BioMarin Pharmaceutical

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) is a global biotechnology company focused on developing and commercializing innovative therapies for rare genetic diseases. Founded in 1997 and headquartered in San Rafael, California, BioMarin has successfully brought eight products to market, primarily for ultra-rare conditions.

BioMarin's product portfolio includes several enzyme replacement therapies and other treatments for rare genetic disorders. Key products include Vimizim for mucopolysaccharidosis IV type A, Naglazyme for mucopolysaccharidosis VI, Brineura for ceroid lipofuscinosis type 2, and Voxzogo for achondroplasia. The company also markets Kuvan and Palynziq for phenylketonuria (PKU). In 2022, BioMarin received European approval for Roctavian, a gene therapy for severe hemophilia A, followed by US approval in 2023.

In recent developments, BioMarin reported strong financial results for 2023, with total revenues of USD 2.42 billion, a 15% increase year-over-year. This growth was driven largely by Voxzogo, which generated USD 470 million in net revenues, a 178% increase from the previous year. As of December 2023, over 2,600 children were being treated with Voxzogo across 41 markets. The company is also expanding Voxzogo's potential by initiating clinical programs for hypochondroplasia and exploring its use in other short stature conditions. For 2024, BioMarin anticipates continued double-digit revenue growth and margin expansion as it focuses on maximizing commercial opportunities and optimizing its R&D pipeline.