Core Insights - BioMarin Pharmaceutical is targeting a preliminary revenue of $3.2 billion for 2025, with Voxzogo expected to contribute $920 million, reflecting a strong performance and a projected 27% year-over-year growth in Q4 [3][6] - The company is focusing on expanding Voxzogo's market presence and enhancing its enzyme therapies portfolio, alongside the strategic acquisition of Amicus, valued at $4.8 billion, which is expected to diversify and accelerate revenue growth [7][8] Revenue and Growth Projections - BioMarin anticipates Voxzogo's growth in achondroplasia will be supported by new country launches and deeper market penetration, particularly emphasizing early treatment decisions for infants [2][6] - The acquisition of Amicus is expected to be accretive within 12 months post-close and substantially accretive starting in 2027, with confidence in outperforming previous growth expectations for enzyme therapies [8][7] Strategic Focus and Pipeline Development - The company's strategy is built on three pillars: innovation, growth, and a value commitment, prioritizing pipeline programs with transformative potential, including BMN 333 and BMN 351 [5][6] - BioMarin plans to accelerate revenue through geographic expansion of Voxzogo and potential label expansions for other therapies, including Palynziq [4][6] Clinical and Regulatory Catalysts - BioMarin is preparing to file for full approval of Voxzogo with the FDA, leveraging extensive safety data and additional efficacy endpoints [11] - Upcoming regulatory actions include an FDA PDUFA date for Palynziq's adolescent label expansion and significant data readouts for BMN 351 and BMN 333 [12][10][13] Competitive Dynamics and Intellectual Property Strategy - The company is committed to defending its intellectual property for Voxzogo, including seeking orphan drug exclusivity and engaging in ongoing litigation to protect its market position [15][16] - BioMarin views BMN 333 as a potential long-term game changer for achondroplasia, based on its promising exposure profile [16]