deferasirox (Exjade)

following an abbreviated submission

deferasirox film-coated tablets (Exjade®) is accepted for restricted use within NHS Scotland.

Indication under review:

• Treatment of chronic iron overload due to frequent blood transfusions (≥7mL/kg/month of packed red blood cells) in patients with beta thalassaemia major aged 6 years and older.

• Treatment of chronic iron overload due to blood transfusions when deferoxamine therapy is contraindicated or inadequate in the following patient groups:

- in paediatric patients with beta thalassaemia major with iron overload due to frequent blood transfusions (≥7mL/kg/month of packed red blood cells) aged 2 to 5 years,

- in adult and paediatric patients with beta thalassaemia major with iron overload due to infrequent blood transfusions (<7mL/kg/month of packed red blood cells) aged 2 years and older,

- in adult and paediatric patients with other anaemias aged 2 years and older.

SMC restriction: deferasirox film-coated tablets are restricted to use as for the SMC advice issued for deferasirox dispersible tablets (No.347/07).

Deferasirox dispersible tablets were accepted for restricted use following a resubmission under the ultra-orphan process (No. 347/07).

Deferasirox film-coated tablets will replace deferasirox dispersible tablets which are to be discontinued. Deferasirox film-coated tablets demonstrated higher bioavailability compared to the deferasirox dispersible tablet formulation and therefore a dose adjustment is required when switching from dispersible tablets to film-coated tablets.

Deferasirox film-coated tablets cannot be accepted for use in 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 as a full submission has not been received by SMC for this indication.