美國BioMarin Pharmaceutical
美國生物制藥公司BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions, focusing on product candidates that:
Address currently unmet medical needs
Suggest a clear-cut development profile
Provide an opportunity to be first-to-market
The company's product portfolio is comprised of three approved products and multiple investigational product candidates. FDA-approved products include Kuvan? (sapropterin dihydrochloride) Tablets for PKU, an oral small molecule therapeutic developed in partnership with Merck Serono; Naglazyme? (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; and Aldurazyme? (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include PEG-PAL for the treatment of phenylketonuria (PKU), and 6R-BH4, currently in Phase 2 clinical development for the treatment of peripheral arterial disease.