FDA Approves Baxter’s RIXUBIS as First Recombinant Factor IX Treatment for Routine Prophylaxis of Hemophilia B
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 54th 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 type of 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
Copyright Business Wire 2013