Avapritinib
Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.
| ID | DRUG-AVAPRITINIB |
|---|---|
| Type | Drug |
| Aliases | AyvakitAyvakytАваприніб |
| Status | reviewed 2026-04-27 | pending_clinical_signoff |
| Diseases | DIS-GIST DIS-MASTOCYTOSIS |
| Sources | SRC-NCCN-GIST-2025 SRC-NCCN-SM-2025 SRC-ONCOKB |
Drug Facts
| Class | Type-I selective KIT/PDGFRA inhibitor (D816V- and D842V-active) |
|---|---|
| Mechanism | Selective KIT and PDGFRA inhibitor designed to bind the active conformation; uniquely active against the imatinib-resistant KIT D816V (advanced systemic mastocytosis) and PDGFRA D842V (GIST) activation-loop mutations. NAVIGATOR (PDGFRA D842V GIST) ORR ~88%; EXPLORER + PATHFINDER (advanced SM) ORR ~75% with deep molecular responses. |
| Typical dosing | PDGFRA D842V GIST: 300 mg PO once daily on empty stomach. Advanced systemic mastocytosis: 200 mg PO once daily on empty stomach (lower dose to mitigate cognitive / cerebral microbleed risk seen at 300 mg in EXPLORER). |
| Ukraine registered | False |
| NSZU reimbursed | False |
| Ukraine last verified | 2026-04-27 |
Warnings
- Intracranial hemorrhage / cerebral microbleeds — dose-related; monitor MRI baseline + on-treatment in advanced SM
Notes
Two distinct dose levels by indication. NAVIGATOR established 300 mg as PDGFRA D842V GIST dose (transformative ORR ~88% vs <5% historical imatinib in this genotype). EXPLORER showed cognitive AEs and cerebral microbleeds at 300 mg in advanced SM, leading to PATHFINDER's 200 mg dose registration. Baseline + on-treatment brain MRI recommended in advanced SM. Not effective in KIT D816V- negative advanced SM (rare FIP1L1-PDGFRA myeloid neoplasm with eosinophilia is treated with imatinib instead).
Used By
Regimens
REG-AVAPRITINIB-ADVSM-1L- Avapritinib monotherapy (advanced systemic mastocytosis, 1L; KIT D816V+)REG-AVAPRITINIB-GIST-1L- Avapritinib monotherapy (GIST advanced/metastatic 1L; PDGFRA D842V)