RYDAPT (midostaurine), tyrosine kinase inhibitor
Reason for request
High clinical benefit in the treatment of newly diagnosed acute myeloid leukaemia with FLT3 gene mutation, combined with chemotherapy, and minor clinical added value over chemotherapy alonein terms of efficacy.
RYDAPT has been granted an MA for the treatment of adults presenting with newly diagnosed acute myeloid leukaemia (AML) with FLT3 gene mutation, combined with standard induction chemotherapy combining daunorubicin and cytarabine, along with high-dose cytarabine consolidation chemotherapy, followed, for patients in complete remission, by a maintenance treatment of RYDAPT monotherapy.
One study demonstrated the superiority, in terms of overall survival, of a therapeutic strategy comprising 3 midostaurin-based treatment phases over a therapeutic strategy without midostaurin. A number of uncertainties remain, however, concerning the amount of effect conferred specifically by RYDAPT insofar as the patients in both treatment groups compared may have received an autologous HSC transplantation.
Improvement in actual benefit