Bagsværd, Denmark, 26 April 2019 – Novo Nordisk today 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 (>=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.
“We are happy to receive the positive opinion for Esperoct® in the EU, and we consider it an important expansion of the treatment options Novo Nordisk can offer people with haemophilia A,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. “We are confident that Esperoct® can provide a less burdensome and simple dosing regimen for prophylaxis and treatment of bleeding episodes, resulting in improved quality of life for people with haemophilia A.”
Esperoct® (turoctocog alfa pegol, N8-GP) is an extended half-life factor VIII molecule for replacement therapy in people with haemophilia A, which provides a 1.6-fold half-life prolongation in adults/adolescents compared to standard half-life factor VIII products.
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.