FDA approves orphan drug for bleeding disorder

October 21, 2015

The FDA this week approved Coagadex, Coagulation Factor X (Human) for hereditary Factor X (10) deficiency. Until the new orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

FDA this week approved Coagadex, Coagulation Factor X (Human) for hereditary Factor X (10) deficiency. Until the new orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

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The medication was also granted fast track designation and priority review. Coagadex is manufactured by Bio Products Laboratory Limited in Elstree, Hertfordshire, U.K.

“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.    

In healthy individuals, the Factor X protein activates enzymes to help with normal blood clotting in the body. Factor X deficiency is an inherited disorder, in which blood does not clot as it should.

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Patients with the disorder are usually treated with fresh-frozen plasma or plasma-derived prothrombin complex concentrates (plasma products containing a combination of vitamin K-dependent proteins) to stop or prevent bleeding. The availability of a purified Factor X concentrate increases treatment options for patients with this rare bleeding disorder.

Coagadex, which is derived from human plasma, is indicated for individuals aged 12 and older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, and for perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management of bleeding in patients with mild hereditary Factor X deficiency.

The safety and efficacy of Coagadex was evaluated in a multi-center, non-randomized study involving 16 participants (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. In a separate study, 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.

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