Novo Nordisk recently announced that they have submitted a Biologics License Applications (BLA) to the U.S. Food and Drug Administration for N8-GP (tuco-tuco alfa pegol), a recombinant, extended half-life factor VIII (FVIII) product intended for the prevention and treatment of bleeding in people with hemophilia A.
The submission was based largely on positive results from a multi-stage clinical trial program known as Pathfinder, which included more than 250 children and adults with hemophilia A worldwide. Pathfinder was designed to evaluate the efficacy and safety of N8-GP in these patients, including those undergoing surgery. Adults treated prophylactically with N8-GP every fourth day experienced a median annualized bleeding rate of 1.3 episodes compared to 30.9 episodes for those who were treated via an on-demand basis. Pediatric participants experienced a median annualized bleeding rate of 1.95 episodes when administered twice weekly. All surgical procedures included in the trial were performed effectively with bleeding controlled through the use N8-GP and with clinical efficacy reported as 'excellent' or 'good' in 43 out of the 45 performed surgeries. In addition, trial investigators reported that N8-GP was safe and well-tolerated across Pathfinder.
“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.”
Source: Globalnewswire.com, February 27, 2018