New Therapy Requires Less Frequent Dosing for Hemophilia B Patients

Interim Phase II/III and III findings of a study conducted by CSL Behring demonstrate an improved pharmacokinetic profile of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) among hemophilia B patients in all age groups. The Phase II/III study of patients ages 12 years to 61 years and the Phase III study of patients ages 1 year to 11 years compared the change in frequency of spontaneous bleeding events between on-demand and weekly prophylaxis regimen in patients previously receiving only on-demand treatment and the number of patients developing inhibitors against factor IX as primary outcome measures. It also compared multiple prophylaxis regimens of seven-day and 14-day treatment intervals. The treatment allows a prolonged routine prophylaxis treatment interval of 14 days or potentially longer compared with the current standard of two to three times per week.

“Patients with hemophilia B and treating physicians are eager for innovative products that are able to decrease the dosing frequency while being effective and reliable in the prevention or treatment of bleeding episodes,” said Elena Santagostino, MD, PhD, lead investigator of the study. “Our interim PK data from two Phase III studies, combined with the Phase I and I/II results, demonstrate that rIX-FP has the potential to satisfy this unmet need by offering a longer dosing interval and fewer injections.”