Roche has announced that pivotal data from the phase III HAVEN 3 study, which evaluated Hemlibra® (emicizumab) prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or older with haemophilia A without factor VIII inhibitors, were published in the 30 August 2018 issue of the New England Journal of Medicine (NEJM).
Data from the HAVEN 3 study showed that Hemlibra prophylaxis administered subcutaneously every week or every two weeks significantly reduced treated bleeds by 96% and 97% respectively, compared to no prophylaxis. Results also showed that 55.6% of people treated with Hemlibra every week and 60% of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% of people treated with no prophylaxis.
In an intra-patient comparison of people who previously received factor VIII prophylaxis in a prospective non-interventional study and switched to Hemlibra prophylaxis, Hemlibra demonstrated a statistically significant reduction of 68% in treated bleeds, making it the first medicine to show superior efficacy to prior factor VIII prophylaxis treatment, the current standard of care.
Earlier this year, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation and Priority Review to Hemlibra for people with haemophilia A without factor VIII inhibitors based on data from the HAVEN 3 study.
Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa said, “In the HAVEN 3 study, Hemlibra showed a significant and clinically meaningful reduction in bleeds in people with haemophilia A without factor VIII inhibitors, while offering multiple subcutaneous dosing options. The publication of these results in the New England Journal of Medicine represents a major advance for haemophilia research and reinforces the potential of Hemlibra to change the standard of care for people with haemophilia A.”
Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development said, “Current prophylactic treatment options for people with haemophilia A can require frequent intravenous infusions, and despite treatment, many continue to have bleeds that can lead to long-term joint damage. Given the challenges many people face managing their haemophilia, we believe Hemlibra could make a meaningful difference, and we are working with health authorities to hopefully make this treatment available to people with haemophilia A without factor VIII inhibitors as soon as possible.”