Baxter Presents ADVATE Data on Prophylaxis Management and Efficacy for Hemophilia A Patients

  Baxter Presents ADVATE Data on Prophylaxis Management and Efficacy for
  Hemophilia A Patients

  ISTH Data Also Reinforce Efficacy as Patients’ Primary Treatment Priority

Business Wire

AMSTERDAM -- July 5, 2013

Baxter International Inc. (NYSE: BAX) today announced data featuring ADVATE
[Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method], a leading
recombinant treatment available worldwide, during the 24^th Annual Congress of
the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam,
The Netherlands. The first study identifies a correlation between peak levels
of factor VIII (FVIII) in the body and the efficacy of treatment, indicating
that frequency of peaks may be important when choosing hemophilia treatments.
A second presentation reinforces that for both hemophilia patients and
parents, efficacy through reduction in bleeds considerably outweighs infusion
convenience as the primary treatment priority.

Exploring the Importance of Managing Both Peaks and Troughs

New findings from a post-hoc analysis of the ADVATE prophylaxis study
presented at ISTH (PB 4.39-1) indicate important insights on the efficacy of
prophylaxis treatment by closely monitoring the levels of recombinant
treatment in a patient’s body.

The study examined ''peaks,'' the highest levels of factor in the body in the
first hours after infusion. Prior studies focused on ''troughs,'' representing
the lowest activity levels as the body metabolizes the treatment (typically
the period just prior to the next dose), and targeted a trough level of >1%
above baseline, but did not consider peak values.^1

The analysis identified a potentially important relationship between higher
peak values, as well as time spent above 30 and 40 percent FVIII levels, (the
“hemostatically effective” non-hemophilic FVIII range) and efficacy for
prophylaxis in hemophilia A patients. This adds to the body of knowledge that
by monitoring how long the factor remains active in the body, clinicians may
be able to design optimal individualized dosing regimens to reduce the
frequency of bleeds.^1

''Efficacy in preventing bleeding is the number one reason for choosing a
hemophilia treatment. This pharmacokinetic research suggests that the dosing
frequency to manage peaks in addition to troughs may aid in maximizing
treatment effectiveness,'' said Leonard A. Valentino, M.D., director, Rush
Hemophilia and Thrombophilia Center and Section of Pediatric
Hematology/Oncology, Rush University Medical Center, Chicago, and lead
investigator of this study. ''The finding may have implications when
considering regimens with less frequent infusions that offer fewer FVIII peaks
and less time within the hemostatically effective range.''

Both Patients and Parents Seek Efficacy as Primary Treatment Goal

Additional research presented at ISTH (#PB 3.36-4) discusses early findings
that achieving low annual bleed rates is the most critical goal for people
living with hemophilia. ^ 2 In the survey of treatment preferences among 76
adults living with hemophilia and 86 parents of children with hemophilia in
the United States, the respondents agreed that achieving zero bleeds per year
was more important than reducing dosing frequency from three times per week to
twice weekly. Further, both groups preferred a “track record of ten years of
product safety over a product new to market,” even one with less frequent

''Overall, these studies emphasize that with regard to hemophilia patient care
regimens, treatment is simply not a ‘one size fits all’ approach, and
individualized regimens can help optimize the effectiveness each patient
achieves,'' said Anders Ullman, M.D., Ph.D., vice president of global research
and development in Baxter's BioScience business. ''Meeting the needs of
individual patients and raising the standard of care are the primary focus of
Baxter’s long-term commitment in hemophilia and our pursuit of a future where
one day, every patient can live a life without bleeds.''


ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] is
indicated for the control and prevention of bleeding episodes in adults and
children (0-16 years) with hemophilia A. ADVATE is also indicated for routine
prophylaxis to prevent or reduce the frequency of bleeding episodes in adults
and children (0-16 years) with hemophilia A. ADVATE is not indicated for the
treatment of von Willebrand disease.

ADVATE is a full-length (derived from the complete FVIII gene) recombinant
FVIII product that is processed without any blood-based additives. Because no
blood-derived components are added at any stage of the manufacturing process,
the potential risk of transmitting pathogens that may be carried in
blood-based additives is eliminated. There have been no confirmed reports of
transmission of HIV, HBV or HCV with rFVIII treatments.

