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Company Overview

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.

The company's product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Approved products include Vimizim® (elosulfase alfa) for MPS IVA, a product wholly developed and commercialized by BioMarin; Naglazyme® (galsulfase) for MPS VI, a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution and Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include drisapersen, an exon skipping oligonucleotide, for which a marketing application has been submitted to FDA and EMA for the treatment of patients with Duchenne muscular dystrophy (DMD) with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping, pegvaliase (PEGylated recombinant phenylalanine ammonia lyase, formerly referred to as BMN 165 or PEG PAL), which is currently in Phase 3 clinical development for the treatment of PKU, talazoparib (formerly referred to as BMN 673), a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, reveglucosidase alfa (formerly referred to as BMN 701), a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase 3 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, BMN 044, BMN 045 and BMN 053, exon skipping oligonucleotides, which are currently in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (exons 44, 45 and 53), cerliponase alfa (formerly referred to as BMN 190), a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of CLN2 disorder, a form of Batten disease, which is currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the treatment of hemophilia A and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB.

2014 Highlights

Total Revenue: $751 Million
Employees: >1900 worldwide
Clinical Research: Conducted internationally
Products: Vimizim® (elosulfase alfa) for MPS IVA, Naglazyme® (galsulfase) for MPS VI, Aldurazyme® (laronidase) for MPS I, Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution and Tablets for phenylketonuria (PKU), and Firdapse® (amifampridine) for Lambert Eaton Myasthenic Syndrome (LEMS) [approved in the EU only]
Commercial Operations: Over 50 countries
Focus: Rare (Orphan) genetic diseases
Strategy: Providing first-in-class or best-in-class treatments for patients with serious unmet medical needs, optimizing powerful biology with demonstrated potential and development clarity, accelerating approval process, strategic pipeline development

Products

BioMarin has developed and commercialized five products since 1997, a remarkable accomplishment in the biopharmaceutical industry, and a testament to the company's passion and dedication to patients with serious unmet medical needs. For complete safety and prescribing information, including boxed warnings for Aldurazyme and Vimizim, please visit www.kuvan.com, www.naglazyme.com, www.aldurazyme.com and www.vimizim.com .