Novo Nordisk has submitted 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 company said that 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, the company said.
“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.”