Phase III Trial for Long-Acting Factor VIII Shows Positive Results
- By BSTQ Staff
Positive results have been shown with the completion of pathfinder2, the first multinational Phase III trial evaluating the safety and efficacy of Novo Nordisk’s long-acting recombinant factor VIII (FVIII), N8-GP (turoctocog alfa pegol), for hemophilia A patients 12 years and older.
In the trial, 175 patients were treated with a prophylactic regimen of 50 U/kg every fourth day, and 11 patients received on-demand treatment when bleedings occurred. Patients were treated for up to 21 months, resulting in median annualized bleeding rates of 1.3 and 30.9 episodes for patients 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 8 percent measured immediately before the next dose for patients on prophylaxis treatment. Among the 186 patients, one patient who responded well to prophylactic treatment throughout the trial developed an FVIII inhibitor, which is in line with expectations in a population of previously treated hemophilia A patients.
“We are very pleased with the results of pathfinder2,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. “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.”
Novo Nordisk is expecting the three remaining trials in the pathfinder program to be finalized within the next 12 months. These trials are investigating N8-GP as a treatment for pediatric patients, surgical procedures and as once-weekly prophylactic treatment.
