New Thalassemia Drug Raises Hopes for Rapid Gulf Approval
Thalassemia patients and medical specialists attending the 12th International TIF Conference for Thalassemia expressed their hope that the breakthrough oral treatment for iron chelation, Exjade, will be available for use in Gulf countries in weeks rather than months or years.
Crucially, Exjade has already received US Food and Drug Administration (FDA) and Swiss approvals late in 2005. Such weighty endorsements are thought to speed the approvals processes in the various Gulf States.
The news comes at a critical time for the region’s thalassemia community – with gene carriers sometimes numbered as high as one in ten residents of some Gulf countries – which is anticipating effective, safe, and more convenient treatments for iron removal following chronic blood transfusions that enable easy compliance, particularly among young patients.
“Compliance is one of the most important issues with any therapy, but with iron chelation, compliance can mean the difference between life and death,” said Dr. Abdullah Al Khayat, Director of Dubai’s Al Wasl Hospital and Thalassemia Center.
“Because Exjade is an oral therapy that can be taken once-daily with a glass of water or orange juice, we are confident that patients of all ages will have no difficulty in adhering to the proper schedule advised by their physicians,” added Dr. Al Khayat.
Patients and parents said that they will closely follow the approvals processes taking place in their home countries.
About Iron Overload and Iron Chelation
Iron overload is a cumulative, potentially life-threatening, unavoidable consequence of chronic blood transfusions used to treat certain types of rare chronic blood disorders, including thalassemia and sickle cell disease, as well as other rare anemia and myelodysplastic syndromes. Signs of iron overload may be detected after transfusion of about 20 units of blood. In many patients the need for transfusions may be life-long. If left undiagnosed or untreated, the excess iron in the body is likely to lead to damage to the liver, heart and endocrine glands.
The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion related iron overload. In iron chelation, an agent binds to iron in the body and tissues and helps remove it through the urine and/or feces.
Approved in November 2005 in the United States and Switzerland, Exjade is the first and only once-daily oral iron chelator and is administered as a drink. To date, deferoxamine is the standard of care for the first-line treatment of transfusional iron overload in most countries around the world. Administration of deferoxamine often requires a subcutaneous infusion, lasting eight to 12 hours per night for five to seven nights a week for as long as the patient continues to receive blood transfusions or has excess iron within the body. Depending on the disease, patients may require hundreds of these infusions each year. As a result, many patients may have stopped or avoided iron chelation therapy, thus risking the toxic effects of iron overload.
Mild, non-progressive increases in serum creatinine, mostly within the normal range, occur in about one-third of Exjade treated patients. These are dose-dependent, often resolve spontaneously and can sometimes be alleviated by reducing the dose. Serum creatinine should be assessed before initiating therapy and should be monitored monthly thereafter to determine if dose modification or discontinuation is necessary. Liver function should be monitored monthly and if there is an unexplained, persistent, or progressive increase in serum transaminase levels Exjade should be interrupted or discontinued.
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