Bio Products Laboratory Receives FDA approval for Coagadex® (Coagulation Factor X, Human)
First-ever FDA-approved treatment for hereditary factor X deficiency
ELSTREE, UK AND DURHAM, NC – October 21, 2015 – Bio Products Laboratory, Ltd. (BPL), a leading manufacturer of plasma-derived therapies, today 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.
The second study collected data on two surgical patients receiving Coagadex perioperatively. Surgical data from three patients in the first study was added and resulted in five patients undergoing seven surgical procedures. For all surgical procedures, Coagadex was assessed by the investigator as excellent in controlling blood loss during and after surgery. 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 two with severe deficiency underwent minor procedures. No patients with moderate or severe disease underwent a major procedure. There were no treatment-related adverse events reported in surgical patients in the second study.
In both studies combined, the most common adverse events were infusion site erythema, infusion site pain, fatigue and back pain.
For information on how to order Coagadex and for Prescribing Information please visit www.coagadex.com or call 1-844-4BPLUSA.
Indications for Coagadex
Coagadex, a plasma-derived blood coagulation factor X concentrate, is indicated in adults and children (aged 12 years and above) with hereditary Factor X deficiency for:
- On-demand treatment and control of bleeding episodes
- Perioperative management of bleeding in patients with mild hereditary Factor X deficiency
Perioperative management of bleeding in major surgery in patients with moderate and severe hereditary Factor X deficiency has not been studied.
Important Safety Information for Coagadex
Coagadex is contraindicated in patients with known hypersensitivity to any of the components of the product.
Allergic type hypersensitivity reactions, including anaphylaxis, are possible with Coagadex. If symptoms occur, patients should discontinue use of the product immediately and contact their physician.
The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking Coagadex for the development of inhibitors by appropriate clinical observations and laboratory tests.
Coagadex is made from human plasma and may contain infectious agents, e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of Coagadex.
In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with Coagadex were infusion site erythema, infusion site pain, fatigue and back pain.
For complete Prescribing Information for Coagadex please see www.coagadex.com.
About Bio Products Laboratory, Ltd.
Bio Products Laboratory, Ltd. (BPL) is a leading manufacturer of plasma-derived protein therapies with global headquarters in Elstree, England, US headquarters in Durham, NC, and a presence in more than 45 countries worldwide. The company has over 60 years of experience developing and manufacturing plasma-derived therapies since being established as part of the Lister Institute in 1950, and currently markets a wide range of products including coagulation factors, human immunoglobulins, and albumin. BPL is committed to continued investment in research and development to maintain its key position as a reliable supplier of high-quality products to patients and healthcare providers worldwide.
FDA Approval News Release