RYDAPT (midostaurin), tyrosine kinase inhibitor

Low clinical benefit in the treatment of aggressive systemic mastocytosis (ASM), systemic mastocytosis associated with a second malignant blood disorder (SM-SBD) or mast cell leukaemia (MCL), but no demonstrated clinical advantage in the therapeutic strategy.

• RYDAPT has been granted a marketing authorisation for the treatment of adults presenting with aggressive systemic mastocytosis (ASM), systemic mastocytosis associated with a second malignant blood disorder (SM-SBD), or mast cell leukaemia (MCL), in monotherapy.

• Efficacy data are limited to one non-comparative phase II study showing an overall response rate of nearly 60%, though lacking any conclusive data showing an increase in overall survival or quality of life.

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