DURHAM, N.C. & ELSTREE, UK--(BUSINESS WIRE)--Bio Products Laboratory, Ltd. (BPL), a leading manufacturer of plasma-derived protein therapies, today announced the results from a phase III registration study examining the pharmacokinetics, efficacy, and safety of its FACTOR X coagulation product in subjects with hereditary FACTOR X deficiency. The data were presented at the Hemostasis and Thrombosis Research Society annual meeting in New Orleans, LA, and have been submitted to the U.S. Food and Drug Administration (FDA) for review.
Hereditary FACTOR X deficiency is a rare bleeding disorder that affects approximately 400-600 patients in the United States. 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. There is currently no FACTOR X product specifically approved by the FDA for the treatment of patients with hereditary FACTOR X deficiency.
The data presented at the conference were generated from an open-label, multicenter, prospective study that enrolled sixteen patients with severe or moderate hereditary FACTOR X deficiency. Patients were treated on-demand for spontaneous or traumatic bleeding episodes, or were given specific short-term preventative therapy. The primary efficacy endpoints were pharmacokinetic measures including recovery rate and half-life, while secondary measures included the patient’s overall assessment of efficacy and the number of infusions needed to treat a bleed.
BPL said the criteria for treatment success were met, and the pharmacokinetic parameters obtained from the study were consistent with previously published data. The mean in-vivo recovery rate was 2.07 IU/dL per IU/kg, and the half-life was approximately 30 hours. On the clinical efficacy measures, 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 the FACTOR X product.
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 event each of infusion site pain and back pain. 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.
Dr. Steve Austin, a bleeding disorder expert at St. George's Hemophilia Centre in London, was an investigator in the study. “The data show that the BPL FACTOR X product helped restore hemostasis and reduced bleeding in Factor X deficient subjects suffering from various bleeds,” said Dr. Austin. “This is an important step in finding treatments for this underserved population.”
“The study completion and data presentation were the culmination of many years of considerable effort from BPL scientists and hemophilia bleeding experts”, said Eric Wolford, PharmD, Vice President of Global Medical for BPL. “We’re very excited to see this work presented, and are eager to continue developing therapies for patients with rare diseases.”
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.