Home » Quality of life improved for blood disorder patients with FDA approval of Exjade®
Nov. 3, 2005 – Texas Children’s Hospital
By Laura Frnka
The U.S. Food and Drug Administration late last night announced the approval of Exjade®, a once-daily oral treatment for chronic iron overload due to blood transfusions.
“Exjade has the potential to increase the quality of life for patients who need frequent blood transfusions because of thalassemia, sickle cell disease, rare anemias or myelodysplastic syndromes,” said Dr. Brigitta Mueller, clinical director of Texas Children’s Cancer Center and Hematology Service and a participating investigator during the clinical trials of Exjade.
“Patients with these chronic blood disorders require repeated blood transfusions because their bodies cannot produce enough healthy, red blood cells. Over time, this can lead to what is known as ‘iron overload,’ which, left untreated or undiagnosed, can cause damage to the liver, heart and endocrine glands,” she continued.
To rid their bodies of this excess iron, chronic blood disorder patients must go through a removal process called ‘iron chelation.’
Mueller, who is also an associate professor of pediatrics at Baylor College of Medicine, said that the approval of Exjade is significant because it is currently the only oral chelator available to blood disorder patients in the United States. Previously, a patient was required to go through an eight to 12-hour infusion per day, for five to seven days a week for as long as the patient continued to receive blood transfusions or had excess iron within his/her body to complete iron chelation.
Exjade, developed by Novartis Pharmaceuticals Corporation, is taken once daily after being dissolved in a glass of water or orange juice.
“It is very hard for patients to continue the regular administration of this infusion, and many start skipping doses, which leads to further problems with iron overload,” said Mueller. “Exjade eliminates the need for time-consuming infusions to rid the body of excess iron.”
The Exjade clinical trial enrolled more than 1,000 pediatric and adult patients globally, including 16 patients from Texas Children’s Sickle Center. It was the largest clinical trial of its type.
Texas Children’s Hematology Service offers comprehensive services for the evaluation and treatment of children and adolescents with acute and chronic blood diseases. The team-based program provides treatment for a broad range of blood disorders including: anemias, thrombocytopenia and neutropenias; congenital and acquired bleeding disorders; hemoglobin disorders, including sickle cell disease and thalassemia; aplastic anemia and other bone marrow failure syndromes; and hypercoagulation syndromes and thrombotic disorders