CHMP recommends approval of Esperoct in haemophilia A

Novo Nordisk announced that the Committee for Medicinal Products for Human Use (CHMP), under the European Medicines Agency (EMA), adopted a positive opinion for the use of Esperoct (turoctocog alfa pegol, N8-GP) recommending marketing authorisation for the treatment of adolescents and adults with haemophilia A. The CHMP recommends Esperoct, the brand name for turoctocog alfa pegol, N8-GP, to be indicated for prophylaxis and on-demand treatment of bleeding as well as for surgical procedures in adolescents (at least 12 years of age) and adults with haemophilia A (congenital factor VIII deficiency). The recommendation is based on the results from the largest pre-registration clinical programme conducted in haemophilia A, with inclusion of 270 previously treated people (PTPs) with severe haemophilia A and more than 5 years of clinical exposure.

Esperoct was shown to provide effective routine prophylaxis in people with severe haemophilia A through a simple, fixed dosing regimen of one injection every 4 days in adults and adolescents. Esperoct provided effective prophylaxis and maintained a low median ABR of 1.18 when dosed at 50 IU/kg every 4 days in adults and adolescents. Furthermore, Esperoct was found to be efficacious in treatment and control of bleeding episodes and perioperative management. Across the clinical trials and age groups, Esperoct was well tolerated and no safety concerns were identified. The overall safety profile of Esperoct is similar to what has been reported for other long-action FVIII products.