FDA approves Factor X deficiency treatment

The U.S. Food and Drug Administration (FDA) on Tuesday approved Coagadex for hereditary Factor X (10) deficiency.

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

The Factor X protein activates enzymes to help with normal blood clotting in the body. When someone has a Factor X deficiency, the blood doesn't clot as it should. It’s an inherited disorder, and patients are treated with fresh-frozen plasma or plasma-derived prothrombin complex concentrates.

Until this approval, no coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

Coagadex comes from human plasma and is indicated for patients 12 years-old and older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, as well as perioperative management of bleeding in patients with mild hereditary Factor X deficiency.

Coagadex was evaluated in a non-randomized study of 16 participants.

The FDA gave Coagadex orphan product designation, which is given to drugs that are created to treat rare diseases in order to promote their development. The FDA also granted Coagadex fast track designation and priority review.

Bio Products Laboratory Limited in Hertfordshire, U.K., manufactures Coagadex.