Exjade deferasirox regulatory update

EMA's CHMP recommended against expanding the label of Exjade deferasirox from Novartis to include first-line treatment of chronic iron overload in patients ages >=6 years with beta thalassemia major who receive infrequent blood transfusions. The committee said the data were insufficient, noting that the data submitted contained methodological weaknesses and did not show that Exjade is at least as effective as deferoxamine in the first-line indication. Exjade is approved for first-line treatment of patients ages >=6 years with beta thalassemia major who receive frequent blood transfusions and second-line treatment, when deferoxamine cannot be used or is inadequate, in patients ages 2-5 years with beta thalassemia major who receive frequent blood transfusions, in patients ages >=2 years with beta thalassemia major who receive infrequent blood transfusions and in patients ages >=2 years who suffer from different types of anemia and receive blood transfusions. The committee did conclude that new data on kidney function in patients treated with Exjade should be added to the product label. ...