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

Pompe disease asset performance and market dynamics

• One year post-approval, patient switching from Nexviazyme to Pombiliti Opfolda is proportional to the existing population, dispelling concerns about losing patients permanently to competitors.

• Most switch patients in the U.S. were on Nexviazyme for one to two years before transitioning, with ongoing efforts to shorten this timeframe.

• Durability of Pombiliti Opfolda is supported by four-year Phase I/II data and two-year Phase III data, with further long-term results expected within 12 months.

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

• Commercial execution focuses on shortening time from prescription to infusion, increasing new patient acquisition, and expanding into new markets, with Q2 revenue at $16 million and 186 patients prescribed or on treatment.

Market growth, diagnosis trends, and reimbursement

• Lysosomal disease therapy markets show steady, durable growth due to underdiagnosis and limited competition, with Pompe projected to grow from $1.2 billion to $2 billion by decade's end.

• Increased awareness, cheaper diagnostics, and more efficacious therapies are driving higher diagnosis rates and market expansion.

• Reimbursement processes for Pombiliti Opfolda have been notably efficient, with NICE recommending approval before MHRA and Germany maintaining launch pricing post-AMNOG review.

• Early and broad access is prioritized over maximizing price, aiming for faster patient uptake and better outcomes.

Galafold growth drivers and patient mix

• Diagnosed Fabry patients have increased from 10,000 to 17,000 in a decade, with 11,000 treated and 6,000 untreated.

• Initial growth focused on switch patients, but now 80% of new starts in key markets are naive or newly diagnosed, indicating a shift to broader patient capture.

• About 40% of Galafold patients are switch patients, with the remainder naive; global share of treated amenable patients is 60%, leaving room for further switches.

• Market for Galafold could exceed $3 billion by the end of the decade, supporting its billion-dollar product potential.