# Avapritinib

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

## TL;DR

Avapritinib is the first drug for GIST driven by the PDGFRA D842V mutation, which resists every other kinase inhibitor; it is also approved for systemic mastocytosis.

## Summary

Avapritinib is a type I kinase inhibitor that binds the active conformation of KIT and PDGFRA, making it potent against the PDGFRA D842V and KIT exon 17 mutations that resist imatinib and every other approved GIST kinase inhibitor. In NAVIGATOR, patients with PDGFRA exon 18 (D842V) GIST had an ORR of 91%, and the FDA approved it in January 2020 for this group. VOYAGER, against regorafenib in unselected third-line GIST, was negative with PFS 4.2 versus 5.6 months (HR 1.25), so the GIST label remains limited to PDGFRA exon 18 mutations. It is now mainly a mastocytosis drug (PATHFINDER, PIONEER), approved for advanced systemic mastocytosis in 2021. Cognitive effects and a small risk of intracranial haemorrhage mean it is avoided in thrombocytopenia; Blueprint Medicines, acquired by Sanofi in 2025, developed it. It is the one drug that works for a specific GIST mutation nothing else touches.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Ayvakit
- Modality: Small-molecule kinase inhibitor (PDGFRA D842V / KIT)
- Mechanism: Type I inhibitor binding the active conformation of KIT/PDGFRA, potent against D842V and exon 17 mutations.
- Approvals: US 2020: Unresectable/metastatic GIST with PDGFRA exon 18 mutation including D842V; US 2021: Advanced systemic mastocytosis; EU 2020: EU brand Ayvakyt
- Dosing: Oral; 300 mg once daily (GIST); 200 mg (advanced SM); 25 mg (indolent SM)

## Sources

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

## Connected records

- biomarkers: [KIT D816V](https://onco.cc/biomarkers/kit-d816v/), [PDGFRA exon 18 mutation (D842V)](https://onco.cc/biomarkers/pdgfra-exon-18-d842v/)
- cancers: [Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia)](https://onco.cc/cancers/advanced-systemic-mastocytosis/), [Gastrointestinal stromal tumour (GIST)](https://onco.cc/cancers/gist/), [Indolent and smouldering systemic mastocytosis](https://onco.cc/cancers/indolent-systemic-mastocytosis/), [PDGFRA D842V-mutant GIST](https://onco.cc/cancers/gist-pdgfra-d842v/), [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/), [Systemic mastocytosis](https://onco.cc/cancers/systemic-mastocytosis/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [KIT](https://onco.cc/targets/kit/), [PDGFRA](https://onco.cc/targets/pdgfra/)
- companies: [CStone Pharmaceuticals](https://onco.cc/companies/cstone/), [Sanofi](https://onco.cc/companies/sanofi/)
- trials: [(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo ](https://onco.cc/trials/nct03731260/), [Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors](https://onco.cc/trials/nct04771520/), [EXPLORER](https://onco.cc/trials/explorer/), [NAVIGATOR](https://onco.cc/trials/navigator/), [VOYAGER](https://onco.cc/trials/voyager/)
- key papers: [EXPLORER: safety and efficacy of avapritinib in advanced systemic mastocytosis (phase 1)](https://onco.cc/key-papers/paper-explorer-avapritinib-advanced-systemic-mastocytosis-nat-med-2021/), [NAVIGATOR: avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour](https://onco.cc/key-papers/paper-navigator-avapritinib-heinrich-lancet-oncol-2020/), [PATHFINDER: efficacy and safety of avapritinib in advanced systemic mastocytosis (phase 2 interim analysis)](https://onco.cc/key-papers/paper-pathfinder-avapritinib-advanced-systemic-mastocytosis-nat-med-2021/), [PIONEER: avapritinib versus placebo in indolent systemic mastocytosis](https://onco.cc/key-papers/paper-pioneer-avapritinib-indolent-systemic-mastocytosis-nejm-evid-2023/)
- terms: [GIST risk stratification (mitotic count, size, site; Miettinen and modified NIH criteria)](https://onco.cc/terms/gist-risk-stratification/)

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