Baxter International recently revealed new data from a phase 3 pivotal study of its investigational drug, BAX 855, an extended half-life recombinant factor VIII treatment for hemophilia A.
Baxter International recently revealed new data from a phase 3 pivotal study of its investigational drug, BAX 855, an extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A.
BAX 855 is based on Advate, a recombinant antihemophilic factor for perioperative management, routine prophylaxis to prevent or reduce the frequency of bleeding episodes, and control and prevention of bleeding episodes in children and adults with hemophilia A.
The 2-arm phase 3 trial evaluated BAX 855 in 137 previously treated hemophilia A patients aged 12 years or older. The new data indicated that BAX 855 met the study's primary endpoint by controlling and preventing bleeding episodes and routine prophylaxis, with patients in the twice-weekly prophylaxis arm experiencing a 95% reduction in median annualized bleed rate compared with those in the on-demand arm.
“These pivotal trial results provide evidence to support the efficacy profile of BAX 855 in controlling, preventing, or reducing the frequency of bleeding episodes when administered prophylactically twice weekly” said Baxter Bioscience vice president John Orloff, MD, in a press release. “Our goal with BAX 855 is to extend the interval between infusions while maintaining a similar efficacy profile to Advate.”
No trial participants developed inhibitors to BAX 855 or experienced treatment-related serious adverse events. However, 7 adverse events—including headache, diarrhea, nausea, and flushing—were reported in 6 participants.
The study findings support Baxter's biologics license application for the approval of BAX 855, which it submitted to the FDA in December 2014.