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Title: New Once-Daily, Oral Iron Chelator Provides Life-Altering Breakthrough for Patients With Iron Overload

"New Once-Daily, Oral Iron Chelator Provides Life-Altering Breakthrough for Patients With Iron Overload"

Exjade* is approved by Health Canada for the management of chronic iron overload in patients with certain types of anemia and other disorders who regularly receive blood transfusions Current treatment requires life-long therapy of a daily subcutaneous infusion lasting eight to 12 hours, starting as early as two years of age Exjade* is the only approved oral iron chelator to provide continuous chelation coverage to remove excess body iron with a once daily regimen1 DORVAL, CANADA -- October 31, 2006 -- Novartis Pharmaceuticals Canada Inc. announced the Health Canada approval of Exjade* (deferasirox), the first and only once-daily oral iron chelator to manage chronic iron overload due to blood transfusions in adults and children aged six years and older, as well as in children aged two to five who cannot be adequately treated with Desferal* (deferoxamine). Health Canada granted Novartis a Priority Review for Exjade* based on an unmet medical need for an alternate therapy to treat chronic iron overload. Iron overload is a condition that can cause life-threatening toxicity resulting in damage to key organs and premature death if undiagnosed or untreated.2 It results from frequent blood transfusions required by patients with certain severe types of anemia, such as thalassemia, sickle cell disease, myelodysplastic syndromes, and other rare types of anemia.3 In Canada, there are an estimated 800 patients treated for iron overload. "Exjade* is a significant advancement in iron chelation therapy that will increase a patient's protection from iron overload toxicity," said Dr. Ian Quirt of Toronto General Hospital & Princess Margaret Hospital, Toronto, Ontario. "Excess iron can lead to serious health complications and, if left untreated, can result in death. Because of its ease of use, Exjade* has the potential to dramatically improve a patient's adherence to treatment for transfusional iron overload." Exjade* is the only oral iron chelator that provides continuous coverage with a single daily dose.1 A breakthrough in iron chelation therapy, Exjade* was developed specifically to meet the high unmet medical need for iron chelation despite the availability of Desferal* (deferoxamine), the current standard therapy used in Canada and around the world. While effective, deferoxamine requires infusions by needle and pump, often lasting eight to 12 hours per night for five to seven nights a week as long as the patient continues to receive blood transfusions or has excess iron within the body. As a result of the inconvenience and pain associated with administration of deferoxamine, compliance with standard chelation therapy is poor.4 Patients have been frustrated for years by the inconvenience and pain that can result from daily insertion of the deferoxamine infusion needle. In many patients, the need for transfusion and chelation therapy may be life-long. "The approval of Exjade* is an extraordinary advance for people like my son Pedro, who has been receiving blood transfusions and iron chelation therapy for most of his life," said Pedro's father, Andre Oliveira. "Currently, Pedro has to use a needle and pump six days a week for ten hours a day. With the availability of Exjade* I believe Pedro's quality of life will improve dramatically." Exjade* is a remarkable therapy for iron overload that will transform the lives of people living with chronic anemia who require blood transfusions," said Mr. Riyad Elbard, Vice-President of the Thalassemia Foundation. "Before Exjade*, most patients required long and painful infusions that interrupted their daily lives." "Infusion therapy is extremely burdensome for older teens and young adults who often will not comply with therapy, even though they see first-hand the risks of iron overload," said Durhane Wong-Rieger, President and Chief Executive Officer, Anemia Institute for Research & Education. "Exjade* is the next generation treatment for transfusional iron overload and its ease of use will provide patients with an alternative therapy they will be more likely to comply with. It is imperative that provinces recognize the importance of this new treatment option and adjust their drug plans accordingly to ensure equal access to this new therapy." About Iron Overload and Iron Chelation Iron overload is a cumulative, potentially life-threatening, consequence