Baxter hemophilia factor VIIa passes Phase III
This article was originally published in Scrip
Executive Summary
Baxter BioScience plans to begin regulatory submissions for the approval of its investigational treatment BAX 817 for hemophiliacs who develop inhibitors, following positive Phase III trial results. The product, a recombinant factor VIIa (rFVIIa) treatment for people with hemophilia A or B, met the primary endpoint of successful resolution of acute bleeding episodes at 12 hours with both on-demand treatment regimens, dosing either 3X90μg/kg or 1X270 μg/kg, with an overall success rate of 92% (98% and 85% in each dosing group, respectively). 89% of patients achieved sustained bleeding control for all acute bleeding episodes 24 hours after infusion. Patients who develop inhibitors to treatments are at increased risk of serious complications from difficult to treat bleeding episodes. No patients developed inhibitors or binding antibodies to BAX817, nor did any discontinue treatment because of an adverse event. The trial was in male patients aged 12-65 covering a six-month period using on-demand therapy. The regulatory submissions will be aligned to ongoing manufacturing expansions.