BioMarin (BMRN) Q4 2025: Voxogo Surges 26% as Pipeline and Amicus Integration Set Up 2026 Inflection

2026-02-23 | By EarningsIQ Research

BioMarin capped 2025 with double-digit revenue growth and robust operating leverage, propelled by Voxogo’s global expansion and durable enzyme therapy demand. Strategic moves—most notably the Amicus acquisition and advancing next-gen assets—signal a pivotal year ahead, with management emphasizing both immediate commercial scale and pipeline-driven growth through 2026 and beyond.

Summary

• Voxogo’s Global Momentum: Deepening market penetration and early-age adoption sustain growth despite looming competition.
• Pipeline and M&A Drive: Integration of Amicus and advancing new therapies expand BioMarin’s rare disease leadership.
• 2026 Setup: Guidance reflects confidence in core franchises and sets expectations for a step-change post-Amicus close.

Performance Analysis

BioMarin delivered a record year, with total revenue reaching $3.22 billion, up 13% year-over-year, driven by broad-based strength across its portfolio. The enzyme therapies franchise, the company’s largest business unit, grew 9% and continues to demonstrate resilience and global reach, now surpassing $2 billion in annual sales and accounting for over 60% of total revenue. Notably, Palynziq, enzyme therapy for PKU, posted 22% growth and is set for further expansion with an adolescent label update pending.

Voxogo, bone growth therapy for achondroplasia, accelerated 26% for the year and 31% in Q4, supported by global execution and early-age patient starts—about 73% of Voxogo revenue was generated outside the U.S., reflecting BioMarin’s operational scale. The company’s fourth quarter was its strongest ever, though management cautioned on Q1 2026 due to order timing and inventory dynamics, with large government orders and stocking inflating Q4.

• Operating Leverage: Non-GAAP EPS climbed 34% excluding special items, and operating cash flow increased 45% to $828 million, underscoring improved profitability and cash generation.
• Special Charges and Portfolio Pruning: The withdrawal of Roctavian led to $240 million in special items, but the impact was isolated and does not affect underlying business momentum.
• Amicus Financing: Secured $3.7 billion in debt at favorable terms, reflecting lender confidence and supporting the pending acquisition’s integration.

BioMarin’s financial model now demonstrates consistent margin expansion and cash flow growth, providing flexibility for both pipeline investment and future M&A.

Executive Commentary

Strategic Positioning

1. Voxogo Franchise: Global Penetration and Early-Age Focus

Voxogo’s growth is anchored in rapid international adoption and a strategic push into treating children under two years old, where it holds market exclusivity for the foreseeable future. BioMarin’s tailored market-by-market approach—leveraging pre-launch activities and clinician engagement—has enabled penetration rates of 40% in new Asia-Pacific markets and 70% in select European countries within a year of launch. This reflects a disciplined, data-driven commercial strategy and positions Voxogo as the backbone of BioMarin’s rare disease portfolio.

2. Enzyme Therapies: Durable, Diversified Revenue Base

The enzyme therapies portfolio, including Palynziq and Vimizin, remains a durable growth engine, with high patient adherence and new starts fueling steady expansion. The upcoming label expansion for Palynziq in adolescents is expected to widen its addressable market, further entrenching BioMarin’s leadership in metabolic disorders.

3. Pipeline Expansion and Next-Gen Therapies

BioMarin is advancing multiple late-stage assets, including BMN333, a long-acting CNP therapy targeting a “best-in-disease” profile for achondroplasia. The pipeline also features pivotal data readouts for Voxogo in hypochondroplasia and BMN401 for ENPP1 deficiency, both representing first-in-class opportunities. Management is preparing for regulatory submissions and aims to launch new indications as early as 2027, with ongoing business development to supplement organic growth.

4. Amicus Acquisition: Scale and Synergy

The pending acquisition of Amicus will add Gallifold (Fabry disease) and Pombility/Alfolda (Pompe disease) to BioMarin’s enzyme therapy platform, leveraging existing commercial infrastructure for immediate scale and anticipated top-line synergies. Management expects a seamless integration, with Amicus assets fitting directly into the current business unit structure.

