Amicus Therapeutics (FOLD) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Pompe disease asset update

• One year post-approval, patient switching from Nexviazyme is proportional to the population, indicating patients are not lost after switching therapies.

• Most U.S. patients switched to Pombiliti Opfolda after 1–2 years on Nexviazyme, with ongoing efforts to shorten this timeframe.

• Durability of effect is supported by up to four years of data, with upcoming four-year results expected within 12 months.

• Real-world evidence and registry data are being collected to reinforce clinical trial findings and support physician confidence.

• 2023 global sales guidance for Pombiliti Opfolda is $62–67 million, with Q2 revenue at $16 million and continued focus on reducing time from prescription to infusion.

Market dynamics and growth drivers

• Lysosomal disease markets show steady, durable growth due to underdiagnosis and limited competition.

• Pompe market projected to grow from $1.2 billion to $2 billion by decade's end, driven by increased diagnosis and more efficacious therapies.

• Launches in new markets, such as Spain, and progress in European reimbursement are key to future uptake.

• Diagnosis rates and awareness are increasing, fueling market expansion and patient identification.

Galafold performance and patient mix

• Diagnosed Fabry patients have increased from 10,000 to 17,000 in a decade, with 11,000 now treated.

• Initial growth focused on switch patients, but now 80% of new starts in major markets are naive or newly diagnosed.

• About 40% of Galafold patients are switch patients; the rest are naive, with significant opportunity remaining among ERT patients.

• Market for Galafold could exceed $3 billion by decade's end, with potential to capture a third to half of the market.