Roche emicizumab positive result for children with haemophilia A

Roche interim results from the phase III HAVEN 2 study shows emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds in children less than 12 years of age with haemophilia A and inhibitors to factor VIII. According to the analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time.

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, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN2 study were injection site reactions and nasopharyngitis. Two additional phase III studies of emicizumab are also in progress ie; HAVEN 3 and HAVEN 4.

MD, Chief Medical Officer and Head of Global Product Development, Sandra Horning said that 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.

HAVEN 2 is a single-arm, multicentre, open-label, phase III study evaluating the efficacy, safety, and pharmacokinetics of once weekly subcutaneous administration of emicizumab. The aim of the study is to evaluate the number of bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life and proxy HRQoL with aspects of caregiver burden.

Emicizumab is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process. It can be administered by an injection of a ready-to-use solution under the skin once weekly. It is being evaluated 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.

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII.