FDA approves Rixubis for hemophilia B

Issue: August 10, 2013

The FDA approved the first recombinant coagulation factor IX indicated specifically for routine prophylaxis of hemophilia B.

Rixubis (Baxter) — approved for use by individuals aged 16 years or older — is indicated for perioperative management, as well as routine use to prevent or reduce the frequency of bleeding episodes.

“Rixubis becomes a new weapon in our arsenal to protect hemophilia B patients,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes.”

Hemophilia B, an inherited blood clotting disorder that primarily affects men, is the second most common type of hemophilia. It is caused by mutations in the Factor IX gene and leads to deficiency of Factor IX, a naturally occurring protein in blood that controls bleeding.

The roughly 3,300 Americans who have hemophilia B often experience internal bleeding, especially in the joints.

Rixubis, a purified protein supplied in single-use vials of freeze-dried powder, is reconstituted with sterile water and then administered by IV injection, according to the FDA. The indication for routine prevention of bleeding episodes calls for twice-weekly doses.

The FDA based its approval on results of a multicenter study that included 73 men aged 12 to 65 years. Patients received Rixubis for routine prophylaxis or as needed in response to symptoms of bleeding episodes.

Patients who used the agent for prophylaxis experienced a 75% reduction in bleeding rates compared with those who historically received on-demand treatment in response to symptoms, according to the FDA.

The most common adverse effects reported in study patients include extremity pain, distorted taste and atypical blood test results.

A separate study designed to evaluate Rixubis in pediatric patients is under way.