In June, the US Food and Drug Administration (FDA) approved Rixubis, a recombinant factor IX (rFIX) therapy for treatment in patients with hemophilia B who are 16 years old or older. Rixubis, manufactured by Baxter Healthcare Corporation, is the first new rFIX treatment in 15 years. It is the only rFIX product approved for the control and prevention of bleeding episodes and for routine prophylaxis (twice weekly) to prevent or reduce the frequency of bleeding episodes. It is also approved for perioperative management.
"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 Hemophilia B patients," said Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research. "This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes."
The efficacy of Rixubis was evaluated in a multicenter study of 73 male patients between 12 and 65 years old. The subjects had severe hemophilia ( 1% FIX) or moderate hemophilia ( or = to 2% FIX), and received the drug for routine prophylaxis or as needed for symptoms of bleeding (on demand) for a minimum of 150 days. The 59 patients in the prophylaxis study had a 75% lower annual bleeding rate compared to the 14 subjects receiving on-demand treatment.
During the study, no patients developed antibodies, or inhibitors, to the drug and there were no reports of anaphylaxis, a life-threatening reaction that can occur in patients with hemophilia B. Side effects occurred in 1% of patients and included distorted taste, pain in an extremity and atypical blood test results. An additional study in a pediatric population is currently being conducted.
Source: FDA news release dated June 27, 2013
