FDA approves Coagadex for hereditary Factor X deficiency

The FDA today approved Coagadex for hereditary Factor X deficiency.

Coagadex (Bio Products Laboratory) — a high-purity human Factor X concentrate — is the first specific coagulation factor replacement therapy available for patients with this condition.

The Factor X protein activates enzymes to help ensure normal clotting in the body. Factor X deficiency, an inherited disorder, prevents the blood from clotting as it should.

Individuals with the condition typically are treated with fresh-frozen plasma or plasma-derived prothrombin complex concentrates to stop or prevent bleeding.

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

Coagadex, derived from human plasma, is indicated for patients aged 12 years or older with hereditary Factor X deficiency who require on-demand treatment and control of bleeding episodes. It also is indicated for perioperative management of bleeding among patients with mild hereditary Factor X deficiency.

The FDA based the approval in part on results of a multicenter study that assessed Coagadex for the treatment of spontaneous, traumatic and heavy menstrual bleeding episodes.

The study included 16 participants who experienced a combined 208 bleeding episodes. Results showed Coagadex effectively controlled bleeding episodes in participants with moderate to severe hereditary Factor X deficiency.

In a separate analysis, perioperative management with Coagadex effectively controlled blood loss during and after surgery in patients with mild Factor X deficiency.

Researchers did not identify any safety concerns in either study.