New data shows substantial quality of life improvements for hemophilia patients

Genentech/Roche announced new longer-term results from the pivotal HAVEN 1 and HAVEN 2 studies of Hemlibra (emicizumab-kxwh) in people with hemophilia A with inhibitors to factor VIII. The data shows once-weekly subcutaneous Hemlibra prophylaxis demonstrated superior efficacy compared to prior treatment with bypassing agents (BPAs) as prophylaxis or on-demand. In updated results from the HAVEN 2 study with six additional months of data and 40 more children (younger than 12 years of age), 94.7 percent (95 percent CI: 85.4; 98.9) of children with hemophilia A with inhibitors who received Hemlibra prophylaxis had zero treated bleeds (n=57). The intra-patient analysis comparing the effects of different therapies in the same child (n=13) showed a 99 percent reduction in treated bleeds with Hemlibra prophylaxis compared to prior treatment with a BPA, either as prophylaxis (n=12) or on-demand (n=1). Substantial improvements in health-related quality of life and aspects of caregiver burden, measured by the haemophilia-specific quality of life short form (Haemo-QoL-SF) and adapted health-related quality of life in hemophilia patients with inhibitors (Inhib-QoL) questionnaires, were also observed with Hemlibra prophylaxis compared to prior BPA prophylaxis.

With nearly 10 additional months of follow-up, updated results from the HAVEN 1 intra-patient analysis of adults and adolescents showed an 88 percent (risk rate [RR]=0.12, 95 percent CI: 0.05; 0.28) reduction in treated bleeds with Hemlibra prophylaxis compared to prior BPA prophylaxis (n=24). The results also showed a 95 percent (RR=0.05, 95 percent CI: 0.02; 0.12) reduction in treated bleeds in patients who received Hemlibra prophylaxis compared to prior on-demand BPA treatment (n=24). After more than one year, substantially more patients continued to experience zero bleeds with Hemlibra prophylaxis compared to their prior prophylaxis or on-demand BPA treatment across bleed endpoints, including treated bleeds and all bleeds. The previously reported improvement in health status after 24 weeks, measured by the haemophilia-specific quality of life (Haem-A-QoL) and EuroQol 5-Dimensions 5-level (EQ-5D-5L) questionnaires, was also maintained with longer follow-up.

These new data from the largest pivotal studies in people with hemophilia A with inhibitors further support Hemlibra as an important new treatment option for these adults, adolescents and children. These data were presented at the 59th American Society of Hematology (ASH) Annual Meeting.

Comment: Based on earlier results from the HAVEN 1 and HAVEN 2 studies, Hemlibra was approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with inhibitors. Data from HAVEN 1 and HAVEN 2 are also being reviewed under accelerated assessment by the EMA and submissions to health authorities around the world are ongoing.