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.
- 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
- YourBlueprint
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
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.
- 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.
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2020 | Unresectable/metastatic GIST with PDGFRA exon 18 mutation including D842V |
| US | 2021 | Advanced systemic mastocytosis |
| 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.
Trials
topRecruiting now (live from ClinicalTrials.gov)
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Landmark trials in OnCo
Latest papers
topQuery 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.
Pages like this
not linked directly; found by shared links- ProductRipretinib
Shares PDGFRA, Gastrointestinal stromal tumour (GIST), KIT, Sarcomas (soft tissue, bone, GIST).
- ProductSunitinib
Shares PDGFRA, Gastrointestinal stromal tumour (GIST), KIT, Sarcomas (soft tissue, bone, GIST).
- ProductPexidartinib
Shares KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
- ProductPazopanib
Shares KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
- TrialSSGXVIII/AIO (adjuvant imatinib in GIST)
Shares KIT, Sarcomas (soft tissue, bone, GIST).
- TrialINVICTUS
Shares KIT, Sarcomas (soft tissue, bone, GIST).
- TargetCSF1R
Shares PDGFRA, KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
- ProductRegorafenib
Shares Gastrointestinal stromal tumour (GIST), KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.