# Avapritinib

Avapritinib (Ayvakyt, Blueprint Medicines (Netherlands) B.V.) has 3 EU-approved indications in 2 tumour types. 1 needs a biomarker result: PDGFR (1).

## Approved indications

### Gastrointestinal stromal tumour

| Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|
| PDGFRA D842V | Monotherapy | Advanced | - |

### Systemic mastocytosis

| Biomarker requirement | Regimen | Setting | Conditions |
|---|---|---|---|
| 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. |
| None | Monotherapy | Any line | Indolent systemic mastocytosis only; Moderate to severe symptoms inadequately controlled on symptomatic treatment |

## Products

### Ayvakyt

- Company: Blueprint Medicines (Netherlands) B.V.
- Target: PDGFRA and KIT kinase inhibitor
- Class: Other protein kinase inhibitors
- ATC: L01EX18
- EU approval: 24 Sep 2020
- Label revised: 29 Oct 2026
- Status: conditional approval, orphan medicine
- SmPC (EN): https://www.ema.europa.eu/en/documents/product-information/ayvakyt-epar-product-information_en.pdf
- Fachinformation (DE): https://www.ema.europa.eu/de/documents/product-information/ayvakyt-epar-product-information_de.pdf
- EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/ayvakyt

Mechanism (SmPC section 5.1): Avapritinib is a type 1 kinase inhibitor that targets mutant PDGFRA and KIT, inhibiting receptor autophosphorylation and the proliferation of tumor cells harboring these mutations.

## Label wording

- **Avapritinib** (Ayvakyt), Gastrointestinal stromal tumour, advanced: “AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation.” [SmPC](https://www.ema.europa.eu/en/documents/product-information/ayvakyt-epar-product-information_en.pdf)
- **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)

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/avapritinib