Emicizumab Beneficial in Hemophilia A Patients With Inhibitors to FVIII

The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P

Genentech announced positive data from its Phase 3 studies (HAVEN 1 and HAVEN 2) for emicizumab, showing substantial and clinically meaningful reduction in bleeds. 

These Phase 3 studies evaluated a once-weekly subcutaneous prophylactic dose of emicizumab for the treatment of hemophilia A with inhibitors to factor VIII. The data will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting.

The HAVEN 1 study compared emicizumab prophylaxis vs. on-demand (no prophylaxis; episodic only) and prophylactic use of bypassing agents (BPA) in adults and adolescents with hemophilia A with inhibitors. The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13;P<0.0001] with emicizumab prophylaxis.  The reduction in bleeding was consistent in all secondary endpoints: treated spontaneous bleeds, treated joint bleeds, and treated target joint bleeds. 

Another study arm included patients who had received BPA prophylaxis converted to emicizumab prophylaxis. An intra-patient analysis was performed in this arm, which included a subset of patients that had previously participated in a non-interventional study (NIS), showing a 79% [RR  0.21; P=0.0003] reduction in treated bleeds in patients receiving emicizumab vs. prior prophylaxis with BPAs. Common adverse events (AEs occurring in >5% of patients) were injection site reactions, headache, fatigue, upper respiratory tract infection, and athralgia. 

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The interim results from the single-arm HAVEN 2 study, in children <12 years of age who received emicizumab prophylaxis, also showed  positive data consistent with results from HAVEN 1. An intra-patient comparison of the subset also enrolled in the NIS, showed patients experienced a 100% reduction in treated bleeds after treatment with emicizumab. The most common AEs in the HAVEN 2 study were mild injection site reactions and nasopharyngitis.

Emicizumab is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X to activate the natural coagulation cascade and restore the clotting process. It is supplied as a ready-to-use solution for use as subcutaneous injection once weekly.

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