61598 Federal Register / Vol. 88, No. 172 / Thursday, September 7, 2023 / Notices 

Estimated Total Annual Burden 

Hours: 1,050. 

Authority: Sec. 5106, Public Law 111– 

320, the Child Abuse Prevention and 

Treatment Act Reauthorization Act of 

2010, and titles IV–B and IV–E of the 

Social Security Act. 

Mary B. Jones, 

ACF/OPRE Certifying Officer. 

[FR Doc. 2023–19228 Filed 9–6–23; 8:45 am] 

BILLING CODE 4184–29–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2023–N–2607] 

Issuance of Priority Review Voucher; 

Rare Pediatric Disease Product 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice. 

SUMMARY: The Food and Drug 

Administration (FDA) is announcing the 

issuance of a priority review voucher to 

the sponsor of a rare pediatric disease 

product application. The Federal Food, 

Drug, and Cosmetic Act (FD&C Act) 

authorizes FDA to award priority review 

vouchers to sponsors of approved rare 

pediatric disease product applications 

that meet certain criteria. FDA is 

required to publish notice of the award 

of the priority review voucher. FDA has 

determined that VEOPOZ (pozelimabbbfg), manufactured by Regeneron 

Pharmaceuticals, Inc., meets the criteria 

for a priority review voucher. 

FOR FURTHER INFORMATION CONTACT: 

Cathryn Lee, Center for Drug Evaluation 

and Research, Food and Drug 

Administration, 10903 New Hampshire 

Ave., Silver Spring, MD 20993–0002, 

301–796–1394, email: Cathryn.Lee@

fda.hhs.gov. 

SUPPLEMENTARY INFORMATION: FDA is 

announcing the issuance of a priority 

review voucher to the sponsor of an 

approved rare pediatric disease product 

application. Under section 529 of the 

FD&C Act (21 U.S.C. 360ff), FDA will 

award priority review vouchers to 

sponsors of approved rare pediatric 

disease product applications that meet 

certain criteria. FDA has determined 

that VEOPOZ (pozelimab-bbfg), 

approved on August 18, 2023, and 

manufactured by Regeneron 

Pharmaceuticals, Inc., meets the criteria 

for a priority review voucher. VEOPOZ 

(pozelimab-bbfg) injection is indicated 

for the treatment of adult and pediatric 

patients 1 year of age and older with 

CD55-deficient protein-losing 

enteropathy (PLE), also known as 

CHAPLE disease. 

For further information about the Rare 

Pediatric Disease Priority Review 

Voucher Program and for a link to the 

full text of section 529 of the FD&C Act, 

go to https://www.fda.gov/ForIndustry/ 

DevelopingProducts

forRareDiseasesConditions/ 

RarePediatricDiseasePriority

VoucherProgram/default.htm. For 

further information about VEOPOZ 

(pozelimab-bbfg), go to the ‘‘Drugs@

FDA’’ website at https://

www.accessdata.fda.gov/scripts/cder/ 

daf/. 

Dated: September 1, 2023. 

Lauren K. Roth, 

Associate Commissioner for Policy. 

[FR Doc. 2023–19287 Filed 9–6–23; 8:45 am] 

BILLING CODE 4164–01–P 

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. 

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: 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 at https://

youtube.com/live/M90IjjxOdQg. 

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 https://www.regulations.gov 

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: 

• 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 


ddrumheller on DSK120RN23PROD with NOTICES1
Federal Register / Vol. 88, No. 172 / Thursday, September 7, 2023 / Notices 61599 

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— 

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 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 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: 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: 

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: 

CBERCTGTAC@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, you should always check 

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 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 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 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 FDAOMA@

fda.hhs.gov 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 CBERCTGTAC@

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/ 

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. 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 


ddrumheller on DSK120RN23PROD with NOTICES1
61600 Federal Register / Vol. 88, No. 172 / Thursday, September 7, 2023 / Notices 

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 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2023–N–2607] 

Issuance of Priority Review Voucher; 

Rare Pediatric Disease Product 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice. 

