FDA Approves Coagadex for Rare Blood Disorder

Factor X deficiency prevents blood from clotting properly.

The FDA on Tuesday approved Coagadex, Coagulation Factor X (Human) to treat hereditary Factor X (10) deficiency. Until this approval, there was no particular coagulation factor replacement therapy available for patients with the condition.

The Factor X protein normally works by activating enzymes to help with normal blood clotting processes in the body. Factor X deficiency is a hereditary condition, affecting men and women at the same rate.

It is characterized by blood that does not clot properly. Patients with the condition are usually treated with fresh-frozen plasma or plasma-derived prothrombin complex concentrates to stop or prevent bleeding.

The availability of a purified Factor X concentrate increases treatment options for patients with this rare bleeding disorder.

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

The new drug is made from human plasma and is intended for individuals aged 12 and 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.

Research was performed to evaluate the safety and efficacy of Coagadex in a multi-center, non-randomized study involving 16 participants (or 208 bleeding episodes) for treatment of spontaneous, traumatic, and heavy menstrual bleeding episodes.

The drug showed efficacy in controlling the bleeding episodes of patients who had severe hereditary Factor X deficiency. Coagadex was also analyzed in 5 patients who had a mild or severe form of the disease who were about to be operated on.

These individuals received the medication for perioperative management of seven surgical procedures. The medication was effective in the management of blood loss both during and after surgery in patients with a mild form of the deficiency.

No individuals with moderate or severe Factor X deficiency received Coagadex for perioperative management of major surgery, and no safety concerns were identified in either study.

The FDA granted orphan product designation to Coagadex for the aforementioned uses. In addition to orphan product designation, Coagadex was granted fast track designation and priority review.