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Issuance of Priority Review Voucher; Rare Pediatric Disease Product; OTARMENI (lunsotogene parvec-cwha)

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, ...

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Department of Health and Human Services
Food and Drug Administration
  1. [Docket No. FDA-2026-N-4588]

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 OTARMENI (lunsotogene parvec-cwha), approved April 23, 2026, manufactured by Regeneron Pharmaceuticals, 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, , 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 ( printed page 24550) 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 OTARMENI (lunsotogene parvec-cwha), manufactured by Regeneron Pharmaceuticals, Inc., meets the criteria for a priority review voucher. OTARMENI (lunsotogene parvec-cwha) is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.

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-diseases-conditions/​rare-pediatric-disease-rpd-designation-and-voucher-programs. For further information about OTARMENI (lunsotogene parvec-cwha), go to the Center for Biologics Evaluation and Research's Approved Cellular and Gene Therapy Products website at https://www.fda.gov/​vaccines-blood-biologics/​cellular-gene-therapy-products/​approved-cellular-and-gene-therapy-products.

Grace R. Graham,

Deputy Commissioner for Policy, Legislation, and International Affairs.

[FR Doc. 2026-08913 Filed 5-5-26; 8:45 am]

BILLING CODE 4164-01-P

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91 FR 24549

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“Issuance of Priority Review Voucher; Rare Pediatric Disease Product; OTARMENI (lunsotogene parvec-cwha),” thefederalregister.org (May 6, 2026), https://thefederalregister.org/documents/2026-08913/issuance-of-priority-review-voucher-rare-pediatric-disease-product-otarmeni-lunsotogene-parvec-cwha.