The Medication(s) listed below have been approved by the Food & Drug Administration (FDA) as orphan products for treatment of this condition. There may be other products available
that are not considered orphan products.
Deferasirox (Brand name: Exjade)
- Manufactured by
Novartis Pharmaceuticals Corporation
FDA-approved indication: 1. Treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes and with a liver iron concentration (LIC) of at least 5 milligrams of iron per gram of liver dry weight (mg Fe/g dw) and a serum ferritin greater than 300 mcg/L. 2. Also treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age or older.
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