5. Commercial Model: Global Infrastructure and Market Access

BioMarin’s commercial execution is supported by a 20-year track record in rare diseases, with 80-country reach and deep stakeholder relationships. The company is navigating routine market access renegotiations in mature markets, which may temporarily reset pricing but are expected to broaden patient access and support long-term revenue growth.

Key Considerations

BioMarin’s 2025 results and 2026 setup reflect a business at an inflection point, balancing strong legacy franchises, new product launches, and transformative M&A. The following considerations are central for investors:

Key Considerations:

• Voxogo’s Competitive Moat: Early-age label, extensive safety data, and global reach underpin resilience against new entrants, especially in the high-value infant segment.
• Order Timing Volatility: Q4 2025 revenue was elevated by large government and stocking orders; Q1 2026 is expected to reset lower, but patient growth trends remain steady.
• Pipeline Value Creation: Upcoming data readouts and label expansions (BMN333, hypochondroplasia, ENPP1 deficiency) are key catalysts for both organic and acquired assets.
• Amicus Integration Synergy: Immediate scale and cross-selling potential as Amicus assets drop into the enzyme therapy unit, with diagnosis initiatives poised to expand patient pools.

Risks

BioMarin faces competitive headwinds in achondroplasia as oral and long-acting competitors approach market entry, potentially pressuring Voxogo’s future growth. Order timing and market access renegotiations introduce quarterly volatility, and integration of Amicus will bring execution and margin dilution risk in 2026. Regulatory and pipeline outcomes, especially for new indications, remain critical swing factors for the growth narrative.

Forward Outlook

For Q1 2026, BioMarin expects:

• Total revenue and Voxogo revenue to be on par with Q1 2025, reflecting typical Q4-to-Q1 step-down and order phasing.
• Q1 non-GAAP EPS to be the lowest of the year, impacted by pre-close Amicus integration costs.

For full-year 2026, management guided:

• Enzyme therapies revenue: $2.225–$2.275 billion
• Voxogo revenue: $975 million–$1.025 billion
• Total revenue: $3.325–$3.425 billion (excluding Amicus contribution)
• Non-GAAP EPS: $4.95–$5.15

Management highlighted:

• High single-digit growth in core franchises at midpoints
• Step-change in total growth rate and margin profile post-Amicus close

Takeaways

BioMarin is executing on a dual-track strategy: maximizing mature franchises while investing in pipeline and M&A for the next growth wave.

• Voxogo’s resilience and early-age focus insulate near-term growth, but competitive landscape requires ongoing innovation and data differentiation.
• Pipeline catalysts and Amicus integration are pivotal for 2026–2027, with regulatory, commercial, and operational execution all under investor scrutiny.
• Investors should monitor: hypochondroplasia data, BMN333 trial progress, Amicus synergy realization, and quarterly revenue volatility tied to order timing and market access resets.

Conclusion

BioMarin enters 2026 with strong commercial momentum, operational leverage, and a robust pipeline, but faces a pivotal year as it integrates Amicus and navigates new competition. The company’s ability to deliver on pipeline milestones and realize acquisition synergies will define its long-term value creation trajectory.

Industry Read-Through

BioMarin’s results underscore the structural advantage of global commercial platforms in rare disease, particularly in scaling new therapies and managing order volatility. The company’s approach to early-age label expansion and data-driven market access negotiations sets a template for peers facing similar competitive threats. Pipeline depth and disciplined M&A remain critical levers for growth as competition intensifies and payers become more sophisticated in pricing negotiations. Competitors in rare disease and specialty pharma should watch BioMarin’s integration of Amicus and its execution on next-generation assets for signals on sustainable growth and margin management in a maturing innovation cycle.

BMRN Healthcare Biotechnology Rare Disease Pipeline Mergers Voxogo Enzyme Therapy