BioMarin's Q4 2024: Contradictions in Voxzogo Growth, BMN 333 Development, and Strategic Focus on CNP

These are the key contradictions discussed in BioMarin Pharmaceutical Inc.'s latest 2024Q4 earnings call, specifically including: Voxzogo's patient growth expectations and strategy, the development timeline for BMN 333, and the strategic focus on CNP:



Record Revenue and Profitability Growth:
- BioMarin Pharmaceuticals reported total revenues of $2.85 billion for 2024, with a 18% year-over-year increase, and an non-GAAP operating margin expansion of 900 basis points.
- The growth was driven by the company's strategic initiatives including the prioritization of pipeline candidates, reorganization of the operating model, and the ongoing $500 million cost transformation program.

VOXZOGO Expansion and Market Penetration:
- VOXZOGO's full-year revenue reached $735 million, growing 56% year-over-year, with significant contributions from new patient starts in infants and young children.
- Expansion in the United States, particularly in the 0-5 age cohort, and continued demand globally, particularly in markets like Germany, contributed to the growth.

Enzyme Therapy Performance:
- The enzyme therapy business surpassed $1.9 billion in full-year revenue, representing a 12% increase over the previous year.
- Growth was supported by expanding patient uptake in the U.S. and Japan for PALYNZIQ and ongoing demand for other enzyme replacement products.

Pipeline Advancements:
- BioMarin's BMN 351 and BMN 333 are progressing, with early clinical data expected later in the year, and potential approvals anticipated in 2027 for BMN 351 and VOXZOGO for hypochondroplasia.
- Advancements are driven by strategic prioritization of promising pipeline candidates and successful enrollment in clinical trials.