DEERFIELD, Ill., December 9, 2013 - Baxter International Inc. (NYSE:BAX) today announced that it has submitted an application to the United States Food and Drug Administration (FDA) for a pediatric indication for RIXUBIS [Coagulation Factor IX (Recombinant)] to treat hemophilia B. The submission was based on the results of a study presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA. RIXUBIS was approved in the United States for adults with hemophilia B earlier this year and the company filed for marketing approval in Europe in November.
"Routine prophylactic infusions can be particularly beneficial for children with hemophilia, particularly if they are started early to help prevent joint bleeding," explained lead investigator Tomasz Urasinski, Pomeranian Medical University, Department of Paediatrics, Haematology and Oncology, Szczecin, Poland. "The clinical evidence suggests that RIXUBIS prophylaxis can offer hemophilia B patients a reduction in bleeding episodes while also being tolerable, an important factor for this young population."
The Phase II/III clinical trial investigated 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 (median dose 56 IU/kg) over six months or for a minimum of 50 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 patients (88.5%) were treated with 1-2 infusions. In hemostatic efficacy, RIXUBIS was rated excellent or good at resolving bleeds in 96 percent of all episodes. Of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous. There were no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events among the study participants.
"These positive results among a pediatric patient population are consistent with those observed in the RIXUBIS pivotal study among adult patients with hemophilia B," noted Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter BioScience. "We submitted these data as part of our application for a pediatric indication for RIXUBIS to advance effective therapeutic solutions for children with hemophilia B."
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 first 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. Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.2
About Baxter in Hemophilia
Baxter has more than 60 years of 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.
1 2011 Annual Global Survey. World Federation of Hemophilia. Accessed on October 16, 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf
2 Rodríguez-Merchán E-C, Valentino LA. New developments in hemophilic arthropathy. In: Rodríguez-Merchán E-C, Valentino LA, eds., Current and Future Issues in Hemophilia Care, Oxford, UK: Wiley-Blackwell; 2011, Ch. 29. doi: 10.1002/9781119979401.