[Federal Register Volume 86, Number 140 (Monday, July 26, 2021)]
[Notices]
[Pages 40059-40061]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-15783]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-N-0651]


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 public docket; request for comments.

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SUMMARY: The Food and Drug Administration (FDA or Agency) announces a 
forthcoming public advisory committee meeting of the Cellular, Tissue 
and Gene Therapies Advisory Committee (CTGTAC). The general function of 
the committee is to provide advice and recommendations to FDA on 
regulatory issues. Matters considered at the meeting will include 
discussion of the toxicity risks of adeno-associated virus (AAV) 
vector-based gene therapy products. The discussion topics include 
oncogenicity risks due to vector genome integration and safety issues 
identified during preclinical and/or clinical evaluation. The meeting 
will be open to the public on both days. FDA is establishing a docket 
for public comment on this document.

DATES: The meeting will be held on September 2 and 3, 2021, from 10 
a.m. to 6 p.m. Eastern Time.

ADDRESSES: Please note that due to the impact of the COVID-19 pandemic, 
all meeting participants will be joining this advisory committee 
meeting via an online teleconferencing platform. Answers to commonly 
asked questions about FDA advisory committee meetings may be accessed 
at: https://www.fda.gov/advisory-committees/about-advisory-committees/common-questions-and-answers-about-fda-advisory-committee-meetings. The 
online web conference meeting will be available at the following links 
on the day of the meeting: Day 1 https://youtu.be/58KjL9_p9Tw and Day 2 
https://youtu.be/yLggQF0XUUY.
    FDA is establishing a docket for public comment on this meeting. 
The docket number is FDA-2021-N-0651. The docket will close on 
September 1, 2021. Submit either electronic or written comments on this 
public meeting on or before September 1, 2021. Please note that late, 
untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 
11:59 p.m. Eastern Time at the end of September 1, 2021. Comments 
received by mail/hand delivery/courier (for written/paper submissions) 
will be considered timely if they are postmarked or the delivery 
service acceptance receipt is on or before that date.
    Comments received on or before August 26, 2021, 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 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:
     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

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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-2021-N-0651 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: Jarrod Collier or Joanne Lipkind, 
Center for Biologics Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 6268, Silver 
Spring, MD 20993-0002, ctgtac@fda.hhs.gov, 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, 
before coming to the meeting, you should always check the Agency'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.

SUPPLEMENTARY INFORMATION:
    Agenda: The meeting presentations will be heard, viewed, captioned, 
and recorded through an online teleconferencing platform. The CTGTAC 
committee will meet in open session on both days to discuss the 
toxicity risks of AAV vector-based gene therapy products. The 
discussion topics include oncogenicity risks due to vector genome 
integration and safety issues identified during preclinical and/or 
clinical evaluation. On September 2, 2021, in the morning, under 
session 1, the CTGTAC committee will meet to discuss and make 
recommendations on vector integration and oncogenicity risks. In the 
afternoon under session 2, the committee will discuss and make 
recommendations on hepatotoxicity issues. On September 3, 2021, in the 
morning under session 3, the committee will meet to discuss and make 
recommendations on thrombotic microangiopathy issues. In the afternoon 
under session 4, the committee will discuss and make recommendations on 
non-clinical findings of neurotoxicity, especially related to the 
dorsal root ganglion toxicity issues. Also, in the afternoon under 
session 5, the committee will discuss and make recommendations on 
clinical findings of neurotoxicity, based on brain magnetic resonance 
imaging studies.
    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.
    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 August 26, 2021, will be provided to the 
committee. Oral presentations from the public will be scheduled twice 
each day between approximately 12:45 p.m. and 1:15 p.m. and 4:05 p.m. 
and 4:35 p.m. on September 2, and between 11 a.m. and 11:30 a.m. and 
1:50 p.m. and 2:20 p.m. on September 3. 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 and addresses of proposed 
participants, and an indication of the approximate time requested to 
make their presentation on or before August 18, 2021. 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 August 19, 2021.
    For press inquiries, please contact the Office of Media Affairs at 
fdaoma@fda.hhs.gov or 301-796-4540.

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    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 Jarrod Collier at ctgtac@fda.hhs.gov (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/advisory-committees/about-advisory-committees/public-conduct-during-fda-advisory-committee-meetings 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: July 16, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-15783 Filed 7-23-21; 8:45 am]
BILLING CODE 4164-01-P


