Ultragenyx sells priority review voucher for $210M

Ultragenyx Pharmaceutical announced it will sell its rare pediatric disease priority review voucher for $210 million.

Ultragenyx received the PRV from the FDA’s August 2026 approval of Genglycos, the first treatment designed to address the underlying cause of glycogen storage disease type Ia (GSDIa). GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream. The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications, requiring rigorous nutritional management that involves a burdensome, around-the-clock regimen of raw cornstarch intake.

The buyer was not revealed, but Ultragenyx say the PRV provide non-dilutive capital to advance its efforts to bring forward first-ever therapies for rare and ultra-rare diseases.

Ultragenyx received an additional PRV from the September approval of Fayuvi, a AAV9 gene therapy, for the the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A).