BioMarin Pharmaceutical is making strategic moves to address the expected decline in revenue from its achondroplasia drug, Voxzogo. The company recently acquired Amicus Therapeutics for $4.9 billion, seeking to diversify its rare disease portfolio as competition intensifies in the achondroplasia market.
Voxzogo has been a significant source of income for BioMarin, accounting for over one-quarter of the company’s revenue in 2024. However, several other companies are developing alternatives that could challenge Voxzogo’s market position with improved dosing schedules and potentially higher efficacy.
According to Mitchell Kapoor, a senior analyst at HC Wainwright, BioMarin’s stock performance is closely tied to Voxzogo: “shares of the company do ‘trade on Voxzogo, mostly, not on earnings beats.’” He noted that while BioMarin is not solely dependent on one product for revenue, investors remain focused on this particular drug.
The acquisition of Amicus brings two commercial therapies into BioMarin's portfolio: Galafold, an oral treatment for Fabry disease, and Pombiliti/Opfolda, a combination therapy for Pompe disease. These drugs generated $599 million in combined sales over the four quarters prior to the deal. Amicus projected that each could reach up to $1 billion in annual sales by the 2030s.
BioMarin set a $4 billion annual revenue goal in September 2024 but later withdrew it due to increasing competition against Voxzogo from companies like Ascendis Pharma and BridgeBio. Wall Street analysts expect Voxzogo sales to remain steady into the next decade; however, Kapoor believes these sales will likely stall soon.
BioMarin did not provide direct comments but referred inquiries to CEO Alexander Hardy's remarks at the 2026 J.P. Morgan Healthcare Conference: Galafold and Pombiliti/Opfolda “just drop perfectly into our enzyme therapy business,” he said. Hardy also stated: “Adding Amicus further strengthens that growth outlook and [diversifies] our revenue base.”
Looking ahead, BioMarin is developing BMN333—a next-generation injectable achondroplasia drug—but only Phase 1 data are available so far. Kapoor described BMN333 as a “prove-it story,” suggesting more evidence is needed before it can be considered a major driver of growth or investor interest.
Since its approval in 2021 by the FDA after showing increased bone growth in children with achondroplasia during Phase 3 trials, Voxzogo has faced new rivals offering alternative treatments. BridgeBio’s oral drug infigratinib demonstrated similar efficacy with average annualized growth rates above 2.5 cm over both 12- and 18-month periods during Phase 2 trials conducted in June 2024.
Ascendis Pharma’s TransCon CNP—an injectable administered once per week rather than daily—is awaiting an FDA decision by February 28 after showing greater effectiveness than placebo (5.89 cm/year versus 4.41 cm/year) according to September data from last year.
Tyra Biosciences is also entering the field with dabogratinib—an oral candidate currently undergoing Phase 2 testing—which may become notable if trial results show annualized growth above seven centimeters within six months.
Kapoor summarized BioMarin's situation: “[BioMarin has] acknowledged there are headwinds… Then it’s like, What’s next?”
