BeneFIX: Phase III data
Top-line data from an open-label, international Phase III trial in 25 patients with moderately severe to severe hemophilia B showed that once-weekly 100 IU/kg BeneFIX as prophylaxis met the primary endpoint of reducing median ABR vs. on-demand treatment with BeneFIX (2 vs. 33.6, p<0.0001). Mean ABR was 3.6 for once-weekly prophylaxis treatment vs. 32.9 for on-demand treatment (p<0.0001). Once-weekly prophylaxis BeneFIX also significantly reduced median spontaneous ABR (1 vs. 22.4) and median traumatic ABR (1 vs. 4.1) vs. on-demand BeneFIX. On a secondary endpoint, none of the 1,254 prophylaxis infusions were associated with an LETE occurrence, defined as a spontaneous bleed occurring within 48 hours of a prophylaxis infusion. Additionally, 82.1% of bleeding episodes in the prophylaxis arm were resolved after 1 infusion. The 2-period trial consisted of a 6-month period of on-demand treatment only followed by a 12-month period of routine prophylaxis with once-weekly 100 IU/kg BeneFIX. ...