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Opinions on drugs - Posted on Sep 10 2014

Reason for request

Extension of indication

Actual benefit


The actual benefit of EXJADE in the treatment of iron overload in patients with non-transfusion-dependent thalassaemia is substantial.

Improvement in actual benefit

IV (mineur)

In the treatment of chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients with non-transfusion-dependent thalassaemia syndromes aged 10 years and older, given :

- the procedures for demonstrating the efficacy of EXJADE in the pivotal study, whose results must be interpreted with caution,

- the lack of alternative therapies in patients who cannot take deferoxamine,

- uncertainties with regard to long-term renal safety, which may prove to be of particular concern in paediatrics,

- the lack of available data on the possibility of retreatment in patients who reaccumlate iron after having achieved a satisfactory body iron level,

- the difficulty of identifying the population for whom deferoxamine therapy is inadequate and therefore the associated risk of misuse,

the Committee believes that the improvement in actual benefit of EXJADE is minor (level IV)