Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. develops and commercializes therapies for serious rare and ultra-rare genetic diseases. Company news commonly covers revenue from approved medicines such as Crysvita, Dojolvi, and Mepsevii, financial guidance, and updates to a portfolio of approved therapies and product candidates for diseases with high unmet medical need and clear biology.
Recurring developments include FDA interactions and clinical data for investigational programs such as UX111 for Sanfilippo syndrome type A, GTX-102 for Angelman syndrome, UX016 for GNE myopathy, and AAV gene therapy candidates for rare metabolic disorders. Updates also include healthcare conference participation, employment inducement equity grants under Nasdaq rules, and corporate restructuring or expense-management actions when disclosed.
Ultragenyx (NASDAQ: RARE) will host a conference call at 5:00 p.m. ET on Thursday, February 12, 2026 to discuss fourth-quarter and full-year 2025 financial results and a corporate update. A live webcast and replay will be available via the company investor website.
The replay will remain available for three months following the call.
Ultragenyx (NASDAQ:RARE) reported up to 8.5 years of follow-up for UX111 (rebisufligene etisparvovec) in MPS IIIA, showing a median 63.98% reduction in CSF heparan sulfate (p<0.001) and sustained functional benefits versus natural history, including a +23.2-point cognitive treatment effect (p<0.0001) in younger patients. UX111 was generally well tolerated (N=33; median follow-up 4.8 years). The company resubmitted a BLA to FDA in January 2026 and expects a PDUFA in Q3 2026 following up to a six-month review.
Ultragenyx (NASDAQ: RARE) resubmitted a Biologics License Application (BLA) for UX111 (rebisufligene etisparvovec) AAV9 gene therapy to treat Sanfilippo syndrome type A (MPS IIIA).
The filing includes longer-term clinical data up to 8.5 years, CSF heparan sulfate and other biomarker evidence, comprehensive CMC responses to a July 2025 Complete Response Letter, and maintains an acceptable safety profile. The FDA granted Priority Review in February 2025; a PDUFA date is expected in Q3 2026 following an anticipated up-to-six-month review.
Ultragenyx (NASDAQ: RARE) announced an inducement equity grant under Nasdaq Listing Rule 5635(c)(4). The company granted 16,355 restricted stock units to nine newly hired non-executive officers, approved by the compensation committee and issued under the Ultragenyx Employment Inducement Plan.
The grants have a grant date of January 16, 2026 and vest over four years with 25% of the underlying shares vesting on each anniversary of the grant date, subject to continued employment as of each vesting date.
Ultragenyx (Nasdaq: RARE) announced topline results from Phase III Orbit and Cosmic studies of setrusumab (UX143) for osteogenesis imperfecta showing both studies failed their primary endpoint of reducing annualized clinical fracture rate versus placebo (Orbit) and versus bisphosphonates (Cosmic).
The studies did show improved bone density but that secondary result did not correlate with lower fracture rates. Management cited a low placebo-group fracture rate as an explanation. Analyst coverage turned cautious with firms, including Barclays, noting limited approval prospects. Levi & Korsinsky has opened an investigation related to the announcement.
Ultragenyx (NASDAQ: RARE) reported preliminary unaudited 2025 results and a corporate update at J.P. Morgan Healthcare Conference on January 12, 2026. Preliminary 2025 total revenue was $672M–$674M, roughly +20% vs 2024, with Crysvita revenue $480M–$482M (+17% vs 2024) and Dojolvi revenue $95M–$97M (+9% vs 2024). Cash and investments were ~ $735M at December 31, 2025. The company completed a rolling BLA submission for DTX401 and expects a PDUFA in Q3 2026; UX111 BLA resubmission is planned for early 2026 with up to a 6-month review. Key 2026 catalysts include potential approvals for two gene therapies and pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome in H2 2026.
Ultragenyx (NASDAQ: RARE) announced that Emil D. Kakkis, M.D., Ph.D., CEO and president, will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026 at 10:30 AM PT. A live webcast and archived replay will be available via the company investor website at https://ir.ultragenyx.com/events-presentations. The webcast replay will remain accessible for 30 days following the presentation.
Ultragenyx (NASDAQ: RARE) completed the rolling submission of its Biologics License Application to the U.S. FDA for DTX401 (pariglasgene brecaparvovec), an AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa), on Dec 30, 2025. The BLA includes non‑clinical, clinical, and completed chemistry, manufacturing, and controls (CMC) modules.
The submission is supported by a clinical program of 52 treated patients with up to six years follow‑up and Phase 3 GlucoGene randomized, double‑blind, placebo‑controlled data showing significant reductions in daily cornstarch intake, maintained low hypoglycemia, improved euglycemia, improved fasting tolerance, and patient‑reported quality‑of‑life benefits; DTX401 was reported as well tolerated with an acceptable safety profile.
Ultragenyx (NASDAQ: RARE) reported Phase 3 results for setrusumab (UX143) in osteogenesis imperfecta from the Orbit and Cosmic studies dated Dec 29, 2025. Neither trial met the primary endpoint of a statistically significant reduction in annualized clinical fracture rate versus placebo (Orbit) or bisphosphonates (Cosmic). Both studies did achieve the secondary endpoint of statistically significant improvements in bone mineral density (BMD) versus comparators. Safety findings showed no change in the safety profile. Ultragenyx said it will implement significant expense reductions while performing additional analyses of other bone-health and clinical endpoints to define next steps for the program.
Ultragenyx (NASDAQ: RARE) granted 13,144 restricted stock units to nine newly hired non-executive officers under the Ultragenyx Employment Inducement Plan.
The awards were approved by the compensation committee with a grant date of December 17, 2025 and reported December 19, 2025, as an inducement under Nasdaq Listing Rule 5635(c)(4). The RSUs vest over four years with 25% vesting each anniversary, subject to continuous employment on each vesting date.