BioMarin Pharmaceutical Inc - Company Profile
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Business Description
BioMarin Pharmaceutical Inc (BioMarin) develops products for unmet medical needs. The company operates through single operating segment focused on the development and commercialization of innovative therapies for people with serious and lifethreatening rare diseases and medical conditions. The company has various approved products which include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Kuvan (sapropterin dihydrochloride), Voxzogo (vosoritide), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec) ,Palynziq and Brineura (cerliponase alfa).
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R&D Overview
BioMarin's pipeline products, if commercialized successfully, could expand its product portfolio and increase its revenue. Its major products under development include Vosoritide in Phase III clinical trial for the treatment of hypochondroplasia and in Phase II trial for the treatment of idiopathic short stature; Pegvaliase in Phase III trial for the treatment of phenylketonuria; BMN 351 in Phase II clinical trial for the treatment of duchenne muscular dystrophy; BMN 293 in Phase I trial for the treatment of MYBPC3 hypertrophic cardiomyopathy; BMN 349 in Phase II trial for the treatment of alpha-1 antitrypsin deficiency; and BMN 333 in pre-clinical stage for multiple growth-related conditions. In FY2023, the company spent US$746.8 million on its R&D, which as a percentage of revenue stood at 30.8%.
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