Notice2023-07518
Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments
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Published
April 11, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA) announces a forthcoming public advisory committee meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee. The general function of the committee is to provide advice and recommendations to FDA on regulatory issues. The committee will discuss the Biologics License Application (BLA) 125781 from Sarepta Therapeutics, Inc. for delandistrogene moxeparvovec with the requested indication for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. The meeting will be open to the public. FDA is establishing a docket for public comment on this document.
Full Text
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<title>Federal Register, Volume 88 Issue 69 (Tuesday, April 11, 2023)</title>
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[Federal Register Volume 88, Number 69 (Tuesday, April 11, 2023)]
[Notices]
[Pages 21685-21687]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-07518]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2023-N-1190]
Cellular, Tissue, and Gene Therapies Advisory Committee; Notice
of Meeting; Establishment of a Public Docket; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice; establishment of a public docket; request for comments.
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SUMMARY: The Food and Drug Administration (FDA) announces a forthcoming
public advisory committee meeting of the Cellular, Tissue, and Gene
Therapies Advisory Committee. The general function of the committee is
to provide advice and recommendations to FDA on regulatory issues. The
committee will discuss the Biologics License Application (BLA) 125781
from Sarepta Therapeutics, Inc. for delandistrogene moxeparvovec with
the requested indication for the treatment of ambulatory patients with
Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD
gene. The meeting will be open to the public. FDA is establishing a
docket for public comment on this document.
DATES: The meeting will be held virtually on May 12, 2023, from 9 a.m.
to 6 p.m. Eastern Time.
ADDRESSES: Please note that due to the impact of COVID-19, all meeting
participants will be joining this advisory committee meeting via an
online teleconferencing platform. Answers to
[[Page 21686]]
commonly asked questions about FDA advisory committee meetings,
including information regarding special accommodations due to a
disability, may be accessed at: <a href="https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm408555.htm">https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm408555.htm</a>. The online web conference
meeting will be available at the following link on the day of the
meeting: <a href="https://youtube.com/live/k33d4h-CpGU">https://youtube.com/live/k33d4h-CpGU</a>.
FDA is establishing a docket for public comment on this meeting.
The docket number is FDA-2023-N-1190. Please note that late, untimely
filed comments will not be considered. The docket will close on May 11,
2023. The <a href="https://www.regulations.gov">https://www.regulations.gov</a> electronic filing system will
accept comments until 11:59 p.m. Eastern Time at the end of May 11,
2023. Comments received by mail/hand delivery/courier (for written/
paper submissions) will be considered timely if they are received on or
before that date.
Comments received on or before May 5, 2023, will be provided to the
committee. Comments received after that date will be taken into
consideration by FDA. In the event that the meeting is canceled, FDA
will continue to evaluate any relevant applications or information, and
consider any comments submitted to the docket, as appropriate.
You may submit comments as follows:
Electronic Submissions
Submit electronic comments in the following way:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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-2023-N-1190 for ``Cellular, Tissue, and Gene Therapies Advisory
Committee; Notice of Meeting; Establishment of a Public Docket; Request
for Comments.'' Received comments, those filed in a timely manner (see
ADDRESSES), will be placed in the docket and, except for those
submitted as ``Confidential Submissions,'' publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets Management Staff between 9 a.m.
and 4 p.m., Monday through Friday, 240-402-7500.
<bullet> 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.'' FDA 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>. Submit both
copies to the Dockets Management Staff. If you do not wish your name
and contact information 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 the 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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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, 240-402-7500.
