Pfizer has reported positive results from its Phase III trial comparing a prophylaxis regimen of BeneFIX Coagulation Factor IX (Recombinant) 100 IU/kg once-weekly to on-demand treatment in people with moderately severe to severe haemophilia B.
The open-label, non-randomised two-period Phase III trial consisted of six months of on-demand therapy only, followed by 12 months of routine prophylaxis with BeneFIX 100 IU/kg once-weekly.
The trial enrolled 25 male patients with a mean age of 31.3 years and moderately severe to severe haemophilia B.
Results showed that the primary endpoint of the trial was met and haemophilia B patients taking once-weekly BeneFIX (100 IU/Kg) showed a statistically significant reduction in the annualised bleeding rate (ABR) relative to on-demand treatment with BeneFIX.
ABR value is a commonly used measure of efficacy for prophylaxis regimens in haemophilia.
In the trial, the median ABR value was 2.0 for the prophylaxis regimen, compared to 33.6 for the on-demand regimen, representing a 94% decrease in bleeding rates.
The company said that the mean ABR value was 3.6 for the prophylaxis period, compared with 32.9 for the on-demand treatment, which represents a reduction of 89%.
Pfizer senior vice-president and medicines development group head of global innovative pharmaceuticals Steven Romano said: "These results are important because they add to the growing body of clinical evidence showing that prophylaxis treatment has the potential to reduce the number of bleeds in a year, the most critical factor in haemophilia management.
"Pfizer remains committed to the research and development of new and innovative products for the haemophilia community."
Results also demonstrated that prophylaxis treatment reduced both spontaneous and traumatic ABR compared to on-demand treatment with BeneFIX.
The secondary endpoints of the trial showed that none of the 1,254 prophylaxis infusions administered were associated with a less than expected therapeutic effect (LETE) occurrence, which was defined as a spontaneous bleed occurring within 48 hours of a prophylaxis infusion.
The company said that adverse events observed in the trial, for both the prophylaxis and on-demand periods, were consistent with the known adverse event profile of BeneFIX.