Baxter International announced their phase 3 pivotal multi-center, open-label study of BAX 855 [recombinant factor VIII (rFVIII)] for the treatment for hemophilia A met its primary endpoint in reducing annualized bleed rates (ABR) in the prophylaxis treatment arm compared to the on-demand treatment arm.
Patients in the multi-center, open-label study received treatment twice weekly (45 IU/kg) or on-demand, and were followed for six months. The primary objective of the study was the reduction in ABR during the treatment period compared to on-demand treatment. The study also evaluated the safety and immunogenicity of the compound when administered on either prophylaxis or on-demand treatment regimens.
Patients in the twice-weekly prophylaxis arm had a 95% reduction in median ABR compared to the on-demand arm (1.9 vs. 41.5, respectively). Baxter reported that BAX 855 was also effective in treating bleeding episodes.
BAX 855 is an extended release version of their currently approved drug Advate
. Studies have shown BAX 855’s half-life to be 1.4 – 1.5 longer than Advate’s.
Baxter expects to submit a Biologics License Application (BLA) for BAX 855 to the U.S. Food and Drug Administration (FDA) before the end of 2014 and plans to present additional data in the coming months. A second trial with BAX 855 is also planned for patients under the age of 12 years.
Johen Orloff, MD, vice president and global head of research and development for Baxter BioScience said, "The positive results of the BAX 855 study reflect our ongoing, long-term commitment to drive innovation and expand treatment options for patients with hemophilia," adding, "We look forward to advancing the BAX 855 program to U.S. regulatory submission by the end of this year."
About Hemophilia A
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life-threatening. There are an estimated 16,000 people in the United States with hemophilia A (and 142,000 world wide). Orphan approved drugs are available to treat bleeding episodes and two orphan drugs - Advate
- are approved for prophylactic use in patients.
Image of vial courtesy of wikimedia commons