Novo Nordisk has announced positive results from a phase 3 trial with long-acting factor IX, glycopegylated recombinant factor IX (N9-GP), for treatment of haemophilia B.
The paradigm 2 multi-centre, blinded trial was designed to assess the safety and efficacy of N9-GP when used for on-demand or prophylactic treatment in haemophilia B patients.
Novo Nordisk executive vice president and chief science officer Mads Krogsgaard Thomsen said the strong results from the trial could represent a paradigm shift in the treatment of haemophilia B.
"The trial demonstrated that once-weekly prophylactic administration of N9-GP can reduce the risk of bleeds by more than 90% compared to on-demand treatment and enable 99% of the few occurring bleeds to be stopped with a single dose," Thomsen added.
The study treated 74 patients for six months on-demand, or 12 months by a prophylactic regimen of 40 U/kg or 10 U/kg N9-GP once a week.
The median bleeding rate of 15.6 episodes per year was noted for patients treated on-demand.
A median annualised bleeding rate of 1.0 and 2.9 episodes per year was noted for patients on prophylaxis, when treated with weekly doses of 40 U/kg and 10 U/kg, respectively.
In the group of patients randomised to receive 40 U/kg N9-GP, 99% of bleeding episodes were treated with only one infusion, and two-thirds experienced complete resolution of bleeding in their target joints, and an improvement in quality of life during the trial was observed.
A steady state half-life of 110 hours was documented in pharmacokinetic data.
N9-GP was safe and well-tolerated in the trial and no patients developed inhibitors, and no apparent differences between the treatment groups with respect to adverse events and standard safety parameters were observed.