In a Phase 3 clinical trial comparing Pfizer's (PFE +1.2%) BeneFIX Coagulation Factor IX (Recombinant) as a treatment for patients with moderately severe-to-severe hemophilia B to on-demand treatment, the once-weekly therapy achieved its primary endpoint of a statistically significant reduction in the annualized bleeding rate (ABR) versus on-demand.
In the study, the median ABR (a measure of efficacy for prophylaxis regimens in hemophilia) for BeneFIX was 2.0 compared to 33.6 for the on-demand treatment. The mean ABR for BeneFIX was 3.6 compared to 32.9 for on-demand.
None of the 1,254 prophylaxis infusions administered in the study were associated with a less-than-expected therapeutic effect (LETE) occurrence.
82.1% of the bleeding episodes in the prophylaxis arm were resolved after one infusion.