Chugai’s HEMLIBRA Receives Priority Review Status from FDA for Hemophilia A Without Inhibitors

Chugai Pharmaceutical announced that the U.S. Food and Drugs Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) and granted Priority Review for HEMLIBRA [US generic name: emicizumab-kxwh] for adults and children with hemophilia A without factor VIII inhibitors. The FDA is expected to make a decision on approval by 4 October, 2018. In the US, Genentech, Inc., a member of Roche Group is the BLA holder and is engaged in the development and distribution of HEMLIBRA, which was approved and launched for hemophilia A with inhibitors in November 2017.

“We are very proud that HEMLIBRA has been granted a second Priority Review for hemophilia A without inhibitors following the previous designation for hemophilia A with inhibitors, which underscores the innovation of this drug,” said Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. “In treatment of hemophilia A without inhibitors, there still remain unmet medical needs in the management of bleeding. Given this Priority Review status, we hope to be able to contribute to people with hemophilia A and their caregivers as soon as possible by providing HEMLIBRA as a new treatment option that can be administered subcutaneously with good bleeding control and eased treatment burden.”

The sBLA filing and Priority Review are based on the results of HAVEN 3 study (NCT02847637), which has been conducted with adults and adolescents with hemophilia A without factor VIII inhibitors under a collaboration between Chugai, Roche and Genentech. In EU, Roche has already filed the results to the European Medicines Agency for a supplemental indication of hemophilia A without inhibitors.

Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in safety or effectiveness of the treatment, prevention or diagnosis of a serious disease. In Japan, HEMLIBRA received regulatory approval in March 2018 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 and, in April 2018, was filed to the Ministry of Health, Labour and Welfare for a supplemental indication of hemophilia A without inhibitors.

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