80 FR 32961 - Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment; Draft Guidance for Industry; Availability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

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment.'' The purpose of this draft guidance is to assist sponsors in the clinical development of drugs for the treatment of X-linked Duchenne muscular dystrophy (DMD) and related dystrophinopathies.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, 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Developing Drugs for Treatment; Draft Guidance for Industry; 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the availability of a draft guidance for industry entitled ``Duchenne 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 The purpose of this draft guidance is to assist sponsors 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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by August 10, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 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section for electronic access to the draft guidance document.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]
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