Amicus Therapeutics (FOLD) Q3 2025: Pompe Revenue Up 45% as Global Launch Accelerates

2025-11-04 | By EarningsIQ Research

Amicus Therapeutics delivered a pivotal Q3 with 45% reported revenue growth in its Pompe franchise, driven by rapid market expansion, new patient starts, and deepening global penetration. The company’s dual rare disease strategy, anchored by Galafold and Pompe therapies, is translating into sustained double-digit growth and a clear path toward blockbuster status. Management’s emphasis on label expansion, evidence generation, and operational discipline underpins confidence in multi-year growth, even as competitive and regulatory forces evolve.

Summary

• Pompe Franchise Momentum: Accelerating global launches and patient switches are driving outperformance in key markets.
• Galafold Penetration: Record new patient starts reflect success in capturing naive and undiagnosed Fabry populations.
• Pipeline Execution: Phase III DMX-200 advances position Amicus for future rare disease portfolio expansion.

Performance Analysis

Amicus Therapeutics posted robust Q3 results, with total revenue up 19% year-over-year and double-digit growth across both Galafold, Fabry disease oral therapy, and its Pompe franchise. Galafold contributed $138.3 million, up 15% in reported terms, supported by a 13% increase in year-over-year patient demand and record high new patient starts. The product now holds approximately 69% global market share for treated Fabry patients with amenable mutations, reflecting its entrenched position as standard of care in mature markets.

The Pompe franchise, branded as Pombility and Opfolda, delivered $30.7 million in Q3 revenue, up 45% in reported terms and 42% at constant exchange rates. Year-to-date, Pompe revenue has grown 61%, with the U.S. accounting for 43% of Q3 franchise sales. Expansion into new geographies, including Japan and several European countries, has doubled the number of patients switching from legacy therapies. Gross margin remained strong in the mid-80% range, with GAAP net income turning positive for the quarter and management reiterating guidance for sustained profitability in the second half of 2025.

• Revenue Mix Shift: Ex-U.S. markets contributed 58% of total revenue, highlighting geographic diversification.
• Operating Leverage: GAAP operating expenses rose 8% versus revenue growth of 19%, supporting improving margins.
• Cash Generation: Cash and securities increased by $32.8 million quarter-over-quarter, reinforcing financial discipline.

Underlying performance reflects increasing commercial traction, successful market access execution, and operational discipline across the portfolio.

Executive Commentary

Strategic Positioning

1. Rare Disease Market Leadership

Amicus is consolidating its position as a leader in rare genetic diseases, leveraging first-mover advantage and deep clinical expertise to drive penetration in Fabry and Pompe segments. Galafold’s dominance is underpinned by robust IP, high compliance rates, and targeted diagnosis campaigns, while Pombility and Opfolda are building momentum through rapid global launches and real-world data generation.

2. Commercial Execution and Global Expansion

Execution in commercial launches is translating into accelerated patient uptake and deeper prescriber engagement. The company now has Pompe reimbursement in 15 countries, with five new markets generating revenue in Q3 and additional launches in Japan, Belgium, Ireland, and Luxembourg. Management is focused on shortening time to reimbursement, expanding prescriber base, and securing broad access in both established and emerging markets.

3. Evidence Generation and Label Expansion

Amicus is investing in long-term data and real-world evidence to differentiate its therapies and support label expansions. Four-year data from the PROPEL extension study in Pompe demonstrated sustained muscle function improvements, while ongoing pediatric and infantile studies are expected to support broader indications. The company is actively pursuing label expansions for adolescent and pediatric populations, which could unlock significant incremental growth.

4. Pipeline Diversification with DMX-200

The late-stage DMX-200 program, targeting FSGS (focal segmental glomerulosclerosis), represents a potential new growth pillar. With over 90% enrollment completed in the pivotal Action 3 trial and FDA alignment on primary endpoints, Amicus is positioned to expand its rare disease portfolio and address additional unmet needs.

Key Considerations

This quarter’s results reflect a multi-pronged strategy balancing commercial execution, evidence generation, and portfolio expansion.

Key Considerations:

• Patient Identification Campaigns: Enhanced diagnostic initiatives are shortening time to diagnosis and increasing the pool of naive patients eligible for Galafold.
• Switch Dynamics in Pompe: Patient-driven advocacy and growing physician comfort are accelerating switches from legacy therapies to Pombility and Opfolda, particularly in the U.S. and Netherlands.
• Label Expansion Timelines: Pediatric and infantile Pompe label submissions are expected in the next 12-24 months, with adolescent expansion anticipated by mid-2026.
• Gross Margin Resilience: Management expects margins to remain at the high end of the mid-80% range, even as product mix evolves and inventory transitions occur.
• Pipeline Optionality: The DMX-200 program provides a de-risked late-stage asset with blockbuster potential in nephrology, diversifying future revenue streams.

Risks

Amicus faces several risks, including competitive dynamics in rare disease markets, regulatory uncertainties around label expansions (especially pediatric and infantile Pompe), and potential supply chain impacts from policy changes such as BioSecure 2.0. While management expresses confidence in securing Ireland-based supply, evolving U.S. manufacturing requirements could introduce complexity. Additionally, the company’s ability to sustain high compliance and switch rates as markets mature will be critical for maintaining growth trajectories.

Forward Outlook

For Q4 2025, Amicus guided to:

• Total revenue growth of 15% to 22% at constant exchange rates
• Galafold revenue growth of 10% to 15%
• Pombility and Opfolda revenue growth of 50% to 65%

For full-year 2025, management reiterated:

• Gross margin in the mid-80% range, likely at the top end
• Non-GAAP operating expenses at the high end of $380-$400 million
• Positive GAAP net income for the second half of 2025

Management highlighted confidence in achieving consensus sales estimates and continued commercial momentum into 2026, with label expansion and new country launches expected to be incremental growth drivers.

• Continued record new patient starts in both Fabry and Pompe franchises
• Ongoing evidence generation to support future reimbursement and uptake

Takeaways

Amicus is executing on a rare disease playbook that marries commercial scale with evidence-driven differentiation.

• Pompe Franchise Scaling: Rapid global launch and high switch rates are positioning Pombility and Opfolda as leading therapies in a competitive landscape.
• Galafold’s Durable Growth: Sustained patient identification and high compliance rates are extending the product’s runway and solidifying blockbuster potential.
• Pipeline Leverage: Late-stage DMX-200 progress and upcoming pediatric label expansions offer optionality for future growth and portfolio resilience.

Conclusion

Amicus Therapeutics delivered a quarter marked by accelerating commercial execution, broad-based revenue growth, and operational discipline. The company’s dual rare disease franchises are on track for blockbuster status, with pipeline and label expansion efforts supporting a multi-year growth outlook.

Industry Read-Through

Amicus’s performance underscores the value of targeted commercial execution and evidence generation in rare disease markets. The company’s success in expanding both naive and switch patient populations highlights the importance of diagnostic initiatives and real-world data for driving uptake. Competitors in rare genetic and orphan drug markets should note the accelerating pace of global launches, the role of patient advocacy in switching dynamics, and the necessity of robust supply chain strategies in the face of evolving regulatory environments. The pipeline-driven approach, exemplified by DMX-200, signals that late-stage portfolio diversification remains a critical lever for sustaining long-term growth in specialty pharma.

FOLD Healthcare Biotechnology Rare Disease Commercial Launch Label Expansion Pipeline Revenue Growth