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.
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Ultragenyx (NASDAQ: RARE) will participate in three investor conferences in March 2026 with Eric Crombez, M.D., Chief Medical Officer, delivering fireside chats and hosting 1x1 meetings.
Events: Cowen Healthcare Conference on March 2 in Boston; Barclays Global Healthcare on March 10 in Miami; Leerink Partners Global Biopharma on March 11 in Miami. Live and archived webcasts will be available at the company investor events page.
Ultragenyx (NASDAQ: RARE) announced the FDA accepted its BLA for DTX401 (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia and granted Priority Review.
The FDA set a PDUFA action date of August 23, 2026. The BLA uses data from 52 treated patients with up to six years follow-up showing reduced daily cornstarch intake, maintained low hypoglycemia, improved euglycemia and fasting tolerance, positive patient-reported quality-of-life results, and an acceptable safety profile. If approved, DTX401 will be manufactured in Bedford, Massachusetts.
Ultragenyx (NASDAQ: RARE) granted 22,917 restricted stock units to six newly hired non-executive officers as an inducement under the Ultragenyx Employment Inducement Plan.
Grants were approved by the compensation committee, dated Feb 16, 2026, and vest 25% annually over four years, subject to continued employment and Nasdaq Rule 5635(c)(4).
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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.