RARE Ultragenyx Pharmaceutical Inc.

42.71
-1.18  -3%
Previous Close 43.89
Open 43.8
Price To Book 3.01
Market Cap 2,464,023,483
Shares 57,691,957
Volume 1,091,845
Short Ratio
Av. Daily Volume 601,818
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NewsSee all news

  1. 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

  2. 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,

  3. 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

  4. 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

  5. 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

Drug Information

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

Phase 1/2 third cohort data due 4Q 2019.
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 planned for 1H 2020.
KRN23 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 second cohort released September 4, 2019.
DTX401
GSD1

Latest News

  1. 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

  2. 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,

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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,