Alnylam Pharmaceuticals (ALNY) 24th Annual Needham Virtual Healthcare Conference summary

Market and regulatory environment

• Exposure to recent tariffs is minimal due to U.S.-based manufacturing.

• Recent FDA and HHS leadership changes create uncertainty, but confidence remains in regulatory processes and continued innovation support.

• Approval of sNDA for vutrisiran and Qfitlia TTR achieved recently, reflecting strong regulatory engagement.

Financial guidance and growth drivers

• 2025 TTR franchise revenue guidance set at $1.6–$1.725 billion, implying 36–41% growth.

• Growth is driven by robust product launches, strong data, and broad label approvals.

• Utilization as a first-line treatment and unmet needs among patients progressing on stabilizers are key growth factors.

Polyneuropathy and cardiomyopathy market dynamics

• Polyneuropathy segment estimated at 25,000–30,000 patients globally, with continued growth expected.

• Market leadership maintained despite new competitors, with category expansion accelerating diagnosis and treatment.

• Cardiomyopathy market estimated at 300,000 patients worldwide, with only 20% currently treated; 18,000 new-to-treatment patients annually.