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Chugai's HEMLIBRA® Receives Breakthrough Therapy Designation from U.S. FDA for Hemophilia A Without Factor VIII InhibitorsChugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its anti-coagulation factor IXa/X humanized bispecific monoclonal antibody / coagulation factor VIII substitute, "HEMLIBRA®" [US generic name: emicizumab-kxwh] for people with hemophilia A without factor VIII inhibitors. Development and distribution of the drug in the US is conducted by Genentech, a member of Roche Group. "We are thrilled that HEMLIBRA has been granted its second Breakthrough Therapy Designation," said Chugai's Executive Vice President, Co-Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. "This will allow us to expedite potential delivery of this new therapy we created to people with hemophilia A without inhibitors in the US following the previous designation for inhibitors. We continue to work closely with Genentech to enable this line extension as soon as possible." This designation is based on the global phase III HAVEN 3 (NCT02847637) study evaluating HEMLIBRA subcutaneous injection once a week and once every two weeks in people with hemophilia A (12 years of age or older) without inhibitors to factor VIII. Hemophilia A is a disease presenting repeated severe bleeding symptoms. In this disease, the blood coagulation reaction does not proceed normally due to the deficiency or functional disorder of coagulation factor VIII. For people with hemophilia A without inhibitors, regular factor VIII replacement therapy has been widely used to prevent bleeding. HEMLIBRA 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, HEMLIBRA provides the cofactor function of factor VIII in people with hemophilia A, who either lack or have impaired coagulation function of factor VIII1, 2). In the HAVEN 3 study, a statistically significant reduction in the frequency of bleeding episodes was observed with HEMLIBRA. With the convenience of subcutaneous administration and the lower frequency of administration, it is expected to be a new treatment option for hemophilia A. This is the sixth Breakthrough Therapy Designation received fr three drugs created by Chugai: ALECENSA® (ALK-positive non-small cell lung cancer with disease progression on crizotinib, and first line treatment for ALK-positive non-small cell lung cancer), ACTEMRA® (systemic sclerosis and giant cell arteritis), and HEMLIBRA (prophylactic treatment for patients 12 years or older with hemophilia A with factor VIII inhibitors). Trademarks used or mentioned in this release are protected by law.
References
About Breakthrough Therapy
Main approval status of the drug EU: In Europe, the drug (EU product name: HEMLIBRA®; Roche) obtained regulatory approval from the European Commission and was marketed for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors in February 2018. Japan: The Ministry of Health, Labour and Welfare has approved HEMLIBRA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with congenital factor VIII deficiency (hemophilia A) with factor VIII inhibitors in March 2018.
About the results of HAVEN 3 study
About Chugai
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