Baxter Announces Phase III Data Evaluating Prophylaxis Treatment of FEIBA NF
for Hemophilia Patients with Inhibitors
Pivotal study to form basis for upcoming biologic license application to the
DEERFIELD, Ill. -- January 8, 2013
Baxter International Inc. (NYSE:BAX) today announced pivotal Phase III study
results evaluating the efficacy and safety of routine prophylaxis compared to
on-demand treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex],
Nanofiltered and Vapor Heated, in patients with hemophilia A or B and
inhibitors. Top-line results from the study showed a reduced median annual
bleed rate (ABR) from 28.7 during FEIBA NF on-demand treatment to 7.9 during
FEIBA NF prophylactic treatment (a 72.5% reduction). The Phase III study will
form the basis of a biologics license application (BLA) to be filed with the
U.S. Food and Drug Administration (FDA) in the first quarter of 2013.
As many as one-third of people with hemophilia develop an inhibitor to a
product used to treat or prevent bleeding episodes. The presence of an
inhibitor makes response to treatment more challenging and patients with
inhibitors have an increased risk of developing complications such as joint
''Treatment with FEIBA NF resulted in a significant reduction in annual bleed
rate (ABR) of all types of bleeds in the prophylaxis arm as compared to the
on-demand arm,'' said lead investigator, Dr. Sandra Antunes MD, UNIFESP, Sao
Paulo, Brazil. ''Three of the 17 intent to treat patients (17.6 %) in the
prophylaxis arm did not experience any bleeding episodes during the study, and
this is very significant for hemophilia patients with inhibitors.''
The Phase III prospective, open label, randomized, multi-center, parallel
study investigated the efficacy, safety and health-related quality of life
benefits of FEIBA NF prophylactic treatment compared to on-demand treatment in
36 patients with hemophilia A or B and inhibitors over a 12-month period. The
most commonly reported adverse reactions in the study were hypersensitivity,
dizziness, headache, rash, hypotension and hepatitis B surface antibody
positive laboratory test result. The occurrence of a transitory increase in
hepatitis B surface antibodies has been seen in certain plasma-derived
products and could be attributed to the passive transfer of antibodies
following FEIBA NF treatment. None of the subjects showed any signs or
symptoms of hepatitis B infection.
This latest study adds to the clinical evidence supporting the prophylactic
use of FEIBA, building on an investigator initiated study showing that FEIBA
can reduce bleeding events in patients with severe hemophilia A and inhibitors
when compared to on-demand treatment (results published in The New England
Journal of Medicine in November 2011).
''One of the greatest remaining challenges in the management of hemophilia is
the development of inhibitors, which can lead to more difficult-to-control and
sometimes life-threatening bleeding. The FEIBA NF prophylaxis study
demonstrates Baxter’s dedication to providing treatment options to the
hemophilia community, including effective inhibitor management,'' said Prof.
Hartmut J. Ehrlich, M.D., vice president of global research and development in
Baxter’s BioScience business.
About FEIBA NF
FEIBA NF is not indicated for prophylaxis use in the United States. Canada,
The Netherlands, Israel, Australia/New Zealand, Japan and South Korea also do
not have a prophylaxis indication.
Indications for FEIBA NF
In the U.S., FEIBA NF [Anti-Inhibitor Coagulant Complex] is indicated for the
control of spontaneous bleeding episodes or to cover surgical interventions in
hemophilia A and hemophilia B patients with inhibitors.
Clinical experience suggests that patients with a Factor VIII inhibitor titer
of less than five Bethesda Units (B.U.) may be successfully treated with
Antihemophilic Factor. Patients with titers ranging between 5 and 10 B.U. may
either be treated with Antihemophilic Factor or FEIBA NF. Cases with Factor
VIII inhibitor titers greater than 10 B.U. have generally been refractory to
treatment with Antihemophilic Factor.
Inadequate response to treatment may result from an abnormal platelet count or
impaired platelet function that were present before treatment with FEIBA NF,
Nanofiltered and Vapor Heated.
Detailed Important Risk Information for FEIBA NF
Thrombotic and thromboembolic events have been reported during postmarketing
surveillance following infusion of FEIBA VH or FEIBA NF, particularly
following the administration of high doses and/or in patients with thrombotic
The use of FEIBA NF is contraindicated:
*In patients who have known anaphylactic or severe hypersensitivity
reactions to the product.
*In patients who are known to have a normal coagulation mechanism.
*For the treatment of bleeding episodes resulting from coagulation factor.
deficiencies in the absence of inhibitors to coagulation factor VIII or
coagulation factor IX.
*In patients with significant signs of disseminated intravascular
*In patients with acute thrombosis or embolism (including myocardial
At first sign or symptoms of an infusion/hypersensitivity reaction or a
thrombotic/thromboembolic event, FEIBA NF administration should be stopped
immediately and diagnostic and therapeutic measures initiated as appropriate.
Allergic-type hypersensitivity reactions, including severe anaphylactoid
reactions, have been reported following the infusion of FEIBA. The symptoms
include urticaria, angioedema, gastrointestinal manifestations, bronchospasm,
and hypotension; these reactions can be severe and can be systemic.
Many of the reported cases of thromboembolic events occurred with doses above
200 units/kg/day or in patients with other risk factors.
Infusion of FEIBA NF should not exceed single dosage of 100 U/kg and daily
doses of 200 U/kg of body weight. Patients receiving more than 100 U/kg of
FEIBA NF must be monitored for the development of DIC and/or symptoms of acute
coronary ischemia. High doses of FEIBA NF should be given only as long as
absolutely necessary to stop bleeding.
