CHMP elicits positive response for Chugai’s HEMLIBRA

For routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors, Chugai Pharmaceuticals sought positive response from EU Committee for Medicinal Products for Human Use for HEMLIBRA  (emicizumab).

Emicizumab is a bispecific monoclonal antibody, which was developed using Chugai’s proprietary antibody engineering technologies. The drug is designed to bind factor IXa and factor X. In doing so, emicizumab provides the cofactor function of factor VIII in people with hemophilia A, who either lack or have impaired coagulation function of factor VIII. In November 2017, the drug (US product name: HEMLIBRA  (Genentech) was approved by the FDA for “routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.

Chugai’s Senior Vice President, Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito said “Existing treatments for people with hemophilia A with inhibitors need frequent intravenous injection every week, which remains a burden for both patients and their caregivers. HEMLIBRA has the benefit of enabling once-weekly subcutaneous (under the skin) injection. We are committed to deliver the drug to patients as early as possible in collaboration with Roche.”

Positive response is based on  two essential studies for hemophilia A with inhibitors: the results of HAVEN1 study (NCT02622321) for adolescents and adults, and the interim analysis of HAVEN2 study (NCT02795767) in children.