The following is an excerpt from the press release from Genentech. Read the full press release here.
Genentech, a member of the Roche Group, announced the full results from the Phase III HAVEN 3 study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis administered every week or every two weeks in people with hemophilia A without factor VIII inhibitors and the Phase III HAVEN 4 study evaluating HEMLIBRA prophylaxis administered every four weeks in people with hemophilia A with or without factor VIII inhibitors. Data from both pivotal studies were presented today as late-breaking abstracts at the World Federation of Hemophilia 2018 World Congress in Glasgow, Scotland.
“HEMLIBRA is the first medicine to show superior efficacy to prior factor VIII prophylaxis, the current standard of care therapy, as demonstrated by a statistically significant reduction in treated bleeds in the HAVEN 3 study intra-patient comparison,” said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. “Even with current prophylactic treatments, many people with hemophilia A continue to have bleeds that can lead to long-term joint damage, and there is a need for more treatment options.”
In the Phase III HAVEN 3 study, adults and adolescents aged 12 years or older without factor VIII inhibitors who received HEMLIBRA prophylaxis every week or every two weeks showed a 96 percent and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis. In addition, 55.6 percent of people treated with HEMLIBRA every week and 60 percent of people treated with HEMLIBRA every two weeks experienced zero treated bleeds, compared to 0 percent of people treated with no prophylaxis. Importantly, in an intra-patient comparison, in people who were previously enrolled in a prospective non-interventional study (NIS), once-weekly HEMLIBRA prophylaxis showed superior efficacy compared to prior factor VIII prophylaxis, the standard of care for people with hemophilia A without factor VIII inhibitors, as demonstrated by a 68 percent reduction in treated bleeds. Additionally, 93.7 percent of all participants who completed a treatment preference survey preferred HEMLIBRA to their previous hemophilia treatment, with 97.8 percent of those in the intra-patient comparison preferring HEMLIBRA to their prior factor VIII prophylaxis. There were no unexpected or serious adverse events (AEs) related to HEMLIBRA, and the most common AEs were consistent with previous studies. The most common AEs occurring in 5 percent or more of people in the HAVEN 3 study were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.
Read the complete press release here.