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Novo Nordisk receives FDA approval

Mads Krogsgaard Thomsen

The US Food and Drug Administration (FDA) has approved Novo Nordisk’s Biologics License Application for ESPEROCT for the treatment of adults and children with haemophilia A.

ESPEROCT is the brand name for turoctocog alfa pegol, N8-GP. It is indicated for use in adults and children with haemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding.

The largest pre-registration clinical programme conducted in haemophilia A

The approval is based on the results from the largest pre-registration clinical programme conducted in haemophilia A, which included 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 or every 3-4 days (twice-weekly) in children. 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.

“We are excited about the approval of ESPEROCT in the US, 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 will provide people with haemophilia A a less burdensome and simple, fixed dosing regimen for prophylaxis and treatment of bleeding episodes, resulting in improved quality of life.”

Due to third-party IP agreements, Novo Nordisk will not be able to launch ESPEROCT before 2020 in the USA.

Photo of Mads Krogsgaard Thomsen: Novo Nordisk