Develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.
BioMarin Pharmaceutical Inc. specializes in developing and bringing to market innovative therapies aimed at addressing severe and life-threatening rare diseases and medical conditions. The company's portfolio includes a range of advanced treatments such as Vimizim for MPS IV type A, Naglazyme for MPS VI, and Kuvan for phenylketonuria (PKU). These therapies represent cutting-edge advancements in enzyme replacement and gene therapies, catering to patients with complex metabolic and lysosomal storage disorders.
In addition to its commercial products, BioMarin is actively advancing several promising candidates through clinical trials. This includes valoctocogene roxaparvovec, currently in Phase III trials for severe hemophilia A, and BMN 307, a gene therapy in Phase 1/2 trials targeting PKU. The company's research pipeline also features BMN 255, under Phase 1/2 investigation for treating primary hyperoxaluria, showcasing its commitment to addressing unmet medical needs across diverse genetic conditions.
BioMarin serves a global market, reaching patients through specialty pharmacies, hospitals, and distribution partners across the United States, Europe, Latin America, and beyond. The company operates under strategic license and collaboration agreements with leading biopharmaceutical entities, fostering synergies that enhance its research capabilities and market reach. Since its inception in 1996 and headquartered in San Rafael, California, BioMarin continues to pioneer advancements in biotechnology aimed at transforming the lives of patients with rare diseases worldwide.
With a steadfast focus on precision medicine and genetic therapies, BioMarin Pharmaceutical Inc. remains at the forefront of innovation in biopharmaceuticals. Its dedication to improving patient outcomes through groundbreaking treatments underscores its pivotal role in advancing the field of rare disease therapeutics, offering hope and tangible solutions to individuals and families facing challenging medical conditions.