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FDA Approves Baxter's RIXUBIS [Coagulation Factor IX (Recombinant)] for Treatment of Children with Hemophilia B

Approval Based on Clinical Data Finding 39% of Pediatric Patients Less Than 12 Years of Age Experienced No Bleeds on Prophylactic Treatment

DEERFIELD, Ill., September 15, 2014 - 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 and prevention of bleeding episodes, and perioperative management in children with hemophilia B. RIXUBIS was the first recombinant factor IX (rFIX) approved for routine prophylaxis and control of bleeding episodes in the U.S. for adults living with this chronic condition.

"In addition to the positive reception we've received from adult RIXUBIS patients, the approval for pediatric patients offers a valuable new option, particularly as our clinical data demonstrated a significant reduction in bleeding episodes for patients who were treated prophylactically, an important factor for this young patient population," said John Orloff, M.D., vice president of global research and development at Baxter BioScience.

The approval is based on the results of a clinical trial investigating the efficacy and safety of RIXUBIS among 23 previously-treated male patients less than
12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (mean dose
56 IU/kg) for a mean treatment duration of six months and a mean of 54 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 bleeding episodes (88.5%) were treated with 1-2 infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants. Common adverse reactions observed in >1% of subjects in clinical studies were dysgeusia, pain in extremity, and positive test for furin antibody. These data were presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA.

Baxter's application for marketing approval for RIXUBIS for adults and pediatric patients is currently under review in the European Union, with a regulatory decision expected later this year. The treatment also recently gained regulatory approval in Australia.

About RIXUBIS
RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor indicated in adults and children with hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis. RIXUBIS is not indicated for induction of immune tolerance in patients with hemophilia B.

RIXUBIS was the first rFIX approved by the FDA for both routine prophylaxis and control of bleeding episodes in adults with this chronic condition. 

Important Risk Information for RIXUBIS

CONTRAINDICATIONS
RIXUBIS is contraindicated in patients who have:

  • Known hypersensitivity to RIXUBIS or its excipients including hamster protein
  • Disseminated Intravascular Coagulation (DIC)
  • Signs of fibrinolysis


WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms such as dyspnea and pruritus) have been reported with RIXUBIS. Anaphylaxis and other hypersensitivity reactions are possible. Early signs of allergic reactions, which can progress to anaphylaxis, include angioedema, chest tightness, hypotension, lethargy, nausea, vomiting, paresthesia, urticaria, rash, restlessness, wheezing, and dyspnea. Immediately discontinue administration and initiate appropriate treatment if allergic- or anaphylactic-type reactions occur.

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

Thromboembolic Complications
The use of factor IX containing products has been associated with the development of thromboembolic complications. Monitor patients for early signs of thromboembolic and consumptive coagulopathy, when administering RIXUBIS to patients with liver disease, with signs of fibrinolysis, peri- and post-operatively, or at risk for thromboembolic events or DIC.

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

Post-marketing Experience
The following class adverse reactions have been seen with another recombinant factor IX: inadequate factor IX recovery, inhibitor development, anaphylaxis, angioedema, 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 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; additional clinical results; changes in laws and regulations; product quality or supply 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.


1Types 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
22011 Annual Global Survey. World Federation of Hemophilia. Accessed on May 10, 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf
3Lee-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