The US Food and Drug Administration has given the green light to Baxter International's Rixubis, the first new recombinant factor IX approved for haemophilia B in more than 15 years.
The agency has approved Rixubis, a purified protein produced by recombinant DNA technology, for the control and prevention of bleeding episodes, perioperative management and routine use to prevent or reduce the frequency of bleeding episodes.
An inherited blood clotting disorder mainly affecting males, haemophilia B is caused by mutations in the factor IX gene and affects about 3,300 people in the USA. Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, noted that as the first recombinant coagulation factor IX indicated specifically for routine prophylaxis to prevent bleeding, "Rixubis becomes a new weapon in our arsenal to protect haemophilia B patients".
Its efficacy was evaluated in a study in which 73 males between 12 and 65 years of age received Rixubis for routine prophylaxis or as needed in response to symptoms of bleeding. Overall, patients had a 75% lower annual bleeding rate when compared to patients who have historically received on-demand treatment. An additional paediatric trial is ongoing.
Ludwig Hantson, president of Baxter's bioscience business, said the approval "moves us one significant step forward in our pursuit of a bleed-free world, one patient at a time".