rIX-FP Regulatory Submissions Now Made in EU and U.S.;
Underscores CSL Behring's Legacy of Improving the Care for
Patients with Bleeding Disorders
KING OF PRUSSIA, Pa.,
March 30, 2015 /PRNewswire/
-- 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.
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.
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.
Contact:
Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Greg.Healy@CSLBehring.com
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