CSL Behring announced today the first in human dosing of recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). The Phase I study will investigate in healthy volunteers the safety and pharmacokinetics of rVIIa-FP in comparison to placebo. CSL Behring, in collaboration with its parent company, CSL Limited (ASX: CSL), is developing rVIIa-FP, a novel therapy to treat hemophilia A and hemophilia B patients who have inhibitors as part of the PROLONG 7- FP clinical study program. For more information about this study, please see www.clinicaltrials.gov
CSL Behring’s albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins due to its inherently long half life, high potential for tolerability, known mechanism of clearance and low potential for immunogenic reactions. CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis.
CSL Behring’s rVIIa-FP was previously granted Orphan Drug Designation by the European Commission and the United States Food and Drug Administration.
Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which render replacement therapy ineffective. This can occur in up to 25 percent of hemophilia A patients and approximately 5 percent of hemophilia B patients. One treatment option for these patients is recombinant activated factor VII (called a “bypassing agent”) which can be used to achieve hemostasis without the need for factor VIII or IX.
About the recombinant fusion protein linking coagulation factor VIIa with recombinant albumin (rVIIa-FP)
Preclinical studies have confirmed that CSL Behring's rVIIa-FP has favorable pharmacokinetic properties compared with the existing recombinant FVIIa product. Significant increases in half-life have been observed across all animal species. The use of a bypassing agent with an extended half-life could offer significant benefit to those affected by hemophilia A or B with inhibitors and may offer patients the opportunity to be treated less frequently than with the currently available product.
CSL Behring's clinical program intends to demonstrate that an extended half-life rVIIa-FP will result in a requirement for fewer doses while providing adequate therapeutic response in patients with hemophilia A and B with inhibitors.
About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of
CSL Limited (ASX:CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For information: www.cslbehring.com.
Sheila A. Burke
Director, Communications & Public Relations