RARE Ultragenyx Pharmaceutical Inc.

59.02
-0.94  -2%
Previous Close 59.96
Open 60
Price To Book 4.69
Market Cap 3,409,639,875
Shares 57,770,923
Volume 470,956
Short Ratio
Av. Daily Volume 617,315
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NewsSee all news

  1. 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. 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. 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. 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. 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,

Drug Information

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

Phase 1/2 third cohort data released January 9, 2020. 6/9 responders across whole trial. Prophylactic steroid cohort to begin 1H 2020 with data due 2H 2020.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency
Approved under priority review November 15, 2017.
MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
Phase 3 data released October 26, 2018 - endpoints not met.
UX007 (triheptanoin)
Glut1 DS patients with the movement disorder phenotype
Phase 3 data released August 22, 2017 - endpoints not met.
Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
Approval announced April 17, 2018.
Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
sBLA filing announced January 13, 2020.
Burosumab
Tumor-induced osteomalacia (TIO)
PDUFA date July 31, 2020.
UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
sBLA filing for children due 2H 2019.
KRN23 Burosumab
X-Linked Hypophosphatemia (XLH)
Phase 1/2 data from expansion cohort due 1H 2020.
DTX401
GSD1
Phase 1/2 enrolment to commence 1H 2020.
GTX-102
Angelman Syndrome

Latest News

  1. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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. 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,