of frequent blood transfusions. Iron starts to build up in the body after as few as 10 transfusions because the body has no mechanism to remove excess iron.5 Iron chelation therapy is the only effective treatment for transfusion-dependent iron overload. Iron chelation therapy, the removal of excess iron, is the only treatment for iron overload6 and is often necessary to prevent the potentially life-threatening complications of excess iron.1 People who receive regular blood transfusions for diseases such as thalassemia, sickle cell disease, myelodysplastic syndromes, and other rare anemias are most at risk. About Exjade* Exjade* has been granted a Notice of Compliance with Conditions by Health Canada for the management of chronic iron overload in patients with transfusion-dependent anemia aged six years or older. Exjade* is also indicated in patients aged two to five who cannot be adequately treated with deferoxamine. The results of key clinical trials have shown that Exjade*, at doses of 20 to 30 mg/kg reduces liver iron concentration (LIC) and serum ferritin. With its simple administration, Exjade* has the potential to significantly improve patient compliance and quality of life. Notice of Compliance with Conditions (NOC/c) Products approved under Health Canada's NOC/c policy are intended for the treatment, prevention or diagnosis of a serious, life-threatening or severely debilitating illness. They have demonstrated promising benefit, are of high quality and possess an acceptable safety profile based on a benefit/risk assessment. In addition, they either respond to a serious unmet medical need in Canada or have demonstrated a significant improvement in the benefit/risk profile over existing therapies. Health Canada has granted conditional market authorization for the availability of this product on the condition that Novartis carries out additional clinical trials to verify and confirm the anticipated benefit within an agreed upon time frame. Therapy with Exjade* should be initiated and maintained by physicians experienced in the treatment of chronic iron overload due to blood transfusions. Contraindications, Warnings, and Adverse Events Exjade* should not be used in patients with hypersensitivity to the active substance, deferasirox, or any of its excipients. Exjade* is not recommended in women who are pregnant or breastfeeding, nor for patients with rare hereditary problems of galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption. Exjade* should not be combined with other iron chelator therapies. Exjade* has not been studied in patients with acute cardiac failure due to iron overload. Therefore the use of Exjade* is not recommended in these patients. Exjade* has not been studied in patients with hepatic or renal impairment and must be used with caution in these types of patients. Auditory and ocular disturbances, as well as elevations of serum transaminase levels (>5xULN) and dose-dependent increases in serum creatinine have been observed. Regular monitoring of hearing, vision, liver function and serum creatinine are recommended. Exjade* was generally well tolerated. The most frequently occurring adverse events are diarrhea, vomiting, nausea, headache, constipation, dyspepsia, abdominal pain, pyrexia, cough, proteinuria, increases in serum creatinine and transaminases, pruritis and skin rash. Gastrointestinal disorders, increases in serum creatinine and skin rash were dose related. Adverse events which most frequently led to dose interruption, dose adjustment, or discontinuation of therapy were skin rash, gastrointestinal disorders, infections, increased creatinine, and increased transaminases. REFERENCES: 1. Exjade Produce Monograph, October 18, 2006 2. Brittenhaum GM et al. Efficacy of deferoxamine in preventing complication of iron overload in patients with thalassemia major. N Eng J Med. 1004. 331 (9): 567-573 3. Hershko C. Treating Iron Overload: The State of the Art. Seminar in Hematology. 2005. 42 (suppl 1): S1-S4 4. Centers for Disease Control and Prevention. Hemochromatosis for Health Care Professionals: Pathophysiology: Iron Overload. Available at: http://www.cdc.gov/hemochromatosis/training/pathophysiology/iron_overload.htm. 5. Hershko C. Treating Iron Overload: The State of the Art. Seminar in Hematology. 2005. 42 (suppl 1): S1-S4 6. Gabutti V, Piga A. Results of Long-Term Iron-Chelating Therapy. Acta Haematologica. 1996. 95:26-36 Exjade* is a registered trademark. Desferal* is a registered trademark. SOURCE: Novartis Pharmaceuticals Canada Inc.

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