Bagsværd, Denmark, 19 February 2019 - Novo Nordisk today announced that the US Food and Drug Administration (FDA) has approved the 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. ESPEROCT® 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 approval of ESPEROCT® 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.
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 and a 1.9-fold half-life prolongation in children, compared to standard half-life factor VIII products.
About the pathfinder clinical programme
ESPEROCT® has been evaluated in 270 people (202 adults/adolescents and 68 children) in five prospective, multi-centre clinical trials in previously treated people (PTPs) with severe haemophilia A (<1% 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 well tolerated across all age groups and indications, and no safety concerns were identified after more than 5 years of clinical exposure.
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Company announcement No 11 / 2019