Amicus Therapeutics (FOLD) Q2 2025: Pombiliti and Opfolda Surge 58% as Global Launch Accelerates

July 31, 2025 | By EarningsIQ Research

Amicus Therapeutics delivered its 17th straight quarter of double-digit revenue growth, powered by robust global uptake in both Fabry and Pompe disease franchises. The company’s rare disease portfolio is expanding rapidly, with Pombiliti and Opfolda launches gaining meaningful traction across new geographies and the DMX200 pipeline asset progressing toward pivotal milestones. Management reaffirmed full-year guidance and signaled confidence in achieving GAAP profitability in the second half, positioning Amicus for sustained growth into 2026 and beyond.

Summary

• Rare Disease Expansion: Global launches of Pombiliti and Opfolda are driving rapid patient adoption and market penetration.
• Pipeline Momentum: DMX200 Phase III enrollment nears completion, reinforcing late-stage pipeline strength.
• Profitability in Sight: Management targets GAAP profitability in H2, underpinned by disciplined cost control and operational leverage.

Performance Analysis

Amicus posted a 22% revenue increase year-over-year, with total revenue reaching $154.7 million for the quarter, demonstrating the resilience and scalability of its rare disease business model. Fabry disease therapy Galafold, an oral small molecule for amenable mutations, remains the anchor, accounting for the majority of sales and maintaining over 69% global market share in treated Fabry patients. Patient demand for Galafold rose 13% YoY, with new patient starts at record highs, reflecting both deeper market penetration and increased diagnostic rates.

Pombiliti and Opfolda, the company’s enzyme replacement and chaperone combination for late-onset Pompe disease, delivered a standout 58% revenue growth at constant currency, fueled by launches in five new markets and robust uptake in established geographies. The U.S. and Europe split sales roughly evenly, with notable momentum in the Netherlands, UK, and Spain. Operating expenses increased sharply, largely due to a $30 million upfront licensing payment for DMX200, a late-stage candidate for FSGS (focal segmental glomerulosclerosis, a rare kidney disease). Excluding this one-time cost, Amicus would have achieved positive GAAP net income for the quarter.

• Galafold Market Leadership: 69% share of treated Fabry patients, with 65% of current patients treatment-naive, highlighting untapped potential.
• Geographic Diversification: 58% of sales now ex-US, underlining expanding global footprint and launch execution.
• Operating Leverage: Gross margin guidance remains in the mid-80s, with high compliance and adherence rates above 90% for both major products.

Cash and equivalents of $231 million provide ample runway, especially as management reiterates its intent to achieve positive GAAP net income in the second half of 2025. The business is showing durable, multi-year growth drivers and operational discipline as it scales.

Executive Commentary

Strategic Positioning

1. Fabry Franchise: Deepening Penetration and Market Expansion

Galafold, oral Fabry therapy, continues to dominate the amenable mutation segment, with patient starts and market share expanding in both established and new markets. Management emphasized that the diagnosed but untreated and underdiagnosed populations represent a significant growth runway, with literature suggesting the true prevalence may exceed 100,000 globally. High compliance rates and new diagnostic initiatives are expected to drive further adoption, and the product’s IP protection remains strong, with recent settlements reinforcing confidence in patent longevity.

2. Pompe Disease: Launch Execution and Differentiation

Pombiliti and Opfolda, combination ERT and chaperone for Pompe disease, are scaling rapidly, with launches in 11 countries and planned expansion into at least 10 new markets this year. The Netherlands launch stands out, with expectations to capture up to 70% of the country’s LOPD (late-onset Pompe disease) population through a centralized treatment site. Real-world evidence, including case studies and head-to-head data, continues to differentiate the therapy, supporting both switches from existing treatments and new patient starts.

3. Pipeline and Portfolio Diversification

DMX200, late-stage FSGS therapy, is a key strategic addition, with pivotal Phase III enrollment on track for completion by year-end. The candidate targets a monocyte-driven inflammatory pathway not addressed by current therapies, and FDA alignment on proteinuria as a primary endpoint de-risks the regulatory path. The asset offers potential for significant expansion into a rare kidney disease market with no approved therapies and is expected to generate value for both patients and shareholders.

