EU approves first hereditary factor X deficiency drug, Coagadex

Privately-held UK manufacturer of plasma-derived protein therapies Bio Products Laboratory has been granted marketing approval by the European Medicines Agency for Coagadex (human coagulation factor X).

Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency. Coagadex is the first and only treatment licensed specifically for this rare bleeding disorder in Europe. It was cleared for this indication by the US Food and Drug Administration last year (The Pharma Letter October 21, 2015).

Factor X deficiency is a rare and serious condition caused by not having enough of the Factor X protein in the blood; this protein plays a crucial role in coagulation (blood clotting) which helps patients stop bleeding. People with Factor X deficiency are at increased risk of bleeding or experience excessive or prolonged bleeding. Severely affected individuals, usually children, have an increased risk of bleeding inside the brain, in the lungs or in the gastrointestinal tract, which can be life-threatening.Hereditary Factor X deficiency is very rare and affects approximately 700 patients in Europe.