Ultragenyx Agrees to Sell Rare Pediatric Disease Voucher for $203 Million

Rare Daily Staff

Ultragenyx Pharmaceutical has agreed to sell a rare pediatric disease priority review voucher for $203 million, marking its second such agreement within a week and providing funding to support its rare-disease medicines and development programs.

The company received the voucher when the U.S. Food and Drug Administration approved Fayuvi, its gene therapy for mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. Fayuvi is the first FDA-approved treatment for the condition, according to Ultragenyx.

The agreement follows a previously disclosed $210 million voucher sale associated with Genglycos.

The FDA’s rare pediatric disease priority review voucher program is designed to encourage companies to develop treatments for serious rare diseases affecting children. A company that secures a qualifying approval may receive a voucher that can be used to obtain priority review of a subsequent marketing application for a different product.

The voucher can also be sold or transferred, allowing a rare-disease drug developer to turn the regulatory incentive into capital.

For Ultragenyx, the $203 million agreement represents non-dilutive funding—money raised without issuing additional shares or reducing existing shareholders’ ownership stakes.

“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,” said Howard Horn, Ultragenyx’s chief financial officer and executive vice president of corporate strategy.

“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,” he said.

Closing of the sale remains subject to customary conditions.