Vutrisiran Health Care Resource Utilization, Costs, Discontinuation, and Mortality: A Real-World Claims Analysis

Vutrisiran was approved by the US Food and Drug Administration for hereditary transthyretin amyloid polyneuropathy (ATTR-PN) in 2022 and for transthyretin amyloid cardiomyopathy in 2025. Real-world data on vutrisiran health care resource utilization (HCRU), costs, discontinuation, and mortality in the USA are limited.

We conducted a retrospective analysis of patients initiating vutrisiran from 2022 to 2024 (when only approved for hereditary ATTR-PN) using Optum's de-identified Clinformatics^® Data Mart (CDM) Database. Patients were followed until death, disenrollment, or last date of available data. Hospitalizations, emergency department (ED) visits, and associated costs (excluding vutrisiran itself) were assessed. Discontinuation analyses were also performed in the Komodo Healthcare Map^® to serve as comparator.

In Optum^® CDM (n = 111), mean (standard deviation [SD]) age was 70.9 years (9.9), 62% were male, 41% were Black, 27% were White, and 70% had heart failure. Mean (SD) follow-up was 1.5 years (0.6). After vutrisiran initiation, 41 (37%) patients had hospitalizations with a median of 9.3 days hospitalized annually and mean (SD) cost of $80,536 ($83,907) per patient; 55 (50%) patients had ED visits not leading to hospitalization, with a mean of 2.4 visits and mean (SD) ED costs of $6339 ($7484) annually per patient. Within 12 months of vutrisiran initiation, 13 (12%) patients died and an additional 13 (12%) discontinued therapy, of which 3 (23%) restarted vutrisiran and 10 (77%) switched ATTR therapy. Results were generally similar in the Komodo database comparator analyses (n = 718); within 12 months of vutrisiran initiation, 22 (3%) patients died and 142 (20%) discontinued therapy, of which 53 (37%) restarted vutrisiran, 40 (28%) switched ATTR therapy, and 47 (33%) discontinued all treatments.

Patients treated with vutrisiran continued to experience significant HCRU and costs related to disease progression despite treatment. New therapies are needed to further reduce burden and costs.