FDA Approves First Long-acting Recombinant Coagulation Factor IX
Concentrate for Patients with Hemophilia B
The U.S. Food and Drug Administration today approved Alprolix, Coagulation Factor IX (Recombinant), Fc Fusion Protein, for use in adults and children who have Hemophilia B. Alprolix is the first Hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding.
Alprolix is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes (prophylaxis). Alprolix consists of the Factor IX molecule linked to a protein fragment, Fc, which is found in antibodies. This makes the product last longer in circulation.
“The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research.
Baxter Receives FDA Approval of FEIBA [Anti-Inhibitor Coagulant Complex] for Prophylactic Treatment of Hemophilia A&B Patients with Inhibitors
Pivotal Phase III data showed 72% reduction in median annual bleed rate with FEIBA prophylactic treatment compared to an on-demand regimen.
DEERFIELD, Ill., December 19, 2013 - Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) granted approval of Baxter's FEIBA [Anti-Inhibitor Coagulant Complex], the first and only FDA-approved treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B who have developed inhibitors.
Inhibitor development is considered one of the most serious complications associated with hemophilia treatment today. As many as one-third of previously untreated patients with severe or moderately severe hemophilia A are at risk for developing inhibitors, which are antibodies produced by the body's immune system in response to factor replacement treatment. The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications.
The approval is based on data from a pivotal Phase III study, known as FEIBA PROOF, in which treatment with a FEIBA prophylactic regimen showed a 72 percent reduction in median annual bleed rate (ABR) compared to treatment with an on-demand regimen. In the intent-to-treat (ITT) analysis, three of the 17 (18%) adult patients in the prophylactic arm reported no bleeding episodes. The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.
"The PROOF study demonstrated that a prophylactic regimen with FEIBA can significantly reduce the rate of bleeding episodes, as compared to an on-demand regimen, in hemophilia patients with inhibitors. This is important among these patients who often have difficult-to-treat bleeds and are at risk of additional complications," said Steven Pipe, M.D., Director, Division of Pediatric Hematology and Oncology, at the C.S. Mott Children's Hospital at the University of Michigan. "This FDA approval of a prophylactic regimen should change the way physicians think about managing hemophilia with inhibitors, validating FEIBA prophylaxis as an effective new option to treat their patients." Pro-FEIBA, a prospective investigator-initiated, randomized, crossover study published in the New England Journal of Medicine in 2011 showed a 62 percent reduction in all bleeding episodes with FEIBA prophylaxis compared to an on-demand regimen. Together with the data from the FEIBA PROOF study, the results provide valuable evidence that prophylaxis with FEIBA can significantly reduce bleeding rates when compared to on-demand treatment.
"FEIBA has been an effective treatment for hemophilia patients with inhibitors for more than 35 years as an on-demand treatment. FEIBA was first licensed in the US as FEIBA VH and then as FEIBA NF. This additional indication for prophylactic treatment is aimed at reducing the number of bleeds among this patient population," said Ludwig Hantson, Ph.D., president of Baxter's BioScience business. "This latest approval reflects our legacy of offering new approaches to help reduce the disease burden of hemophilia, and supports Baxter's ongoing commitment to pursuing a bleed-free world."
FEIBA is approved in more than 60 countries worldwide and is indicated for prophylaxis in more than 40 countries.
FDA Approves TRETTEN® for the Treatment of Congenital Factor XIII A-Subunit Deficiency
Novo Nordisk today announced the US Food and Drug Administration (FDA) has approved TRETTEN® (Coagulation Factor XIII A-Subunit [Recombinant]) for the routine prophylaxis of bleeding in people with congenital factor XIII (FXIII) A-subunit deficiency, a serious, rare bleeding disorder with limited treatment options.
TRETTEN® is the only recombinant treatment for congenital FXIII A-subunit deficiency. TRETTEN® was proven safe and effective, offering patients once-monthly dosing with a short infusion time. Patients with congenital FXIII A-subunit deficiency have a lifelong susceptibility towards bleeding, including spontaneous intracranial hemorrhage. Caused by a lack of the protein clotting factor XIII (FXIII), congenital FXIII deficiency is estimated to occur in one in three to five million births in the United States and affects all ethnicities and both genders equally.1 As of 2011 estimates, only 1,054 patients are diagnosed worldwide, and approximately 115 live in the United States.2 Of those with congenital FXIII deficiency, approximately 95 percent of these patients have a deficiency of the A-subunit.1
"Today marks an exciting milestone for people living with congenital FXIII deficiency, and we are proud to provide a recombinant therapy to people living with this very rare disease," said Mads Krogsgaard, Chief Science Officer, Novo Nordisk. "Through our expanding portfolio of recombinant products, we are committed to serving the hemophilia and rare bleeding disorders community." FDA approved TRETTEN® based on results from a clinical program that demonstrated the safety and efficacy of TRETTEN®. The phase 3 trial that included 41 patients showed that when compared with a historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly 35 IU/kg TRETTEN® injections significantly decreased the number of treatment-requiring bleeding episodes. The most common adverse reactions reported in the clinical trials (> or = 1 percent) were headache, pain in the extremities, injection site pain, and D dimer increase.
