RARE - Ultragenyx Pharmaceutical Inc.

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
55.71
+0.91 (+1.66%)
At close: 4:00PM EST
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Previous Close54.80
Open55.39
Bid49.50 x 1100
Ask65.60 x 800
Day's Range54.99 - 57.40
52 Week Range35.41 - 74.50
Volume283,866
Avg. Volume539,695
Market Cap3.225B
Beta (5Y Monthly)2.46
PE Ratio (TTM)N/A
EPS (TTM)N/A
Earnings DateN/A
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target EstN/A
All
News
Press Releases
  • GlobeNewswire

    Ultragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The FDA has assigned priority review designation with a Prescription Drug User Fee Act (PDUFA) target date of June 18, 2020.

  • GlobeNewswire

    Ultragenyx to Present at SVB Leerink Global Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company’s Chief Business Officer, will present at the SVB Leerink Global Healthcare Conference on Wednesday, February 26, 2020 at 11:00 AM ET in New York, NY. The live and archived webcast of the presentation will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update

    2019 total revenue is $103.7 million; 2019 Crysvita® (burosumab) revenue to Ultragenyx is $87.3 million 2020 Crysvita revenue in Ultragenyx territories guidance of $125.

  • GlobeNewswire

    Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2019 Financial Results and Corporate Update

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, February 13, 2020 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2019. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

  • GlobeNewswire

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

    GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. "The FDA’s clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community,” said Paula Evans, Chief Executive Officer at GeneTx.

  • GlobeNewswire

    Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories.

  • GlobeNewswire

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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February 2020. “Approximately half of patients with TIO have tumors that cannot be surgically removed, leaving them with no other current treatment options,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

  • GlobeNewswire

    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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. In Cohort 3 (n=3), there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status.

  • GlobeNewswire

    Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco. The live and archived webcast of the presentation will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Royalty Pharma today announced that Ultragenyx has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland. Crysvita has received conditional marketing authorization in Europe for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.

  • GlobeNewswire

    Ultragenyx to Present at Piper Jaffray 31st Annual Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 2:00 PM ET in New York, NY. The live and archived webcast of the presentation will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx to Present at Upcoming Investor Conferences

    Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Credit Suisse 28th Annual Healthcare Conference on Tuesday, November 12, 2019 at 11:30 AM MT in Scottsdale, Arizona. Tom Kassberg, the company’s Chief Business Officer, will present at the Stifel Healthcare Conference on Wednesday, November 20, 2019 at 1:50 PM ET in New York, NY. The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm.

  • GlobeNewswire

    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.

  • GlobeNewswire

    Ultragenyx to Host Conference Call for Third Quarter 2019 Financial Results and Corporate Update

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, November 5, 2019 at 5pm ET to discuss third quarter 2019 financial results and provide a corporate update. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

  • GlobeNewswire

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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.

  • GlobeNewswire

    Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, the company’s Chief Executive Officer and President will present at the Jefferies Gene Therapy/Editing Summit on Tuesday, October 8, 2019 at 9:45 AM ET in New York, NY. The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for Crysvita® (burosumab). The label has been updated to include new clinical data demonstrating superiority of treatment with Crysvita versus oral phosphate and active vitamin D (conventional therapy) in pediatric patients with XLH, and improvement in stiffness, and maintenance of efficacy of Crysvita in adult patients with longer-term treatment. Crysvita is an antibody that binds to and inhibits the biological activity of fibroblast growth factor 23 (FGF23), restoring renal phosphate reabsorption and increasing the serum concentration of 1,25 dihydroxy vitamin D. It was first approved in the U.S. in April 2018 for the treatment of XLH in adult and pediatric patients one year of age and older.

  • GlobeNewswire

    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 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and.

  • GlobeNewswire

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

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 2 have shown a clinical response with improvements in glucose control and other metabolic parameters compared to baseline.

  • GlobeNewswire

    GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102

    GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. “The granting of these designations from the FDA is an enormous milestone for the Angelman community,” stated Paula Evans, Chief Executive Officer of GeneTx.

  • GlobeNewswire

    Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and GeneTx Biotherapeutics LLC, today announced a partnership to develop GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. GTX-102 is currently in late preclinical development with an investigational new drug (IND) application expected to be filed with the U.S. Food and Drug Administration (FDA) in the first half of 2020.

  • GlobeNewswire

    Ultragenyx to Present at Wedbush PacGrow Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company’s Chief Business Officer, will present at the Wedbush PacGrow Healthcare Conference on Wednesday, August 14, 2019 at 1:20 PM ET in New York, NY. The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update

    Strong Crysvita® (burosumab) Launch Continues with Approximately 960 Patients on Reimbursed Commercial Therapy in the United States Ultragenyx Submits New Drug Application to.

  • GlobeNewswire

    Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that it has submitted to the U.S. Food and Drug Administration (FDA) a New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA previously granted Rare Pediatric Disease Designation and Fast Track designation, which enables eligibility for Priority Review, if relevant criteria are met.