Last €75.42 EUR
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Volume 2.0
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As of 2:16 AM 12/29/14 All times are local (Market data is delayed by at least 15 minutes).
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Company Description

Contact Info

770 Lindaro Street

San Rafael, CA 94901

United States

Phone: 415-506-6700

Fax: 415-382-7889

BioMarin Pharmaceutical Inc. develops and commercializes pharmaceuticals for serious diseases and medical conditions. The company selects product candidates for diseases and conditions that represent an unmet medical need and provide an opportunity to be first-to-market or offer a benefit over existing products. Its product portfolio includes five approved products and multiple investigational product candidates. Approved products include Naglazyme (galsulfase), Kuvan (sapropterin dihydrochloride), Aldurazyme (laronidase), Firdapse (amifampridine phosphate), and VIMIZIM (elosulfase alpha). Commercial Products Naglazyme Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with mucopolysaccharidosis VI (MPS VI). Patients with MPS VI become progressively worse and experience multiple severe and debilitating symptoms resulting from the build-up of carbohydrate residues in tissues in the body. These symptoms include inhibited growth, spinal cord compression, enlarged liver and spleen, joint deformities and reduced range of motion, skeletal deformities, impaired cardiovascular function, upper airway obstruction, reduced pulmonary function, frequent ear and lung infections, impaired hearing and vision, sleep apnea, malaise and reduced endurance. The company markets Naglazyme in the U.S., the EU, Canada, Latin America, Turkey, and other areas using its own sales force and commercial organization. Additionally, it uses local distributors in various other regions to help the company pursue registration and/or market Naglazyme on a named patient basis. Kuvan Kuvan is a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase (PAH), indicated for patients with PKU. Kuvan is granted marketing approval for the treatment of PKU in the U.S. The company markets Kuvan in the U.S. and Canada using its own sales force and commercial organization. Kuvan has been granted orphan drug status in the U.S., which confers seven years of market exclusivity in the U.S. for the treatment of PKU, expiring in December 2014. The company sells Kuvan to Merck Serono S.A. and Merck Serono S.A. resells the product to end-users outside the U.S., Canada, and Japan. Aldurazyme Aldurazyme has been approved for marketing in the U.S., the EU, and other countries for patients with mucopolysaccharidosis I (MPS I). Patients with MPS I become progressively worse and experience multiple severe and debilitating symptoms resulting from the build-up of carbohydrate residues in all tissues in the body. These symptoms include inhibited growth, delayed and regressed mental development (in the severe form of the disease), enlarged liver and spleen, joint deformities and reduced range of motion, impaired cardiovascular function, upper airway obstruction, reduced pulmonary function, frequent ear and lung infections, impaired hearing and vision, sleep apnea, malaise and reduced endurance. The company developed Aldurazyme through collaboration with Genzyme Corporation, a wholly-owned subsidiary of Sanofi. Under its collaboration agreement, the company is responsible for manufacturing Aldurazyme and supplying it to Genzyme Corporation. Firdapse Firdapse is a form of 3, 4-diaminopyridine (amifampridine phosphate or 3, 4-DAP) for the treatment of Lambert Myasthenic Syndrome (LEMS). Firdapse is granted marketing approval in the EU. In addition, Firdapse has been granted orphan drug status in the EU. The company licensed to Catalyst Pharmaceutical Partners, Inc. the North American rights to develop and market Firdapse. VIMIZIM VIMIZIM is an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder. MPS IV A is a disease characterized by deficient activity of Nacetylgalactosamine- 6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans, such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Other symptoms might include hearing loss, corneal clouding, and he

 

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TEV/Sales 18.3x
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