Roche announces data analysis of the phase III HAVEN 2 study

Roche has announced data from the primary analysis of the phase III HAVEN 2 study evaluating Hemlibra® (emicizumab) prophylaxis in children 11 years and younger with haemophilia A with factor VIII inhibitors. The data showed that the drug provided sustained bleed control, with close to 77% of children receiving Hemlibra once weekly experiencing zero treated bleeds. Hemlibra once weekly reduced treated bleeds by 99% compared to prior bypassing agents in a prospective intra-patient comparison, and Hemlibra every two weeks and every four weeks also showed clinically meaningful control of bleeding.
Guy Young, MD, Director of Haemostasis and Thrombosis Centre, Children’s Hospital Los Angeles, and Professor of Paediatrics, University of Southern California Keck School of Medicine, Los Angeles, said: “Children with inhibitors are at increased risk of life-threatening bleeds and may experience frequent, repeated bleeding into joints. These updated data from HAVEN 2 showed that the majority of children with haemophilia A with factor VIII inhibitors treated with emicizumab had zero treated bleeds across three different dosing schedules, reinforcing the ability of this medicine to provide sustained, effective bleed control.”

Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development, said: “The updated analysis from the HAVEN 2 study supports the potential of Hemlibra to control bleeds at less frequent subcutaneous dosing, providing parents and their children more flexibility to choose a treatment schedule that is right for them. Many children with haemophilia A with factor VIII inhibitors have already experienced the benefits of Hemlibra, and with these new positive data, we are confident that this treatment will continue to make a meaningful difference in their lives.”