Hemlibra results from HAVEN 3 trial for haemophilia A prophylaxis in patients without factor VIII inhibitors was published in NEJM.- Genentech/Roche.

Genentech, a member of the Roche Group announced that pivotal data from the Phase III HAVEN 3 study, which evaluated Hemlibra (emicizumab-kxwh) prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors, were published in the August 30, 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 hemophilia A without factor VIII inhibitors, while offering multiple subcutaneous dosing options,� said Dr. 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 hemophilia research and reinforces the potential of Hemlibra to change the standard of care for people with hemophilia A.�

The Phase III HAVEN 3 study in people with hemophilia A without factor VIII inhibitors met its primary endpoint and key secondary endpoints. Data from the study showed: 1.Hemlibra prophylaxis every week or every two weeks resulted in a 96 percent (RR=0.04; p<0.0001) and 97 percent rr="0.03;" p><0.0001) reduction in treated bleeds respectively compared to no prophylaxis. 2. 55.6 percent 95 percent ci: 38.1 72.1 of people treated with hemlibra every week and 60 percent 95 percent ci: 42.1 76.1 of people treated with hemlibra every two weeks experienced zero treated bleeds compared to 0 percent 95 percent ci: 0.0 18.5 of people treated with no prophylaxis. 3. 91.7 percent 95 percent ci: 77.5 98.2 of people treated with hemlibra prophylaxis every week and 94.3 percent 95 percent ci: 80.8 99.3 of people treated with hemlibra a prophylaxis every two weeks experienced three or fewer treated bleeds compared to 5.6 percent 95 percent ci: 0.1 27.3 of people treated with no prophylaxis. 4. hemlibra prophylaxis every week or every two weeks resulted in a 95 percent rr="0.05;" p><0.0001) and 95 percent rr="0.05;" p><0.0001) reduction in treated target-joint bleeds respectively compared to no prophylaxis. 5. hemlibra prophylaxis every week or every two weeks resulted in a 95 percent rr="0.05;" p><0.0001) and 94 percent rr="0.06;" p><0.0001) reduction in all bleeds respectively compared to no prophylaxis .6. hemlibra prophylaxis every week demonstrated a statistically significant reduction of 68 percent rr="0.32;" p><0.0001) in treated bleeds compared to prior factor viii prophylaxis based on an intra-patient comparison of people who were previously enrolled in a prospective non-interventional study.>

There were no unexpected or serious AEs related to Hemlibra, and the most common AEs were consistent with previous studies. No thrombotic events or cases of thrombotic microangiopathy were observed. The most common AEs occurring in 5 percent or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.