Date: 2014-03-19
Type of information: Initiation of preclinical development
phase: 3
Announcement: completion of the study
Company: Novo Nordisk (Denmark)
Product: N8-GP (turoctocog alfa pegol)
Action
mechanism: N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life. The modification that renders N8-GP long-acting is located in the B-domain whereby the active factor VIII generated by thrombin activation is identical to endogenous FVIII and to activated turoctocog alfa.
Disease: hemophilia A
Therapeutic area: Hematologic diseases - Genetic diseases - Rare diseases
Country:
Trial
details: PathfinderT is a Novo Nordisk registered trademark for trials conducted with N8-GP. The programme includes more than 200 patients with hemophilia A investigating pharmacokinetics, safety and efficacy of N8-GP in adults and children as well as patients undergoing surgery.
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.
Latest
news: * On March 19, 2014, Novo Nordisk has 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 annualised 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.
Novo Nordisk is expecting the three remaining trials in the pathfinderT programme to be finalised within the next 12 months. These trials investigate N8-GP as a treatment for paediatric patients, surgical procedures and as once-weekly prophylactic treatment.
