The company is conducting clinical trials on various investigational product candidates for the treatment of various diseases, including Kyndrisa (drisapersen), an exon-51 skipping compound for the potential treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping; pegvaliase, an enzyme substitution therapy for the treatment of PKU; reveglucosidase alfa, an enzyme replacement therapy for Pompe disease, a glycogen storage disorder; vosoritide, a peptide therapeutic for the treatment of achondroplasia, the main cause of dwarfism; BMN 044, BMN 045 and BMN 053 for the treatment of DMD (exons 44, 45 and 53); cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis, a lysomal storage disorder primarily affecting the brain; and BMN 270, an AAV VIII vector and Factor VIII gene therapy drug development candidate, for the treatment of hemophilia A. Pegvaliase is an investigational enzyme substitution therapy that the company is developing as a subcutaneous injection for the treatment of PKU. Reveglucosidase alfa is a fusion protein of acid alpha glucosidase with a peptide derived from insulin-like growth factor 2. The company is conducting or planning to conduct preclinical development of various other product candidates for genetic and other metabolic diseases, including a fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2, for the treatment of Sanfilippo B syndrome, or mucopolysaccharidosis type IIIB. Research and Development For the year ended December 31, 2015, the company’s research and development expense included $634.8 million. Acquisitions In 2015, the company completed the acquisition of Prosensa Holding N.V., a public limited liability company. Sale of Talazoparib to Medivation, Inc. In 2015, the company completed the sale of talazoparib, an orally available poly-ADP ribose polymerase inhibitor for the treatment of patients with certain cancers, to Medivation, Inc. (Medivation), under which Medivation acquired the worldwide rights to talazoparib. Sales and Marketing The company has established a commercial organization, including a sales force, to support its product lines directly in the U.S., Europe, South America and certain other significant markets. For other selected markets, it has signed agreements with other companies to act as distributors of Vimizim, Naglazyme and Kuvan. Most of these agreements generally grant the distributor the right to market the product in the territory and the obligation to secure all necessary regulatory approvals for commercial or named patient sales. In the U.S., the company’s products are marketed through its commercial teams, including sales representatives and supporting staff members, who promote Vimizim, Naglazyme and Kuvan, directly to physicians in specialties appropriate for each product. Outside of the U.S., its sales representatives and supporting staff members market Vimizim, Naglazyme, Kuvan and Firdapse. Genzyme has the exclusive right to distribute, market and sell Aldurazyme globally and is required to purchase its requirements exclusively from the company. Customers The company’s Vimizim, Naglazyme, Kuvan and Firdapse customers include a limited number of specialty pharmacies and end-users, such as hospitals and foreign government agencies. It also sells Vimizim and Naglazyme to its authorized distributors and to certain larger pharmaceutical wholesalers globally, which act as intermediaries between it and end-users and generally do not stock significant quantities of its products. During 2015, 42% of its net Vimizim, Naglazyme, Kuvan and Firdapse product revenues were generated by three customers. Genzyme is its sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third-parties. Patents and Proprietary Rights As of January 25, 2016, the number of the company’s worldwide issued patents stands at 633, including 82 patents issued by the U.S. Patent and Trademark Office (the USPTO). Furthermore, its portfolio of pending patent applications totals 407 applications, including 86 pending U.S. app
biomarin pharmaceutical inc
770 Lindaro Street
San Rafael, CA 94901
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