Baxter Submits Application for European Approval of RIXUBIS, Recombinant Factor IX (Nonacog Gamma) for Hemophilia B

Baxter International Press Release:

Baxter Submits Application for European Approval of RIXUBIS, Recombinant Factor IX (Nonacog Gamma) for Hemophilia B  

DEERFIELD, Ill., October 31, 2013 - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for approval of RIXUBIS, recombinant factor IX (nonacog gamma) for the treatment and prophylaxis of bleeding in patients of all ages with hemophilia B. Hemophilia B, also known as Christmas disease, is the second most common type of hemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding.¹ RIXUBIS was approved for adult patients and launched in the United States and Puerto Rico earlier in 2013.
The filing is based on results of research with hemophilia B patients previously treated with other therapies. A global Phase I/III study of adult patients with severe or moderately severe hemophilia B demonstrated that twice-weekly prophylactic treatment with RIXUBIS for six months achieved a median annualized bleed rate (ABR) of 2.0, with 43 percent (24/56) of patients experiencing no bleeds. In the study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. The most common adverse reactions observed in >1% of subjects of this study were dysgeusia, and pain in extremity. The application is further supported by data from a study of 23 hemophilia B patients less than twelve years old.²
"Our focus at Baxter is to strengthen and expand the portfolio of treatments for patients with bleeding disorders, particularly when there are limited treatment options, such as for patients with hemophilia B," said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter's BioScience business. "We are actively working to extend access to these treatments and look forward to introducing RIXUBIS in Europe and other countries around the world."
Current Indications for RIXUBISIn the United States, 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 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 RIXUBISRIXUBIS 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 BHemophilia 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.³  Approximately 26,000 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 hospitalization.⁵
About Baxter in HemophiliaWith more than 60 years of experience in hemophilia, Baxter has introduced a number of therapeutic firsts for people with hemophilia. With the broadest portfolio of hemophilia treatments in the industry, Baxter is able to meet individual therapy choices, providing a range of options at each treatment stage. The company is dedicated to 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 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 approval and other requirements; actions of regulatory bodies and other governmental authorities; product quality or patient safety issues; 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.

¹Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on April 20, 2012 Available at: http://www.wfh.org/index.asp?lang=EN 
²Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ?2%) haemophilia B. Haemophilia. 2013 Jul 9.; pediatric study not yet published
³Types 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 
⁴2011 Annual Global Survey. World Federation of Hemophilia. Accessed on May 10, 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf
⁵Lee-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