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Exjade

Treatment for Hemosiderosis, Iron Overload

Novartis Files Exjade New Drug Applications for Treatment of Chronic Iron Overload Due to Blood Transfusions


- Innovative, once-a-day oral formulation offers life-altering treatment option to broad range of iron overload patients

EAST HANOVER, N.J., May 03, 2005 -- Novartis has filed regulatory submissions for Exjade (deferasirox), the first and only once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions, in the United States and the European Union (EU). Submissions in other countries will follow shortly.

Exjade, also known as investigational agent ICL670, has been granted fast- track status in the US and Switzerland. Priority review has been requested in the US. Furthermore, Exjade has received Orphan Drug status in the US, EU and Australia.

An easy to administer novel oral iron chelator, Exjade is simply taken once daily, after dispersing tablets in a glass of water. Exjade was developed to extend the benefits of iron chelation to a greater number of patients receiving blood transfusions and to address the needs of thousands of adult and pediatric patients who have been using Desferal (deferoxamine). 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.

"Novartis has demonstrated a long-term commitment to help improve the lives of patients at risk for iron overload. First by developing a highly effective drug, deferoxamine, and then by conducting research on hundreds of new compounds to find an easy-to-take oral alternative to this product," said Diane Young, MD, vice president and global head of Clinical Development at Novartis Oncology. "We understand the needs of patients and know that the burdensome administration of deferoxamine limits its use. In an effort to bring the benefits of effective iron chelation to more patients, we will work diligently with health authorities to expedite the approval of this important advancement."

Iron overload is a life-threatening cumulative toxicity which results from lifesaving blood transfusions required to treat certain types of anemias and other disorders, including thalassemia, sickle cell disease, other rare anemias, and myelodysplastic syndromes. If left undiagnosed or untreated, iron overload can lead to damage to the liver, heart and endocrine glands. Transfused patients may require concomitant removal of excess iron with a type of drug therapy called iron chelation, to treat iron overload. Deferoxamine, the current standard of care in iron chelation, is effective but typically requires subcutaneous infusion lasting eight to twelve hours per day, for five to seven days a week for as long as the patient continues to receive blood transfusions.

"Blood transfusions can be a sickle cell disease patient's lifeline - they reduce the occurrence of recurrent pain episodes, as well as the risk of stroke and other life-threatening complications. But with each blood transfusion, more iron can accumulate in the body until it becomes toxic and can lead to serious organ damage," said Willarda V. Edwards, MD, president and chief operating officer, Sickle Cell Disease Association of America, Inc. "The availability of a once-daily oral iron chelator like Exjade would make it possible to eliminate excess iron without the need for the physical and emotional burden of an uncomfortable pump."

Filing data
The Exjade global clinical trials program enrolled more than 1,000 patients and is the largest ever prospectively implemented for an investigational iron chelator. The filings are based on the results of pivotal clinical trials, including a Phase III head-to-head trial vs. deferoxamine, which showed that Exjade significantly reduced liver iron concentration (LIC), an accepted indicator for body iron content, in adult and pediatric patients receiving blood transfusions. Findings from the clinical trial program were presented in December 2004 at the annual meeting of the American Society of Hematology. The studies demonstrated that Exjade led to the maintenance or reduction of absolute LIC in regularly transfused patients with different underlying diseases. Additional data on Exjade will be presented this month at three important meetings: the annual meeting of the American Society of Pediatric Hematology/Oncology in Washington, D.C. (May 14-16, 2005); the 8th International Symposium on Myelodysplastic Syndromes in Nagasaki, Japan (May 12-15, 2005); and the First Congress of the International BioIron Society in Prague, Czech Republic (May 22-27, 2005); and in June at the 10th Congress of the European Hematology Association in Stockholm, Sweden (June 2-5, 2005).

In the clinical studies in both adults and children as young as two years of age, Exjade was generally well tolerated, with the most frequently reported adverse events being nausea, vomiting, diarrhea, abdominal pain, skin rash and mild stable increases in serum creatinine, usually within the normal range.

Orphan drug designation and fast-track status
In the EU, the filing for Exjade was submitted to the European Medicines Agency under the centralized procedure. Exjade was granted Orphan Drug status in both the US and EU in 2002. The intent of the Orphan Drug designation is to stimulate the research, development, and approval of products that treat rare diseases. In the EU, the term "Orphan Drug" refers to a product that treats a serious or life-threatening disease that affects fewer than five people per 10,000 population. In the US, the term "Orphan Drug" refers to a product that treats a disease that affects fewer than 200,000 people in the country. Exjade also was granted fast-track status in the US and Switzerland. The fast-track designation is generally reserved for drugs intended for the treatment of a serious or life-threatening condition that demonstrate the potential to address unmet medical needs for that condition.

Additional information
Exjade is still in clinical development and not yet approved by the U.S. Food and Drug Administration (FDA). The brand name "Exjade" is also subject to approval by the FDA.

Some patients may be eligible to enroll in ongoing clinical trials. To learn more about Exjade clinical trials, patients, caregivers and their health care providers can call 800-340-6843, Monday through Friday, between 8:30 am and 5:30 pm Eastern Time.

About Novartis
Novartis Pharmaceuticals Corporation researches, develops, manufacturers and markets leading innovative prescription drugs used to treat a number of diseases and conditions, including central nervous system disorders, organ transplantation, cardiovascular diseases, dermatological diseases, respiratory disorders, cancer and arthritis. The company's mission is to improve people's lives by pioneering novel healthcare solutions.

Posted: May 2005

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