Positive long-term safety data using rIX-FP for Haemophilia-CSL Behring

CSL Behring presented data from Phase III studies evaluating the efficacy and long-term safety of rIX-FP (recombinant coagulation factor IX with recombinant albumin). The data support the use of rIX-FP for routine prophylaxis, dosed once up to every 14 days, and for on-demand treatment of bleeding episodes in previously-treated adults and children with hemophilia B. The findings also include efficacy and safety results supporting the use of rIX-FP in patients undergoing surgical procedures. In patients who started on-demand treatment then switched to prophylaxis treatment, annualized spontaneous bleeding rates (AsBR) decreased by 100 percent from a median 15.43 during the on-demand treatment phase to 0.00 in the prophylaxis treatment phase (p<0.0001).>

In a second study, 97 percent of bleeding episodes were successfully managed by one or two infusions, with 89 percent treated with only one infusion. Finally in a third study, results showed that a single dose of rIX-FP was sufficient to maintain hemostasis during surgery. Over the 14-day post-surgical study period, patients needed anywhere from two to seven infusions. Both the consumption and dosing frequency of rIX-FP were considered remarkably low based on the type of surgery performed. Data were presented at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress in Toronto.

Comment: rIX-FP has been submitted in both the US and EU for Haemophilia treatment.