Roche (SIX: RO, ROG; OTCQX: RHHBY) today 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).
“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,” said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. “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.”
“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,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “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.”
Data from the HAVEN 3 study showed that Hemlibra prophylaxis administered subcutaneously every week or every two weeks significantly reduced treated bleeds by 96% (rate ratio [RR]=0.04; p<0.0001) and 97% (RR= 0.03; p<0.0001), respectively, compared to no prophylaxis. Results also showed that 55.6% (95% CI: 38.1; 72.1) of people treated with Hemlibra every week and 60% (95% CI: 42.1; 76.1) of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% (95% CI: 0.0; 18.5) 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% (RR=0.32; p<0.0001) in treated bleeds, making it the first medicine to show superior efficacy to prior factor VIII prophylaxis treatment, the current standard of care. There were no unexpected or serious adverse events (AEs) related to Hemlibra in the HAVEN 3 study, and the most common AEs were consistent with previous studies. The most common AEs occurring in 5% or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.
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. The FDA is expected to make a decision on approval by 4 October 2018. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate substantial improvement over existing therapies. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease. In addition, the company’s Marketing Authorisation Application (MAA) variation of Hemlibra for haemophilia A without factor VIII inhibitors is under review by the European Medicines Agency (EMA). Submissions to other regulatory authorities around the world are ongoing.