Emerging drugs for hemophilia A: insights into phase II and III clinical trials

Authors: Kizilocak, H; Young, G.

Affiliations: Children’s Hospital Los Angeles, Hemostasis and Thrombosis Center, Cancer and Blood Disease Institute, Los Angeles, CA, USA ; University of Southern California, Keck School of Medicine, Los Angeles, CA, USA.

Publication: Expert opinion on emerging drugs. October 2021

ABSTRACT: INTRODUCTION: Hemophilia is a lifelong, genetic bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia. AREAS COVERED: This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies. EXPERT OPINION: Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.