OnCo
drugsProductApproved2020🇪🇺🇬🇧🇨🇳🇦🇺

Avapritinib

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.

NAVIGATOR: ORR 91% in PDGFRA exon 18 (D842V) GIST; approved January 2020. VOYAGER (vs regorafenib in third-line GIST) was negative for PFS. Now mainly a mastocytosis drug (PATHFINDER, PIONEER). Blueprint Medicines, acquired by Sanofi 2025.

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Avapritinib
PubChem
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.
Brand / code
Ayvakit
Dosing & schedule
Route
Oral
Schedule
300 mg once daily (GIST); 200 mg (advanced SM); 25 mg (indolent SM)
Monitoring
Cognitive effects, intracranial haemorrhage risk (avoid with thrombocytopenia), oedema
Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).

Commercial insurance
covered with prior authorisation

Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. PDGFRA exon 18 mutation (GIST) or systemic mastocytosis diagnosis.

Assistance programmes

Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

Not yet researchedSearch NICE
Notes
Licensed 2020 for PDGFRA D842V-mutant GIST and advanced systemic mastocytosis; NICE positions not yet researched.

Sources: NICE search: avapritinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US2020Unresectable/metastatic GIST with PDGFRA exon 18 mutation including D842V
US2021Advanced systemic mastocytosis

Safety

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Toxicity profile
Adverse eventAny gradeGrade 3+
Cognitive effects
NAVIGATOR 300-400 mg
40%
Intracranial haemorrhage
in GIST cohorts
1%
Periorbital oedema
45%

Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
13 studies6 recruiting12 Phase 21 Phase 3
Search “Avapritinib” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Avapritinib
intervention: Avapritinib
Open on ClinicalTrials.gov →

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Landmark trials in OnCo

Latest papers

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Literature trend44 papers in the last 12 months+26% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Avapritinib" OR ABSTRACT:"Avapritinib" OR TITLE:"Ayvakit" OR ABSTRACT:"Ayvakit") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Avapritinib, not a curated reading list.

Connected

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