Reason for request
Renewal of inclusion
The actual benefit of EXJADE in the Marketing Authorization indications is substantial.
Improvement in actual benefit
|III (modéré)||Given its oral administration and : - the failure to strictly demonstrate the non-inferiority of the efficacy of EXJADE relative to DESFERAL regarding the primary efficacy endpoint, - a sustained rate of adverse effect reporting more than 6 years after the Marketing Authorization, - the seriousness of the adverse effects observed, - the need to continue long-term monitoring of renal safety, especially in children, - the lack of demonstration of the impact of EXJADE on compliance and quality of life observed under actual conditions of use, - the lack of available data on the possibility of retreatment in patients who re accumulate iron after having achieved a satisfactory body iron level,|
|IV (mineur)||the Committee believes that the improved actual benefit (IAB) of EXJADE is : - minor (level IV) relative to deferoxamine (DESFERAL) in the treatment of chronic iron overload due to frequent blood transfusions (≥7 ml/kg/month of packed red blood cells) in patients with beta thalassemia major aged 6 years and older and - moderate (level III) in the treatment of chronic iron overload due to blood transfusions when deferoxamine therapy (DESFERAL) is contraindicated or inadequate in the following patient groups: • with other types of anaemia, • aged 2 to 5 years, • with beta thalassemia major with chronic iron overload due to infrequent blood transfusions (<7 ml/kg/month of packed red blood cells). In the situation where FERRIPROX can be used (iron overload in patients who have thalassemia major for whom a treatment with deferoxamine is contraindicated or inadequate), the Transparency Committee cannot rule on the respective performances of the two medicinal products due to lack of comparative data. In the indication "treatment of chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients with non-transfusion dependent thalassemia syndromes aged 10 years and older" the Transparency Committee finds an IAB IV (minor). For the rationale, refer to the specific opinion delivered for this extension of indication of 22 January 2014.|