Novo Nordisk reports positive results from Phase-3 haemophilia B study of N9-GP

Paradigm 2 is a multi-centre, blinded study designed to assess the safety and efficacy of the glycopegylated recombinant factor IX as on-demand or prophylactic treatment against haemophilia B.

Novo Nordisk executive vice president and chief science officer Mads Krogsgaard Thomsen said, "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."

A total of 74 patients were treated on-demand for six months or by a prophylactic regimen of 40U/kg or 10U/kg N9-GP once a week for 12 months.

According to the data, 15.6 episodes per year was the median bleeding rate for patients treated on-demand.

Median annualised bleeding rate for patients on prophylaxis was 1.0 and 2.9 episodes per year, when treated with weekly doses of 40 U/kg and 10 U/kg, respectively.

Among patients randomised with 40 U/kg N9-GP, 99% of bleeding episodes were treated with only one infusion, two-thirds of the patients experienced complete resolution of bleeding in their target joints in addition to an improvement in quality of life during the trial.

A steady state half-life of 110 hours was documented in the pharmacokinetic data.

N9-GP was safe and well-tolerated in the trial where patients developed inhibitors.

No apparent differences related to adverse events and standard safety parameters between the treatment groups were observed.