BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) 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 Vimizim™ (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia.
|Total Revenue:||$500.7 Million|
|Clinical Research:||Conducted internationally|
|Products:||Naglazyme® for MPS VI, Aldurazyme® for MPS I, Firdapse™ (currently approved in the EU only) for LEMS, KUVAN® Tablets for PKU|
|Commercial Operations:||Over 40 countries|
|Clinical Pipeline:||Vimizim™ (GALNS) for Morquio A Syndrome (MPS IVA), PEG-PAL for PKU, BMN-701 for Pompe disease, BMN-673 for genetically-defined cancers, BMN-111 for achondroplasia|
|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|
BioMarin has developed and commercialized four 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 a boxed warning for Aldurazyme,
please visit www.kuvan.com,