83_FR_7083 83 FR 7050 - Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment; Guidance for Industry; Availability

83 FR 7050 - Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment; Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 33 (February 16, 2018)

Page Range7050-7051
FR Document2018-03225

The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled ``Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment.'' The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of X-linked Duchenne muscular dystrophy (DMD) and related dystrophinopathies. This guidance finalizes the draft guidance of the same name issued on June 10, 2015.

Federal Register, Volume 83 Issue 33 (Friday, February 16, 2018)
[Federal Register Volume 83, Number 33 (Friday, February 16, 2018)]
[Notices]
[Pages 7050-7051]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-03225]


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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; 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 guidance for industry entitled ``Duchenne 
Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for 
Treatment.'' The purpose of this guidance is to assist sponsors in the 
clinical development of drugs for the treatment of X-linked Duchenne 
muscular dystrophy (DMD) and related dystrophinopathies. This guidance 
finalizes the draft guidance of the same name issued on June 10, 2015.

DATES: The announcement of the guidance is published in the Federal 
Register on February 16, 2018.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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-2015-D-1884 for ``Duchenne Muscular Dystrophy and Related 
Dystrophinopathies: Developing Drugs for Treatment; Guidance for 
Industry; Availability.'' 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 this 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-

[[Page 7051]]

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 guidance document.

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 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 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. This guidance finalizes the draft 
guidance of the same name issued June 10, 2015 (80 FR 32961). It 
reflects FDA's consideration of public comments on the draft guidance 
and makes minor clarifying changes.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents 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. This 
guidance is not subject to Executive Order 12866.

II. 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. Electronic Access

    Persons with access to the internet may obtain the guidance at 
either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: February 12, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-03225 Filed 2-15-18; 8:45 am]
BILLING CODE 4164-01-P



