[Federal Register Volume 88, Number 69 (Tuesday, April 11, 2023)]
[Notices]
[Pages 21685-21687]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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

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commonly asked questions about FDA advisory committee meetings, 
including information regarding special accommodations due to a 
disability, may be accessed at: https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm408555.htm. The online web conference 
meeting will be available at the following link on the day of the 
meeting: https://youtube.com/live/k33d4h-CpGU.
    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 https://www.regulations.gov 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:
     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-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 https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. 
and 4 p.m., Monday through Friday, 240-402-7500.
     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 https://www.regulations.gov. 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: https://www.govinfo.gov/content/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, 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, [email protected], 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 
https://www.fda.gov/AdvisoryCommittees/default.htm 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 
https://www.fda.gov/AdvisoryCommittees/Calendar/default.htm. 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 
[email protected] 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 [email protected] (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 https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm111462.htm 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


