| BioMarin Pharmaceuticals, Inc. | (NQ: BMRN) |
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May 18, 2013
BioMarin Pharmaceutical Inc. (BMRN), a developer of enzyme-related therapies (ERT), co-markets Aldurazyme with Genzyme Corporation (GENZ) for the treatment of MPS-I (mucopolysaccharidosis). MPS-I is a rare genetic enzyme deficiency disorder, afflicting roughly 3,000 people worldwide, caused by the lack of a specific enzyme involved in lysosomal storage. More common names for mucopolysaccharidosis are Hurler's Syndrome, Hurler-Scheie, and Scheie's Disease. The company's other developed product, Naglazyme, for MPS-VI, recently entered the market. MPS-VI is also a rare genetic enzyme deficiency disorder, with an estimated 1,100 people suffering from it worldwide. The company's third lead drug, Kuvan, received FDA approval for the treatment of PKU in December 2007. With the recent launch of Kuvan, the company now has three products on the market. The company also receives royalties and license revenue on sales of the Orapred franchise from Alliant Pharmaceuticals. In 2004, the company acquired Orapred, a grape-flavored oral corticosteroid liquid for the treatment of severe asthma in children and subsequently licensed the franchise to Alliant Pharmaceuticals in March 2006. BioMarin, headquartered in Novato, CA, employs roughly 300 people.
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