Novo Nordisk announced the completion of pathfinderT2, the first Phase 3 trial with long-acting recombinant factor VIII, N8-GP (turoctocog alfa pegol) for hemophilia A patients. PathfinderT2 is a multi-national trial evaluating safety and efficacy of N8-GP, when administered for prophylaxis and on-demand treatment in patients with hemophilia A who are 12 years or 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% measured immediately before next dose for patients on prophylaxis treatment.
N8-GP appeared to have a safe profile and to be well tolerated. Among the 186 patients in the trial, one patient who responded well to prophylactic treatment throughout the trial developed an FVIII inhibitor. This is in line with expectations in a population of previously treated hemophilia A patients.
“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,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.
Novo Nordisk is expecting the three remaining trials in the pathfinderT 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.
Date: March 19, 2014
Source: Novo Nordisk