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Novo Nordisk files for regulatory approval of long-acting factor VIII (N8-GP) in the US and the EU for treatment of haemophilia A
Bagsværd, Denmark, 27 February 2018 - Novo Nordisk today announced the submission of a Biologics License Applications (BLA) to the US Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for N8-GP, an extended half-life factor VIII for treatment of people with haemophilia A.
The submission is based on results from the pathfinder clinical trial programme. The pathfinder programme included more than 250 people with haemophilia A and investigated efficacy and safety of N8-GP in adults and children as well as people undergoing surgery. In the trial, adults treated prophylactically with N8-GP every fourth day experienced a median annualised bleeding rate of 1.3 episodes compared to 30.9 episodes for people treated on-demand. Paediatric participants experienced a median annualised bleeding rate of 1.95 episodes when administered twice weekly. In the surgery trial, all surgeries were effectively performed with N8-GP, and clinical efficacy evaluated by haemostatic response was reported as 'excellent' or 'good' in 43 out of the 45 performed surgeries.
Across the pathfinder clinical trial programme, N8-GP demonstrated a safe and well-tolerated profile.
"We are excited to file the regulatory application for long-acting N8-GP in the US and EU," said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. "Based on the results from the global pathfinder clinical trial programme, we believe N8-GP can reduce the burden of treatment by decreasing the number of intravenous infusions while achieving the benefits in terms of efficacy and safety for people with haemophilia A."
N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life. The site specific glycopegylation is within the truncated B-domain. N8-GP is a B-domain modified form of turoctocog alfa and hence the active factor VIII generated by thrombin activation is identical to both activated endogenous FVIII and turoctocog alfa.
About the pathfinder phase 3a clinical trial programme
The pathfinder programme included more than 250 people with haemophilia A and investigated safety and efficacy of N8-GP, treatment of bleeds, prophylaxis and surgery, in adults as well as children.
Pathfinder 2 was a global trial evaluating safety and efficacy of N8-GP, when administered for prophylaxis and on-demand treatment in people with haemophilia A, who are 12 years or older. 175 people were treated with a prophylactic regimen of 50 IU/kg every fourth day, and 12 people received on-demand treatment. People were treated for up to 21 months, resulting in median annualised bleeding rates of 1.3 and 30.9 episodes for people treated prophylactically and on-demand, respectively. The pharmacokinetic data documented a single dose half-life of 18.4 hours and a mean trough level of 4% measured immediately before next dose for people on prophylaxis treatment.
Pathfinder 3 was a surgery trial which was performed as an open-label, multi-national trial evaluating the efficacy and safety of N8-GP when administered for perioperative management in previously treated patients with severe haemophilia A who were 12 years or older. The trial included 33 people who underwent 45 major surgeries. People received a single preoperative dose of N8-GP, at a median of 51 IU/kg. All surgeries were effectively performed with N8-GP, and clinical efficacy evaluated by haemostatic response was reported as 'excellent' or 'good' in 43 out of the 45 performed surgeries. N8-GP appeared to have a safe and well-tolerated profile.
Pathfinder 5 was a paediatric trial which was performed as a global trial evaluating safety and efficacy for N8-GP, when administered for prophylaxis in previously treated children with haemophilia A, who were between 0 to 11 years old. In the trial, 34 children 0-5 years of age and 34 children 6-11 years of age received prophylactic treatment and treatment of bleeding episodes. All children were treated with a regimen of 50-75 IU/kg twice weekly for 26 weeks. Median annualised bleeding rates of 1.95 episodes were obtained in the trial. N8-GP appeared to have a safe and well-tolerated profile.
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Company announcement No 21 / 2018Attachment: