Amicus Therapeutics (FOLD) investor relations material

Business performance and product updates

• Achieved over $500 million in annual revenue for the first time, driven by double-digit growth in Galafold for Fabry disease and 50%-65% revenue growth in Pombiliti/Opfolda for Pompe disease.

• Galafold continues to expand in a Fabry market now estimated at $2.4 billion, with patient numbers and diagnosis rates rising due to improved genetic testing and broader disease recognition.

• Pombiliti/Opfolda sales exceeded $100 million, with strong momentum in the U.S. and Europe, and increasing patient switches from competitor products as eligibility widens.

• Achieved GAAP profitability in Q3, with a focus on maintaining full-year profitability and disciplined financial management.

• Cash position exceeds $260 million, supporting ongoing growth and business development initiatives.

Market dynamics and growth drivers

• Fabry market growth is fueled by increased diagnosis, especially among underrepresented populations, and advances in AI-driven patient identification.

• Treatment guidelines are evolving to support earlier intervention, with Galafold gaining first-line status in some regions.

• Pombiliti/Opfolda benefits from delayed competitor launches and patient/physician switching dynamics, with U.S. and European markets showing robust adoption.

• Real-world evidence and mandated switches in certain European centers are accelerating market share gains.

• Investor consensus for 2026 revenue targets has adjusted to more achievable levels, aligning with current momentum.

Pipeline and strategic initiatives

• Licensed late-stage DMX-200 for FSGS, a rare kidney disease, with U.S. commercial rights and no upfront payment until phase III data readout.

• FDA has agreed that proteinuria is an appropriate primary endpoint for DMX-200, with top-line data expected in early 2028 based on current enrollment.

• Monitoring peer regulatory outcomes and planning for potential accelerated approval pathways.

• Business development focus remains on late-stage, de-risked, synergistic rare disease assets, with continued financial discipline.

• Multiple independent parties have validated the DMX-200 opportunity, supporting confidence in its potential.