The US Food and Drug Administration (FDA) has approved the first long-lasting recombinant coagulation factor IX product (Alprolix, Biogen Idec) for use in children and adults with hemophilia B.
Alprolix is indicated to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes, the FDA said.
It is the first hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding, the agency notes. According to the company, the therapy is shown to reduce bleeding episodes with prophylactic infusions starting at least a week apart.
Alprolix fuses factor IX to the Fc portion of IgG subclass I, which is believed to prolong time in circulation, Biogen Idec says.
"The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B," said Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research, in a statement.
The safety and efficacy of Alprolix were demonstrated in a multicenter clinical trial involving 123 patients with severe hemophilia B who were followed for up to a year and a half, as reported by Medscape Medical News.
"The studies demonstrated the effectiveness of Alprolix in the prevention and treatment of bleeding episodes and during perioperative management of patients undergoing a surgical procedure. No safety concerns were identified in this trial," the FDA notes.
Biogen says common adverse reactions seen in clinical trials were headaches and oral paresthesia.
Hemophilia B is a rare blood-clotting disorder caused by defects in the factor IX gene. The disorder affects about 3300 people in the United States, primarily males. People with hemophilia B experience repeated episodes of potentially serious bleeding, mainly into the joints, which can be destroyed by the bleeding.
Alprolix received orphan-drug status for hemophilia B by the FDA because it is intended for treatment of a rare disease.
The drug was approved in Canada on March 23. The company says it expects to make Alprolix available in the United States in early May.
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Cite this: First Long-Acting Hemophilia B Drug (Alprolix) Clears FDA - Medscape - Mar 31, 2014.