Ultragenyx Agrees to Sell FDA Rare Pediatric-Disease Priority Review Voucher for $203 Million

Ultragenyx Pharmaceutical said in an SEC filing on Wednesday that it agreed to sell a rare pediatric disease priority review voucher tied to its newly approved gene therapy FAYUVI for $203 million in cash, marking the company’s second disclosed voucher sale this month and underscoring how biotechs use the transferable FDA awards to raise non-dilutive funding.

The disclosure came in a Form 8-K accepted by the Securities and Exchange Commission on Oct. 14 and describing an Oct. 8 event under “Entry into a Material Definitive Agreement.” Ultragenyx said it entered into an asset purchase agreement, referred to in the filing as the PRV Asset Purchase Agreement, to sell the voucher.

A rare pediatric disease priority review voucher is awarded by the Food and Drug Administration when a company wins approval for a treatment for a qualifying rare childhood disorder. The sponsor can use the voucher to secure a speedier FDA review for another drug application or sell it to another company. Ultragenyx received the voucher on Sept. 17, when the FDA approved FAYUVI for pediatric mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. The company has said FAYUVI is the first approved treatment for that disease.

Under the newly disclosed agreement, the buyer will pay $203 million in cash at closing, according to the 8-K. Ultragenyx did not identify the buyer. The transaction is subject to customary closing conditions, including expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, the U.S. law that requires certain deals to undergo antitrust review. Ultragenyx said the full agreement will be filed with its 2026 annual report on Form 10-K.

The FAYUVI-related deal is Ultragenyx’s second announced voucher sale in October. In a separate Oct. 6 Form 8-K, the company said it agreed to sell another rare pediatric disease priority review voucher, this one tied to the Aug. 19 approval of GENGLYCOS, for $210 million. If both transactions close, the two deals would bring in $413 million in gross cash consideration, based on the company’s filings.

The two filings differ in one notable respect. The Oct. 6 disclosure said 20% of the gross proceeds from the GENGLYCOS-related voucher sale would be payable to the National Institutes of Health under a 2018 patent license. The new Oct. 14 filing related to FAYUVI does not mention any comparable NIH payment obligation.

Priority review vouchers have become a recurring financing tool for biotechnology companies because they can be monetized without issuing stock or taking on debt. Ultragenyx highlighted that dynamic in discussing the earlier GENGLYCOS-related sale. Chief Financial Officer Howard Horn said in an Oct. 7 press release that “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.”