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Mkt Cap

$1.43B

Volume

2.24M

Next Earnings

November 3rd

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RA

RAREUltragenyx Pharmaceutical Inc.

RARE

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RA

Mkt Cap

$1.50B

Volume

2.24M

Next Earnings

November 3rd

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RARE's Fayuvi Approval Spurs Second PRV Sale Worth $203M in a WeekUltragenyx 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.Zacks Investment Research · Oct 15, 2026

Can RARE's Approved Product Portfolio Drive Its Next Growth Phase?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.Zacks Investment Research · Oct 14, 2026

Ultragenyx to Sell Rare Pediatric Disease PRV for $210M to Boost CashUltragenyx 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.Zacks Investment Research · Oct 8, 2026

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of OSI Systems, Inc. - OSISPomerantz 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.GlobeNewswire Inc. · Oct 6, 2026

INVESTOR ALERT: Pomerantz Law Firm Investigates Claims On Behalf of Investors of Ultragenyx Pharmaceutical Inc. - RAREPomerantz 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.GlobeNewswire Inc. · Oct 6, 2026

Why Is Ultragenyx (RARE) Up 6.4% Since Last Earnings Report?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.Zacks Investment Research · Sep 3, 2026

Amlogenyx to Present at the Wells Fargo 21ˢᵗ Annual Healthcare ConferenceAmlogenyx, 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.GlobeNewswire Inc. · Sep 2, 2026

Fabry Disease Treatment Market 2026-2031 - Growth Driven by Increasing Adoption of Enzyme Replacement TherapyThe 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.GlobeNewswire Inc. · Aug 25, 2026

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)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.GlobeNewswire Inc. · Jun 19, 2026

ULTRAGENYX URGENT DEADLINE: Bragar Eagel & Squire, P.C. Reminds Ultragenyx (NASDAQ:RARE) Investors of the April 6th Lead Plaintiff Deadline and Urges Investors to Contact the FirmA 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.GlobeNewswire Inc. · Apr 6, 2026

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