BioMarin Pharmaceutical Inc (NASDAQ:BMRN) plans to land $4 billion in revenue by 2027.

The company is targeting an adjusted operating margin of low-to-mid 40%'s (starting at 40% in 2026).

The new structure, announced at an investor event Wednesday, covers three key business units. They include skeletal conditions, enzyme therapies, and Roctavian — its hemophilia A gene therapy.

BioMarin has been implementing a $500 million "cost transformation program" to contribute to non-GAAP operating margin targets set for 2026 and beyond.

CEO Alexander Hardy highlighted five experimental programs through 2031 for Voxzogo beyond achondroplasia, a type of genetic condition that causes dwarfism.

Voxzogo sales reached $470 million in 2023. According to BioMarin, the drug could prove a $5 billion opportunity with a total addressable patient population of around 420,000 across its global markets.

The next most advanced Voxzogo program is in hypochondroplasia, another form of dwarfism. Phase 3 data readouts are expected in 2026, and a targeted launch is expected in 2027.

BioMarin plans to launch two products outside its approved medicines by 2027 or 11 by 2034. The company's pipeline includes programs for diseases such as Duchenne muscular dystrophy, other forms of dwarfism, and von Willebrand disease.

Recently, BioMarin updated its Roctavian business to focus commercial operations on three markets: the U.S., Germany, and Italy.

These markets are where the medicine is approved and reimbursed as a severe hemophilia A treatment.

The company will adjust its Roctavian efforts and reduce additional investments in development and manufacturing.

BioMarin anticipates reducing annual direct Roctavian expenses to approximately $60 million beginning in 2025.

In July, the FDA approved BioMarin's Brineura (cerliponase alfa) 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 indicated in symptomatic children three years of age and older with late infantile CLN2 disease. This expanded indication now includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or presymptomatic.

William Blair says, "However, BioMarin has a rich pipeline, only three programs could potentially contribute to its top line in the next three years, which includes label expansion of Palynziqinto adolescents and Voxzogo into HCH. Therefore, we think commercial execution and continued differentiation from competitors will continue to drive the stock in the near term."

Price Action: BMRN stock closed at $89.86 on Wednesday.

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