SUMMARY: The Food and Drug 

Administration (FDA) is announcing the 

issuance of a priority review voucher to 

the sponsor of a rare pediatric disease 

product application. The Federal Food, 

Drug, and Cosmetic Act (FD&C Act) 

authorizes FDA to award priority review 

vouchers to sponsors of approved rare 

pediatric disease product applications 

that meet certain criteria. FDA is 

required to publish notice of the award 

of the priority review voucher. FDA has 

determined that SOHONOS 

(palovarotene), manufactured by Ipsen 

Biopharmaceuticals, Inc., meets the 

criteria for a priority review voucher. 

FOR FURTHER INFORMATION CONTACT: 

Cathryn Lee, Center for Drug Evaluation 

and Research, Food and Drug 

Administration, 10903 New Hampshire 

Ave., Silver Spring, MD 20993–0002, 

301–796–1394, email: Cathryn.Lee@

fda.hhs.gov. 

SUPPLEMENTARY INFORMATION: FDA is 

announcing the issuance of a priority 

review voucher to the sponsor of an 

approved rare pediatric disease product 

application. Under section 529 of the 

FD&C Act (21 U.S.C. 360ff), FDA will 

award priority review vouchers to 

sponsors of approved rare pediatric 

disease product applications that meet 

certain criteria. FDA has determined 

that SOHONOS (palovarotene), 

approved on August 16, 2023, and 

manufactured by Ipsen 

Biopharmaceuticals, Inc., meets the 

criteria for a priority review voucher. 

SOHONOS (palovarotene) capsules are 

indicated for reduction in volume of 

new heterotopic ossification in adults 

and pediatric patients (aged 8 years and 

older for females and 10 years and older 

for males) with fibrodysplasia ossificans 

progressiva. 

For further information about the Rare 

Pediatric Disease Priority Review 

Voucher Program and for a link to the 

full text of section 529 of the FD&C Act, 

go to http://www.fda.gov/ForIndustry/ 

Developing

ProductsforRareDiseasesConditions/ 

RarePediatricDisease

PriorityVoucherProgram/default.htm. 

For further information about 

SOHONOS (palovarotene), go to the 

‘‘Drugs@FDA’’ website at http://

www.accessdata.fda.gov/scripts/cder/ 

daf/. 

Dated: September 1, 2023. 

Lauren K. Roth, 

Associate Commissioner for Policy. 

[FR Doc. 2023–19289 Filed 9–6–23; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket Nos. FDA–2023–D–3132, FDA– 

2023–D–3133, and FDA–2023–D–3134] 

Modernizing the Food and Drug 

Administration’s Premarket 

Notification Program; Draft Guidances 

for Industry and Food and Drug 

Administration Staff; Availability 

AGENCY: Food and Drug Administration, 

HHS. 

ACTION: Notice of availability. 

SUMMARY: The Food and Drug 

Administration (FDA or Agency) is 

announcing the availability of three 

draft guidances entitled ‘‘Evidentiary 

Expectations for 510(k) Implant 

Devices,’’ ‘‘Recommendations for the 

Use of Clinical Data in Premarket 

Notification [510(k)] Submissions,’’ and 

‘‘Best Practices for Selecting a Predicate 

Device to Support a Premarket 

Notification [510(k)] Submission.’’ FDA 

is issuing these guidances to improve 

the predictability, consistency, and 

transparency of the 510(k) premarket 

review process. The draft guidances are 

not final nor are they for 

implementation at this time. 

DATES: Submit either electronic or 

written comments on the draft guidance 

by December 6, 2023 to ensure that the 

Agency considers your comment on this 

draft guidance before it begins work on 

the final version of the guidance. 

ADDRESSES: You may submit comments 

on any guidance at any time 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 Docket No. FDA–2023–D– 

3132 for ‘‘Evidentiary Expectations for 

510(k) Implant Devices,’’ Docket No. 

FDA–2023–D–3133 for 

‘‘Recommendations for the Use of 

Clinical Data in Premarket Notification 

[510(k)] Submissions,’’ or Docket No. 

FDA–2023–D–3134 for ‘‘Best Practices 

for Selecting a Predicate Device to 

Support a Premarket Notification 

[510(k)] Submission.’’ Received 

comments 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 


ddrumheller on DSK120RN23PROD with NOTICES1