                                               7050                          Federal Register / Vol. 83, No. 33 / Friday, February 16, 2018 / Notices

                                               statement of the general nature of the                  announcing the availability of a                      2015–D–1884 for ‘‘Duchenne Muscular
                                               evidence or arguments they wish to                      guidance for industry entitled                        Dystrophy and Related
                                               present, the names and addresses of                     ‘‘Duchenne Muscular Dystrophy and                     Dystrophinopathies: Developing Drugs
                                               proposed participants, and an                           Related Dystrophinopathies: Developing                for Treatment; Guidance for Industry;
                                               indication of the approximate time                      Drugs for Treatment.’’ The purpose of                 Availability.’’ Received comments will
                                               requested to make their presentation on                 this guidance is to assist sponsors in the            be placed in the docket and, except for
                                               or before April 18, 2018. Time allotted                 clinical development of drugs for the                 those submitted as ‘‘Confidential
                                               for each presentation may be limited. If                treatment of X-linked Duchenne                        Submissions,’’ publicly viewable at
                                               the number of registrants requesting to                 muscular dystrophy (DMD) and related                  https://www.regulations.gov or at the
                                               speak is greater than can be reasonably                 dystrophinopathies. This guidance                     Dockets Management Staff between 9
                                               accommodated during the scheduled                       finalizes the draft guidance of the same              a.m. and 4 p.m., Monday through
                                               open public hearing session, FDA may                    name issued on June 10, 2015.                         Friday.
                                               conduct a lottery to determine the                      DATES: The announcement of the                           • Confidential Submissions—To
                                               speakers for the scheduled open public                  guidance is published in the Federal                  submit a comment with confidential
                                               hearing session. The contact person will                Register on February 16, 2018.                        information that you do not wish to be
                                               notify interested persons regarding their               ADDRESSES: You may submit either                      made publicly available, submit your
                                               request to speak by April 19, 2018.                     electronic or written comments on                     comments only as a written/paper
                                                  Persons attending FDA’s advisory                     Agency guidances at any time as                       submission. You should submit two
                                               committee meetings are advised that                     follows:                                              copies total. One copy will include the
                                               FDA is not responsible for providing                                                                          information you claim to be confidential
                                               access to electrical outlets.                           Electronic Submissions                                with a heading or cover note that states
                                                  For press inquiries, please contact the                Submit electronic comments in the                   ‘‘THIS DOCUMENT CONTAINS
                                               Office of Media Affairs at fdaoma@                      following way:                                        CONFIDENTIAL INFORMATION.’’ The
                                               fda.hhs.gov or 301–796–4540.                              • Federal eRulemaking Portal:                       Agency will review this copy, including
                                                  FDA welcomes the attendance of the                   https://www.regulations.gov. Follow the               the claimed confidential information, in
                                               public at its advisory committee                        instructions for submitting comments.                 its consideration of comments. The
                                               meetings and will make every effort to                  Comments submitted electronically,                    second copy, which will have the
                                               accommodate persons with disabilities.                  including attachments, to https://                    claimed confidential information
                                               If you require accommodations due to a                  www.regulations.gov will be posted to                 redacted/blacked out, will be available
                                               disability, please contact LaToya Bonner                the docket unchanged. Because your                    for public viewing and posted on
                                               (see FOR FURTHER INFORMATION CONTACT)                   comment will be made public, you are                  https://www.regulations.gov. Submit
                                               at least 7 days in advance of the                       solely responsible for ensuring that your             both copies to the Dockets Management
                                               meeting.                                                comment does not include any                          Staff. If you do not wish your name and
                                                  FDA is committed to the orderly                      confidential information that you or a                contact information to be made publicly
                                               conduct of its advisory committee                       third party may not wish to be posted,                available, you can provide this
                                               meetings. Please visit our website at                   such as medical information, your or                  information on the cover sheet and not
                                               https://www.fda.gov/                                    anyone else’s Social Security number, or              in the body of your comments and you
                                               AdvisoryCommittees/                                     confidential business information, such               must identify this information as
                                               AboutAdvisoryCommittees/                                as a manufacturing process. Please note               ‘‘confidential.’’ Any information marked
                                               ucm111462.htm for procedures on                         that if you include your name, contact                as ‘‘confidential’’ will not be disclosed
                                               public conduct during advisory                          information, or other information that                except in accordance with 21 CFR 10.20
                                               committee meetings.                                     identifies you in the body of your                    and other applicable disclosure law. For
                                                  Notice of this meeting is given under                comments, that information will be                    more information about FDA’s posting
                                               the Federal Advisory Committee Act (5                   posted on https://www.regulations.gov.                of comments to public dockets, see 80
                                               U.S.C. app. 2).                                           • If you want to submit a comment                   FR 56469, September 18, 2015, or access
                                                 Dated: February 9, 2018.                              with confidential information that you                the information at: https://www.gpo.gov/
                                               Leslie Kux,                                             do not wish to be made available to the               fdsys/pkg/FR-2015-09-18/pdf/2015-
                                                                                                       public, submit the comment as a                       23389.pdf.
                                               Associate Commissioner for Policy.
                                                                                                       written/paper submission and in the                      Docket: For access to the docket to
                                               [FR Doc. 2018–03237 Filed 2–15–18; 8:45 am]                                                                   read background documents or the
                                                                                                       manner detailed (see ‘‘Written/Paper
                                               BILLING CODE 4164–01–P
                                                                                                       Submissions’’ and ‘‘Instructions’’).                  electronic and written/paper comments
                                                                                                                                                             received, go to https://
                                                                                                       Written/Paper Submissions                             www.regulations.gov and insert the
                                               DEPARTMENT OF HEALTH AND                                                                                      docket number, found in brackets in the
                                                                                                         Submit written/paper submissions as
                                               HUMAN SERVICES                                                                                                heading of this document, into the
                                                                                                       follows:
                                               Food and Drug Administration                              • Mail/Hand delivery/Courier (for                   ‘‘Search’’ box and follow the prompts
                                                                                                       written/paper submissions): Dockets                   and/or go to the Dockets Management
                                               [Docket No. FDA–2015–D–1884]                            Management Staff (HFA–305), Food and                  Staff, 5630 Fishers Lane, Rm. 1061,
                                                                                                       Drug Administration, 5630 Fishers                     Rockville, MD 20852.
                                               Duchenne Muscular Dystrophy and                         Lane, Rm. 1061, Rockville, MD 20852.                     You may submit comments on any
                                               Related Dystrophinopathies:                               • For written/paper comments                        guidance at any time (see 21 CFR
                                               Developing Drugs for Treatment;                         submitted to the Dockets Management                   10.115(g)(5)).
daltland on DSKBBV9HB2PROD with NOTICES




