Ultragenyx sells second rare pediatric PRV in a week for $203M
October 22, 2026
Ultragenyx has entered into a definitive agreement to sell a rare pediatric disease Priority Review Voucher (PRV) for $203 million, its second voucher sale in the space of a week.
The US Food and Drug Administration’s (FDA) rare pediatric disease PRV programme is designed to encourage development of treatments for serious conditions that affect children. A sponsor that wins approval for such a product may receive a voucher, which can be redeemed for priority review of a later application for a different product, or sold on.
The Novato, California-based company received the voucher following the FDA’s approval of FAYUVI (rebisufligene etisparvovec-hopf). The gene therapy is the first FDA-approved treatment for mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. The buyer has not been disclosed.
“The sale of this PRV delivers meaningful non-dilutive funding that strengthens our ability to provide innovative treatments for rare and ultra-rare diseases while supporting our objective of achieving profitability,” Howard Horn, chief financial officer and executive vice president of corporate strategy at Ultragenyx, said. “FAYUVI itself benefited from capital generated from a previous PRV sale, demonstrating the important role the PRV program plays in helping companies develop transformative therapies for rare disease patients.”
The deal follows Ultragenyx’s agreement to sell the voucher it earned from the approval of GENGLYCOS for glycogen storage disease type Ia for $210 million. Together, the two transactions bring in $413 million for the biotech.
“Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitability,” Horn said.
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