Novo Nordisk Completes Phase 3 Trial Of Hemophilia A Drug
Novo Nordisk announced excellent results from its recently completed Phase III trial evaluating ong-acting recombinant factor VIII, N8-GP (turoctocog alfa pegol) in patients with hemophilia A.
The trial labeled PathfinderT2 is a multi-national trial for the assessment of safety and efficacy of N8-GP when administered for the prevention and on-demand treatment in eligible patients with hemophilia A twelve years of age and older.
Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk, said, “We are very pleased with the results of pathfinderT2. These results show that N8-GP has the potential to reduce the burden of treatment by decreasing the number of intravenous infusions while achieving strong results in terms of efficacy and safety for people with hemophilia A.”
N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life. N8-GP contains a modification that renders it a long-acting treatment. This can be found in the B-domain where thrombin activation-generated active factor VIII is similar to endogenous FVIII and activated turoctocog alfa. Pathfinder is a registered trademark for trials conducted with N8-GP. The program involves more than 200 participants with hemophilia A evaluating pharmacokinetics, safety, and efficacy of N8-GP in adult and pediatric patients including those undergoing surgery.
175 patients were treated with prophylactic regimen of 50 U/kg every fourth day during the study, with 11 patients receiving on-demand treatment in cases of bleeding events. Patients were treated for up to 21 months, resulting in median annualized bleeding rates of 1.3 episodes for patients treated prophylactically and 30.9 for patients on-demand. The drug appeared to have sufficient safety and tolerability profile, said Novo Nordisk.
The company stated, “Novo Nordisk is expecting the three remaining trials in the pathfinder program to be finalized within the next 12 months. These trials investigate N8-GP as a treatment for pediatric patients, surgical procedures and as once-weekly prophylactic treatment.”