Biotech company BioMarin Pharmaceutical (NASDAQ: BMRN) reported Q1 CY2025 results exceeding the market’s revenue expectations, with sales up 14.8% year on year to $745.1 million. The company expects the full year’s revenue to be around $3.15 billion, close to analysts’ estimates. Its non-GAAP profit of $1.13 per share was 18.6% above analysts’ consensus estimates.
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BioMarin Pharmaceutical (BMRN) Q1 CY2025 Highlights:
- Revenue: $745.1 million vs analyst estimates of $738.1 million (14.8% year-on-year growth, 1% beat)
- Adjusted EPS: $1.13 vs analyst estimates of $0.95 (18.6% beat)
- Adjusted Operating Income: $265.7 million vs analyst estimates of $158.5 million (35.7% margin, 67.7% beat)
- The company reconfirmed its revenue guidance for the full year of $3.15 billion at the midpoint
- Management reiterated its full-year Adjusted EPS guidance of $4.30 at the midpoint
- Operating Margin: 30%, up from 13.6% in the same quarter last year
- Free Cash Flow Margin: 21.2%, up from 3.2% in the same quarter last year
- Market Capitalization: $11.17 billion
StockStory’s Take
BioMarin’s first quarter results were shaped by continued growth in its VOXZOGO therapy for achondroplasia and solid contributions from its Enzyme Therapies portfolio. Management highlighted that VOXZOGO achieved strong year-over-year revenue gains, supported by expanded patient access across 49 countries and increased adoption in younger age groups following recent guideline updates. Enzyme Therapies also delivered, with PALYNZIQ and ALDURAZYME posting notable growth, and operating expenses reflecting benefits from recent cost transformation initiatives.
Looking forward, leadership reaffirmed full-year guidance and emphasized plans for increased investment in research and development, particularly for ongoing VOXZOGO indications and pipeline programs like BMN 351 and BMN 333. CEO Alexander Hardy stressed BioMarin’s resilience to sector headwinds, citing the company’s global revenue base and limited Medicare exposure. BioMarin anticipates higher revenues in the second half of the year, with new product launches and clinical milestones expected to drive future growth.
Key Insights from Management’s Remarks
Management attributed first quarter outperformance to robust commercial execution and strategic focus on expanding access for key therapies. The following major themes emerged from the call:
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VOXZOGO international expansion: BioMarin’s VOXZOGO therapy for achondroplasia continued to grow, now available in 49 countries. Management noted that international markets contributed about 75% of VOXZOGO revenues, with ongoing efforts to open access in over 60 countries by 2027.
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Adoption in younger patients: New international guidelines recommending early diagnosis and treatment have driven increased uptake among infants and young children in the U.S., following regulatory approval for these age groups at the end of last year.
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Enzyme Therapies growth: The Enzyme Therapies unit delivered 8% year-over-year growth, with PALYNZIQ and ALDURAZYME showing strong demand. Management highlighted PALYNZIQ’s potential for expanded adolescent indications, supported by positive pivotal data and regulatory submissions planned for later this year.
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Operational efficiency impact: Cost transformation efforts led to lower year-over-year R&D and SG&A expenses in Q1, boosting operating margins. However, spending is expected to rise through the rest of the year as BioMarin invests in new launches and pipeline programs.
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Minimal tariff exposure: Leadership explained that BioMarin’s global manufacturing footprint and small U.S. Medicare patient base limit exposure to potential pharmaceutical tariffs or policy changes, insulating the business from some external risks.
Drivers of Future Performance
BioMarin’s outlook for the remainder of the year is anchored in expanding indications for existing therapies, new product pipeline progress, and continued global market penetration.
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Pipeline advancement: Management expects clinical milestones from late-stage pipeline programs—such as VOXZOGO in hypochondroplasia, PALYNZIQ for adolescents, and initial data from BMN 351 for Duchenne Muscular Dystrophy—to support future growth and potential product launches.
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Commercial execution in new markets: The company aims to further expand access to VOXZOGO and Enzyme Therapies worldwide, with strategic focus on increasing prescriber awareness and broadening reimbursement pathways, particularly outside the U.S.
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Operational investment: Planned increases in R&D and SG&A spending are intended to accelerate growth in core franchises and support launches, but management cautioned that this could moderate margins in coming quarters. External risks such as evolving tariff policies remain an area of ongoing monitoring.
Top Analyst Questions
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Philip Nadeau (TD Cowen): Asked if VOXZOGO revenue could decline in Q2 and how future pharmaceutical tariffs might affect financials. Management clarified that order timing dynamics could make Q2 flat or down, and said they are modeling tariff scenarios but have limited current exposure.
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Tommie Reerink (Goldman Sachs): Inquired about business development timing and the detail of upcoming BMN 333 data. CEO Alexander Hardy reiterated a focus on executing at least one business development transaction this year, and R&D leadership expects topline pharmacokinetic results for BMN 333 later in 2025.
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Jenny Gonzalez-Armenta (Leerink Partners): Sought details on strategies to expand VOXZOGO adoption. Chief Commercial Officer Cristin Hubbard described targeted awareness campaigns, expanded field teams, and prescriber base expansion, especially among pediatric endocrinologists.
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Zaki Molvi (Jefferies): Questioned BMN 351’s safety profile given preclinical liver signals. Chief R&D Officer Greg Friberg said the candidate was engineered for safety and detailed ongoing clinical monitoring, with full safety and efficacy data to be presented later this year.
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Jessica Fye (JPMorgan): Asked how BioMarin would address market concerns about VOXZOGO’s long-term value beyond 2030. Management emphasized the large and growing dataset on efficacy and safety, broad international footprint, and next-generation candidates like BMN 333.
Catalysts in Upcoming Quarters
The StockStory team will be watching (1) progress on key clinical milestones, including data releases for VOXZOGO in hypochondroplasia and BMN 351 for Duchenne Muscular Dystrophy, (2) expansion of VOXZOGO access in new geographies and patient age groups, and (3) the outcome of regulatory submissions for PALYNZIQ adolescent indications in the U.S. and Europe. Developments in business development activities and tariff policy changes may also influence future performance.
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