Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company held a successful meeting with the U.S. Food and Drug Administration (FDA or the Agency) during which the company reached a ...

Ultragenyx Pharmaceutical Inc. (RARE) , along with partner Mereo BioPharma (MREO), announced new positive 14-month data from the phase II portion of the phase II/III Orbit study evaluating setrusumab (UX143) in patients with osteogenesis imperfecta (OI).The data showed that the large reduction in fracture rates previously reported in patients treated for a minimum of six months was sustained in patients treated for at least 14 months.OI is a group of genetic disorders that impact bone metabolism. It is char ...

Ariawan Armoko/iStock via Getty Images Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is a good long-term biotech to own in my opinion. That's because it has already been able to receive regulatory approvals for several of its drugs in its pipeline. Matter of fact, the drugs being sold by the company are Crysvita and Dojolvi. In Q1 of 2024, it generated $109 million in total revenue, which was an 8% growth year-over-year. As such, if sales continue like this, it will continue to build shareholder value. I ...

NOVATO, Calif., June 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 45th Annual Global Healthcare Conference on Tuesday, June 11, 2024, at 10:00 a.m. ET. The live and archived webcast of the panel will ...

Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at 5:00 p.m. ET NOVATO, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced positive topline results from the Phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational gene therapy for the treatment of patients aged eight years and older with glycog ...

NOVATO, Calif., May 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 17,180 restricted stock units of the company's common stock to ten newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducem ...

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q1 2024 Earnings Conference Call May 2, 2024 5:00 PM ET Company Participants Joshua Higa - Vice President of Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Howard Horn - Chief Financial Officer Eric Crombez - Chief Medical Officer Conference Call Participants Yigal Nochomovitz - Citigroup Anupam Rama – JPMorgan Tazeen Ahmad - Bank of America Securities Gena Wang – Barclays Dae Gon Ha – Stifel Kristen K ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other j ...

Exhibit 99.1 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Presented positive interim Phase 1/2 data ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File No. 001-36276 Ultragenyx Pharmaceutical Inc. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other jurisdiction of (I. ...