                                               Guidance for Industry; Availability                     Staff, FDA will post your comment, as                    Submit written requests for single
                                               AGENCY:    Food and Drug Administration,                well as any attachments, except for                   copies of this guidance to the Division
                                               HHS.                                                    information submitted, marked and                     of Drug Information, Center for Drug
                                               ACTION:   Notice of availability.                       identified, as confidential, if submitted             Evaluation and Research, Food and
                                                                                                       as detailed in ‘‘Instructions.’’                      Drug Administration, 10001 New
                                               SUMMARY: The Food and Drug                                Instructions: All submissions received              Hampshire Ave., Hillandale Building,
                                               Administration (FDA or Agency) is                       must include the Docket No. FDA–                      4th Floor, Silver Spring, MD 20993–


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                                                                             Federal Register / Vol. 83, No. 33 / Friday, February 16, 2018 / Notices                                           7051

                                               0002. Send one self-addressed adhesive                  III. Electronic Access                                Electronic Submissions
                                               label to assist that office in processing                                                                       Submit electronic comments in the
                                               your requests. See the SUPPLEMENTARY                      Persons with access to the internet
                                                                                                       may obtain the guidance at either                     following way:
                                               INFORMATION section for electronic                                                                              • Federal eRulemaking Portal:
                                               access to the guidance document.                        https://www.fda.gov/Drugs/Guidance
                                                                                                       ComplianceRegulatoryInformation/                      https://www.regulations.gov. Follow the
                                               FOR FURTHER INFORMATION CONTACT:                        Guidances/default.htm or https://                     instructions for submitting comments.
                                               Colleen Locicero, Center for Drug                       www.regulations.gov.                                  Comments submitted electronically,
                                               Evaluation and Research, Food and                                                                             including attachments, to https://
                                                                                                         Dated: February 12, 2018.
                                               Drug Administration, 10903 New                                                                                www.regulations.gov will be posted to
                                               Hampshire Ave., Bldg. 22, Rm. 4242,                     Leslie Kux,                                           the docket unchanged. Because your
                                               Silver Spring, MD 20993–0002, 301–                      Associate Commissioner for Policy.                    comment will be made public, you are
                                               796–1114.                                               [FR Doc. 2018–03225 Filed 2–15–18; 8:45 am]           solely responsible for ensuring that your
                                               SUPPLEMENTARY INFORMATION:
                                                                                                       BILLING CODE 4164–01–P                                comment does not include any
                                                                                                                                                             confidential information that you or a
                                               I. Background                                                                                                 third party may not wish to be posted,
                                                                                                       DEPARTMENT OF HEALTH AND                              such as medical information, your or
                                                  FDA is announcing the availability of                HUMAN SERVICES                                        anyone else’s Social Security number, or
                                               a guidance for industry entitled
                                                                                                                                                             confidential business information, such
                                               ‘‘Duchenne Muscular Dystrophy and                       Food and Drug Administration                          as a manufacturing process. Please note
                                               Related Dystrophinopathies: Developing
                                                                                                                                                             that if you include your name, contact
                                               Drugs for Treatment.’’ DMD and other                    [Docket No. FDA–2017–D–0040]                          information, or other information that
                                               dystrophinopathies result from genetic
                                                                                                                                                             identifies you in the body of your
                                               mutations in the dystrophin gene that                   How To Prepare a Pre-Request for                      comments, that information will be
                                               decrease levels of dystrophin and/or                    Designation; Guidance for Industry;                   posted on https://www.regulations.gov.
                                               cause dysfunction of the dystrophin                     Availability                                            • If you want to submit a comment
                                               protein, leading to muscle degeneration,                                                                      with confidential information that you
                                               including cardiac and respiratory                       AGENCY:    Food and Drug Administration,
                                                                                                                                                             do not wish to be made available to the
                                               muscles, and greatly decreased life                     HHS.
                                                                                                                                                             public, submit the comment as a
