# Midostaurin

Source: https://onco.cc/drugs/midostaurin/  
OnCo record `midostaurin` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

The first drug to improve survival in FLT3-mutated AML, added to standard chemotherapy: median survival went from about two years to more than six.

## Summary

RATIFY (n=717, FLT3-ITD or TKD, age 18-59): midostaurin with 7+3 and as maintenance improved OS (median 74.7 vs 25.6 months, HR 0.78). Approved 2017 for newly diagnosed FLT3-mutated AML and for systemic mastocytosis. Now being displaced in FLT3-ITD by quizartinib (QuANTUM-First) and challenged by gilteritinib in frontline trials; still the only option labelled for FLT3-TKD.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Rydapt
- Modality: Small-molecule multikinase inhibitor (FLT3)
- Mechanism: Type I multikinase inhibitor of FLT3 (ITD and TKD), KIT, PDGFR, VEGFR2, and PKC.
- Approvals: US 2017: Newly diagnosed FLT3-mutated AML with 7+3 and consolidation; advanced systemic mastocytosis
- Dosing: Oral; 50 mg twice daily on days 8-21 of each induction and consolidation cycle, then 50 mg twice daily continuously for up to 12 cycles of maintenance
- Toxicity (grade 3+): Febrile neutropenia 84%; Rash/desquamation 14%

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Midostaurin
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Midostaurin

## Connected records

- biomarkers: [FLT3-ITD (internal tandem duplication)](https://onco.cc/biomarkers/flt3-itd/), [FLT3-TKD (D835 and I836 tyrosine kinase domain mutations)](https://onco.cc/biomarkers/flt3-tkd/)
- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute myeloid leukaemia in older or unfit patients](https://onco.cc/cancers/aml-older-unfit/), [Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia)](https://onco.cc/cancers/advanced-systemic-mastocytosis/), [FLT3-mutated acute myeloid leukaemia](https://onco.cc/cancers/aml-flt3/), [Systemic mastocytosis](https://onco.cc/cancers/systemic-mastocytosis/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [FLT3](https://onco.cc/targets/flt3/), [KIT](https://onco.cc/targets/kit/), [Protein kinase C family (PKC)](https://onco.cc/targets/pkc/), [STAT5 (STAT5A, STAT5B)](https://onco.cc/targets/stat5/)
- companies: [Novartis](https://onco.cc/companies/novartis/)
- trials: [A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML](https://onco.cc/trials/nct03591510/), [RATIFY (CALGB 10603)](https://onco.cc/trials/ratify/)
- pairings: [FLT3 inhibitor + intensive chemotherapy](https://onco.cc/pairings/flt3i-plus-7-3/)
- key papers: [ADMIRAL: gilteritinib pills versus chemotherapy for relapsed FLT3-mutated acute myeloid leukaemia](https://onco.cc/key-papers/paper-admiral-gilteritinib-flt3-nejm-2019/), [Efficacy and safety of midostaurin in advanced systemic mastocytosis](https://onco.cc/key-papers/paper-gotlib-midostaurin-advanced-systemic-mastocytosis-nejm-2016/), [QuANTUM-First: quizartinib added to intensive chemotherapy and continued as maintenance in newly diagnosed FLT3-ITD AML](https://onco.cc/key-papers/paper-quantum-first-quizartinib-lancet-2023/), [RATIFY: midostaurin added to chemotherapy for acute myeloid leukaemia with a FLT3 mutation](https://onco.cc/key-papers/paper-ratify-midostaurin-nejm-2017/)
- terms: [7+3 induction chemotherapy](https://onco.cc/terms/seven-plus-three/)
- pathways: [Acute myeloid leukaemia (KEGG map)](https://onco.cc/pathways/aml-signalling/)

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