Turoctocog alfa and drug development for hemophilia A

Introduction: Prophylactic replacement therapy with factor VIII (FVIII) is recommended as the foundation of treatment for severe hemophilia A; however, access to such therapy remains limited in many parts of the world. Introducing new products into the market may increase treatment accessibility across the global hemophilia community. Turoctocog alfa, a new and recently developed B-domain–truncated recombinant FVIII concentrate, further expands the therapeutic armamentarium for the prevention and treatment of bleeding episodes in hemophilia A. The lessons learned and insights gained throughout the nonclinical and clinical development of turoctocog alfa may offer guidance for future research in hemophilia A. Areas covered: This review summarizes the development of turoctocog alfa from production methods through to Phase III clinical development and the outcomes achieved in the pivotal guardian™ trial program. Key references relating to each step of the development process have been considered. Expert opinion: The turoctocog alfa clinical development program has led not only to the availability of a new, effective FVIII product for the treatment of hemophilia A but it has also demonstrated that large multinational trials can be successfully and quickly completed in this rare patient population.