Ultragenyx Pharmaceutical Inc - Company Profile
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Ultragenyx Pharmaceutical Inc (Ultragenyx) is a biotechnology company that develops novel medicines for rare and ultra-rare genetic diseases. It has four 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); Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). 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
Website
www.ultragenyx.com
Telephone
1 415 4838800
No of Employees
1,276
Industry
Pharmaceuticals and Healthcare
Ticker Symbol & Exchange
RARE (NASD)
Revenue (2022)
$434.2M
19.5%
(2022 vs 2021)
EPS
XYZ
Net Income (2022)
XYZ
14.2%
(2022 vs 2021)
Market Cap*
$3.9B
Net Profit Margin (2022)
XYZ
28.3%
(2022 vs 2021)
* As of and is in US$
Ultragenyx Pharmaceutical Inc premium industry data and analytics
Products and Services
| Products | Brands |
|---|---|
| Crysvita - X-linked hypophosphatemia and Tumor-Induced Osteomalacia | Crysvita |
| Mepsevii - Mucopolysaccharidosis VII | Mepsevii |
| Dojolvi - Long Chain Fatty-Acid Oxidation Disorders | Dojolvi |
| XYZ | XYZ |
| XYZ | XYZ |
| XYZ | XYZ |
History
History section provides information on new products, mergers, acquisitions, expansions, approvals, and many more key events.
| Year | Event | Description |
|---|---|---|
| 2023 | Contracts/Agreements | In December, the company selected Andelyn Biosciences to execute late-stage process performance qualification manufacturing of the UX111 gene therapy. |
| 2023 | Corporate Changes/Expansions | In June, the company opened its gene therapy manufacturing facility in Bedford, Massachusetts, the US. |
| 2023 | Regulatory Approval | In May, the company received the U.S. Food and Drug Administration (FDA) to expand global phase 1/2 trial evaluating GTX-102 in patients with Angelman Syndrome in the U.S. |
Competitor Comparison
| Key Parameters | Ultragenyx Pharmaceutical Inc | Pfizer Inc | Sanofi | Amgen Inc | Moderna 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 | Cambridge |
| State/Province | California | New York | Ile-de-France | California | Massachusetts |
| No. of Employees | 1,276 | 88,000 | 87,994 | 26,700 | 5,600 |
| Entity Type | Public | Public | Public | Public | Public |
Key Financial Charts
Sales Growth
Net Income Growth
Executives
| Name | Position | Board | Since | Age |
|---|---|---|---|---|
| Daniel G. Welch | Chairman | Executive Board | 2015 | 65 |
| Emil D. Kakkis, M.D., Ph.D. | Chief Executive Officer; President; Director | Executive Board | 2010 | 63 |
| Howard Horn | Chief Financial Officer; Executive Vice President - Corporate Strategy | Senior Management | 2023 | - |
| Karah Parschauer | Executive Vice President; Chief Legal Officer; General Counsel | Senior Management | 2016 | 45 |
| Eric Crombez | Executive Vice President; Chief Medical Officer | Senior Management | 2023 | - |
| Non Dignissim Eros | Proin vel | Convallis | 2024 | XY |
| Non Dignissim Eros | Proin vel | Convallis | 2024 | XY |
| Non Dignissim Eros | Proin vel | Convallis | 2024 | XY |
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