                                               expectancy. There remains a high-level                  ACTION:   Notice of availability.                     written/paper submission and in the
                                               unmet medical need for effective drug                                                                         manner detailed (see ‘‘Written/Paper
                                               treatments for DMD and other                            SUMMARY:   The Food and Drug                          Submissions’’ and ‘‘Instructions’’).
                                               dystrophinopathies. This guidance                       Administration (FDA) is announcing the
                                               addresses FDA’s current thinking                        availability of a guidance for industry               Written/Paper Submissions
                                               regarding the clinical development                      entitled ‘‘How to Prepare a Pre-Request                  Submit written/paper submissions as
                                               program and clinical trial designs for                  for Designation (Pre-RFD).’’ The purpose              follows:
                                               drugs to support an indication for the                  of this guidance is to explain the Pre-                  • Mail/Hand delivery/Courier (for
                                               treatment of dystrophinopathies. This                   RFD process at the FDA Office of                      written/paper submissions): Dockets
                                               guidance finalizes the draft guidance of                Combination Products (OCP), describe                  Management Staff (HFA–305), Food and
                                               the same name issued June 10, 2015 (80                  and help a sponsor understand the type                Drug Administration, 5630 Fishers
                                               FR 32961). It reflects FDA’s                            of information that the sponsor should                Lane, Rm. 1061, Rockville, MD 20852.
                                               consideration of public comments on                     include in a Pre-RFD, and assist                         • For written/paper comments
                                               the draft guidance and makes minor                      sponsors in obtaining a preliminary                   submitted to the Dockets Management
                                               clarifying changes.                                     assessment from FDA through the Pre-                  Staff, FDA will post your comment, as
                                                  This guidance is being issued                        RFD process. The Pre-RFD process is                   well as any attachments, except for
                                               consistent with FDA’s good guidance                     available to provide informal, non-                   information submitted, marked and
                                               practices regulation (21 CFR 10.115).                   binding feedback regarding the                        identified, as confidential, if submitted
                                               The guidance represents the current                     regulatory identity or classification of a            as detailed in ‘‘Instructions.’’
                                               thinking of FDA on developing drugs for                 human medical product as a drug,                         Instructions: All submissions received
                                               the treatment of DMD. It does not                       device, biological product, or                        must include the Docket No. FDA–
                                               establish any rights for any person and                 combination product. In addition, this                2017–D–0040 for ‘‘How to Prepare a Pre-
                                               is not binding on FDA or the public.                    informal process provides information                 Request for Designation (Pre-RFD);
                                               You can use an alternative approach if                  about a non-combination or                            Guidance for Industry.’’ Received
                                               it satisfies the requirements of the                    combination product’s assignment to                   comments will be placed in the docket
                                               applicable statutes and regulations. This               the appropriate Agency Center (Center                 and, except for those submitted as
                                               guidance is not subject to Executive                    for Drug Evaluation and Research                      ‘‘Confidential Submissions,’’ publicly
                                               Order 12866.                                            (CDER), Center for Devices and                        viewable at https://www.regulations.gov
                                               II. Paperwork Reduction Act of 1995                     Radiological Health (CDRH), or Center                 or at the Dockets Management Staff
                                                                                                       for Biologics Evaluation and Research                 between 9 a.m. and 4 p.m., Monday
                                                 This guidance refers to previously                    (CBER)) for premarket review and                      through Friday.
                                               approved collections of information that                regulation.                                              • Confidential Submissions—To
                                               are subject to review by the Office of                                                                        submit a comment with confidential
daltland on DSKBBV9HB2PROD with NOTICES




                                               Management and Budget (OMB) under                       DATES: The announcement of the                        information that you do not wish to be
                                               the Paperwork Reduction Act of 1995                     guidance is published in the Federal                  made publicly available, submit your
                                               (44 U.S.C. 3501–3520). The collections                  Register on February 16, 2018.                        comments only as a written/paper
                                               of information in 21 CFR parts 312 and                  ADDRESSES:   You may submit either                    submission. You should submit two
                                               314 have been approved under OMB                        electronic or written comments on                     copies total. One copy will include the
                                               control numbers 0910–0014 and 0910–                     Agency guidances at any time as                       information you claim to be confidential
                                               0001, respectively.                                     follows:                                              with a heading or cover note that states


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Document Created: 2018-02-16 00:55:33
Document Modified: 2018-02-16 00:55:33
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 of availability.
DatesThe announcement of the guidance is published in the Federal Register on February 16, 2018.
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 Citation83 FR 7050 

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