Emicizumab is an investigational bispecific monoclonal antibody developed to bring together factors IXa and X, which are proteins needs to activate the natural coagulation cascade and restore the blood clotting process.
Roche’s Haven 1 trial compared emicizumab prophylaxis with on-demand and prophylactic use of bypassing agents (BPAs) in adults and adolescents with haemophilia A with inhibitors.
According to the company, the results demonstrated a statistically significant and clinically meaningful reduction in bleed rate of 87% with emicizumab prophylaxis compared with on-demand treatment with BPAs.
Interim results from the single arm Haven 2 study in children younger than 12 years of age with haemophilia A with inhibitors who secured emicizumab prophylaxis are in line with the positive results from the Haven 1 study.
Haven 1 is a randomised, multicentre, open-label and phase III trials designed to assess the efficacy, safety, and pharmacokinetics of emicizumab prophylaxis compared to on-demand BPA in adults and adolescents with haemophilia A with inhibitors to factor VIII.
The trial is comprised of 109 patients with haemophilia A with inhibitors to factor VIII, who were previously treated with on-demand or prophylactic BPAs
The study’s primary endpoint is the number of treated bleeds over time with emicizumab prophylaxis (Arm A) compared with no prophylaxis (Arm B).
Haven 2 is a single-arm, multicentre, open-label and phase III study designed to assess the efficacy, safety, and pharmacokinetics of once-weekly subcutaneous administration of emicizumab.
The interim analysis after a median of 12 weeks of treatment is comprised of 19 children younger than 12 years of age with haemophilia A with inhibitors to factor VIII, who require treatment with BPAs.
It is designed to assess the number of treated bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life (HRQoL) and proxy HRQoL with aspects of caregiver burden.
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