CSL Behring Continues Improving Patient Well-Being; Key
Milestone Achieved in PROLONG-9FP, Company's Recombinant
Factor IX Fusion Protein Development Program
KING OF PRUSSIA, Pa.,
Dec. 16, 2014 /PRNewswire/ -- CSL
Behring announced today it has submitted a biologics license
application (BLA) to the United States Food and Drug Administration
(FDA) for the marketing authorization of its long-acting fusion
protein linking recombinant coagulation factor IX with recombinant
albumin (rIX-FP). Once approved by the FDA, rIX-FP
(Coagulation Factor IX {Recombinant}, Albumin Fusion Protein) will
provide people with hemophilia B and their physicians a long-acting
treatment option with dosing intervals up to 14 days.
"As we mentioned at our recent R&D investor briefing,
submission of our BLA to the FDA for rIX-FP is a significant
milestone for CSL Behring's recombinant factor IX development
program and moves us one step closer to bringing this innovative
therapy to hemophilia B patients in the U.S.," said Dr.
Andrew Cuthbertson, Chief Scientific
Officer and R&D Director, CSL Limited. "Our strong
partnership with and commitment from the hemophilia community led
us to develop rIX-FP based on novel recombinant albumin fusion
technology. This technology has led to a long-acting treatment
candidate that continues our legacy of improving the well-being of
patients with bleeding disorders and other rare diseases."
About PROLONG-9FP Clinical Development Program
CSL Behring's BLA is based on the results from the PROLONG-9FP
Phase II/III (patients ages 12 to 61 years) study. 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 severe 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
http://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
unique 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
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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