Emicizumab for the prevention of bleeds in hemophilia A

Abstract

Introduction: The management of hemophilia A with and without inhibitors is challenging with high treatment burden of prophylactic regimens, musculoskeletal complications, poor treatment compliance, poor venous access and therapies with suboptimal levels. Areas covered: Emicizumab is a bispecific monoclonal antibody recently approved for prevention of bleeds in hemophilia A patients with and without inhibitors. This review is a synthesis of several recently completed emicizumab clinical trials with the emphasis of its mechanism of action, efficacy, safety, and pharmacokinetic profile when used for prevention of bleeds in hemophilia A patients of all age groups with and without inhibitors. We also review the role of emicizumab in the era of rapidly evolving novel therapies in hemophilia A. Expert opinion: Data from completed clinical studies indicate that emicizumab is poised to address current unmet needs in hemophilia A. It has high efficacy in the prevention of bleeds and a favorable safety profile. Subcutaneous dosing and versatile dosing regimens make emicizumab an ideal drug to address current unmet needs in hemophilia A management across all age groups. How emicizumab fits in the current rapidly evolving hemophilia A therapeutic landscape will be exciting to watch in the coming period.

Keywords: Emicizumab; Haven study; bispecific monoclonal antibody; clinical trials; efficacy; hemophilia A; pharmacokinetic; prophylaxis; safety.