The FDA has approved Bio Product Laboratory’s Coagadex for the treatment of adults and children (aged 12 years and over) with hereditary factor X deficiency for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in patients with mild hereditary Factor X deficiency.
Coagadex is the first FDA-approved product to treat hereditary factor X deficiency and is now the only specific factor X concentrate available in the US.
What is Hereditary factor X deficiency?
Factor X deficiency is a disorder caused by too little of a protein called factor X in the blood. It leads to problems with coagulation. Inherited factor X deficiency affects 1 out of every 500,000 people.
Previously, treatments have included controlling blood loss by getting infusions of fresh or frozen plasma or concentrates of clotting factors into the blood.
Coagadex was approved based on data generated from 2 open-label, multicenter, prospective studies. The first study enrolled patients with moderate to severe hereditary factor X deficiency who were treated on-demand for spontaneous or traumatic bleeding episodes.
The criteria for treatment success were satisfied in the study. The overall mean in-vivo recovery rate was 2.0 IU/dL per IU/kg and the half-life was approximately 30 hours. There were 187 assessable bleeds in the study with patients rating the treatment as “excellent” in 170 (91%) cases, “good” in 14 (7.5%) cases, and “poor” in 2 (1.1%) cases.
In addition, most bleeding episodes (155/187 [82.9%]) were effectively treated with only one infusion of Coagadex.
The second study collected data on 2 surgical patients receiving Coagadex preoperatively. Surgical data from three patients in the first study was added and resulted in 5 patients undergoing seven surgical procedures. All patients undergoing major procedures were diagnosed with mild factor X deficiency (i.e., factor X level >5 IU/dL and < 20 IU/dL). One patient with moderate deficiency and 2 with severe deficiency underwent minor procedures. No patients with moderate or severe disease underwent a major procedure.
Two patients in the fist study reported six adverse events considered possibly related to the medication: two events of fatigue in one patient, two events of infusion site erythema in one patient, and one of infusion site pain and back pain in each patient. No adverse effects were reported in the second study.
Coagadex is expected to be available to patients in the U.S. in December through a network of specialty pharmacies and authorized distributors.
Bio Products Laboratory Receives FDA approval for Coagadex® (Coagulation Factor X, Human) [news release]. Elstree, UK; Bio Products Laboratory: October 21, 2015. http://www.businesswire.com/news/home/20151021005989/en/Bio-Products-Laboratory-Receives-FDA-approval-Coagadex