RARE Ultragenyx Pharmaceutical Inc.

64.43
-0.28  -0%
Previous Close 64.71
Open 64.74
Price To Book 5.43
Market Cap 3303903264
Shares 51,278,958
Volume 1,142,855
Short Ratio
Av. Daily Volume 683,161

Drug Information

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

Phase 1/2 high dose Cohort 3 data due mid-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)
Data released September 2018.
KRN23 Burosumab
Tumor-induced osteomalacia (TIO)
NDA to be filed mid-2019.
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 positive low dose cohort data released January 4, 2019. Data from second cohort due mid-2019.
DTX401
GSD1

Latest News

  1. Ultragenyx's (RARE) UX007 Gets Fast Track Status by FDA
  2. Ultragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders
  3. Ultragenyx to Present Corporate Update at Analyst and Investor Day on April 17 in New York
  4. Global X Launches GNOM ETF to Tap Booming Genomics Space
  5. See what the IHS Markit Score report has to say about Ultragenyx Pharmaceutical Inc.
  6. Have Insiders Been Selling Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Shares?
  7. Ultragenyx Announces Approval of Crysvita® (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children
  8. New Research Coverage Highlights UnitedHealth Group, Ultragenyx Pharmaceutical, Navistar International, REGENXBIO, Par Pacific, and Athenex — Consolidated Revenues, Company Growth, and Expectations for 2019
  9. Why Is Ultragenyx (RARE) Up 12.4% Since Last Earnings Report?
  10. Ultragenyx to Present at Upcoming Investor Conferences
  11. Ultragenyx Announces Pricing of Public Offering of Common Stock
  12. Ultragenyx Announces Proposed Public Offering of Common Stock
  13. Sell-Side Picks Top Gene Therapy Takeout Candidates Following Spark Therapeutics Deal
  14. MarketPulse: Roche's Bumper Deal for Spark Therapeutics Boosts Healthcare Stocks
  15. Edited Transcript of RARE earnings conference call or presentation 19-Feb-19 10:00pm GMT
  16. Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
  17. Report: Exploring Fundamental Drivers Behind Canopy Growth, Adobe, Ultragenyx Pharmaceutical, Humana, Ship Finance International, and NexGen Energy — New Horizons, Emerging Trends, and Upcoming Developments
  18. Here's Why This Small Biotech Stock Just Hit A Four-Month High
  19. Ultragenyx (RARE) Posts Narrower-Than-Expected Loss in Q4