BioMarin Pharmaceutical Inc (BMRN) is aiming high, setting its sights on reaching $4 billion in revenue by 2027. The company is making bold moves to achieve this ambitious goal, including a focus on expanding existing products and launching new therapies. To ensure profitability, BioMarin is targeting an adjusted operating margin of between 40% and 45% starting in 2026. This plan is built around three key business units: skeletal conditions, enzyme therapies, and Roctavian, its innovative hemophilia A gene therapy.
To support this growth strategy, BioMarin has implemented a comprehensive $500 million “cost transformation program.” This initiative is crucial for achieving the non-GAAP operating margin targets set for 2026 and beyond.
One of BioMarin’s key growth drivers is Voxzogo, a treatment for achondroplasia, a genetic condition causing dwarfism. Voxzogo sales reached a significant $470 million in 2023, and BioMarin believes it has the potential to become a $5 billion opportunity with a global patient population of approximately 420,000. The company is actively exploring five experimental programs for Voxzogo beyond achondroplasia, aiming to expand its reach and impact even further. Phase 3 data readouts for a related treatment for hypochondroplasia, another form of dwarfism, are anticipated in 2026, with a potential launch in 2027.
BioMarin is also actively developing its pipeline, with plans to launch two new products outside its currently approved medicines by 2027, and a total of 11 by 2034. These pipeline programs address critical diseases such as Duchenne muscular dystrophy, other forms of dwarfism, and von Willebrand disease.
In a recent strategic shift, BioMarin has narrowed the focus of its Roctavian business, concentrating commercial operations on the U.S., Germany, and Italy. These are the markets where Roctavian is approved and reimbursed as a treatment for severe hemophilia A. This adjustment will lead to reduced investments in development and manufacturing, with annual direct Roctavian expenses anticipated to decline to approximately $60 million beginning in 2025.
Adding to its growth momentum, BioMarin received FDA approval in July for its Brineura (cerliponase alfa) treatment. Brineura is now indicated to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Previously, Brineura was only approved for symptomatic children three years of age and older with late infantile CLN2 disease. This expanded indication now extends to children of all ages with CLN2 disease, regardless of their symptom status.
While BioMarin’s pipeline holds significant promise, William Blair analysts point out that only three programs are projected to contribute to the company’s top line within the next three years. These include label expansion of Palynziq into adolescents and Voxzogo into HCH. Despite this, William Blair remains optimistic about BioMarin’s future, believing that its commercial execution and differentiation from competitors will drive the stock in the near term.
BMRN stock closed at $89.86 on Wednesday, reflecting the market’s response to BioMarin’s ambitious growth plan and strong product pipeline.
