Roche's emicizumab treats hemophilia A in children


HAVEN 2 is the second phase III study in the emicizumab clinical development programme to report results. Emicizumab was administered by subcutaneous injection, once weekly.

Roche said managing hemophilia A with inhibitors to factor VIII was especially challenging for children and their caregivers because bleeding was hard to control and current treatments required frequent intravenous infusions. HAVEN 1, in adolescent and adults aged 12 years and older with heophilia A and factor VIII inhibitors, showed that emicizumab prophylaxis resulted in a statistically significant, and clinically meaningful reduction in the number of bleeds when compared with no prophylaxis, or in comparison with prior prophylaxis with bypassing agents. HAVEN 2 is an open-label study and aims to enrol 60 children who receive emicizumab prophylaxis for 52 weeks. Data from both HAVEN 1 and the interim data from HAVEN 2 studies will be presented at an upcoming medical meeting and submitted to health authorities for approval consideration.

"At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time", Roche said in a statement.

Roche claimed that the patient's death was due to rectal hemorrhage, which is not related to emicizumab treatment.

Interim data from the Phase 3 HAVEN 2 study assessing Roche's (OTCQX:RHHBY) Genentech unit's emicizumab for reducing bleeding episodes in children younger than 12 years of age with hemophilia A showed a clinically meaningful treatment effect.

Hemophilia patients, whose blood does not clot properly, need life-saving infusions of clotting factors, but development of inhibitors in many of those being treated can interfere with efforts to control their bleeding.

The drug, called ACE910, was originally developed by Roche's majority-owned Chungai, and it is created to restore the normal blood clotting process and to bind IX and X factors.



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