Novo Nordisk announced the completion of a phase 3 clinical trial of N8-GP (turoctocog alfa pegol), a third-generation, long acting recombinant factor VIII (rFVIII) product intended for prevention and treatment of bleeding in people with hemophilia A. Known as pathfinderT2, the multinational trial was designed to evaluate the safety and efficacy of N8-GP when used for prophylaxis and in on-demand situations in patients with hemophilia A who are 12 years of age or older.
In the trial, 175 patients were treated with a prophylactic regimen every fourth day, while 11 received on-demand treatment in response to bleeds. Patients were treated for up to 21 months. Their median annualized bleeding rates were 1.3 and 30.9 episodes for patients treated prophylactically and on-demand, respectively.
Pharmacokinetic data showed a single dose half-life of 18.4 hours. In addition, N8-GP appeared to have a safe profile and to be well tolerated. In all, one patient who responded well to prophylactic treatment throughout the trial developed an 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,” 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 pathfinderT program to be finalized within the next 12 months. These trials investigate N8-GP as a treatment for pediatric patients, in surgical procedures and as a weekly prophylactic treatment.
Source: Drug Discovery and Development, March 19, 2014
