Document ID: FDA-2015-N-0430-0001
Agency: fda
Document Type: Notice
Title: Measuring Dystrophin in Dystrophinopathy Patients and Interpreting the Data; Public Scientific Workshop; Request for Comments
Posted Date: 2015-03-03T05:00Z

[Federal Register Volume 80, Number 41 (Tuesday, March 3, 2015)]
[Notices]
[Pages 11454-11455]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2015-04384]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-N-0430]

Measuring Dystrophin in Dystrophinopathy Patients and 
Interpreting the Data; Public Scientific Workshop; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public scientific workshop; request for comments.

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    The Food and Drug Administration (FDA) is announcing a public 
scientific workshop to discuss dystrophin protein quantification 
methodologies for human tissue. This workshop is being cosponsored by 
the National Institutes of Health (NIH). The purpose of the workshop is 
to discuss currently available methodologies and to identify scientific 
knowledge gaps and opportunities for improving dystrophin protein 
detection in the context of drug development. The intended audiences 
for this workshop are scientists and clinicians involved in the 
acquisition, measurement, and analysis of proteins associated with 
Duchenne Muscular Dystrophy (DMD).

[[Page 11455]]

DATES: Dates and Time: The scientific workshop will be held on March 
20, 2015, from 8:30 a.m. to 5:30 p.m.

ADDRESSES: The scientific workshop will be held at FDA's White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503A), Silver Spring, MD 20993-0002. Participants must enter 
through Building 1 and undergo security screening. For parking and 
security information, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Contact Persons: Mary Gross, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Silver Spring, MD 20993-0002, 301-796-3519, mary.gross@fda.hhs.gov; or 
Georgiann Ienzi, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 
301-796-3515, georgiann.ienzi@fda.hhs.gov.
    If you need special accommodations due to a disability, contact 
Mary Gross or Georgiann Ienzi at least 7 days in advance.
    Registration: The scientific workshop is free and seating will be 
on a first-come, first-served basis. It may be necessary to limit both 
the number of attendees from individual organizations and the total 
number of attendees based on space limitations. Email registrations 
should be sent to Dystrophin_Workshop@fda.hhs.gov by March 17, 2015. If 
you cannot attend in person, the meeting will be Webcast live. 
Information about how to access the Webcast will be located at: http://www.fda.gov/Drugs/NewsEvents/ucm432429.htm.
    Comments and Meeting Summary: Submit electronic comments to http://www.regulations.gov by May 20, 2015. Submit written comments to the 
Division of Dockets Management (HFA-305), Food and Drug Administration, 
5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Please identify your 
comments with the docket number found in brackets in the heading of 
this document. It is only necessary to send one set of comments. 
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.
    A summary of the scientific workshop's highlights will be made 
available for review at the Division of Dockets Management and at 
http://www.regulations.gov. You may submit a request to obtain a hard 
copy by sending a request to the Division of Freedom of Information 
(ELEM-1029), Office of Management Programs, Food and Drug 
Administration, 12420 Parklawn Dr., Element Bldg., Rockville, MD 20857.

SUPPLEMENTARY INFORMATION: FDA and NIH are cosponsoring this scientific 
workshop to discuss current methodologies being used in drug 
development and scientific research for DMD. Recent scientific advances 
present an opportunity for the development and validation of robust 
methods for the objective, reliable, and quantitative measurement of 
DMD-associated proteins.

I. Background

    Dystrophinopathies result from genetic mutations in the dystrophin 
gene that decrease dystrophin protein expression levels and result in 
altered dystrophin function. These changes can lead to muscle 
degeneration and, in many patients, downstream pathologies including 
inflammation and fibrosis that interfere with muscle regeneration, loss 
of movement, orthopedic complications, and ultimately respiratory and 
cardiac failure.

II. Scope of the Scientific Workshop

    The workshop will include sessions which will focus on current 
technologies used in the detection of dystrophin. Presentations will 
provide overviews of the technologies (including limitations, detection 
sensitivities, linearity, and reproducibility). A panel discussion will 
help identify development challenges for each method. Muscle biopsy 
collection, sample handling, reference materials, and image analysis 
will also be discussed.
    FDA will post the agenda and other background material 
approximately 2 days before the public scientific workshop at: http://www.fda.gov/Drugs/NewsEvents/ucm432429.htm.

    Dated: February 24, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-04384 Filed 3-2-15; 8:45 am]
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