FDA Okays Coagadex for Rare Bleeding Disorder


October 20, 2015

The US Food and Drug Administration (FDA) today approved the first replacement therapy for hereditary Factor X deficiency, a rare bleeding disorder, the agency announced.

The new therapy, Coagulation Factor X (Coagadex, Bio Products Laboratory), is derived from human plasma. It is indicated for individuals aged 12 years or older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, and for perioperative management of bleeding in patients with a mild form of the condition.

A protein, Factor X turns on enzymes to help with normal blood clotting. Congenital Factor X deficiency, which causes blood not to clot properly, affects an estimated 1 in 500,000-1,000,000 individuals worldwide. The traditional treatment is either fresh frozen plasma or plasma-derived prothrombin complex concentrates.

The FDA evaluated the safety and efficacy of the Factor X replacement therapy in a multicenter, nonrandomized study involving 16 participants who were treated for spontaneous, traumatic, and heavy menstrual bleeding. The therapy effectively controlled bleeding in participants with moderate-to-severe cases of hereditary Factor X deficiency. It also controlled blood loss before and after surgery in five individuals with a mild deficiency. The agency did not identify any safety concerns.

The FDA designated the factor replacement therapy as an orphan drug, which gives manufacturers economic benefits that make commercializing a product for a small number of patients more feasible. The agency expedited approval by granting the drug both fast-track and priority-review status.

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


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