BioMarin Pharmaceutical Inc. engages in the development and commercialization of pharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Its approved products are Vimizim (elosulfase alpha), Naglazyme (galsulfase), Kuvan (sapropterin dihydrochloride), Aldurazyme (laronidase) and Firdapse (amifampridine phosphate). Vimizim is an enzyme replacement therapy for the treatment of mucopolysaccharidosis IV Type A (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. It has identified approximately 1,800 patients worldwide suffering from MPS IV A and estimate that the total number of patients suffering from MPS IV A worldwide could be as many as 3,000. Vimizim was granted marketing approval in the United States (the U.S.) and the European Union (the EU) in February 2014 and April 2014, respectively, and subsequently in various other countries. The company markets Vimizim in the U.S., the E.U., and other areas using its own existing sales force and commercial organization. Additionally, the company uses local distributors in various other regions to help it pursue registration and/or market Vimizim on a named patient basis. Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with mucopolysaccharidosis VI. Mucopolysaccharidosis VI is a debilitating life-threatening genetic disease for which no other drug treatment exists and is caused by the deficiency of arylsulfatase B, an enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans. 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. Naglazyme was granted marketing approval in the U.S. in 2005, in the EU in 2006, and subsequently in other countries. 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, the company uses local distributors in various other regions to help it pursue registration and/or market Naglazyme on a named patient basis. Kuvan is a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase, indicated for patients with phenylketonuria (PKU). Kuvan is the drug for the treatment of PKU, which is an inherited metabolic disease that affects approximately 50,000 diagnosed patients under the age of 40 in the developed world. In 2005, the company entered into the license agreement with PKU Franchise from Ares Trading S.A. (Merck Serono) for the further development and commercialization of Kuvan and any other product containing 6R-BH4, and pegvaliase for PKU. Aldurazyme was approved for marketing in the U.S., and the EU in 2003, and subsequently in other countries for patients with mucopolysaccharidosis I (MPS I). MPS I is a progressive and debilitating life-threatening genetic disease, for which no other drug treatment exists, that is caused by the deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of glycosaminoglycans. The company developed Aldurazyme through collaboration with Genzyme Corporation (Genzyme), a wholly-owned subsidiary of Sanofi. Under its collaboration agreement with Genzyme, the company is responsible for manufacturing Aldurazyme and supplying it to Genzyme. Firdapse is a form of 3,4-diaminopyridine (amifampridine phosphate or 3,4-DAP) for the treatment of Lambert Myasthenic Syndrome (LEMS). Firdapse was originally developed by AGEPS, the pharmaceutical unit of the Paris Public Hospital Authority.
biomarin pharmaceutical inc
770 Lindaro Street
San Rafael, CA 94901
The information and data displayed in this profile are created and managed by S&P Global Market Intelligence, a division of S&P Global. Bloomberg.com does not create or control the content.
|No competitor information is available for BM8.|
|View Industry Companies|
|Price/Cash Flow||NM||Not Meaningful|
Sponsored Financial Commentaries
To contact BIOMARIN PHARMACEUTICAL INC, please visit www.bmrn.com. Company data is provided by S&P Global Market Intelligence. Please use this form to report any data issues.