• Total Revenue: Full-year 2024 revenue grew 18% to $2.85 billion; Q4 2024 revenue increased 16% year over year to $747 million.
• VOXZOGO Revenue: Full-year 2024 revenue of $735 million, with 56% year-over-year growth; Q4 2024 revenue of $208 million.
• Enzyme Therapy Revenue: Full-year 2024 revenue over $1.9 billion, a 12% increase from 2023.
• Non-GAAP Operating Margin: Full-year 2024 margin expanded by 9.2 percentage points to 28.6%; Q4 2024 margin at 31.1%.
• Non-GAAP Diluted EPS: Full-year 2024 EPS increased 69% to $3.52; Q4 2024 EPS increased 88% to $0.92.
• Operating Cash Flow: Full-year 2024 operating cash flow of $573 million, a 260% increase over 2023.
• 2025 Revenue Outlook: Expected total revenue between $3.1 billion and $3.2 billion, representing 10% growth.
• 2025 Non-GAAP Operating Margin Guidance: Expected between 32% and 33%.
• 2025 Non-GAAP Diluted EPS Guidance: Expected between $4.20 and $4.40 per share.

• Warning! GuruFocus has detected 2 Warning Sign with BMRN.

Release Date: February 19, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Biomarin Pharmaceutical Inc (NASDAQ:BMRN) reported a record growth and profitability in 2024, with a topline growth of 18% year over year.
• The company achieved a significant expansion in non-GAAP operating margin by over 900 basis points and a 69% increase in non-GAAP diluted earnings per share.
• VOXZOGO for achondroplasia showed impressive growth of 56% year over year, with plans to expand into hypochondroplasia and other skeletal conditions.
• The enzyme therapy business grew by 9% in the fourth quarter, nearing $2 billion in annual revenues, supporting a high single-digit CAGR outlook.
• Biomarin's strategic pipeline prioritization led to advancements in promising candidates like BMN 351 for Duchenne Muscular Dystrophy and BMN 333, with early clinical data expected later this year.

Negative Points

• Despite strong growth, VOXZOGO's growth rate is expected to decelerate due to the law of large numbers as the revenue base increases.
• The enzyme therapy business experienced unusual high contributions from ALDURAZYME in 2024, which may not be sustainable in 2025.
• Non-GAAP SG&A expenses increased due to a bad debt reserve and higher commercialization expenses, impacting overall profitability.
• There is a potential risk of competition in the achondroplasia treatment market, which could affect VOXZOGO's market share.
• The company faces challenges in expanding patient access and awareness, particularly in the US, where a significant portion of eligible patients remain untreated.

Q & A Highlights

Q: Can you elaborate on the expected growth for VOXZOGO and the factors influencing its guidance? A: Brian Mueller, CFO, explained that while VOXZOGO's growth is expected to be around $200 million year-over-year, the growth rate is decelerating due to the law of large numbers as the revenue base increases. Cristin Hubbard, Chief Commercial Officer, added that growth is anticipated from the U.S., Germany, and Brazil, with efforts to expand into over 60 geographies by 2027. The focus is on increasing prescriber base and awareness, especially in the U.S. where the 0 to 5 age cohort is a significant growth driver.

Q: What are BioMarin's priorities for business development, and is there a focus on pipeline versus commercial assets? A: Alexander Hardy, CEO, stated that BioMarin is interested in both pre-clinical and clinical assets, focusing on genetically defined conditions and leveraging their global footprint. The company is looking to strengthen its growth outlook and had numerous meetings at JPMorgan, reflecting interest in BioMarin's capabilities.

Q: Could you discuss the durability and safety of the growth hormone and CNP combo, and what are you looking for in the BMN 333 data? A: Greg Friberg, Chief R&D Officer, mentioned that the goal is to develop a single-agent CNP drug that offers best-in-class growth and safety. For BMN 333, they are looking for sustained CNP levels in healthy volunteers, similar to animal models, to deliver superior efficacy while maintaining safety.

Q: Can you provide an update on the enzyme therapy business and any expected growth patterns? A: Brian Mueller noted that the enzyme therapy business is expected to grow at a high single-digit CAGR over the mid-term, contributing to the $4 billion revenue target by 2027. Growth is expected to be weighted towards the second half of the year, with PALYNZIQ being a key driver.

Q: How is BioMarin addressing potential competition in the VOXZOGO market, and what is the expected impact on market dynamics? A: Cristin Hubbard emphasized that competition is factored into their projections and can raise awareness about treatment options. BioMarin aims to maintain leadership through the trusted profile of VOXZOGO, high compliance rates, and a strong global footprint, especially in markets outside the U.S.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.