恩瑞格的获批适应症

Approved indications: In November 2005, the U.S. FDA approved Enrig for the treatment of patients 2 years old and older with chronic iron overload caused by blood transfusion. In December 2012, Deferasirox was approved by the European Commission for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndrome who require chelation therapy due to contraindications or insufficiency of deferoxamine mesylate therapy. On January 23, 2013, the FDA approved a new indication for deferasirox for the treatment of chronic iron overload in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT).

Non-transfusion-dependent thalassemia (NTDT) is a mild form of thalassemia that does not require frequent blood transfusions. But over time, NTDT patients are at risk for iron overload, which can lead to fatal organ damage. According to the National Heart, Lung, and Blood Institute, an estimated 1,000 people in the United States have thalassemia. Thalassemia causes the body to produce fewer healthy red blood cells and hemoglobin, which carries oxygen to various parts of the body and transports carbon dioxide from various parts of the body back to the lungs, where it is then exhaled. Some people with thalassemia require frequent blood transfusions to keep their hemoglobin at an acceptable level. Iron overload is a common phenomenon in these patients.

Deferasirox () was previously approved for the treatment of iron overload due to transfusion in patients 2 years of age and older, and this approval allows deferasirox to be used in the treatment of iron overload in patients with NTDT. Deferasirox should be used in patients with NTDT who have at least 5 mg of iron per 1 g of desiccated liver tissue. The new indication for deferasirox was approved by the FDA through the accelerated approval process, which allows patients to have early access to this promising new drug to treat serious or life-threatening diseases, while the company conducts additional studies to ensure the clinical effectiveness of deferasirox.