Federal Register #2026-17932

FDA Awards Priority Review Voucher for GENGLYCOS Gene Therapy (Ultragenyx)

The Food and Drug Administration (FDA), part of the Department of Health and Human Services, has issued a priority review voucher for a rare pediatric disease product. - Government buyer: - Food and Drug Administration (FDA), Department of Health and Human Services - OEM highlighted: - Ultragenyx Pharmaceutical, Inc. - Product awarded: - GENGLYCOS (parisglasgene brecaparvovec-opnr) - Adeno-associated virus (AAV) vector-based gene therapy - Indicated to reduce daily cornstarch intake as an adjunct to nutritional management for adult and pediatric patients (8 years and older) with Glycogen Storage Disease Type 1a (GSD1a) - Notable requirements: - Product meets criteria for rare pediatric disease designation under the Federal Food, Drug, and Cosmetic Act - Award is for a regulatory priority review voucher, not a procurement of goods or services - No purchase quantities or part numbers specified - No competing products or vendors listed, as this is a regulatory award

Description

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 voucher is awarded under the Federal Food, Drug, and Cosmetic Act to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined that GENGLYCOS (parisglasgene brecaparvovec-opnr), approved on August 19, 2026, and manufactured by Ultragenyx Pharmaceutical, Inc., meets the criteria for a priority review voucher. GENGLYCOS is an adeno-associated virus vector-based gene therapy indicated to reduce daily cornstarch intake as an adjunct to nutritional management in patients 8 years and older with Glycogen Storage Disease Type 1a.

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