Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.

ABSTRACT:

Abstract

Fitusiran is an investigational small interfering RNA therapeutic that targets antithrombin (AT) to rebalance hemostasis in people with hemophilia. Here, we present the results of a completed phase 2 open-label extension study, which evaluated the long-term safety and efficacy of fitusiran in participants with moderate or severe hemophilia A or B, with or without inhibitors. Male participants who had completed the phase 1 study (ClinicalTrials.gov identifier: NCT02035605) were enrolled. Participants received monthly subcutaneous fitusiran (50 or 80 mg) under the original dose regimen until a voluntary dosing pause in 2020, after which the AT-based dose regimen was introduced, targeting the recommended AT activity levels of 15% to 35%. Thirty-four participants (hemophilia A

SUBMITTER: Pipe SW 

PROVIDER: S-EPMC11914172 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature