Regulatory approval

Published by the European Medicines Agency.

The European Medicines Agency (EMA) has authorized midostaurin 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 maintence therapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive. Midostaurin's product information further states that AML patients must have confirmation of the FLT3 mutation (internal tandem duplication [ITD] or tyrosine kinase domain [TKD]) using a validated test.

This is written in the approval document as:

Rydapt is indicated in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation-positive.

Citation

Novartis Europharm Ltd. Rydapt (midostaurin) [product information]. European Medicines Agency website. https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf. Revised July 2023. Accessed March 22, 2024.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type	Biomarker(s)	Cancer type	Therapy(ies)
Sensitivity (+)	FLT3-ITD	Acute Myeloid Leukemia	Cytarabine, Daunorubicin, Midostaurin