Emicizumab shows positive results in hemophilia A

Positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII were announced yesterday by Swiss pharma giant Roche (ROG: SIX).

Data from both studies will be presented on July 10 at the 26th International Society on Thrombosis and Hemostasis (ISTH) Meeting in Berlin, Germany.

The Phase III HAVEN 1 study compared emicizumab prophylaxis with on-demand (no prophylaxis; episodic use only) and prophylactic use of bypassing agents (BPAs) in adults and adolescents with hemophilia A with inhibitors. The primary endpoint was treated bleeds, and results showed a statistically-significant and clinically meaningful reduction in bleed rate of 87% (risk rate [RR]=0.13, p<0.0001) with emicizumab prophylaxis compared with on-demand treatment with BPAs.