deferasirox (Exjade)

following a resubmission considered under the ultra-orphan process

deferasirox (Exjade®) is accepted for restricted use within NHS Scotland.

Indication under review: Treatment of chronic iron overload due to blood transfusions when deferoxamine therapy is contraindicated or inadequate, in adult and paediatric patients aged 2 years and older with rare acquired or inherited anaemias. 

The current advice relates only to use in the myelodysplastic syndrome (MDS) population.

SMC restriction: use in patients with MDS with an International Prognostic Scoring System (IPSS) score of low or intermediate -1 risk.

Plasma ferritin levels were statistically significantly reduced from baseline to end of study in two phase II/III open-label, single-arm studies of patients with MDS with an IPSS score of  low or intermediate -1 risk. 

SMC has previously accepted deferasirox for restricted use for the treatment of chronic iron overload associated with the treatment of rare acquired or inherited anaemias requiring recurrent blood transfusions. 

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.