Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (US generic name: emicizumab-kxwh), a treatment for hemophilia A created by Chugai, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors, administered once weekly, every two weeks, or every four weeks. The FDA has also approved additional dosing options of every two weeks or every four weeks in adults and children with hemophilia A with factor VIII inhibitors. The US application was submitted by Genentech, a member of Roche Group.
“We are very pleased that HEMLIBRA has obtained its first regulatory approval for people with hemophilia A without inhibitors,” said Chugai’s President & CEO, Tatsuro Kosaka. “Now people with hemophilia A in the US can be offered flexibility in HEMLIBRA’s dosing interval from multiple options depending on their needs, regardless of their inhibitor expression. We anticipate that HEMLIBRA will make an even greater contribution to the advancement of treatment of hemophilia A.”
This regulatory approval is based on results from two Phase lll studies HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160), conducted jointly with Roche and Genentech. HAVEN 3 study was conducted to evaluate the reduction of bleed rate of 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. HAVEN 4 study was conducted to evaluate efficacy, safety, and pharmacokinetics of HEMLIBRA subcutaneous injection every four weeks in people with hemophilia A (12 years of age or older), with and without inhibitors to factor VIII.
HEMLIBRA was granted Priority Review and Breakthrough Therapy Designation by the FDA in hemophilia A without inhibitors, following the prior designations in hemophilia A with inhibitors. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention or diagnosis of a serious disease. In Japan and the EU, applications have been filed to regulatory authorities and are currently under review for an additional indication of prophylactic treatment for people with hemophilia A without inhibitors, as well as for additional dosage and administration as a biweekly or every four-week treatment for people with hemophilia A with inhibitors to factor VIII.