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FDA Approves New Recombinant Factor IX Therapy for Hemophilia B

March 9, 2016

The US Food and Drug Administration (FDA) has approved a new therapy for patients with hemophilia B. Idelvion®, manufactured by CSL Behring, combines or “fuses” recombinant factor IX (rFIX) with albumin, a protein found in blood plasma that helps factor IX stay active in the bloodstream longer.

Idelvion® is indicated for on-demand control and prevention of bleeding episodes, management of bleeding following surgery (perioperative) and as a routine preventive (prophylaxis) measure to reduce the incidence of bleeding episodes in patients with hemophilia B, or FIX deficiency. According to the FDA release, Idelvion® potentially requires less frequent infusions than unmodified rFIX when used for prevention.

The safety and efficacy of Idelvion® were evaluated in two multicenter clinical studies, which included a total of 90 adult and pediatric patients with hemophilia B between 1 and 61 years old. The therapy was demonstrated to be effective in controlling bleeding episodes and in managing perioperative bleeding. When used prophylactically, Idelvion® led to a significant reduction in the rate of spontaneous bleeding episodes per year, despite less frequent infusions of the product. Lastly, no safety concerns were reported. The most common side effect observed was headache.

“The approval of Idelvion® provides another important therapeutic option for children and adults with hemophilia B to help prevent or control bleeding and reduce the frequency of bleeding episodes,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research.   

Source: FDA news release dated March 4, 2016