Global biotherapeutics leader CSL Behring announced today that the company has submitted its new drug application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for its investigational fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). rIX-FP is a long-acting recombinant albumin fusion protein for people with hemophilia B, a congenital bleeding disorder characterized by deficient or defective factor IX. Based on the 2011 National Survey of Coagulation Disorder, hemophilia B occurs in approximately 1 to 2 of every 100,000 male births in Japan.
“CSL was formed nearly 100 years ago with a promise to develop and deliver innovative specialty biotherapies that help people with serious medical conditions live full lives,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. “rIX-FP demonstrates our longstanding commitment to innovation and patient care and we are pleased to submit our new drug application to PMDA. We look forward to, upon approval, bringing this innovative specialty biotherapy to patients with hemophilia B in Japan.”
About PROLONG 9-FP
The submission is based on the PROLONG-9FP clinical development program. PROLONG-9FP includes Phase I through Phase III open-label, multicenter studies evaluating the safety and efficacy of rIX-FP in adults and children (ages 1 to 61 years) with hemophilia B (FIX ≤ 2%) who were previously treated with other factor IX products.
The data from PROLONG-9FP showed median annualized spontaneous bleeding rates (AsBR) of 0.00 in patients using rIX-FP prophylactically. This result was achieved for both 14-day dosing and 7-day dosing. The data for on-demand therapy showed that 93.6 percent of bleeds were controlled with one infusion, while 98.6 percent were controlled with one or two infusions. Across the completed Phase III studies, none of the patients developed inhibitors to factor IX or antibodies to rIX-FP. The most common adverse reaction in clinical trials was headache.
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.
The U.S. Food and Drug Administration, European Medicines Agency and Swissmedic are currently reviewing CSL Behring’s license applications for rIX-FP. For more information about CSL Behring's recombinant coagulation factor products in development to treat hemophilia, visit http://www.cslbehring.com/products/bleeding-disorders/novel-recombinant-hemophilia-treatments.
CSL (ASX:CSL) is a leading global biotherapeutics company with a dynamic portfolio of life-saving innovations, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL — including our two businesses CSL Behring and Seqirus — operates in over 30 countries with more than 14,000 employees. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.
For more information about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.
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