Amicus Therapeutics (FOLD) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key business highlights

• Galafold delivered $111 million in quarterly sales, with annual sales guidance raised to 14%-18% growth.

• Pombiliti and Opfolda generated $16 million in Q2 revenue, targeting $62-67 million for the year.

• On track to achieve first full year of non-GAAP profitability, reflecting strong financial discipline.

• Focus remains on expanding market share and deepening penetration in core rare disease markets.

• Tightened non-GAAP OpEx guidance to $345-$360 million for the year.

Galafold market dynamics and growth drivers

• Global Fabry disease patient diagnosis has grown from 10,000 in 2016 to 17,000 today.

• 80% of new Galafold patients in major markets are newly diagnosed or previously untreated.

• Holds 60% global market share among treated amenable patients, with mature markets at 80%-90%.

• Growth expected from diagnosed but untreated patients and improved diagnosis pathways.

• AI and machine learning seen as future tools to identify more patients.

Pombiliti and Opfolda launch and market expansion

• Pompe disease market growth driven by newborn and high-risk screening, especially in the U.S.

• Spain launch outpaced competitors, with strong momentum and potential for faster ramp in select EU markets.

• U.S. and Germany expected to reach 25%-30% market share in two years.

• Prescription-to-infusion time reduced from 65 to 30-45 days, improving patient access.

• Growth balanced between new and repeat prescribers, with strong net patient adds.