Thursday, August 24, 2017
Chugai Pharmaceutical Co., Ltd. announced that the US Food and Drugs Administration (FDA) has accepted the Biologics License Application (BLA) and has granted Priority Review for an investigational antibody emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. The FDA is expected to make a decision on approval by February 23, 2018.
“We expect that FDA’s review process for emicizumab has been expedited with the Breakthrough Therapy Designation granted in September 2015,” said Chugai’s Senior Vice President, Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. “There are limited treatment options for patients with hemophilia A with inhibitors. We are thrilled that the day is approaching when we can deliver this antibody with a novel mode of action to patients given this Priority Review status.”
Both the filing and the Priority Review designation were made 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 Genentech. HAVEN 1 is for adult and adolescent patients and HAVEN 2 is for pediatric patients.
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. Outside US, emicizumab has been filed in the EU in June 2017 and in Japan in July 2017.
