Alnylam Pharmaceuticals, Inc.

ATTR amyloidosis, AHP, and PH1 are fatal or severely debilitating diseases requiring indefinite treatment, driving near-complete patient persistence once therapy is initiated. Net product revenues grew from $844M in FY2021 to $1.646B in FY2024 without a single annual decline, including through the COVID-19 disruption.

The TTR franchise (ONPATTRO and AMVUTTRA) represented approximately 74% of net product revenues in FY2024 and rose toward 86% in Q4 FY2025 as ATTR-CM approvals drove AMVUTTRA adoption. The Rare franchise (GIVLAARI and OXLUMO) contributed approximately $423M in FY2024, providing a secondary revenue line insufficient to offset the dominant TTR concentration.

AMVUTTRA, ONPATTRO, GIVLAARI, and OXLUMO each hold regulatory approvals in the US, EU, and Japan, providing meaningful multi-region commercial presence. US pricing premiums typical of specialty orphan drugs likely place the US above 60% of consolidated net product revenues, consistent with home-country dependence in the absence of disclosed geographic splits.

The RNAi platform allows multiple drugs to be developed and commercialized using the same chemistry and GalNAc delivery infrastructure, sharing sunk R&D costs across a growing portfolio. FY2025 marked the company's first profitable year (income from operations of $501.6M versus a loss of $176.9M in FY2024), demonstrating emerging operating leverage as product revenues scale ahead of fixed costs.

ATTR amyloidosis, AHP, and PH1 are progressive, life-threatening diseases for which Alnylam's products are primary or sole disease-modifying options, making them mission-critical for the patient population across a lifelong treatment horizon. Revenue is generated through prescription fills rather than formal multi-year contracts, which is the only material departure from the highest revenue-quality profile.