BioMarin Pharmaceutical Inc. engages in the development and commercialization of pharmaceuticals for serious diseases and medical conditions. Product Portfolio 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). The company is in the process of conducting clinical trials on several investigational product candidates for the treatment of various diseases, including: drisapersen, an exon-51 skipping compound for the potential treatment of Duchenne muscular dystrophy (DMD); pegvaliase (formerly referred to as PEG PAL), an enzyme substitution therapy for the treatment of phenylketonuria (PKU); reveglucosidase alfa (formerly referred to as BMN 701), an enzyme replacement therapy for Pompe disease, a glycogen storage disorder; talazoparib (formerly referred to as BMN 673), an orally available poly-ADP ribose polymerase (PARP) inhibitor for the treatment of patients with certain cancers; BMN 111, a peptide therapeutic for the treatment of achondroplasia, the cause of dwarfism; BMN 044, BMN 045 and BMN 053 for the treatment of DMD (exons 44, 45 and 53); and cerliponase alfa (formerly referred to as BMN 190) for the treatment of late infantile neuronal ceroid lipofuscinosis (CLN2), a lysomal storage disorder primarily affecting the brain. Commercial Products 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. Vimizim was granted marketing approval in the U.S. and the EU in February 2014 and April 2014, respectively, and subsequently in other countries. Naglazyme Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with mucopolysaccharidosis VI (MPS VI). MPS 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 (GAGs). 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. 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 phenylketonuria (PKU). In 2013, the U.S. Food and Drug Administration (FDA) approved the use of Kuvan powder for oral solution which would be provided in a dose sachet packet allowing faster dissolution of powder in solution compared to the tablet form. The company commenced the commercial launch of this new form of Kuvan in 2014. 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 market exclusivity in the U.S. for the treatment of PKU, expiring in June 2015. Aldurazyme Aldurazyme has been approved for marketing in the U.S., the EU and 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 currently exists, that is caused by the deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of GAGs. The company developed Aldurazyme through collaboration with Genzyme Corporation (Genzyme), a wholly-owned subsidiary of Sanofi. Firdapse Firdapse is a form of 3,4-diaminopyridine (amifampridine phosphate or 3,4-DAP) for the treatment of Lambert Myasthenic Syndrome (LEMS). Firdapse was granted marketing approval in the European Union (EU) in 2009. In addition, Firdapse has been granted orphan drug status in the EU, which confers 10 years of market exclusivity in the EU. The company launched Firdapse on a country-by-country basis in Europe in 2010. Customers The company’s Vimizim, Naglazyme, Kuvan and Firdapse customers include specialty pharmacies and end-users, such as hospitals and foreign government agencies. The company also sells Vimizim and Naglazyme to authorized European distributors and to certain larger pharmaceutical wholesalers, which act as intermediaries between the company and end-users and do not stock significant quantities of Naglazyme and Vimizim. Genzyme is a sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third-parties. In certain countries particularly in Latin America, governments place large periodic orders for Vimizim and Naglazyme. Intellectual Property The number of the company’s worldwide issued patents stands at approximately 500, including approximately 64 patents issued by the U.S. Patent and Trademark Office (the USPTO). Furthermore, its portfolio of pending patent applications totals approximately 458 applications, including approximately 78 pending U.S. applications. Government Regulation The company’s products require approval from the Food and Drug Administration, the European Medicines Agency, and corresponding agencies in other countries before they could be marketed. History BioMarin Pharmaceutical Inc. was founded in 1996. The company was incorporated in Delaware in 1996 and began its operations in 1997.
biomarin pharmaceutical inc
770 Lindaro Street
San Rafael, CA 94901
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