# Systemic mastocytosis

The EU has approved 3 indications of 2 drugs in systemic mastocytosis. None of them needs a biomarker result.

## Approved indications

### No biomarker required

| Drug | Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|---|
| [Avapritinib](https://oncolabels.com/drugs/avapritinib) (Ayvakyt) | None | Monotherapy | 2nd line | Advanced systemic mastocytosis only (ASM, SM-AHN or MCL); After at least one systemic therapy Ambiguous in the label (stage): check the SmPC. |
| [Avapritinib](https://oncolabels.com/drugs/avapritinib) (Ayvakyt) | None | Monotherapy | Any line | Indolent systemic mastocytosis only; Moderate to severe symptoms inadequately controlled on symptomatic treatment |
| [Midostaurin](https://oncolabels.com/drugs/midostaurin) (Rydapt) | None | Monotherapy | Any line | Advanced systemic mastocytosis only (ASM, SM-AHN or MCL) |

## Label wording

- **Avapritinib** (Ayvakyt), Systemic mastocytosis, 2nd line: “AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/ayvakyt-epar-product-information_en.pdf)
- **Avapritinib** (Ayvakyt), Systemic mastocytosis, any line: “AYVAKYT is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment (see section 5.1).” [SmPC](https://www.ema.europa.eu/en/documents/product-information/ayvakyt-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/tumours/systemic-mastocytosis