Novo Nordisk’s Esperoct Approved to Treat Individuals with Hemophilia A

In February, the U.S. Food and Drug Administration approved Esperoct (antihemophilic factor[recombinant], glycopegylatedexei), an extended half-life factor VIII molecule for replacement therapy in people with hemophilia A, which provides a 1.6- fold half-life prolongation in adults and adolescents and a 1.9-fold half-life prolongation in children, compared to standard half-life factor VIII products. Esperoct is specifically indicated for use in adults and children with hemophilia A for on-demand treatment and control of bleeding episodes, perioperative management of bleeding and routine prophylaxis to reduce the frequency of bleeding episodes.

Approval was based on the PATHFINDER program, five prospective, multicenter clinical trials in 270 previously treated patients (202 adults/adolescents and 68 children) with severe hemophilia A (less than 1 percent endogenous FVIII activity) and no history of inhibitors. Total exposure to Esperoct was 80,425 exposure days corresponding to 889 patient years of treatment. Esperoct was shown to provide effective routine prophylaxis in people with severe hemophilia A through a fixed dosing regimen of one injection every four days in adults and adolescents or every three to four days (twice-weekly) in children. It provided effective prophylaxis and maintained a low median ABR of 1.18 when dosed at 50 IU/kg every four days in adults and adolescents. It was also found to be efficacious in treatment and control of bleeding episodes and perioperative management.