Bio Products Laboratory, Ltd. (BPL), a manufacturer of plasma-derived therapies, has announced that the U.S. Food and Drug Administration (FDA) has approved 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 and only FDA-approved product to treat hereditary factor X deficiency and is the only specific factor X concentrate available in the U.S. Coagadex is expected to be available to patients in the U.S. in December 2015 through a network of specialty pharmacies and authorized distributors.
Hereditary factor X deficiency is a rare bleeding disorder that affects approximately 300-600 patients in the U.S. Affected individuals often have inadequate amounts of circulating factor X, an important component of the coagulation system. Factor X deficient patients are at increased risk of bleeding and need to be managed similarly to hemophilia patients.
“Completing the clinical studies and bringing Coagadex to the U.S. market is the culmination of years of considerable effort from BPL scientists and input from world-renowned coagulation experts,” said Eric Wolford, PharmD, Vice President of Global Medical at BPL. “We’re excited to provide the first approved treatment to this underserved patient population.”
“The goal of treatment in patients with hereditary factor X deficiency is to replace their missing factor X without adding other clotting factors these patients do not need,” said Miguel Escobar, MD, Medical Director at Gulf States Hemophilia and Thrombophilia Center and Coagadex clinical trial investigator. “Coagadex is a significant advancement for U.S. patients with hereditary factor X deficiency.”
Coagadex was approved based upon data generated from two 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 primary efficacy endpoints were pharmacokinetic measures including recovery rate and half-life, and secondary endpoints included overall assessment of efficacy and the number of infusions needed to treat a bleed.
The criteria for treatment success were satisfied in the study, and the pharmacokinetic parameters were consistent with previously published data. 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.
Two patients in the 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. There were no other drug-related adverse events, no serious drug-related adverse events, and no patients discontinued from the study due to adverse events.