FDA Approves Baxter's RIXUBIS as First Recombinant Factor IX Treatment for Routine Prophylaxis of
Approval brings patients first new rFIX treatment option in more than
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 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.
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.
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