An update of the current pharmacotherapeutic armamentarium for hemophilia A

Abstract

Introduction: For several decades, we have seen unprecedented advances in novel therapy development for hemophilia A. These advances address the unmet need of replacement therapy, and they include the development of recombinant products with improved pharmacokinetics, subcutaneously administered products, and those with better efficacy and safety profiles in hemophilia A management.

Areas covered: In this update of hemophilia A treatment, the author summarizes data from completed standard half-life FVIII products, extended half-life FVIII products and FVIII mimetic studies. All products have an acceptable safety profile. The standard half-life products, EHL-FVIII products and emicizumab are efficacious in the prevention and treatment of bleeds and for EHL-FVIII in the perisurgical setting.

Expert opinion: Advances in pharmacotherapy for hemophilia A have been characterized by changing care goals from supportive care to eliminating infections, preventing inhibitors, and more recently achieving zero bleeds in many patients. While gene therapy has the potential for functional cure in hemophilia A, it has many limitations which need to be addressed. Therefore, pharmacotherapy is likely to remain the mainstay in the management of hemophilia A and promises to get better with currently available therapies. Evolving factor and non-factor replacement therapies may also improve current unmet needs in hemophilia A management.

Keywords: emicizumab; extended half-life FVIII; hemophilia A; pharmacotherapy; rFVIII; standard half-life FVIII.