CSL Behring announced today that the European Medicines Agency (EMA) has started the Centralized Procedure for reviewing the company's Marketing Authorization Application (MAA) for its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). Upon European Commission approval, rIX-FP will provide hemophilia B patients in the European Union (EU), as well as the European Economic Area (EEA) countries, with a long-acting treatment option with dosing intervals up to 14 days.
"The EMA beginning its Centralized Procedure for rIX-FP is a significant milestone for CSL Behring's recombinant factor IX clinical development program and moves us one step closer to bringing this innovative therapy to hemophilia B patients in the EU and EEA," said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. "CSL Behring's rIX-FP for hemophilia B patients further illustrates the company's protein science capabilities and strong partnership with the hemophilia community."
In February 2015, the U.S. Food and Drug Administration accepted for review CSL Behring's Biologics License Application for rIX-FP. Pivotal data for rIX-FP will be presented during the International Society on Thrombosis and Haemostasis (ISTH) Congress in Toronto in June 2015.
CSL Behring engineered rIX-FP to extend the half-life of recombinant factor IX through genetic fusion with recombinant albumin. CSL Behring selected recombinant albumin as its recombinant genetic fusion partner for its coagulation factor proteins due to its long physiological half-life. In addition, recombinant albumin has been shown to have a good tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance. The cleavable linker connecting recombinant factor IX and recombinant albumin has been specifically designed to preserve the native function of the coagulation factor in the fusion protein, while benefiting from recombinant albumin's long physiological half-life.
About PROLONG-9FP Clinical Development Program
CSL Behring's MAA is based on data from the PROLONG-9FP clinical development program, covering patients from the age of 1 to 61 years. Studies in the program were conducted as open-label, multicenter, safety and efficacy studies of rIX-FP in previously treated patients with hemophilia B (FIX ≤ 2%).
The Phase II/III pivotal study (patients ages 12 to 61 years) was designed to compare the change in frequency of spontaneous bleeding events between on-demand treatment and a weekly prophylaxis regimen in patients previously receiving only on-demand treatment; and the number of patients developing inhibitors against factor IX as primary outcome measures. The study evaluated multiple prophylaxis regimens, including 7-day and 14-day intervals. A sub-study evaluated the prevention and control of bleeding in patients with hemophilia B undergoing a surgical procedure.
The primary outcome measures of the Phase III children study (patients ages 1 to 11 years) are PK parameters of rIX-FP and the number of subjects developing inhibitors against factor IX. All patients received a weekly prophylaxis regimen.
Study design details for rIX-FP (CSL654) are available at clinicaltrials.gov.
About CSL Behring's Recombinant Factor Development Program
rIX-FP for the treatment of hemophilia B is a part of CSL Behring's Recombinant Factor Development program. The AFFINITY clinical development program is studying CSL Behring's recombinant Factor VIII SingleChain (rVIII-SingleChain) to treat hemophilia A. CSL Behring also continues to advance its long-acting recombinant fusion protein linking recombinant coagulation factor VIIa with recombinant albumin (rVIIa-FP) to control bleeding episodes in hemophilia patients who have inhibitors.
About Hemophilia B
Hemophilia B (congenital factor IX deficiency) is characterized by deficient or defective factor IX and affects approximately 1 in 25,000 to 50,000 people. Hemophilia B is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. Nearly all hemophilia B patients are male.
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 used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. 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 global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.