# Midostaurin

Midostaurin (Rydapt, Novartis Europharm Ltd) has 4 EU-approved indications in 2 tumour types. 3 need a biomarker result: FLT3 (3).

## Approved indications

### Acute myeloid leukaemia

| Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|
| FLT3 mutation | + standard daunorubicin and cytarabine | Induction | Then maintenance monotherapy |
| FLT3 mutation | Monotherapy | Maintenance | After standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy; Complete response |
| FLT3 mutation | + high-dose cytarabine | Consolidation | After standard daunorubicin and cytarabine induction chemotherapy |

### Systemic mastocytosis

| Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|
| None | Monotherapy | Any line | Advanced systemic mastocytosis only (ASM, SM-AHN or MCL) |

## Products

### Rydapt

- Company: Novartis Europharm Ltd
- Target: Multikinase inhibitor (FLT3, KIT)
- Class: Other protein kinase inhibitors
- ATC: L01EX10
- EU approval: 18 Sep 2017
- Label revised: 14 Jan 2026
- Status: orphan medicine
- SmPC (EN): https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf
- Fachinformation (DE): https://www.ema.europa.eu/de/documents/product-information/rydapt-epar-product-information_de.pdf
- EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/rydapt

Mechanism (SmPC section 5.1): Midostaurin binds the catalytic domain of receptor tyrosine kinases including FLT3 and KIT, inhibiting downstream signalling to cause cell cycle arrest and apoptosis.

## Label wording

- **Midostaurin** (Rydapt), Acute myeloid leukaemia, induction: “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 (see section 4.2);” [SmPC](https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf)
- **Midostaurin** (Rydapt), Acute myeloid leukaemia, consolidation: “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 (see section 4.2);” [SmPC](https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf)
- **Midostaurin** (Rydapt), Acute myeloid leukaemia, maintenance: “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 (see section 4.2);” [SmPC](https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf)
- **Midostaurin** (Rydapt), Systemic mastocytosis, any line: “as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL).” [SmPC](https://www.ema.europa.eu/en/documents/product-information/rydapt-epar-product-information_en.pdf)

Source: section 4.1 of each summary of product characteristics (SmPC), EMA data as of 5 Nov 2026. AI models structured these entries and no person has checked them yet. oncolabels is a non-commercial reference for education and research and gives no medical advice; the current SmPC takes precedence.

Data under CC BY 4.0: credit oncolabels and the European Medicines Agency. Page: https://oncolabels.com/drugs/midostaurin