FDA grants Priority Review to Genentech's emicizumab for Hemophilia A with inhibitors

Application based on positive results of Phase III study in adults and adolescents with hemophilia A with inhibitors and interim Phase III results in children.

“Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “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 the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible.”

The BLA for emicizumab is based on results from the Phase III HAVEN 1 study in adults and adolescents 12 years of age and older, as well as interim results from the Phase III HAVEN 2 study in children younger than 12 years of age. Results from HAVEN 1 were published in The New England Journal of Medicine (NEJM) and results from both studies were presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Congress in July 2017.

The FDA is expected to make a decision on approval by 23 February 2018. 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. The FDA granted Breakthrough Therapy Designation for emicizumab in adults and adolescents with hemophilia A with inhibitors in September 2015. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate substantial improvement over existing therapies.

Data from both HAVEN 1 and HAVEN 2 have also been submitted for approval consideration to the European Medicines Agency (EMA) and will be reviewed under accelerated assessment. Additional studies evaluating emicizumab in people with hemophilia A both with and without inhibitors and exploring less frequent dosing regimens are ongoing.