Ultragenyx has agreed to sell its rare pediatric disease priority review voucher for $203 million, marking the company’s second PRV sale within one week.
Ultragenyx received the PRV from the FDA’s September 2026 approval of Fayuvi, an AAV9 gene therapy for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). The approval marked the first-ever FDA-approved treatment for the indication, a rare fatal lysosomal storage disease that primarily affects the brain.
The buyer was not revealed.
The news comes just a week after Ultragenyx sold the PRV received from the FDA’s August 2026 approval of Genglycos for $210 million. Genglycos, a one-time AAV8 based gene therapy, is 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.
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