Roche's emicizumab treats hemophilia A in children

Barsaba Taglieri
Aprile 18, 2017

Emicizumab was administered by subcutaneous injection, once weekly.

HAVEN 3 is evaluating emicizumab prophylaxis given once a week to patients 12 years old or older with hemophilia A but without inhibitors to factor VII; and HAVEN 4, which will evaluate emicizumab dosed every four weeks in patients 12 years old or older with hemophilia A with or without inhibitors to factor VII.

Release of the interim HAVEN 2 results coincides with World Hemophilia Day on April 17th. In that study, emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared with non-prophylaxis, as well as compared to prior prophylaxis with bypassing agents.

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.

Roche published encouraging data on the drug in December from a phase III study with adults. 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. 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.

According to Genetic Engineering & Biotechnology News, in February, the pharmaceutical company reported on the European Haemophilia Consortium website that a patient enrolled in HAVEN 1 had died, although they insisted that the death was unrelated to emicizumab treatment. Emicizumab prophylaxis is an investigational bispecific monoclonal antibody designed join factors IXa and X to restore the blood clotting process.

Emicizumab was originally discovered by Roche's majority-owned Chugai, and is in development by Roche, Chugai and Roche's wholly owned Genentech operation.

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