Notice2023-19284
Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments-Sickle Cell Disease
Primary source
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Published
September 7, 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 Committee). The general function of the Committee is to provide advice and recommendations to FDA on regulatory issues. On October 31, 2023, the Committee will discuss and make recommendations on biologics license application (BLA) 125787 from Vertex Pharmaceuticals, Inc. for exagamglogene autotemcel (exa-cel). The applicant has requested an indication for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso- occlusive crises. 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 172 (Thursday, September 7, 2023)</title>
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[Federal Register Volume 88, Number 172 (Thursday, September 7, 2023)]
[Notices]
[Pages 61598-61600]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-19284]
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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--
Sickle Cell Disease
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 Committee). The general function of
the Committee is to provide advice and recommendations to FDA on
regulatory issues. On October 31, 2023, the Committee will discuss and
make recommendations on biologics license application (BLA) 125787 from
Vertex Pharmaceuticals, Inc. for exagamglogene autotemcel (exa-cel).
The applicant has requested an indication for the treatment of sickle
cell disease in patients 12 years and older with recurrent vaso-
occlusive crises. 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 on October 31, 2023, from 9 a.m. to 5
p.m. Eastern Time.
ADDRESSES: All meeting participants will be heard, viewed, captioned,
and recorded for this advisory committee meeting via an online
teleconferencing and/or video conferencing platform. Answers to
commonly asked questions about FDA advisory committee meetings 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 at <a href="https://youtube.com/live/M90IjjxOdQg">https://youtube.com/live/M90IjjxOdQg</a>.
FDA is establishing a docket for public comment on this meeting.
The docket number is FDA-2023-N-1190. The docket will close on October
30, 2023. Please note that late, untimely filed comments will not be
considered. 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
October 30, 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 October 24, 2023, will be provided
to the Committee. Comments received after that date and on October 30,
2023, will be taken into consideration by FDA. In the event that the
meeting is cancelled, 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
[[Page 61599]]
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--Sickle Cell Disease, Meeting Date: October 31, 2023.''
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 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 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: Cicely Reese or Marie DeGregorio,
Center for Biologics Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1246, Silver
Spring, MD 20993-0002, 301-796-9025, email: <a href="/cdn-cgi/l/email-protection#b0f3f2f5e2f3e4f7e4f1f3f0d6d4d19ed8d8c39ed7dfc6"><span class="__cf_email__" data-cfemail="b8fbfafdeafbecffecf9fbf8dedcd996d0d0cb96dfd7ce">[email protected]</span></a>, 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 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 and/or video
conferencing platform. On October 31, 2023, the Committee will discuss
and make recommendations on BLA 125787 from Vertex Pharmaceuticals,
Inc. for exagamglogene autotemcel (exa-cel). The applicant has
requested an indication for the treatment of sickle cell disease in
patients 12 years and older with recurrent vaso-occlusive crises.
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 and/or video conference meeting 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 and video
components to allow the presentation of materials in a manner that most
closely resembles an in-person advisory committee meeting.
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 October 24, 2023, will be provided to the
Committee. Oral presentations from the public will be scheduled between
approximately 12:35 p.m. and 1:35 p.m. Eastern Time on October 31,
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, along
with the names, email addresses, and direct contact phone numbers of
proposed participants, and an indication of the approximate time
requested to make their presentation on or before 12 p.m. Eastern Time
on October 16, 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 October 18, 2023.
For press inquiries, please contact the Office of Media Affairs at
<a href="/cdn-cgi/l/email-protection#85c3c1c4cac8c4c5e3e1e4abededf6abe2eaf3"><span class="__cf_email__" data-cfemail="c58381848a888485a3a1a4ebadadb6eba2aab3">[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 Cicely Reece or Marie DeGregorio at <a href="/cdn-cgi/l/email-protection#591a1b1c0b1a0d1e0d181a193f3d387731312a773e362f"><span class="__cf_email__" data-cfemail="aae9e8eff8e9feedfeebe9eacccecb84c2c2d984cdc5dc">[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. 1001 et seq.). This meeting notice also serves
as notice that, pursuant to 21 CFR 10.19, the requirements in 21 CFR
14.22(b), (f), and (g) relating to the location of advisory committee
meetings are hereby waived to allow for this
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meeting to take place using an online meeting platform. This waiver is
in the interest of allowing greater transparency and opportunities for
public participation, in addition to convenience for advisory committee
members, speakers, and guest speakers. No participant will be
prejudiced by this waiver, and that the ends of justice will be served
by allowing for this modification to FDA's advisory committee meeting
procedures.
Dated: September 1, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-19284 Filed 9-6-23; 8:45 am]
BILLING CODE 4164-01-P
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