[Federal Register Volume 87, Number 72 (Thursday, April 14, 2022)]
[Notices]
[Pages 22215-22217]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2022-08022]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2022-N-0470]


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 scientific issues. 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 June 9, 2022, from 10 a.m. to 6 p.m. 
and June 10, 2022, from 10 a.m. to 4 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/AdvisoryCommittees/AboutAdvisoryCommittees/ucm408555.htm.
    The online web conference meeting will be available at the 
following link on the day of the meeting: Day 1 June 9 link: https://youtu.be/RvtTK3KNl5g and Day 2 June 10 link: https://youtu.be/Eo2BXnGienc.
    FDA is establishing a docket for public comment on this meeting. 
The docket number is FDA-2022-N-0470. The docket will close on June 8, 
2022. Submit either electronic or written comments on this public 
meeting on or before June 8, 2022. Please note that late, untimely 
filed comments will not be considered. Electronic comments must be 
submitted on or before June 8, 2022. The https://www.regulations.gov 
electronic filing system will accept comments until 11:59 p.m. Eastern 
Time at the end of June 8, 2022. 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 June 2, 2022, 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:
    <bullet> 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.
    <bullet> If you want to submit a comment with confidential 
information that you do not wish to be made available to the

[[Page 22216]]

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-2022-N-0470 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.
    <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 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: Christina Vert or Tonica Burke, Center 
for Biologics Evaluation and Research, Food and Drug Administration, 
10903 New Hampshire Ave., Bldg. 71, Rm. 1244, Silver Spring, MD 20993-
0002, <a href="/cdn-cgi/l/email-protection#ed8e998a998c8ead8b898cc385859ec38a829b"><span class="__cf_email__" data-cfemail="11726576657072517775703f7979623f767e67">[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 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 coming to 
the meeting.

SUPPLEMENTARY INFORMATION: Agenda: The meeting presentations will be 
heard, viewed, captioned, and recorded through an online 
teleconferencing platform. The committee will discuss two biologics 
license applications (BLAs) from bluebird bio, Inc.: (1) BLA 125755 for 
elivaldogene autotemcel (autologous CD34+ stem cells genetically 
modified with a lentiviral vector to contain an adenosine triphosphate-
binding cassette, sub-family D, member 1 gene that encodes a functional 
adrenoleukodystrophy protein); the applicant has requested an 
indication for the treatment of patients younger than 18 years of age 
with early cerebral adrenoleukodystrophy who do not have an available 
and willing human leukocyte antigen-matched sibling hematopoietic stem 
cell donor and (2) BLA 125717 for betibeglogene autotemcel (autologous 
CD34+ stem cells genetically modified with a lentiviral vector to 
contain a gene encoding functional beta-globin); the applicant has 
requested an indication for the treatment of patients with [beta]-
thalassemia who require regular red blood cell transfusions.
    The morning session of June 9, 2022, will include presentations of 
the effectiveness and product-specific safety results from the clinical 
trials in BLA 125755. The afternoon session will include presentations 
of safety concerns relevant to both products, followed by committee 
discussion of BLA 125755. On June 10, 2022, the morning session will 
include presentations of the effectiveness and product-specific safety 
results from the clinical trials in BLA 125717. The afternoon session 
will include committee discussion of BLA 125717.
    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 June 2, 2022, will be provided to the 
committee. Oral presentations from the public will be scheduled between 
approximately 1 p.m. and 2 p.m. Eastern Time on June 9, 2022, and 
between approximately 1 p.m. and 2 p.m. Eastern Time on June 10, 2022. 
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 and 
addresses of proposed participants, and an indication of the 
approximate time requested to make their presentation on or before May 
25, 2022. 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 May 26, 2022.
    For press inquiries, please contact the Office of Media Affairs at 
<a href="/cdn-cgi/l/email-protection#422426232d2f23022426236c2a2a316c252d34"><span class="__cf_email__" data-cfemail="a6c0c2c7c9cbc7e6c0c2c788ceced588c1c9d0">[email protected]</span></a> or 301-796-4540.

[[Page 22217]]

    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 Christina Vert at <a href="/cdn-cgi/l/email-protection#eb889f8c9f8a88ab8d8f8ac5838398c58c849d"><span class="__cf_email__" data-cfemail="20435447544143604644410e4848530e474f56">[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 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 11, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-08022 Filed 4-13-22; 8:45 am]
BILLING CODE 4164-01-P


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