Hemophilia A candidate emicizumab filed for approval in Japan

Japanese drugmaker Chugai Pharmaceutical (TYO: 4519) says it has filed a new drug application for its anti-coagulation factor IXa/X humanized bispecific monoclonal antibody emicizumab to the Ministry of Health, Labor and Welfare (MHLW).

The planned indication for the drug, under development by Chugai and its majority owner, Swiss pharma giant Roche (ROG: SIX), is 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.”

This filing is based on the results of HAVEN 1 study (NCT02622321) and the interim analysis of HAVEN 2 study (NCT02795767), both of which have been conducted under a collaboration between Chugai, Roche and the latter’s US subsidiary Genentech. The two studies have been carried out in hemophilia A patients with factor VIII inhibitors in order to evaluate the efficacy, safety and pharmacokinetics of the once-weekly subcutaneous injection of emicizumab, while HAVEN 1 is for adult and adolescent patients (12 years of age or older) and HAVEN 2 is for pediatric patients (younger than 12 years of age).