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) reported an inducement grant of 10,839 restricted stock units to two newly hired non-executive officers under the Ultragenyx Employment Inducement Plan, with a grant date of March 16, 2026.
The awards were approved by the compensation committee and vest over four years, with 25% of underlying shares vesting on each anniversary, subject to continued employment, in accordance with Nasdaq Listing Rule 5635(c)(4).
Ultragenyx (NASDAQ: RARE) reported positive 36-week Phase 3 results for DTX301 in OTC deficiency, showing a statistically significant 18% reduction in 24-hour plasma ammonia AUC0-24 versus placebo (p=0.018) and maintenance of average ammonia in the normal range through Week 36.
Patients reduced ammonia scavenger drugs by a mean 27% and increased protein intake ~13% while on DTX301; safety was acceptable with one treatment-related acute hepatitis that resolved. Additional 64-week treatment-burden data are expected in H1 2027.
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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.