Ultragenyx Pharmaceutical Inc - Company Profile
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Business Description
Ultragenyx Pharmaceutical Inc (Ultragenyx) identifies, acquires, develops and markets novel products for the treatment of various rare and ultra-rare genetic diseases.
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R&D Overview
Ultragenyx conducts research to advance its pipeline including biologics that include a monoclonal antibody and an enzyme replacement therapy; and small-molecule substrate replacement therapies for the treatment of various serious rare and ultra-rare genetic diseases with unmet medical needs. The company’s product pipeline has been either in-licensed from academic institutions or derived from partnerships with other pharmaceutical companies. In FY2022, the company spent US$702.6 million on R&D activities an increase of 41.4%. It has 705 employees working on its research and development.
Product Categories
Overview
It is a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). It is a subcutaneous injection that binds and reduces the biological activity of fibroblast growth factor 23 (FGF23). - It is approved in the US, the European Union and other regions for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older; and in Mexico and Brazil for the treatment of XLH in adult and pediatric patients one year of age and older. In the EU and the UK, Crysvita is conditionally approved for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents.
Performance
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Overview
Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride designed to produce medium-chain, odd-carbon fatty acids for patients with long-chain fatty acid oxidation disorders (LC-FAOD). - It is approved in the US by the FDA for the treatment of LC-FAOD, a set of rare metabolic diseases that prevent the conversion of fat into energy.
Performance
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Overview
It is the first product approved for the treatment of adults and children with mucopolysaccharidosis VII (MPS VII) (Sly syndrome). Mepsevii is an intravenous (IV) enzyme replacement therapy for the treatment of MPS VII. Deficiency of the lysosomal enzyme, beta-glucuronidase causes MPS VII. - Mepsevii is approved in the US for the treatment of children and adults. In the EU and the UK, it is approved under exceptional circumstances for the treatment of non-neurological manifestations of MPS VII in patients of all ages. In Brazil, it is approved for the treatment of patients of all ages.
Performance
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Other Break-up
Performance
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Performance
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Geographical Segments
Performance
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Performance
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Performance
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Performance
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