The United States Food and Drug Administration (FDA) has approved Baxter International's Rixubis [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control of bleeding episodes, and perioperative management in adults with haemophilia B. Rixubis is the first new recombinant factor IX (rFIX) approved for haemophilia B in more than 15 years and is the only rFIX indicated for both routine prophylaxis and control of bleeding episodes in the US for adult patients living with this chronic condition.
Haemophilia B is the second most common type of haemophilia and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.
"Baxter has long been a leader in the support of people living with bleeding disorders with its innovative products and programmes," said Val Bias, chief executive officer of the National Haemophilia Foundation (NHF). "We applaud their ongoing commitment to the haemophilia community, demonstrated once again with now the first prophylactic treatment approved in the US for adults living with haemophilia 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 per cent 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 Haematology (ASH) in Atlanta.
"Rixubis is a welcome addition to our leading portfolio of haemophilia treatments, providing a much-needed treatment option for the thousands of people living with haemophilia 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.
The approval is based on a pivotal phase I/ III prospective, open-label, uncontrolled, multi-centre study that investigated the pharmacokinetics, efficacy and safety of Rixubis in 73 previously-treated patients (between 12 and 65 years of age) with severe (factor IX level <1 per cent) or moderately severe (factor IX level =2 per cent) haemophilia B. Patients received Rixubis either for prophylaxis and/or for the treatment of bleeding episodes on an on-demand basis, and were exposed to a factor IX-containing product on =150 days. Fifty-nine patients received Rixubis for prophylaxis twice-weekly, and 56 of them received treatment for a minimum of three months. An additional 14 patients received Rixubis for the treatment of bleeding episodes only, and had to have at least 12 documented bleeding episodes requiring treatment within 12 months prior to enrollment.
The majority of patients taking Rixubis had arthropathy at screening (88 percent) and target joints (66 percent). Treatment was individualized based on the severity, cause and site of bleed and of the total 249 bleeding episodes, the majority (211; 84.7 percent) were treated with 1-2 infusions.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with haemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions.