Findings of a late-stage study show back the use of once-weekly treatment with Pfizer’s BeneFix to cut down bleeding episodes in patients with haemophilia B.
The international Phase III trial showed that patients given Pfizer’s shot prophylactically experienced a significant reduction in the annualised bleeding rate (ABR) - the primary goal of disease management - compared to those treated on an ‘on demand’ basis.
The median ABR values were 2.0 for the prophylaxis period and 33.6 for the on-demand period, while during the 52 weeks of the prophylaxis period, 36 percent of patients experienced no bleeding events of any kind, and 48 percent of patients experienced no spontaneous bleeding events.
On the safety side, adverse events reported for the prophylactic regimen were similar to those reported for on-demand treatment, the most common being: arthralgia, back pain, headache, joint swelling, local swelling, nasopharyngitis, pharyngitis, pyrexia, toothache and upper respiratory tract infection. Neither inhibitors nor thrombotic events were reported, Pfizer noted.
BeneFix (recombinant coagulation factor IX) was first launched in 1997. In the US it is a indicated for the control and prevention of bleeding episodes in adult and paediatric patients with haemophilia B, but is not cleared for prophylactic use, but in Europe is cleared for both treatment and prophylaxis.