FDA Approves Idelvion for Hemophilia B

March 04, 2016

The US Food and Drug Administration (FDA) today approved a drug for children and adults with hemophilia B called albutrepenonacog alfa (Idelvion, CSL Behring), which combines albumin with factor IX to reduce injection frequency.

Factor IX is a naturally occurring clotting factor that's deficient in people with hemophilia B.

Albutrepenonacog alfa is the first recombinant coagulation factor IX product approved by the FDA that contains albumin, a protein found in blood. It is the second FDA-approved product that combines the clotting factor with any sort of protein that prolongs circulation time. The first was eftrenonacog alfa ( Alprolix , Biogen Idec), which fuses factor IX to the Fc portion of IgG subclass I. Like albumin, this bloodstream protein gives factor IX extra staying power, potentially translating into less frequent injections when the drug is used to prevent bleeding episodes.

European Union regulators recommended approval of both albutrepenonacog alfa and eftrenonacog alfa last month.

Albutrepenonacog alfa is indicated specifically for on-demand control and prevention of bleeding episodes, management of postoperative bleeding, and as prophylaxis to reduce the frequency of bleeding episodes.

The FDA determined the new drug for hemophilia B to be safe and effective based on two multicenter studies involving 90 patients aged between 1 and 61 years. Albutrepenonacog alfa effectively controlled bleeding episodes, including those after surgery. Used prophylactically, it significantly reduced the rate of spontaneous bleeding episodes per year despite less frequent infusions.

Headache was the only adverse event observed in the studies, which did not identify any safety concerns.

More information about today's FDA announcement is available on the agency's website.


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