California-based Ultragenyx Pharmaceutical (Nasdaq: RARE) announced an agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for USD 210 million to an undisclosed buyer, following the US FDA's accelerated approval of its gene therapy Genglycos (pariglasgene brecaparvovec-opnr, DTX401) for glycogen storage disease type Ia (GSDIa) in August 2026.
Under the transaction terms disclosed in an SEC filing, Ultragenyx will pay 20% of the gross proceeds, equivalent to USD 42 million, to the US National Institutes of Health under a 2018 patent licensing agreement, leaving USD 168 million before transaction expenses. The sale remains subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.
The US FDA granted accelerated approval to Genglycos on August 19, 2026, to reduce daily cornstarch intake as an adjunct to nutritional management in patients aged 8 years and older with GSDIa. Approval was supported by the Phase III GlucoGene study, in which treated patients achieved a statistically significant 31% mean reduction from baseline in daily cornstarch intake relative to placebo at Week 48. Ultragenyx is required to collect additional post-marketing data to confirm clinical benefit.