FDA Issues Priority Review Voucher for LOARGYS (pegzilarginase-nbln)

Regs.gov: Food and Drug Administration

Published April 7th, 2026

Detected April 7th, 2026

Summary

The FDA announced issuance of a priority review voucher to Immedica Pharma AB for LOARGYS (pegzilarginase-nbln), approved February 23, 2026. The drug treats hyperargininemia in adults and pediatric patients 2 years and older with Arginase 1 Deficiency. The voucher was awarded under section 529 of the FD&C Act for rare pediatric disease products meeting specified criteria.

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What changed

The FDA published notice of a priority review voucher award for LOARGYS (pegzilarginase-nbln), an enzyme replacement therapy for Arginase 1 Deficiency, a rare pediatric disease. The voucher, issued under the Rare Pediatric Disease Priority Review Voucher Program established by section 529 of the FD&C Act, allows the sponsor to receive priority review for a future marketing application. This is an administrative designation that does not alter drug approval requirements but accelerates FDA review timelines.

For Immedica Pharma AB and other rare pediatric disease product sponsors, this notice confirms the mechanics of the voucher program and highlights an incentive pathway for developing treatments for rare pediatric conditions. Sponsors developing qualifying rare pediatric disease products may be eligible for similar vouchers upon approval. The announcement is informational and creates no new compliance obligations.

What to do next

Monitor FDA Rare Pediatric Disease Priority Review Voucher Program updates
Review program eligibility criteria for future submissions
Consult FDA guidance on rare pediatric disease product development

Source document (simplified)

Content

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 LOARGYS (pegzilarginase-nbln), approved February
23, 2026, manufactured by Immedica Pharma AB, 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, Quyen.Tran1@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 LOARGYS (pegzilarginase-nbln), manufactured
by Immedica Pharma AB, meets the criteria for a priority review voucher. LOARGYS (pegzilarginase-nbln) injection is indicated
for the treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency
(ARG1-D), in conjunction with dietary protein restriction.

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 LOARGYS (pegzilarginase-nbln), go to the “Drugs@FDA” website at https://www.accessdata.fda.gov/scripts/cder/daf/.

Grace R. Graham, Deputy Commissioner for Policy, Legislation, and International Affairs. [FR Doc. 2026-06722 Filed 4-6-26; 8:45 am] BILLING CODE 4164-01-P