"The FDA approval of TRETTEN® marks the culmination of years of research and development to help bring this much-needed treatment to market," said Eddie Williams, Corporate Vice President, BioPharmaceuticals, Novo Nordisk. "We continue to build on our rich hemophilia heritage and remain committed to serving the bleeding disorders community."
FDA Approves Baxter's RIXUBIS for Routine Prophylaxis Treatment for Adults with Hemophilia B
The U.S. Food and Drug Administration (FDA) has approved Baxter's RIXUBIS [Coagulation Factor IX (Recombinant)], on June 26, 2013, making it the only FDA-approved recombinant factor IX indicated in the US to treat adults with hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes, control of bleeding episodes, and perioperative management. 1
RIXUBIS is not indicated for induction of immune tolerance in patients with Hemophilia B. You should not use RIXUBIS if you are allergic to hamsters or any ingredients in RIXUBIS. The efficacy and safety of RIXUBIS were evaluated in a prospective, open-label, uncontrolled, multicenter combined study of 73 male previously treated patients (PTPs) between 12 and 65 years of age who received RIXUBIS either for prophylaxis or for the treatment of bleeding episodes. The prophylaxis efficacy of RIXUBIS was studied in 56 of the 73 PTPs for a mean treatment duration of 6 months. 1
In the RIXUBIS prophylaxis study, the overall median annualized bleed rate (ABR) was 2.0 (range: 0.0-23.4). The total median ABR for spontaneous bleeds with RIXUBIS was 0.0 (range: 0.0-15.6) and for joint bleeds was 0.0 (range: 0.0-21.5). 43% of patients (24 of 56) achieved zero bleeds during 6 months of prophylactic treatment.
Some common side effects that have been reported with RIXUBIS include: unusual taste in the mouth and limb pain.
"Baxter's commitment to leadership and support of the hemophilia community began more than 60 years ago, and for more than 20 years, we have provided recombinant technologies that include the most widely used recombinant factor eight. Now we can also offer a product for prophylaxis for adults with hemophilia B," said Jacopo Leonardi, Vice President of Sales and Marketing of US Hemophilia, Baxter Healthcare Corporation.
RIXUBIS, a third-generation recombinant factor IX product, is a purified protein produced by recombinant DNA technology. 1,2 No human or animal proteins are added during any stage of manufacturing or formulation of RIXUBIS. The BAXJECT II Needle-less Transfer device can be used for mixing RIXUBIS. 1 The recombinant factor IX will be available starting October 2013 in compact packaging. RIXUBIS has a maximum infusion rate of 10 mL/minute. 1
Click here to read the entire document, including detailed important risk information about RIXUBIS.
Novo Nordisk Announces New Findings to Broaden Understanding of Improving Outcomes for People with Hemophilia
Data Presentations at ISTH Highlight Impact of Hemophilia on Health Care Utilization
PLAINSBORO, N.J., July 3, 2013 – Novo Nordisk today announced findings from the Hemophilia Experiences, Results, and Opportunities (HERO) study showing that bleed frequency and treatment regimen choice impacted the use of health care resources and comprehensive care services among adults living with hemophilia. These results were presented in an oral presentation at the XXIV Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam, the Netherlands. Additionally, the company sponsored eight poster presentations that highlighted the real-life psychosocial challenges faced by adult hemophilia patients and caregivers of affected children.
The data, based on responses from 515 adult patients living with hemophilia in eight countries, showed patients with hemophilia who treat with prophylaxis are likely to visit a comprehensive hemophilia treatment center (HTC) more frequently than those who use intermittent, on-demand treatment.1 Those on prophylaxis also reported more frequent involvement of hemophilia nurses, and to a lesser extent social workers, in management of their disease compared with patients treating on-demand.1 Irrespective of treatment regimen, those with more frequent bleeding were less likely to engage in higher-risk physical activities but had a similar number of HTC visits per year.2
"This is the first scientific survey of this type and scale to illuminate psychosocial aspects of life with hemophilia," said Eddie Williams, Corporate Vice President, Biopharmaceuticals. "The HERO initiative underscores Novo Nordisk's commitment to the hemophilia community, and we look forward to sharing new data as they become available."
