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Ultragenyx Pharmaceutical Inc: Overview

Ultragenyx Pharmaceutical Inc (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

Headquarters United States of America

Address 60 Leveroni Ct, Novato, California, 94949-5746


Telephone 1 415 4838800

No of Employees 1,119

Industry Pharmaceuticals and Healthcare

Ticker Symbol & Exchange RARE (NASD)

Revenue (2020) $351.4M 29.7% (2020 vs 2019)

EPS XXX

Net Income (2020) XXX -143.4% (2020 vs 2019)

Market Cap* $3.7B

Net Profit Margin (2020) XXX -87.7% (2020 vs 2019)

* As of and is in US$

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Clinical Trials

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Catalyst Calendar

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Lead Sheet

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Pipeline Drugs

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Sales & Consensus Forecasts

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Regulatory Milestones

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Marketed Drugs

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Products and Services

Products Brands
Crysvita - X-linked hypophosphatemia and Tumor-Induced Osteomalacia Ultragenyx
Mepsevii - Mucopolysaccharidosis VII Crysvita
Dojolvi - Long Chain Fatty-Acid Oxidation Disorders Mepsevii
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History

History section provides information on new products, mergers, acquisitions, expansions, approvals, and many more key events.

Year Event Description
2022 Acquisitions/Mergers/Takeovers In July, the company acquired GeneTx BioTherapeutics for US$75 million.
2022 Stake Sale In July, the company announced the sale of 30% royalty interest from Kyowa Kirin Co., Ltd on the future sales of Crysvita (burosumab) in the U.S. and Canada to OMERS.
2022 Acquisitions/Mergers/Takeovers In July, the company acquired marketing rights of Mepsevii in Japan from Amicus Therapeutics.
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Competitor Comparison

Key Parameters Ultragenyx Pharmaceutical Inc Pfizer Inc Sanofi Amgen Inc Acer Therapeutics Inc
Headquarters United States of America United States of America France United States of America United States of America
City Novato New York Paris Thousand Oaks Newton
State/Province California New York - California Massachusetts
No. of Employees 1,119 79,000 95,442 24,200 35
Entity Type Public Public Public Public Public
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Executives
Name Position Board Since Age
Daniel G. Welch Chairman Executive Board 2015 64
Emil D. Kakkis, M.D., Ph.D. Director; President; Chief Executive Officer Executive Board 2010 61
Mardi C. Dier Chief Financial Officer; Executive Vice President Senior Management 2020 58
Karah Parschauer Executive Vice President; General Counsel; Chief Legal Officer Senior Management 2016 44
Camille L. Bedrosian, M.D. Chief Medical Officer; Executive Vice President Senior Management 2018 69
Non Dignissim Eros Proin vel Convallis 2022 XX
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