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CSL Behring Announces Orphan Drug Designation for rFIX Therapy

Last month CSL Behring announced that the US Food and Drug Administration (FDA) had granted orphan drug designation for its recombinant factor IX (FIX) product, a novel recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). rIX-FP therapy is for treatment of bleeding episodes in patients with congenital factor IX deficiency (hemophilia B). It includes routine prophylactic treatment, control and prevention of bleeding episodes, and prevention and control of bleeding in perioperative settings.


The FDA’s Office of Orphan Products Development grants orphan drug status to unique drugs or biologics to treat rare diseases, those affecting fewer than 200,000 people. Approved products go through an expedited review process, ensuring that therapies become available earlier to patients with rare, life-threatening diseases who need effective treatments.


rIX-FP is engineered to extend the half-life of factor IX through genetic fusion with a recombinant form of albumin, a water-soluble protein found in blood. According to CSL, albumin was chosen as the ideal recombinant genetic fusion partner for coagulation factor proteins due to its long half-life. Albumin is well tolerated and has a low potential to trigger an inhibitor response from the immune system.


“CSL Behring is pleased to have achieved this important regulatory milestone for our recombinant factor IX,” said Val Romberg, Senior Vice President, Research and Development at CSL Behring. “It represents yet another advance that our company is making in the area of recombinant factor development and is extremely encouraging.”


Source: CSL Behring news release dated June 8, 2012