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FDA Approves Therapy for Rare Clotting Factor Deficiency

The first replacement factor therapy for hereditary Factor X deficiency is on its way to the market

Coagulation factor X (human) (Coagadex, Bio Products Laboratory) has been granted FDA approval for hereditary factor X deficiency in patients 12 years and older for on-demand control of bleeding episodes and for perioperative bleeding management in those with mild conditions. Prior to the approval, there was no specific coagulation factor therapy available for patients, who were primarily treated with fresh frozen plasma or prothrombin complex concentrates.

According to the FDA, the safety and efficacy of Coagadex was evaluated in a multicenter, nonrandomized study involving 16 participants (who experienced 208 bleeding episodes) for treatment of spontaneous, traumatic and heavy menstrual (menorrhagic) bleeding episodes. Coagadex was demonstrated to be effective in controlling bleeding episodes in participants with moderate to severe hereditary factor X deficiency.

Coagadex was also evaluated in five participants with mild to severe factor X deficiency who were undergoing surgery. The five individuals received Coagadex for perioperative management of seven surgical procedures. Coagadex was demonstrated to be effective in controlling blood loss during and after surgery in participants with mild deficiency. No individuals with moderate or severe factor X deficiency received Coagadex for perioperative management of major surgery, and no safety concerns were identified in either study.

“The approval of Coagadex is a significant advancement for patients who suffer from this rare but serious disease,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research.  The FDA had granted Coagadex an orphan product designation for this use. 

Source: FDA, October 20, 2015; MedPage Today, October 20, 2015.

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