BioMarin Pharmaceutical (BMRN 3.73%), a biotechnology company focused on therapies for rare genetic conditions, reported results for Q2 2025 on August 4, 2025. GAAP revenue was $825 million, far above expectations of $761.66 million, and non-GAAP Earnings Per Share (EPS) of $1.44 compared to the anticipated $0.82. Both figures marked significant beats against analyst estimates, with non-GAAP EPS and GAAP revenue exceeding consensus, with GAAP revenue up 16% year-over-year and non-GAAP EPS up 50% year-over-year. Management raised full-year 2025 guidance following the strong Q2, including updated targets for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS, driven by robust commercial execution for its leading products VOXZOGO and Enzyme Therapies.
| Metric | Q2 2025 | Q2 2025 Estimate | Q2 2024 | Y/Y Change |
|---|---|---|---|---|
| EPS (Non-GAAP) | $1.44 | $0.82 | $0.96 | 50.0% |
| Revenue (GAAP) | $825 million | $761.66 million | $712 million | 16% |
| Non-GAAP Operating Margin | 39.9% | 31.2% | 8.7 pp | |
| GAAP Net Income | $241 million | $107 million | 125.2% |
Source: Analyst estimates provided by FactSet. Management expectations based on management’s guidance, as provided in Q1 2025 earnings report.
Business Overview and Recent Priorities
BioMarin Pharmaceutical develops, manufactures, and markets medicines for people with rare genetic diseases. Its primary focus is on short stature conditions, enzyme deficiencies, and gene therapies for specific inherited disorders. The business is centered around flagship product families like VOXZOGO (an injectable therapy for achondroplasia, the most common form of dwarfism), and multiple Enzyme Therapies targeting specific genetic disorders.
Recently, the company has concentrated on expanding its commercial reach, accelerating its innovation pipeline, and achieving regulatory approvals in new regions and indications. Key success factors include advancing pipeline candidates toward approval, defending and growing market leadership for VOXZOGO, and maintaining robust manufacturing and supply chain infrastructure to support global launches. Commercial momentum and pipeline innovation are both critical drivers for BioMarin’s growth strategy.
Highlights and Trends from the Quarter
During Q1 2025, BioMarin delivered substantial gains in both sales and profitability. Revenue (GAAP) was up 16% year over year, driven by strong double-digit year-over-year growth for both the Skeletal Conditions and Enzyme Therapies franchises. VOXZOGO, a biologic therapy for children with achondroplasia, generated $221 million in GAAP revenue (a 20% increase compared to the prior-year period). Patient numbers grew as new children began therapy worldwide, particularly in the US among infants and toddlers after a label expansion allowed for earlier treatment. By the end of Q2 2025, VOXZOGO had reached patients in 51 countries, with a target of surpassing 60 countries by 2027.
The Enzyme Therapies segment, which includes products like VIMIZIM (for Morquio A syndrome), PALYNZIQ (for phenylketonuria), ALDURAZYME, NAGLAZYME, and BRINEURA, delivered combined GAAP revenue of $555 million, up 15% year-over-year. VIMIZIM delivered over 20% GAAP revenue growth, while PALYNZIQ grew 20%, and ALDURAZYME rose 44% year-over-year on a GAAP basis, though management noted that timing of large government orders contributed to these surges. In contrast, KUVAN (for phenylketonuria) revenues continued to fall as generic alternatives entered the market.
Profitability (non-GAAP) expanded even faster than revenue, with GAAP net income reached $241 million (a 125% increase year over year) and the non-GAAP operating margin jumped to 39.9%, up 8.7 percentage points from the prior year. The company attributed this to increased gross profit, lower selling, general, and administrative costs (after lapping severance and restructuring expenses from 2024), and disciplined research and development (R&D) spending following portfolio reprioritization in 2024. Operating cash flow was $185 million, up 55% compared to the same period in 2024, bringing total cash and investments to approximately $1.9 billion.
BioMarin continued advancing its innovation pipeline. VOXZOGO reached new clinical milestones and expanded its label for younger patients, while development progressed on additional indications including hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. The Phase 1 study for BMN 333 (a long-acting C-type Natriuretic Peptide for achondroplasia) in healthy volunteers demonstrated high drug levels and no safety concerns, as reported in Q2 2025, setting the stage for a pivotal trial in 2026. The acquisition of Inozyme in July introduced BMN 401, a potential new enzyme replacement therapy, with pivotal trial data expected next year. Regulatory submissions are planned for PALYNZIQ expansion in adolescents. ROCTAVIAN (an adeno-associated virus gene therapy for hemophilia A) remained a minor contributor to overall revenues.
Manufacturing and supply chain stability remained strong. The company’s drug substance for VOXZOGO is sourced in the United States, and it operates major facilities in both the US and Ireland. Management highlighted minimal exposure to US tariffs and global trade disruptions, with the majority of revenue coming from outside the US. There were no reported supply chain bottlenecks during the period.
Forward Guidance and What to Watch
The company raised its full-year 2025 guidance. Revenue (GAAP) is now projected at $3,125 million to $3,200 million (up from previous guidance starting at $3,100 million), and expected VOXZOGO sales were lifted to a range of $900 million to $935 million. Non-GAAP operating margin guidance was increased from 32–33% to 33–34%, and Non-GAAP EPS is forecast at $4.40 to $4.55. This outlook reflects continued strong demand for VOXZOGO and expanding contributions from enzyme therapies, alongside tight operational discipline.
Management is maintaining its approach of reinvesting profits in R&D and business development. Key items for future quarters include monitoring competitive threats to VOXZOGO, progress on pivotal clinical trial readouts (particularly for pipeline programs BMN 333 and BMN 401), and the commercial ramp for new product launches or label expansions. Investors should also pay attention to any update regarding accounting impacts from the recent Inozyme acquisition and ongoing efforts to penetrate new global markets for lead therapies.
Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted.
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