RARE Ultragenyx Pharmaceutical Inc.

Drug Information

Drug catalyst information is displayed when you hover over / tap on the stage bar graph.

Latest News

1. 15 January 2020 GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

   SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products

2. 13 January 2020 Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

   Preliminary 2019 total revenue is approximately $102 million to $104 million Preliminary 2019 Crysvita revenue to Ultragenyx of approximately $86 million to $88 million 2020 Crysvita revenue in Ultragenyx

3. 13 January 2020 Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

   NOVATO, Calif. and TOKYO, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

4. 09 January 2020 Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

   More uniform response in Cohort 3 with two confirmed responders and one potential responder New female responder in Cohort 2 for a total of three confirmed female responders across all cohorts Up to six responders

5. 19 December 2019 Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

   NOVATO, Calif., Dec. 19, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

6. 18 December 2019 Ultragenyx Announces Sale of Future European Royalties on Crysvita® (burosumab) for $320 Million to Royalty Pharma

   NOVATO, Calif. and NEW YORK, Dec. 18, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

7. 08 November 2019 Ultragenyx to Present at Upcoming Investor Conferences

   NOVATO, Calif., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

8. 05 November 2019 Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update

   Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Quarter UX007 (triheptanoin) NDA for Treatment of

9. 30 October 2019 Ultragenyx to Host Conference Call for Third Quarter 2019 Financial Results and Corporate Update

   NOVATO, Calif., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

10. 14 October 2019 Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders

    NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today

11. 02 October 2019 Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

    NOVATO, Calif., Oct. 02, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

12. 30 September 2019 Ultragenyx and Kyowa Kirin Announce U.S. FDA Approves Label Update for Crysvita® (burosumab) for the Treatment of X-Linked Hypophosphatemia (XLH)

    Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older Expanded Label

13. 10 September 2019 Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and

14. 04 September 2019 Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

    Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses

15. 03 September 2019 GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102

    SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel

16. 30 August 2019 Ultragenyx to Present at Upcoming Investor Conferences

    NOVATO, Calif., Aug. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,