FDA Approves Baxter’s RIXUBIS as First Recombinant Factor IX Treatment for Routine Prophylaxis of Hemophilia B

  FDA Approves Baxter’s RIXUBIS as First Recombinant Factor IX Treatment for
  Routine Prophylaxis of Hemophilia B

Approval brings patients first new rFIX treatment option in more than 15 years

Business Wire

DEERFIELD, Ill. -- June 27, 2013

Baxter International Inc. (NYSE:BAX) today announced that the United States
Food and Drug Administration (FDA) has approved RIXUBIS [Coagulation Factor IX
(Recombinant)] for routine prophylactic treatment, control of bleeding
episodes, and perioperative management in adults with hemophilia B. RIXUBIS is
the first new recombinant factor IX (rFIX) approved for hemophilia B in more
than 15 years and is the only rFIX indicated for both routine prophylaxis and
control of bleeding episodes in the U.S. for adult patients living with this
chronic condition. Hemophilia B is the second most common type of hemophilia
and is the result of insufficient amounts of clotting factor IX, a naturally
occurring protein in blood that controls bleeding.^1

''Baxter has long been a leader in the support of people living with bleeding
disorders with its innovative products and programs,'' said Val Bias, chief
executive officer of the National Hemophilia Foundation (NHF). ''We applaud
their ongoing commitment to the hemophilia community, demonstrated once again
with now the first prophylactic treatment approved in the U.S. for adults
living with hemophilia B.''

The approval is based on a Phase I/III study demonstrating that twice-weekly
prophylactic treatment with RIXUBIS for six months achieved a median
annualized bleed rate (ABR) of 2.0 with 43 percent of patients experiencing no
bleeds. In this study, no patients developed an inhibitory antibody to FIX and
no cases of anaphylaxis were reported. One patient developed a transient
antibody to furin. The most common adverse reactions observed in >1% of
subjects in clinical studies were: dysgeusia, pain in extremity, and positive
test for furin antibody. The study was presented at the 54^th Annual Meeting
and Exposition of the American Society of Hematology (ASH) in Atlanta.

''RIXUBIS is a welcome addition to our leading portfolio of hemophilia
treatments, providing a much-needed treatment option for the thousands of
people living with hemophilia B,'' said Ludwig Hantson, Ph.D., president of
Baxter's BioScience business. ''This approval moves us one significant step
forward in our pursuit of a bleed-free world, one patient at a time.''

For the prophylaxis regimen to prevent or reduce frequency of bleeding
episodes, RIXUBIS dosing of 40 to 60 IU per kg twice weekly may be used. Five
dosage options will be available to provide the opportunity for patients to
use a single vial to dose RIXUBIS. A BAXJECT II Needle-less Transfer device
will be included in each package of RIXUBIS.

Baxter was granted orphan-drug designation by the FDA as part of the RIXUBIS
application, a special status given to a product that would address an unmet
need for people with a rare disease or condition. Baxter expects to file for
marketing authorization for RIXUBIS in the European Union later this year.

About the Pivotal Study

The approval is based on a pivotal Phase I/III prospective, open-label,
uncontrolled, multicenter study that investigated the pharmacokinetics,
efficacy and safety of RIXUBIS in 73 previously-treated patients (between 12
and 65 years of age) with severe (factor IX level <1 percent) or moderately
severe (factor IX level ≤2 percent) hemophilia B. Patients received RIXUBIS
either for prophylaxis and/or for the treatment of bleeding episodes on an
on-demand basis, and were exposed to a factor IX-containing product on ≥150
days. Fifty-nine patients received RIXUBIS for prophylaxis twice-weekly, and
56 of them received treatment for a minimum of three months. An additional 14
patients received RIXUBIS for the treatment of bleeding episodes only, and had
to have at least 12 documented bleeding episodes requiring treatment within 12
months prior to enrollment.

The majority of patients taking RIXUBIS had arthropathy at screening (88
percent) and target joints (66 percent). Treatment was individualized based on
the severity, cause and site of bleed and of the total 249 bleeding episodes,
the majority (211; 84.7 percent) were treated with 1-2 infusions.

