Cost-effectiveness of iptacopan for paroxysmal nocturnal hemoglobinuria.

ABSTRACT:

Abstract

Iptacopan, a novel oral factor B inhibitor, recently obtained US Food and Drug Administration approval for treating paroxysmal nocturnal hemoglobinuria, a rare blood disorder characterized by persistent complement-mediated hemolytic anemia. The standard-of-care (SOC) has traditionally relied on complement C5 inhibitors eculizumab and ravulizumab, which are limited by persistent anemia from extravascular hemolysis and requirement for intravenous infusion. Recent publication of phase 3 studies in this arena reinforces iptacopan as an effective anticomplement monotherapy compared with SOC. Given ongoing price negotiations and limited literature showing its cost-ineffectiveness in the anti-C5-treated population, we conducted a comprehensive cost-effectiveness analysis of iptac

SUBMITTER: Ito S 

PROVIDER: S-EPMC11738035 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature