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FDA grants priority review for emicizumab, an investigational bispecific monoclonal antibody, for treating hemophilia A with factor VIII inhibitors.
Genentech, a member of the Roche Group, announced on August 23, 2017 that FDA has accepted its biologics license application (BLA) and granted priority review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for hemophilia A with factor VIII inhibitors. Nearly one in three people with hemophilia A develop inhibitors to standard factor VIII replacement therapies, which limits treatment options and increases the risk of life-threatening bleeds and repeated bleeds, particularly in joints, that cause long-term damage, according to the company.
FDA is expected to make a decision on approval by Feb. 23, 2018. Priority review designation is granted to medicines that 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. Earlier in Sept. 2015, the agency granted breakthrough therapy designation for emicizumab in this indication.
Data from the Phase III clinical trials on which the BLA is based was submitted in June 2017 for approval consideration to the European Medicines Agency and will be reviewed under accelerated assessment. In addition, it was filed for approval consideration in Japan in July 2017. Additional studies evaluating emicizumab in people with hemophilia A both with and without inhibitors and exploring less frequent dosing regimens are ongoing.
“Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, in a company press release. “Results of our Phase III study in adults and adolescents as well as early Phase III results in children showed that emicizumab has significant potential to help people with hemophilia A with inhibitors, who face major challenges in preventing and treating bleeds. We are working with FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible.”
Emicizumab is an investigational bispecific monoclonal antibody designed to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood clotting process. The biologic was created by Chugai Pharmaceutical, also a member of the Roche Group, and is being co-developed by Chugai, Roche, and Genentech.