ADVATE is approved in 58 countries worldwide, including the United States,
Canada, 27 countries in the European Union, Argentina, Australia, Brazil,
Chile, China, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau,
Malaysia, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South
Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela.

Detailed Important Risk Information for ADVATE

ADVATE is contraindicated in patients with known anaphylaxis to mouse or
hamster protein or other constituents of the product.

Allergic-type hypersensitivity reactions, including anaphylaxis, are possible
and have been reported with ADVATE. Symptoms have manifested as dizziness,
paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus.
Discontinue use if hypersensitivity symptoms occur and administer appropriate
emergency treatment.

Carefully monitor patients treated with AHF products for the development of
FVIII inhibitors by appropriate clinical observations and laboratory tests.
Inhibitors have been reported following administration of ADVATE predominantly
in previously untreated patients (PUPs) and previously minimally treated
patients (MTPs).

If expected plasma FVIII levels are not attained, or if bleeding is not
controlled with an expected dose, perform an assay that measures FVIII
inhibitor concentration.

The serious adverse reactions seen with ADVATE are hypersensitivity reactions
and the development of high-titer inhibitors necessitating alternative
treatments to FVIII.

The most common adverse reactions observed in clinical trials (frequency
greater than or equal to 10 percent of subjects) were pyrexia, headache,
cough, nasopharyngitis, vomiting, arthralgia, and limb injury.

Please see full prescribing information for ADVATE at:

About Hemophilia A

Hemophilia is a rare genetic^3 blood clotting disorder and the most severe
forms of the disease primarily affect males.^4 People living with hemophilia
do not have enough of, or are missing, one of the blood clotting proteins
naturally found in blood.^5 Two of the most common forms of hemophilia are A
and B. In people with hemophilia A, clotting factor VIII is not present in
sufficient amounts or is absent.^5 Without enough FVIII, people with
hemophilia can experience spontaneous, uncontrolled internal bleeding that is
painful, debilitating, damaging to joints and potentially fatal.^4,6 According
to the World Federation of Hemophilia, it is estimated that more than 400,000
people in the world have hemophilia. ^ 7 All races and economic groups are
affected equally.^8

About Inhibitors

As many as one-third of patients with severe or moderately severe hemophilia A
are at risk for developing inhibitors,^9 which are antibodies produced by the
body's immune system in response to factor replacement therapy.^10 Inhibitors
cause the body to work against the factor replacement therapy, neutralizing
its effect and preventing an individual's blood from clotting.^9 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.^9

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 studies related to
the use of ADVATE and the treatment of hemophilia, including with regard to
findings related to the efficacy of prophylaxis treatment and the potential
impact on dosing regimens. 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: actions of
regulatory bodies and other governmental authorities; additional analysis of
clinical results or the development of other clinical evidence; 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

^1 2013 ISTH Congress. E-poster presentation. Peak FVIII levels and time spent
in ''hemostatically effective'' FVIII range post-infusion correlates with
improved efficacy for prophylaxis in hemophilia A: a closer look at the other
end of the curve.

^2 2013 ISTH Congress. E-poster presentation. Patient and parent preferences
for efficacy, inhibitor risk, safety, and infusion frequency associated with
prophylactic Factor VIII treatment.

^3 How do you get hemophilia? World Federation of Hemophilia. Accessed on:
June 3, 2013. Available at:

^4 Frequently Asked Questions About Hemophilia. World Federation of
Hemophilia. Accessed on: June 3, 2013. Available at:

^5 What is Hemophilia? World Federation of Hemophilia. Accessed on: June 3,
2013. Available at:

^6 Lee, C. A. Hemophilia Care in the Modern World, in Current and Future
Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino),

^7 Treatment. World Federation of Hemophilia. Accessed on June 3, 2013.
Available at:

^8 What is Hemophilia? Hemophilia Federation of America. Accessed on June 3,
2013. Available at:

^9 Leissinger, Cindy A. Prevention of Bleeds in Hemophilia Patients With
Inhibitors: Emerging Data and Clinical Direction. American Journal of
Hematology. 2004.

^10 What are Inhibitors? World Federation of Hemophilia. Accessed on June 3,
2013. Available at:


Baxter International Inc.
Media Contacts
Brian Kyhos or Deborah Spak, (224) 948-5353
Investor Contacts
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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