FOR FURTHER INFORMATION CONTACT: Marie DeGregorio or Christina Vert,
Center for Biologics Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1246, Silver
Spring, MD 20993-0002, <a href="/cdn-cgi/l/email-protection#dab9aebdaebbb99abcbebbf4b2b2a9f4bdb5ac"><span class="__cf_email__" data-cfemail="accfd8cbd8cdcfeccac8cd82c4c4df82cbc3da">[email protected]</span></a>, 240-701-9119, or FDA
Advisory Committee Information Line, 1-800-741-8138 (301-443-0572 in
the Washington, DC area). A notice in the Federal Register about last-
minute modifications that impact a previously announced advisory
committee meeting cannot always be published quickly enough to provide
timely notice. Therefore, you should always check the FDA's website at
<a href="https://www.fda.gov/AdvisoryCommittees/default.htm">https://www.fda.gov/AdvisoryCommittees/default.htm</a> and scroll down to
the appropriate advisory committee meeting link, or call the advisory
committee information line to learn about possible modifications before
the meeting.
SUPPLEMENTARY INFORMATION:
Agenda: The meeting presentations will be heard, viewed, captioned,
and recorded through an online teleconferencing platform. On May 12,
2023, the committee will discuss BLA 125781 from Sarepta Therapeutics,
Inc. for delandistrogene moxeparvovec. The applicant has requested an
indication for the treatment of ambulatory patients with DMD with a
confirmed mutation in the DMD gene.
FDA intends to make background material available to the public no
later than 2 business days before the meeting. If FDA is unable to post
the background material on its website prior to the meeting, the
background material will be made publicly available on FDA's website at
the time of the advisory committee meeting. Background material and the
link to the online teleconference meeting room will be available at
<a href="https://www.fda.gov/AdvisoryCommittees/Calendar/default.htm">https://www.fda.gov/AdvisoryCommittees/Calendar/default.htm</a>. Scroll
down to the appropriate advisory committee meeting link. The meeting
will include slide presentations with audio components to allow the
presentation of materials in a manner that most closely resembles an
in-person advisory committee meeting.
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Procedure: Interested persons may present data, information, or
views, orally or in writing, on issues pending before the committee.
All electronic and written submissions submitted to the Docket (see
ADDRESSES) on or before May 5, 2023, will be provided to the committee.
Comments received after May 5, 2023, and by May 11, 2023, will be taken
into consideration by FDA. Oral presentations from the public will be
scheduled between approximately 12:30 p.m. and 1:30 p.m. Eastern Time
on May 12, 2023. Those individuals interested in making formal oral
presentations should notify the contact person and submit a brief
statement of the general nature of the evidence or arguments they wish
to present; the names, phone numbers, and email addresses of proposed
participants; and an indication of the approximate time requested to
make their presentation on or before 12 p.m. Eastern Time on April 26,
2023. Time allotted for each presentation may be limited. If the number
of registrants requesting to speak is greater than can be reasonably
accommodated during the scheduled open public hearing session, FDA may
conduct a lottery to determine the speakers for the scheduled open
public hearing session. The contact person will notify interested
persons regarding their request to speak by 6 p.m. Eastern Time on
April 28, 2023.
For press inquiries, please contact the Office of Media Affairs at
<a href="/cdn-cgi/l/email-protection#a0c6c4c1cfcdc1e0c6c4c18ec8c8d38ec7cfd6"><span class="__cf_email__" data-cfemail="66000207090b0726000207480e0e1548010910">[email protected]</span></a> or 301-796-4540.
FDA welcomes the attendance of the public at its advisory committee
meetings and will make every effort to accommodate persons with
disabilities. If you require accommodations due to a disability, please
contact Marie DeGregorio at <a href="/cdn-cgi/l/email-protection#0c6f786b786d6f4c6a686d2264647f226b637a"><span class="__cf_email__" data-cfemail="95f6e1f2e1f4f6d5f3f1f4bbfdfde6bbf2fae3">[email protected]</span></a> (see FOR FURTHER
INFORMATION CONTACT) at least 7 days in advance of the meeting.
FDA is committed to the orderly conduct of its advisory committee
meetings. Please visit our website at <a href="https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm111462.htm">https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm111462.htm</a> for procedures
on public conduct during advisory committee meetings.
Notice of this meeting is given under the Federal Advisory
Committee Act (5 U.S.C. app. 2).
Dated: April 5, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-07518 Filed 4-10-23; 8:45 am]
BILLING CODE 4164-01-P
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