 have been approved under OMB AGENCY: Food and Drug Administration, Drugs for Treatment.’’ control numbers 0910–0014 and 0910– HHS. DMD and other dystrophinopathies 0001, respectively. ACTION: Notice. result from genetic mutations in the dystrophin gene that decrease levels of III. Comments SUMMARY: The Food and Drug dystrophin and/or cause dysfunction of Interested persons may submit either Administration (FDA or Agency) is the dystrophin protein, leading to electronic comments regarding this announcing the availability of a draft muscle degeneration, including cardiac document to http://www.regulations.gov guidance for industry entitled and respiratory muscles, and greatly or written comments to the Division of ‘‘Duchenne Muscular Dystrophy and decreased life expectancy. There Related Dystrophinopathies: Developing Dockets Management (see ADDRESSES). It remains a high level unmet medical Drugs for Treatment.’’ The purpose of is only necessary to send one set of need for effective drug treatments for this draft guidance is to assist sponsors comments. Identify comments with the DMD and other dystrophinopathies. in the clinical development of drugs for docket number found in brackets in the This draft guidance addresses FDA’s the treatment of X-linked Duchenne heading of this document. Received current thinking regarding the clinical muscular dystrophy (DMD) and related comments may be seen in the Division development program and clinical trial dystrophinopathies. of Dockets Management between 9 a.m. designs for drugs to support an DATES: Although you can comment on indication for the treatment of and 4 p.m., Monday through Friday, and any guidance at any time (see 21 CFR dystrophinopathies. Development of will be posted to the docket at http:// 10.115(g)(5)), to ensure that the Agency this draft guidance was greatly www.regulations.gov. considers your comment on this draft facilitated by the efforts of Parent guidance before it begins work on the IV. Electronic Access Project Muscular Dystrophy to final version of the guidance, submit coordinate a consortium of stakeholders Persons with access to the Internet either electronic or written comments including patients, parents and may obtain the document at either on the draft guidance by August 10, caregivers, clinicians, academic experts, 2015. http://www.fda.gov/Drugs/Guidance and industry representatives in ComplianceRegulatoryInformation/ ADDRESSES: Submit written requests for producing a proposed draft guidance Guidances/default.htm or http:// single copies of the draft guidance to the with extensive background information www.regulations.gov. Division of Drug Information, Center for about DMD. That stakeholder proposal Drug Evaluation and Research, Food was submitted to FDA and made Dated: June 4, 2015. and Drug Administration, 10001 New available for comment through a Leslie Kux, Hampshire Ave., Hillandale Building, Federal Register notice seeking public asabaliauskas on DSK5VPTVN1PROD with NOTICES Associate Commissioner for Policy. 4th Floor, Silver Spring, MD 20993– comment. The comments received were [FR Doc. 2015–14100 Filed 6–9–15; 8:45 am] 0002. Send one self-addressed adhesive also considered in writing this draft BILLING CODE 4164–01–P label to assist that office in processing guidance. your requests. See the SUPPLEMENTARY This draft guidance is being issued INFORMATION section for electronic consistent with FDA’s good guidance access to the draft guidance document. practices regulation (21 CFR 10.115). Submit electronic comments on the The draft guidance, when finalized, will draft guidance to http:// represent the current thinking of FDA VerDate Sep<11>2014 16:46 Jun 09, 2015 Jkt 235001 PO 00000 Frm 00033 Fmt 4703 Sfmt 9990 E:\FR\FM\10JNN1.SGM 10JNN1


Document Created: 2015-12-15 15:17:24
Document Modified: 2015-12-15 15:17:24
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice.
DatesAlthough 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 August 10, 2015.
ContactColleen Locicero, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 4242, Silver Spring, MD 20993-0002, 301- 796-1114.
FR Citation80 FR 32961 
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