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.

Ultragenyx (RARE) reported Q2 2026 earnings that beat expectations with a narrower loss of $0.90 per share versus consensus of $1.27, and revenues of $214 million surged 28.1% year-over-year, marking the company's highest quarterly revenues. However, despite the strong results, estimate revisions have trended downward over the past month, and the stock received a Zacks Rank #3 (Hold) rating with expectations for in-line returns in the coming months.

Amlogenyx, a biotechnology company developing gene therapies for Alzheimer's disease, announced it will present at the Wells Fargo 21st Annual Healthcare Conference on September 8, 2026. The company is developing AM805, a novel protease therapy designed to reduce amyloid accumulation in the brain, and is currently raising Series A financing for Phase 2a clinical proof of concept trials.

The global Fabry disease treatment market is projected to grow from USD 2.63 billion in 2025 to USD 4.09 billion by 2031, with a CAGR of 7.63%. Growth is driven by expanded screening identifying more patients, advances in gene and mRNA therapies, and sustained enzyme replacement therapy demand. However, high treatment costs remain a significant barrier to patient access, particularly in emerging markets. Asia-Pacific is expected to record the fastest growth at 8.28% CAGR.

Ultragenyx Pharmaceutical Inc. granted 44,409 restricted stock units to 15 newly hired non-executive officers on June 16, 2026, as an inducement for employment. The awards were approved by the compensation committee under the Ultragenyx Employment Inducement Plan and comply with Nasdaq Listing Rule 5635(c)(4). The restricted stock units vest over four years with 25% vesting annually, contingent on continuous employment.

A class action lawsuit has been filed against Ultragenyx Pharmaceutical for allegedly providing misleading statements about setrusumab's efficacy in treating Osteogenesis Imperfecta. The company claimed the drug would reduce fracture rates, but Phase III Orbit and Cosmic studies failed to achieve statistical significance on primary endpoints. Ultragenyx stock plummeted 42.32% on December 29, 2025, following the announcement of study failures. Investors who purchased shares between August 3, 2023, and December 26, 2025, have until April 6, 2026, to apply as lead plaintiffs.

Rosen Law Firm is notifying investors in BlackRock TCP Capital Corp. (TCPC) and other companies about ongoing securities class action lawsuits. The firm is seeking a lead plaintiff by the April 6, 2026 deadline. According to the lawsuit, BlackRock TCP made materially false statements regarding investment valuations, portfolio quality, and net asset value, resulting in investor losses.

Rosen Law Firm has filed securities class action lawsuits against Ultragenyx Pharmaceutical, Kyndryl Holdings, and Vital Farms, urging investors with significant losses to secure legal counsel before upcoming deadlines. The Ultragenyx case alleges the company made misleading statements about setrusumab's effectiveness in treating Osteogenesis Imperfecta, claiming bone density increases did not correlate to reduced fracture rates as promised.