Federal Register / Vol. 89, No. 8 / Thursday, January 11, 2024 / Notices 1925 

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 FILSUVEZ (birch 

triterpenes), manufactured by Amryt 

Pharmaceuticals, 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. 

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 FILSUVEZ (birch triterpenes), 

manufactured by Amryt 

Pharmaceuticals, meets the criteria for a 

priority review voucher. FILSUVEZ 

(birch triterpenes) gel is indicated for 

the treatment of wounds associated with 

dystrophic and junctional epidermolysis 

bullosa in adult and pediatric patients 6 

months of age and older. 

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/ 

DevelopingProductsforRare

DiseasesConditions/RarePediatric

DiseasePriorityVoucherProgram/

default.htm. For further information 

about FILSUVEZ (birch triterpenes), go 

to the ‘‘Drugs@FDA’’ website at http:// 

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

daf/. 

Dated: January 8, 2024. 

Lauren K. Roth, 

Associate Commissioner for Policy. 

[FR Doc. 2024–00400 Filed 1–10–24; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2020–N–0026] 

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 ADZYNMA 

(ADAMTS13, recombinant-krhn), 

manufactured by Takeda 

Pharmaceuticals U.S.A., Inc., meets the 

criteria for a priority review voucher. 

FOR FURTHER INFORMATION CONTACT: 

Myrna Hanna, Center for Biologics 

Evaluation and Research, Food and 

Drug Administration, 10903 New 

Hampshire Ave., Bldg. 71, Rm. 7301, 

Silver Spring, MD 20993–0002, 240– 

402–7911. 

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 ADZYNMA (ADAMTS13, 

recombinant-krhn), manufactured by 

Takeda Pharmaceuticals U.S.A., Inc., 

meets the criteria for a priority review 

voucher. 

ADZYNMA (ADAMTS13, 

recombinant-krhn) is indicated for 

prophylactic or on-demand enzyme 

replacement therapy in adult and 

pediatric patients with congenital 

thrombotic thrombocytopenic purpura. 

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/industry/ 

developing-products-rare-diseasesconditions/rare-pediatric-disease-rpddesignation-and-voucher-programs. For 

further information about ADZYNMA 

(ADAMTS13, recombinant-krhn), go to 

the Center for Biologics Evaluation and 

Research’s Approved Blood Products 

website at https://www.fda.gov/ 

vaccines-blood-biologics/adzynma. 

Dated: January 8, 2024. 

Lauren K. Roth, 

Associate Commissioner for Policy. 

[FR Doc. 2024–00401 Filed 1–10–24; 8:45 am] 

BILLING CODE 4164–01–P 

DEPARTMENT OF HEALTH AND 

HUMAN SERVICES 

Food and Drug Administration 

[Docket No. FDA–2022–D–2512] 

Q5A(R2) Viral Safety Evaluation of 

Biotechnology Products Derived From 

Cell Lines of Human or Animal Origin; 

International Council for 

Harmonisation; Guidance for Industry; 

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 a final 

guidance for industry entitled ‘‘Q5A(R2) 

Viral Safety Evaluation of Biotechnology 

Products Derived from Cell Lines of 

Human or Animal Origin.’’ The 

guidance was prepared under the 

auspices of the International Council for 

Harmonisation of Technical 

Requirements for Pharmaceuticals for 

Human Use (ICH). The guidance is 

intended to describe risk-based 

principles and mitigation strategies to 

assure the viral safety of biotechnology 

products, including the data necessary 

to submit in a marketing application. 

The guidance also finalizes the updates 

based on advances in scientific 

knowledge and regulatory expectations 

to the first version of the ICH guidance 

for industry ‘‘Q5A Viral Safety 

Evaluation of Biotechnology Products 

Derived from Cell Lines of Human or 

Animal Origin,’’ issued in September 

1998. Lastly, the guidance replaces the 

draft guidance ‘‘Q5A(R2) Viral Safety 

Evaluation of Biotechnology Products 

Derived from Cell Lines of Human or 

Animal Origin’’ issued on November 14, 

2022. 

DATES: The announcement of the 

guidance is published in the Federal 

Register on January 11, 2024. 

ADDRESSES: You may submit either 

electronic or written comments on 

Agency guidances 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 


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