midostaurin (Rydapt) for AML

following a full submission assessed under the ultra-orphan and end of life medicine process:

midostaurin (Rydapt®) is accepted for use within NHS Scotland.

Indication under review: In combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by midostaurin single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FMS like tyrosine kinase 3 (FLT3) mutation-positive.

In a randomised, double-blind, phase III study of adults (aged <60 years) with FLT3 mutation positive AML, the addition of midostaurin to standard intensive chemotherapy regimen resulted in improved overall survival when compared with addition of placebo.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of midostaurin. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.

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