About RIXUBIS

RIXUBIS [Coagulation Factor IX (Recombinant)] is a recombinant factor IX
(rFIX) protein for both routine prophylaxis and control of bleeding episodes
in adults with hemophilia B. RIXUBIS is the first new rFIX approved to treat
hemophilia B in more than 15 years and is the only rFIX approved by the FDA
for both routine prophylaxis and control of bleeding episodes in this chronic
condition.

Indications for RIXUBIS

RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor
indicated for:

  *Control and prevention of bleeding episodes in adults with hemophilia B.
  *Perioperative management in adults with hemophilia B.
  *Routine prophylaxis to prevent or prevent or reduce the frequency of
    bleeding episodes in adults with hemophilia B.

RIXUBIS is not indicated for induction of immune tolerance in patients with
hemophilia B.

Important Risk Information for RIXUBIS

RIXUBIS is contraindicated in patients who have:

  *Known hypersensitivity to RIXUBIS or its excipients including hamster
    protein
  *Disseminated intravascular coagulation (DIC)
  *Signs of fibrinolysis

Hypersensitivity reactions, including anaphylaxis, have been reported with
factor IX-containing products. Early signs of allergic reactions, which can
progress to anaphylaxis, include angioedema, chest tightness, hypotension,
lethargy, nausea, vomiting, paresthesia, restlessness, wheezing, and dyspnea.
Immediately discontinue administration and initiate appropriate treatment if
allergic- or anaphylactic-type reactions occur.

Development of neutralizing antibodies (inhibitors) to factor IX may occur.
Regularly evaluate patients for the development of factor IX inhibitors by
appropriate clinical observations and laboratory tests. If expected factor IX
plasma activity levels are not attained, or if bleeding is not controlled with
an expected dose, perform an assay that measures factor IX inhibitor
concentration. Patients with factor IX inhibitors are at an increased risk of
severe hypersensitivity reactions or anaphylaxis if re-exposed to RIXUBIS.

Nephrotic syndrome has been reported following attempted immune tolerance
induction in hemophilia B patients with factor IX inhibitors. The safety and
efficacy of using RIXUBIS for immune tolerance induction have not been
established.

The use of factor IX containing products has been associated with the
development of thromboembolic complications.

The most common adverse reactions observed in >1% of subjects in clinical
studies were: dysgeusia, pain in extremity, and positive test for furin
antibody.

The following class adverse reactions have been seen with another recombinant
factor IX: inadequate factor IX recovery, inhibitor development, anaphylaxis,
angioedema, dyspnea, hypotension, and thrombosis.

Please see the RIXUBIS full Prescribing Information at:
www.baxter.com/downloads/healthcare_professionals/products/RIXUBIS_PI.pdf.

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.^1
Approximately 26,000 people worldwide, including more than 4,000 in the U.S.,
have been diagnosed with hemophilia B.^2 Hemophilia B is often a debilitating,
chronic disease with complications that include bleeding episodes, hemophilic
arthropathy (bleeding into a joint) and hospitalization.^3

About Baxter in Hemophilia

Baxter has more than 60 years experience in hemophilia and has introduced a
number of therapeutic firsts for hemophilia patients. The company 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.
Its diverse pipeline includes longer-acting therapies for hemophilia A,
potential new treatments for hemophilia B (including gene therapy), von
Willebrand disease and a recombinant treatment for patients with inhibitors.

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 RIXUBIS, including
expectations with regard to anticipated 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 approval and other
requirements; actions of regulatory bodies and other governmental authorities;
changes in law and regulations; 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 Types of Bleeding Disorders: Hemophilia B. National Hemophilia Foundation.
Accessed on May 10, 2013. Available at:
http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=181&contentid=46&rptname=bleeding

^2 2011 Annual Global Survey. World Federation of Hemophilia. Accessed on May
10, 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf

^3 Lee-Rodríguez-Merchán, E.-C. and Valentino, L. A. (2011) New Developments
in Hemophilic Arthropathy, in Current and Future Issues in Hemophilia Care
(eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford,
UK. doi:10.1002/9781119979401.ch29

Contact:

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