Chugai Pharmaceutical Co., Ltd. announced that it filed applications for its anti-coagulation factor IXa/X humanized bispecific monoclonal antibody/coagulation factor VIII substitute, “Hemlibra subcutaneous injection 30 mg, 60 mg, 90 mg, 105 mg, and 150 mg” [generic name: emicizumab (genetical recombination)] to the Ministry of Health, Labour and Welfare (MHLW) for an additional indication of prophylactic treatment for people with haemophilia A without inhibitors to factor VIII, as well as for additional dosage and administration as a biweekly or every four-week treatment for people with haemophilia A with inhibitors to factor VIII.

These filings are based on the results from HAVEN 3 study (NCT02847637) and HAVEN 4 study (NCT03020160). HAVEN 3 study is a global phase III study evaluating Hemlibra subcutaneous injection, once a week and once every two weeks, in people with haemophilia A (12 years of age or older) without inhibitors to factor VIII. HAVEN 4 study is a global phase III study evaluating efficacy, safety, and pharmacokinetics of Hemlibra subcutaneous injection every four weeks in people with haemophilia A (12 years of age or older), with and without inhibitors to factor VIII. Based on the results from these studies, the filings were submitted for two purposes including: 1) expansion of the indication to include weekly, biweekly, or every four-week subcutaneous treatment for people with haemophilia A without inhibitors to factor VIII; and 2) addition of dosage and administration to include the biweekly or every four-week subcutaneous treatment for people with haemophilia A with inhibitors to factor VIII for whom the weekly injection has previously been approved.

“Hemlibra has recently been designated as a Breakthrough Therapy from the US Food and Drug Administration (FDA) for the treatment of haemophilia A without inhibitors to factor VIII. We are very glad that the filing to include this indication has been successfully submitted in Japan,” said Chugai’s executive vice president, co-head of project & lifecycle management unit, Dr. Yasushi Ito. “The purpose of the two filings is to obtain approval for weekly, biweekly, and every four-week subcutaneous injections of Hemlibra regardless of the presence of inhibitors to factor VIII. Chugai is committed to seek approval so that we may deliver Hemlibra to people with haemophilia A without inhibitors as a new therapeutic option, as well as to further improve convenience for people with inhibitors by extending the dosing interval as soon as possible.”