Positive phase III results for Roche?s Hemlibra for haemophilia A without factor VIII inhibitors published in New England Journal of Medicine
Positive phase III results for Roche?s Hemlibra for haemophilia A without factor VIII inhibitors published in New England Journal of Medicine
- Hemlibra prophylaxis significantly reduced bleeds compared to no prophylaxis
- Hemlibra is the first medicine to demonstrate superior efficacy to prior factor VIII prophylaxis based on a statistically significant reduction in treated bleeds in an intra-patient comparison
- Hemlibra is currently under Priority Review by the FDA for people with haemophilia A without factor VIII inhibitors
About HAVEN 3 (NCT02847637)
HAVEN 3 is a randomised, multicentre, open-label, phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with haemophilia A without factor VIII inhibitors. The study included 152 patients with haemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis. Patients previously treated with on-demand factor VIII were randomised in a 2:2:1 fashion to receive subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk for at least 24 weeks (Arm A), subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks (Arm B) for at least 24 weeks or no prophylaxis (Arm C) for at least 24 weeks. Patients previously treated with factor VIII prophylaxis received subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm D). Episodic treatment of breakthrough bleeds with factor VIII therapy was allowed per protocol. The phase III HAVEN 3 study in people with haemophilia A without factor VIII inhibitors met its primary endpoint and key secondary endpoints. Data from the study showed:- Hemlibra prophylaxis every week or every two weeks resulted in a 96% (RR=0.04; p<0.0001) and 97% (RR=0.03; p<0.0001) reduction in treated bleeds, respectively, compared to no prophylaxis.
- 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.
- 91.7% (95% CI: 77.5, 98.2) of people treated with Hemlibra prophylaxis every week and 94.3% (95% CI: 80.8, 99.3) of people treated with Hemlibra prophylaxis every two weeks experienced three or fewer treated bleeds, compared to 5.6% (95% CI: 0.1, 27.3) of people treated with no prophylaxis.
- Hemlibra prophylaxis every week or every two weeks resulted in a 95% (RR=0.05; p<0.0001) and 95% (RR=0.05; p<0.0001) reduction in treated target-joint bleeds, respectively, compared to no prophylaxis.
- Hemlibra prophylaxis every week or every two weeks resulted in a 95% (RR=0.05; p<0.0001) and 94% (RR=0.06; p<0.0001) reduction in all bleeds, respectively, compared to no prophylaxis.
- Hemlibra prophylaxis every week demonstrated a statistically significant reduction of 68% (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% or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.