4. Manufacturing and Supply Chain Resilience

Amicus is actively diversifying its manufacturing base, bringing new capacity online in Ireland and the U.S. to mitigate geopolitical and tariff risks. The company has fully stockpiled U.S. commercial and clinical supply for the year, and future supply chain moves are designed to optimize both cost of goods sold and supply security. Management’s forward-looking approach to global manufacturing positions Amicus to scale efficiently and adapt to evolving regulatory and trade environments.

5. Financial Discipline and Capital Allocation

Cost management remains a core focus, with non-GAAP operating expense guidance unchanged and a clear path to profitability even as the company invests in launches and pipeline assets. Cash reserves are sufficient to fund operations through upcoming milestones, and management reiterated its commitment to late-stage, de-risked pipeline expansion through business development, following the DMX200 model.

Key Considerations

This quarter marks a pivotal period for Amicus, as it transitions from a single-product rare disease company to a diversified, multi-asset commercial and late-stage pipeline player. The company’s ability to execute across launches, supply chain, and clinical development will be critical as it seeks to sustain high growth and deliver on profitability targets.

Key Considerations:

• Untapped Patient Pools: The diagnosed but untreated and underdiagnosed Fabry and Pompe populations represent significant upside if diagnostic initiatives succeed.
• Geographic Expansion Pace: Successful launches in new countries, especially in Europe and Asia Pacific, will determine the trajectory of Pombiliti and Opfolda revenue growth.
• Pipeline Read-Through: DMX200’s regulatory progress and Phase III results could unlock a new rare disease vertical, but timelines and endpoints must be met.
• Manufacturing Flexibility: Ongoing investments in U.S. and Ireland facilities provide insulation from tariff volatility and supply chain shocks.
• IP and Legal Defensibility: Recent settlements bolster the IP estate, but ongoing litigation remains a watchpoint for long-term exclusivity.

Risks

Key risks include delays in country-level reimbursement and patient access for new launches, potential setbacks in DMX200 clinical or regulatory progress, and ongoing exposure to litigation on core product IP. Manufacturing transitions and political volatility could impact supply chain stability, while competitive dynamics in rare disease markets may pressure share gains or pricing over time. Investors should monitor execution on global launches and pipeline milestones closely.

Forward Outlook

For Q3 2025, Amicus guided to:

• Continued double-digit revenue growth across both core franchises
• Further country launches for Pombiliti and Opfolda, including Japan

For full-year 2025, management reiterated guidance:

• Total revenue growth of 15% to 22% at constant exchange rates
• Galafold revenue growth of 10% to 15%
• Pombiliti and Opfolda revenue growth of 50% to 65%
• Gross margin in the mid-80s
• Positive GAAP net income in the second half

Management highlighted several factors that will shape results:

• Accelerating patient starts and market share in both Fabry and Pompe disease
• Progress on DMX200 enrollment and regulatory interactions

Takeaways

Amicus is executing a rare disease growth playbook, combining commercial execution, pipeline advancement, and operational discipline to drive sustainable value creation.

• Launch Momentum: Pombiliti and Opfolda’s rapid uptake and broad geographic rollout are outpacing expectations, providing a new multi-year growth engine.
• Pipeline Optionality: DMX200 introduces a late-stage, high-potential asset, with FDA endpoint alignment reducing regulatory uncertainty.
• Profitability Watch: Investors should track GAAP profitability in H2 and the pace of operational leverage as new launches scale.

Conclusion

Amicus Therapeutics is entering a new phase of growth, with commercial momentum in core rare disease markets and a late-stage pipeline asset that could diversify revenue streams. Execution on launches and manufacturing, coupled with prudent capital allocation, sets the stage for multi-year value creation.

Industry Read-Through

Amicus’s results underscore the value of focused rare disease portfolios with global reach and strong IP protection. The company’s ability to drive high compliance, rapid geographic expansion, and differentiated real-world evidence sets a benchmark for peers in rare disease and specialty pharma. Pipeline de-risking through FDA endpoint alignment and strategic partnerships highlights the importance of regulatory clarity and late-stage asset acquisition. Manufacturing flexibility and supply chain localization are emerging as critical levers for all biotechs navigating geopolitical and trade uncertainties.

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