Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FORZINITY (Elamipretide)

---
identifier: "/us/fr/2025-23409"
source: "fr"
legal_status: "authoritative_unofficial"
title: "Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FORZINITY (Elamipretide)"
title_number: 0
title_name: "Federal Register"
section_number: "2025-23409"
section_name: "Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FORZINITY (Elamipretide)"
positive_law: false
currency: "2025-12-19"
last_updated: "2025-12-19"
format_version: "1.1.0"
generator: "[email protected]"
agency: "Health and Human Services Department"
document_number: "2025-23409"
document_type: "notice"
publication_date: "2025-12-19"
agencies:
  - "Health and Human Services Department"
  - "Food and Drug Administration"
fr_citation: "90 FR 59530"
fr_volume: 90
docket_ids:
  - "Docket No. FDA-2025-N-4683"
fr_action: "Notice."
---


#  Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FORZINITY (Elamipretide)

**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 FORZINITY (elamipretide), approved September 19, 2025, manufactured by Stealth BioTherapeutics Inc., meets the criteria for a priority review voucher.

**FOR FURTHER INFORMATION CONTACT:**

Quyen Tran, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Room 5324, Silver Spring, MD 20993-0002, 301-796-2771, *[email protected].*

**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 FORZINITY (elamipretide) manufactured by Stealth BioTherapeutics Inc., meets the criteria for a priority review voucher. FORZINITY (elamipretide) injection is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.

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/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm.* For further information about FORZINITY (elamipretide), go to the “Drugs@FDA” website at *https://www.accessdata.fda.gov/scripts/cder/daf/.*

Lowell M. Zeta,

Acting Deputy Commissioner for Policy, Legislation, and International Affairs.