Amicus Therapeutics (FOLD) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Business performance and growth outlook

• Achieved over $150 million in Q2 sales, driven by Galafold and Pombiliti/Opfolda therapies.

• Galafold projected to grow 10%-15% in 2025; Pombiliti/Opfolda expected to grow 50%-65%.

• Targeting GAAP profitability in the second half of 2025, marking a key financial milestone.

• Operating leverage expected to increase as top-line grows faster than OpEx over the next five years.

• Focused on late-stage, accretive business development to accelerate growth.

Product updates and market dynamics

• Galafold maintains double-digit growth, benefiting from increased diagnosis and treatment of Fabry disease, especially among women and late-onset patients.

• Pombiliti expanding rapidly, with launches in multiple countries and strong momentum in the Netherlands and Japan.

• U.S. Pombiliti growth driven by switches from competing therapies as patients reach two-year treatment milestones.

• No significant payer or logistical barriers for Pombiliti; focus is on physician and patient education.

• Real-world evidence and long-term data support Pombiliti’s differentiated clinical profile.

Regulatory, legal, and reimbursement landscape

• Galafold’s IP protected through at least 2037 via Teva settlement; Aurobindo litigation ongoing with trial set for September 29.

• U.K. VPAG rebate rate set at 22.9% for 2025, with limited expected year-over-year impact.

• Ongoing FDA interactions for pediatric studies and potential label expansion for Pombiliti.

• Tariffs and MFN seen as manageable risks, with mitigation strategies in place.