FEIBA VH or FEIBA NF should be used with particular caution and only if there
are no therapeutic alternatives in patients at risk of DIC, arterial or venous
If clinical signs of intravascular coagulation occur, which include changes in
blood pressure, changes in pulse rate, respiratory distress, chest pain and/or
cough, infusion of FEIBA NF should be stopped promptly.
Non-hemophilic patients with acquired inhibitors against factors VIII, IX or
XII may have both a bleeding tendency and an increased risk of thrombosis at
the same time.
FEIBA NF is made from human plasma. It may carry a risk of transmitting
infectious agents, e.g., viruses and theoretically, the Creutzfeldt-Jakob
disease (CJD) agent.
Adverse reactions reported in clinical studies with FEIBA were anamnestic
response, somnolence, dizziness, dysgeusia, dyspnea, hypoesthesia, nausea,
chills, pyrexia, chest pain and chest discomfort.
Please see full prescribing information for FEIBA NF at:
Licenses and licensing conditions may vary from country to country; therefore
please always consult your local full prescribing information. Please check
FEIBA NF website for information on indications approved in other countries.
About Hemophilia A
Hemophilia is a rare genetic blood clotting disorder that primarily affects
males.^1 People living with hemophilia do not have enough of, or are missing,
one of the blood clotting proteins naturally found in blood.^1 Two of the most
common forms of hemophilia are A and B.^2 In people with hemophilia A,
clotting factor VIII is not present in sufficient amounts or is absent.^2
Without enough FVIII, people with hemophilia can experience spontaneous,
uncontrolled internal bleeding that is painful, debilitating, damaging to
joints and potentially fatal.^2 According to the World Federation of
Hemophilia, more than 400,000 people in the world have hemophilia.^2 All races
and economic groups are affected equally.^1
About Hemophilia B
Hemophilia B is the second most common typeof hemophilia (also known as
Christmas disease) and is the result of insufficient amounts of clotting
factor IX, a naturally occurring protein in blood that controls bleeding.^3
Approximately 25,000 people worldwide, including more than 4,000 in the U.S.,
have been diagnosed with hemophilia B. ^ 4 Hemophilia B is often a
debilitating, chronic disease with complications that include bleeding
episodes, hemophilic arthropathy (bleeding into a joint) and
As many as one-third of patients with severe or moderately severe hemophilia A
are at risk for developing inhibitors, which are antibodies produced by the
body’s immune system in response to factor replacement therapy. Inhibitors
cause the body to work against the factor replacement therapy, neutralizing
its effect and preventing an individual’s blood from appropriate clotting.^6
Individuals who have inhibitors have a form of hemophilia that is more
difficult to control, with an increased risk of uncontrolled bleeding,
compared to patients without inhibitors. Inhibitor development is considered
one of the most serious complications associated with hemophilia treatment,
and may include other associated complications such as impaired movement,
increased need for surgery and greater complexity or risk associated with
surgery, lower life expectancy and poor health-related quality of life. ^6,7
About Baxter in Hemophilia
Baxter has more than 60 years experience in hemophilia and has introduced a
number of therapeutic firsts for hemophilia patients. Baxter has the broadest
portfolio of hemophilia treatments in the industry and is able to meet
individual therapy choices, providing a range of options at each treatment
stage. The company’s work is focused on optimizing hemophilia care and
improving the lives of people living with hemophilia A and B worldwide.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures
and markets products that save and sustain the lives of people with
hemophilia, immune disorders, cancer, infectious diseases, kidney disease,
trauma and other chronic and acute medical conditions. As a global,
diversified healthcare company, Baxter applies a unique combination of
expertise in medical devices, pharmaceuticals and biotechnology to create
products that advance patient care worldwide.
This release includes forward-looking statements concerning the company’s
Phase III study evaluating the efficacy and safety of routine prophylaxis
compared to on-demand treatment of FEIBA NF in hemophilia patients with
inhibitors, including expectations regarding related regulatory filings.The
statements are based on assumptions about many important factors, including
the following, which could cause actual results to differ materially from
those in the forward-looking statements: satisfaction of regulatory and other
requirements; actions of regulatory bodies and other governmental authorities;
changes in laws and regulations; product quality or patient safety issues; and
other risks identified in Baxter's most recent filing on Form 10-K and other
SEC filings, all of which are available on Baxter's website. Baxter does not
undertake to update its forward-looking statements.
^1 What is Hemophilia? World Federation of Hemophilia. Accessed on: May 24,
2012. Available at: http://www.wfh.org/en/page.aspx?pid=646.
^2 Frequently Asked Questions About Hemophilia. World Federation of
Hemophilia. Accessed on: May 24, 2012. Available at:
^3 Frequently Asked Questions About Hemophilia.World Federation of
Hemophilia.Accessed on April 20, 2012 Available at:
^4 World Federation of Hemophilia Report on the Annual Global Survey 2010.
World Federation of Hemophilia. Accessed on April 20, 2012. Available at:
^5 Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and
Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A.
Valentino), Wiley-Blackwell, Oxford, UK. Accessed on April 20, 2012. Screen
shot of page available here
^6 What are Inhibitors (section)? World Federation of Hemophilia. Accessed on
December 14, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=651.
^7 Leissinger, Cindy A. Prevention of Bleeds in Hemophilia Patients With
Inhibitors: Emerging Data and Clinical Direction. American Journal of
Hematology. 2004; 77:187-193.
Baxter International Inc.
Brian Kyhos, (224) 948-4210
Deborah Spak, (224) 948-2349
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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