Emicizumab and unmet needs of patients with hemophilia a who are managed with replacement therapies.

Abstract

INTRODUCTION Hemophilia A is managed with coagulation clotting factor VIII (FVIII) therapy that poses significant challenges, such as a high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Various non-factor therapies, such as several natural anticoagulant inhibitors and factor FVIII mimetics, have been developed to address these unmet needs. However, the role of emicizumab in addressing these unmet needs remains underexplored. AREAS COVERED This review delves into the evolution of hemophilia A replacement clotting therapy from plasma-derived products to recombinant products and, more recently, nonfactor therapies. It underscores the unmet needs of replacement therapy and explores the nonfactor therapies developed to address them. The review then comprehensively summarizes the clinical trial and real-world experience data, demonstrating how emicizumab tackles these unsatisfied demands. EXPERT OPINION Replacement clotting factor therapies as the standard of care has exposed several needs that have yet to be addressed. However, data from numerous emicizumab clinical trials and real-world experience offer a promising outlook, suggesting that it may effectively address many unmet needs. As hemophilia treatment goals continue to evolve, the role of currently developed nonfactor therapies in hemophilia management is yet to be fully defined.