The US Food and Drug Administration has approved a purified clotting factor for patients with hereditary Factor X deficiency. This is the first drug to be approved for the rare, inherited bleeding condition.
Previously, patients had to rely on treatment with blood plasma or plasma concentrates to help manage bleeding. The new drug consists of purified Factor X extracted from blood plasma. It is indicated for patients aged 12 years and over for on-demand treatment and control of bleeding episodes, and for management of bleeding when patients need an operation.
Factor X is an enzyme that is part of the coagulation cascade in the blood. People who inherit two defective copies of the gene either do not make enough Factor X or have a malfunctioning version. This can prevent normal blood clotting and lead to uncontrolled bleeding.
“The approval of Coagadex is a significant advancement for patients who suffer from this rare but serious disease,” says Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research.
The drug is manufactured by Bio Products Laboratory and marketed as Coagadex. It is currently under consideration by the European Medicines Agency and has been given “orphan” designation.