CSL Behring recently announced the results of a Phase I study evaluating recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in patients with severe hemophilia B. Results of the study, which were presented February 2, during an oral session at the Gesellschaft für Thrombose- und Hämostaseforschung e.V. congress in St. Gallen, Switzerland, showed that rIX-FP was well tolerated in all patients and lasted longer in the body, due to its prolonged half-life, compared with current factor IX (FIX) treatment options.
CSL Behring, in collaboration with its parent company, CSL Limited, is developing rIX-FP for the prophylaxis and treatment of bleeding episodes in patients with congenital FIX deficiency as part of the PROLONG-9FP clinical study program. No adverse events were reported during the first phase of the study.
“Hemophilia B is a rare and serious bleeding disorder that prevents normal blood clotting and requires frequent infusion of Factor IX concentrates to restore clotting ability,” said Elena Santagostino, MD, PhD, Professor in the Medical School of Clinical and Experimental Hematology at the University of Milan/IRCCS Maggiore Hospital. “The results of this study suggest that rIX-FP is a promising investigational agent for improvement of prophylactic and on-demand treatment for patients with hemophilia B.”
In a separate announcement made on February 16th, CSL reported that its novel recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation has been granted for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia and inhibitors to coagulation factor VIII or IX. CSL Behring is also developing this therapy in collaboration with CSL Limited.
The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare, life-threatening diseases who need effective treatments.
“CSL Behring’s albumin fusion technology uses albumin as the ideal recombinant genetic fusion partner for coagulation factor proteins because of its high tolerability, inherently long half-life, low potential for immunogenic reactions and known mechanism of clearance,” said Russell Basser, MD, Senior Vice President, Global Clinical R&D at CSL Behring. “CSL Behring’s rVIIa albumin fusion protein is expected to exhibit a good tolerability profile and improved pharmacokinetics that may enable prophylaxis. We welcome Orphan Drug Designation for our rVIIa-FP and will work closely with the FDA to make this important therapy available for people in the U.S. with hemophilia A and hemophilia B with inhibitors.”
Source: CSL Behring news release dated February 16, 2012