CSL Behring Achieves Another Key Milestone in PROLONG-9FP,
its Recombinant Factor IX Fusion Protein Development Program;
Continues to Advance CSL Behring's Legacy of Improving Patient
Well-Being
KING OF PRUSSIA, Pa.,
Feb. 4, 2015 /PRNewswire/ -- CSL
Behring announced today that the U.S. Food and Drug Administration
(FDA) has accepted for review its Biologics License Application
(BLA) for the marketing authorization of its long-acting fusion
protein linking recombinant coagulation factor IX with recombinant
albumin (rIX-FP). Upon FDA approval, rIX-FP will provide
hemophilia B patients with a long-acting treatment option with
dosing intervals up to 14 days.
"FDA's decision to accept for review the rIX-FP BLA brings CSL
Behring one step closer to providing this innovative therapy to
hemophilia B patients in the U.S.," said Dr. Andrew Cuthbertson, Chief Scientific Officer and
R&D Director, CSL Limited. "The development of rIX-FP
underscores CSL Behring's protein science capabilities, thorough
understanding of the hemophilia community, and commitment to
improving the well-being of patients with hemophilia B."
About PROLONG-9FP Clinical Development Program
CSL Behring's BLA is based on the results from the Phase II/III
study (patients ages 12 to 61 years) in the PROLONG-9FP program.
The Phase II/III pivotal study was an open-label, multicenter,
safety, pharmacokinetic (PK) and efficacy study of rIX-FP in
previously treated patients with hemophilia B (FIX <
2%).
This study 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.
Study design details for rIX-FP (CSL654) are available at
clinicaltrials.gov.
About rIX-FP
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.
In 2012, the FDA granted Orphan Drug Designation for rIX-FP for
the treatment and prophylaxis of bleeding episodes in patients with
hemophilia B. The designation includes routine prophylaxis
treatment, control and prevention of bleeding episodes, and
prevention and control of bleeding in perioperative settings. The
FDA's Orphan Drug Designation program provides orphan status to
drugs and biologics defined as those intended for the safe and
effective treatment or prevention of rare diseases that affect
fewer than 200,000 people in the U.S. Orphan designation qualifies
the sponsor of the product for important tax credits, elimination
of FDA license application fees and certain marketing
incentives.
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'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 trial 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 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
http://www.cslbehring.com/.
Contact:
Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Greg.Healy@cslbehring.com
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