US FDA approves first new haemophilia B drug in over 15 years

The FDA has approved Baxter Healthcare's hemophilia B drug for use in patients aged 16 years of age and older in the United States. This approval represents the first new treatment for this genetic disorder in more than 15 years.

Hemophilia B is an inherited blood clotting disorder that mainly affects males – approximately 25,000 people worldwide are affected each year. The condition is caused by mutations in the Factor IX gene, which can lead to deficiency of Factor IX. Patients with hemophilia B can experience potentially serious bleeding.

Rixubis is the first recombinant coagulation factor IX specifically indicated for routine use in preventing bleeding episodes perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management, and routine use to prevent or reduce the frequency of bleeding episodes (prophylaxis).

"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. This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes."

Karen Midthun, M.D., director of the FDA's Center for Biologics Evaluation and Research.

Baxter's Rixubis is a purified protein produced by recombinant DNA technology. The efficacy of this drug was evaluated in a clinical trial of 73 male patients between 12 and 65 years of age, who received the Rixubis for routine prophylaxis or as needed in response to symptoms of bleeding (on-demand). Overall, patients in the prophylaxis study had a 75% lower annual bleeding rate when compared to patients who have historically received on-demand treatment.

An additional study in a pediatric population is currently ongoing.