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. (NASDAQ: RARE) is a biopharmaceutical company focused on novel therapies for serious rare and ultra-rare genetic diseases. The RARE news feed highlights company announcements on commercial performance, clinical development, and key corporate decisions that shape its rare disease portfolio.
Investors and followers of Ultragenyx can find updates on revenue trends from its approved products Crysvita, Dojolvi, Mepsevii, and Evkeeza in select territories, as well as guidance ranges and commentary on operating expenses and cash position. Earnings-related press releases and Form 8-K summaries detail product sales, royalty revenue, and royalty financing transactions, including agreements involving future Crysvita royalties with OMERS.
The news stream also covers Ultragenyx’s late-stage pipeline and regulatory milestones. Readers can track progress of AAV gene therapy programs such as DTX401 for glycogen storage disease type Ia, UX111 for Sanfilippo syndrome type A, and UX701 for Wilson disease, along with monoclonal antibody UX143 for osteogenesis imperfecta and antisense oligonucleotide GTX-102 for Angelman syndrome. Company releases describe Phase 3 study results, longer-term follow-up data, regulatory designations, rolling BLA submissions, and responses to FDA communications such as Complete Response Letters.
In addition, the RARE news page features items on conference presentations, investor events, and equity inducement grants under Nasdaq Listing Rule 5635(c)(4). By reviewing these updates, users can follow how Ultragenyx manages its commercial portfolio, advances its clinical pipeline, and executes financing and collaboration strategies in the rare and ultra-rare disease space.
Summary not available.
Ultragenyx (NASDAQ: RARE) reported 2025 total revenue of $673 million (+20% YoY), including Crysvita $481 million and Dojolvi $96 million. Net loss was $575 million; cash and marketable securities totaled $737 million at year-end.
The company provided 2026 guidance of $730–$760 million from current products, outlined a strategic restructuring (10% workforce reduction, ~130 roles) and reiterated a path to profitability in 2027.
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.