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Roche's emicizumab reduces bleeds in children with hemophilia A

PBR Staff Writer Published 18 April 2017

Interim data from the phase 3 HAVEN 2 study evaluating Roche's emicizumab for reducing bleeding episodes in children younger than 12 years of age with hemophilia A demonstrated a clinically meaningful treatment effect.

According to the company, emicizumab prophylaxis demonstrated a clinically meaningful reduction in the number of bleeds over time at this interim analysis after a median of 12 weeks of treatment.

The latest findings are in line with results from the phase III Haven 1 study in adults and adolescents (12 years of age or older) with haemophilia A and inhibitors to factor VIII.

The company is also currently carrying out two additional phase III studies, including Haven 3 and Haven 4, for emicizumab.

Haven 2 is a single-arm, multicentre, open-label and phase III study, which was designed to assess the efficacy, safety, and pharmacokinetics of once weekly subcutaneous administration of emicizumab.

The trial’s objectives include the evaluating the number of bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life (HRQoL) and proxy HRQoL with aspects of caregiver burden.

Emicizumab is an investigational bispecific monoclonal antibody that will bring together factors IXa and X, which are proteins needed to activate the natural coagulation cascade and restore the blood clotting process.

It is being assessed in pivotal phase III studies in people 12 years of age and older, both with and without inhibitors to factor VIII, and in children under 12 years of age with factor VIII inhibitors

Roche’s global product development head and chief medical officer Dr Sandra Horning said: “Managing haemophilia A with inhibitors to factor VIII is especially challenging for children and their caregivers, because bleeding is difficult to control and current treatments require frequent intravenous infusions.”


Image: Site Roche Basel. Photo: courtesy of F. Hoffmann-La Roche Ltd.