The FDA has approved Rixubis, the first recombinant coagulation factor IX, for use in people 16 years of age and older with hemophilia B.
Rixubis, which is manufactured by Baxter Healthcare in Westlake Village, Calif., is indicated for the control and prevention of bleeding episodes, perioperative management and routine prevention of bleeding episodes. It is administered by intravenous injection twice weekly.
Hemophilia B affects about 3,300 people in the U.S., and is caused by mutations in the Factor IX gene that lead to a deficiency of the protein Factor IX. The disorder can lead to serious bleeding and can be fatal or debilitating if bleeding occurs in the brain or joints.
A multicenter study of 73 male patients was used to evaluate Rixubis. Patients received the treatment routinely as a prophylactic measure or in response to episodes of serious bleeding. Results showed that patients in the prophylaxis study had a 75 percent lower annual bleeding rate compared with those who received on-demand treatment.
Most common side effects were distorted taste, pain in an extremity and atypical blood tests, though life-threatening allergic reactions can occur.
“This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a release.