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.
Ultragenyx (NASDAQ: RARE) announced the U.S. FDA has accepted the resubmitted BLA for UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a PDUFA action date of September 19, 2026 and previously granted Priority Review in February 2025.
Updated long-term clinical data in the filing show up to 8 years of follow-up with durable clinical improvements versus natural history and supportive biomarker evidence, and the submission reports an acceptable safety profile. If approved, UX111 would be the first therapy for MPS IIIA and be manufactured in the U.S.
Ultragenyx (NASDAQ: RARE) announced FDA clearance of an Investigational New Drug application for UX016, a sialic acid prodrug for treatment of GNE myopathy. The externally funded program plans a first-in-human Phase 1/2 study starting in the second half of 2026 to enroll ~24 adults.
UX016 is designed to improve muscle delivery of sialic acid via a fatty-acid prodrug tail; the study will assess safety, pharmacokinetics, and muscle delivery, plus efficacy measures through Week 48.
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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).