RARE

Ultragenyx Pharmaceutical agreed to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million following FDA approval of Fayuvi, a gene therapy for mucopolysaccharidosis type IIIA. This marks the company's second PRV sale in one week, with the previous Genglycos voucher sale generating $210 million. Both transactions provide non-dilutive funding to support pipeline development and profitability goals.

Ultragenyx Pharmaceutical reported $350 million in first-half 2026 revenues, up 14% year-over-year, driven by Crysvita ($249M) and strong growth in Evkeeza (up 56%). The company received FDA approvals for two new gene therapies—Genglycos and Fayuvi—and sold a priority review voucher for $210 million.

However, RARE stock has underperformed, declining 35.1% year-to-date, and faces competition from Amgen and Denali Therapeutics. The company carries a Zacks Rank #3 (Hold) rating.

Ultragenyx Pharmaceutical has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million following FDA approval of its gene therapy Genglycos. After a $42 million NIH payment obligation, approximately $168 million in non-dilutive capital will support the company's research, development, and commercialization efforts, helping it move toward profitability without equity financing.