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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.
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.
Type I inhibitor binding the active conformation of KIT/PDGFRA, potent against D842V and exon 17 mutations. Connects to KIT.
1.Avapritinib slips into a pocket on KIT.
Background: ADC sequencing, Antigen escape (antigen loss, lineage switch), BCG-unresponsive, Castration-resistant prostate cancer (CRPC), Circulating tumour DNA (ctDNA). Also on OnCo: Resistance: how tumours escape each drug class · Lines of therapy.
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).
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.
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.
Sources: NICE TA1012. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
Compare all products across the US, EU, UK, Japan, China and Australia →
| Region | Year | Indication |
|---|---|---|
| US | 2020 | Unresectable/metastatic GIST with PDGFRA exon 18 mutation including D842V |
| US | 2021 | Advanced systemic mastocytosis |
| EU | 2020 | EU brand Ayvakyt |
| Adverse event | Any grade | Grade 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.
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Patients with indolent systemic mastocytosis whose symptoms persist on antihistamines and mast cell stabilisers now have a disease-modifying option.
Avapritinib is the most active drug in advanced systemic mastocytosis; platelet counts must be checked before and during treatment.
Avapritinib is the preferred first targeted therapy for advanced systemic mastocytosis in patients with adequate platelet counts.
Avapritinib 300 mg is the standard first-line treatment for advanced PDGFRA D842V GIST, and mutation testing before starting imatinib is essential to identify these patients.
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.
Shares NAVIGATOR: avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour, NAVIGATOR, PDGFRA D842V-mutant GIST, Gastrointestinal stromal tumour (GIST).
Shares Indolent and smouldering systemic mastocytosis, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia), KIT, Gastrointestinal stromal tumour (GIST).
Shares Indolent and smouldering systemic mastocytosis, Systemic mastocytosis, Sanofi.
Shares Indolent and smouldering systemic mastocytosis, Systemic mastocytosis, Sanofi.
Shares PDGFRA, KIT, Gastrointestinal stromal tumour (GIST), Sarcomas (soft tissue, bone, GIST).
Shares KIT, Gastrointestinal stromal tumour (GIST), Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
Shares GIST risk stratification (mitotic count, size, site; Miettinen and modified NIH criteria), PDGFRA D842V-mutant GIST, KIT, Gastrointestinal stromal tumour (GIST).