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.

Pomerantz LLP announced investigations into potential securities fraud and unlawful business practices at OSI Systems, lululemon athletica, and Ultragenyx Pharmaceutical. The investigation into OSI was triggered by the company's August 20, 2026 earnings report that missed revenue expectations due to deferred product deliveries, resulting in a 5.21% stock price decline.

Pomerantz LLP announced investigations into potential securities fraud at Ultragenyx Pharmaceutical, OSI Systems, and lululemon athletica. The investigation into Ultragenyx was triggered by the company's failure to meet primary endpoints in a Phase 3 clinical trial for apazunersen, resulting in a 44% stock price decline on September 3, 2026.