NTDT2

EXJADE® significantly reduced LIC in patients with NTDT

NTDT Efficacy_Mean change in LIC

aP value adusted with the Dunnet method.

NTDT, non–transfusion–dependent thalassemia.

Data from THALASSA, a 1-year, prospective, randomized, double-blind, placebo-controlled phase 2 study in 166 patients with β-thalassemia intermedia, α-thalassemia, or HbE/β-thalassemia with liver iron concentration (LIC) ≥5 mg Fe/g dw and serum ferritin (SF) >300 μg/l. Patients were randomized to EXJADE 5 mg/kg/day (n=55) or placebo (n=28) and EXJADE 10 mg/kg/day (n=55) or placebo (n=28).1

Core EXJADE Study

  • Significant LIC reduction—mean reduction from baseline in the EXJADE 10 mg/kg/day group was 3.8 mg Fe/g dw, at least 10 times the accumulation of 0.38 mg Fe/g dw with placebo1
  • These results demonstrate that iron accumulates in the liver at a rate of ~0.38 mg Fe/g dw per year1

SF may underestimate iron overload in patients with NTDT—however, it can still be used as an efficacy marker.2

Absolute change in LIC over time3
NTDT Efficacy_Absolute change in LIC

Data from a 1-year, prospective, randomized, double-blind, placebo-controlled phase 2 study (N=166) and an open-label, 1-year extension of THALASSA (N=133) in patients with β-thalassemia intermedia, α-thalassemia, or HbE/β-thalassemia with LIC 5 mg Fe/g dw and SF >300μg/l. Patients were randomized to EXJADE 5 mg/kg/day or placebo and EXJADE 10 mg/kg/day or placebo.3

SAFETY PROFILE ACROSS ANEMIAS

Learn more