The depth and breadth of the HERO findings, which is the largest multinational, comprehensive study of hemophilia to date, targeting over 1,200 people in 10 countries, is intended to broaden understanding of the factors that impact people living with hemophilia and their loved ones and identify ways in which the community can improve the quality of life for people living with this chronic disease, as well as their caregivers and families.
Click here to read the entire Novo Nordisk Press Release dated July 3, 2013
Novo Nordisk Files for Regulatory Approval of Turoctocog Alfa for Haemophilia A in the US and EU
Novo Nordisk today announced the submission of the regulatory application for turoctocog alfa (NN7008) to the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). Turoctocog alfa is a third-generation recombinant coagulation factor VIII intended for prevention and treatment of bleeding in people with haemophilia A.
"We are very excited about having reached this goal. Turoctocog alfa represents a new treatment alternative for people with haemophilia A and is one of the first important outcomes of the haemophilia research strategy we embarked upon in 2006," says Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.
Turoctocog alfa demonstrates Novo Nordisk's commitment to the wider haemophilia community as the new alternative in factor VIII treatment. Based on the most advanced protein and purification technology, the product has been designed to expand reliability, safety and portability for people with haemophilia A.
The decision to apply for marketing authorisation for turoctocog alfa is based on the results of the clinical trials guardian™1 and guardian™3, which were completed in 2011. More than 200 people with haemophilia A around the world were enrolled, making guardian™ the largest clinical pre-registration trial program conducted in haemophilia A.
The phase 3 trials included previously treated adults and children with severe haemophilia A and demonstrated efficacy in preventing and treating bleeds with no development of inhibitors.
In the coming months, applications for regulatory approval in other countries
will be submitted.
Click here to read the Novo Nordisk Press Release dated October 16, 2012
FDA Approves Longer Storage for Bayer’s Kogenate® FS
In April, Bayer HealthCare Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) approved Kogenate® FS, a recombinant factor VIII therapy, for storage at room temperature (up to 77 degrees F) for up to one year. Kogenate® FS is indicated for the treatment of hemophilia A. The previous storage time at room temperature was three months.
“As convenience with medication is important to people with hemophilia A, we're pleased to offer a new storage temperature option, which complements other convenience features, including Grab and Go packaging with BIO-SET®, a compact and complete reconstitution system for Kogenate® FS,” said Paul Bedard, Vice President/General Manager, Hematology, Bayer HealthCare Pharmaceuticals. “This new option demonstrates Bayer’s ongoing commitment to the hemophilia A community.”
Source: PRNewswire, April 25, 2011
CSL Behring Receives NORD Corporate Award
Last month the National Organization for Rare Disorders (NORD) honored CSL Behring with its 2011 Corporate Award for “new treatments brought to market for patients with rare diseases.” The award was presented during the NORD Partners in Progress Celebration 2011 on May 17, at the Andrew W. Mellon Auditorium in Washington, DC.
CSL Behring develops and manufactures therapies for several types of bleeding disorders. In February 2011 the company's product Corifact, a plasma-derived factor XIII (FXIII) therapy, received U.S. Food and Drug Administration approval for the routine prophylactic treatment of congenital FXIII deficiency.
Factor XIII deficiency, a very rare disorder affecting approximately 150 people in the U.S, can be associated with prolonged, trauma-related bleeding. More severely affected patients are at risk for head bleeds. Women who go untreated risk spontaneous abortion. Men with the deficiency may show signs of infertility. Common symptoms include soft tissue bleeds, menorrhagia, joint bleeding and persistent bleeding during circumcision or at the site of the umbilical cord.
“CSL Behring is honored to receive this NORD Corporate Award,” said Paul Perreault, CSL Behring Executive Vice President for Worldwide Commercial Operations and incoming president. “People with rare diseases often face a host of challenges in being accurately diagnosed and in gaining ongoing access to appropriate medical care. CSL Behring focuses on these areas and partners with groups such as NORD to improve patients' lives. We commend NORD for their outstanding achievements and dedication to supporting people with rare diseases.”
NORD is a patient advocacy organization that advances the causes of people with rare diseases. It provides support for orphan product research used to treat serious conditions that affect fewer than 200,000 people. It develops and advocates on public policy issues before Congress and health agencies.
Source: CSL Behring news release dated May 17, 2011