BioMarin Pharmaceutical Inc. (BMRN) is a U.S.-based biotechnology company specialising in treatments for rare genetic and metabolic disorders.

Founded in 1997, it has built a portfolio of eight approved therapies, including groundbreaking enzyme replacement and gene therapies such as VOXZOGO for achondroplasia and ROCTAVIAN for severe haemophilia A, the first gene therapy approved for this condition. The company’s pipeline continues to expand, targeting additional growth disorders, muscular dystrophy, and liver diseases. BioMarin’s innovation-driven model focuses on addressing unmet medical needs with high barriers to entry, translating genetic research into life-changing therapies.

Revenues have grown steadily over the past five years, supported by global expansion and new product launches; however, they are currently down 21% year to date, following slower uptake in select markets. Given its strong R&D capabilities, diversified product base, and long-term growth drivers, we see potential undervaluation in the stock and meaningful upside as the market reappraises BioMarin’s earnings trajectory and pipeline progress.