rFVIII Treatment for Hemophilia A Receives Positive Results
- By BSTQ Staff
Baxter’s Phase III clinical trial of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A based on ADVATE (Antihemophilic Factor [Recombinant]), has met its primary endpoint in reducing annualized bleeding rates (ABR) in the prophylaxis arm compared with the on-demand arm.
The multi-center, open-label study evaluated BAX 855 among 138 adolescent and adult patients with previously treated hemophilia A. Patients received prophylaxis treatment twice weekly (45 IU/kg) or on-demand, and were followed for six months. Patients in the prophylaxis arm experienced a 95 percent reduction in median ABR compared with those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating bleeding episodes, 96 percent of which were controlled with one or two infusions. The half-life of BAX 855 was 1.4 to 1.5 times that of ADVATE, consistent with the findings from the Phase I study. No patients developed inhibitors to BAX 855, and no treatment-related serious adverse events, including hypersensitivity, were reported. The most common (three patients) product-related adverse event was headache.
Baxter expects to submit a Biologics License Application for BAX 855 to the U.S. Food and Drug Administration before the end of 2014 and will present additional data in the coming months. In addition to an ongoing continuation study for patients who have completed the pivotal trial, the company is initiating a Phase III prospective, open-label, multi-center study to evaluate the safety and efficacy of BAX 855 among 60 previously treated patients under age 12 with severe hemophilia A.
