FDA Approves Baxter's RIXUBIS as First Recombinant Factor IX Treatment for Routine Prophylaxis of Hemophilia B

Baxter International Inc.

today announced that the United States Food and Drug Administration (FDA) has approved RIXUBIS [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control of bleeding episodes, and perioperative management in adults with hemophilia B. RIXUBIS is the first new recombinant factor IX (rFIX) approved for hemophilia B in more than 15 years and is the only rFIX indicated for both routine prophylaxis and control of bleeding episodes in the U.S. for adult patients living with this chronic condition. Hemophilia B is the second most common type of hemophilia and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.1 ''Baxter has long been a leader in the support of people living with bleeding disorders with its innovative products and programs,'' said Val Bias, chief executive officer of the National Hemophilia Foundation (NHF). ''We applaud their ongoing commitment to the hemophilia community, demonstrated once again with now the first prophylactic treatment approved in the U.S. for adults living with hemophilia B.'' The approval is based on a Phase I/III study demonstrating that twice-weekly prophylactic treatment with RIXUBIS for six months achieved a median annualized bleed rate (ABR) of 2.0 with 43 percent of patients experiencing no bleeds. In this study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. One patient developed a transient antibody to furin. The most common adverse reactions observed in >1% of subjects in clinical studies were: dysgeusia, pain in extremity, and positive test for furin antibody. The study was presented at the 54th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta. ''RIXUBIS is a welcome addition to our leading portfolio of hemophilia treatments, providing a much-needed treatment option for the thousands of people living with hemophilia B,'' said Ludwig Hantson, Ph.D., president of Baxter's BioScience business. ''This approval moves us one significant step forward in our pursuit of a bleed-free world, one patient at a time.'' For the prophylaxis regimen to prevent or reduce frequency of bleeding episodes, RIXUBIS dosing of 40 to 60 IU per kg twice weekly may be used. Five dosage options will be available to provide the opportunity for patients to use a single vial to dose RIXUBIS. A BAXJECT II Needle-less Transfer device will be included in each package of RIXUBIS. Baxter was granted orphan-drug designation by the FDA as part of the RIXUBIS application, a special status given to a product that would address an unmet need for people with a rare disease or condition. Baxter expects to file for marketing authorization for RIXUBIS in the European Union later this year.