Positive Ph III results with Novo Nordisk’s N8-GP, a long-acting hemophilia A agent

Danish insulin giant Novo Nordisk (NOV: N) says it has completed the pathfinderT2, the first Phase III 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 50U/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.

Turoctocog alfa pegol is a glycopegylated form of turoctocog alfa designed for prolonged half-life. Turoctocog alfa was approved by the US Food and Drug Administration last year under the trade name NovoEight (The Pharma Letter October 16, 2013) and is awaiting a European decision following a positive opinion from a European Medicines Agency advisory panel. The product will join a market sector valued at around $5 billion a year. The major treatments for hemophilia A include recombinant factor VIII drugs such as NovoSeven (from Novo Nordisk), Kogenate, Advate, ReFacto, Xyntha and Feiba.