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 Pharmaceutical (NASDAQ: RARE) has received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for UX111, a gene therapy treatment for Sanfilippo syndrome type A. The FDA cited specific chemistry, manufacturing and controls (CMC) related observations that require resolution.
While the FDA acknowledged the robustness of clinical data and supportive biomarker evidence, this delay pushes the potential approval timeline to 2026. The company believes the CMC observations are addressable and primarily relate to facilities and processes, not product quality. After resolving these issues, Ultragenyx plans to resubmit the BLA, which will be followed by an estimated 6-month review period.
Ultragenyx Pharmaceutical (NASDAQ: RARE) and Mereo BioPharma announced that their Phase 3 Orbit study of UX143 (setrusumab) for osteogenesis imperfecta (OI) will continue to final analysis, expected around year-end. The Data Monitoring Committee confirmed UX143's acceptable safety profile.
The study evaluates UX143 in pediatric and young adult OI patients. The parallel Cosmic study in younger patients is also progressing well, with safety consistent with other studies. Both trials will conclude after patients complete 18 months of therapy, with significance thresholds set at p<0.04 for Orbit and p<0.05 for Cosmic.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has received FDA Breakthrough Therapy Designation for GTX-102 (apazunersen) in treating Angelman syndrome. The designation is based on promising Phase 1/2 study results involving 74 patients aged 4-17 years, showing consistent developmental improvements across multiple symptom domains for up to 3 years.
The company's Phase 3 Aspire study, which began in December 2024, aims to enroll approximately 120 children with Angelman syndrome. Additionally, the Aurora study, evaluating GTX-102 in other Angelman syndrome genotypes and ages, is expected to start in the second half of 2025.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 44,805 restricted stock units (RSUs) to 20 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of May 16, 2025. The RSUs were granted as employment inducements in compliance with Nasdaq Listing Rule 5635(c)(4). The vesting schedule spans four years, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel therapies for rare and ultra-rare genetic diseases, announced that CFO and Executive VP Howard Horn will participate in a fireside chat at Bank of America's 2025 Healthcare Conference. The event is scheduled for Tuesday, May 13, 2025, at 8:00 a.m. PT. Investors and interested parties can access both the live and archived webcast of the panel through Ultragenyx's investor relations website.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a leading biopharmaceutical company specializing in rare and ultra-rare genetic diseases, has scheduled its first quarter 2025 financial results conference call.
The earnings call will take place on Tuesday, May 6, 2025, at 5:00 p.m. ET. During this call, the company will present its financial performance for the quarter ending March 31, 2025, along with a corporate update.
Investors and interested parties can access the live webcast through the company's investor relations website at https://ir.ultragenyx.com/events-presentations. A replay of the conference call will remain available for three months following the presentation.