Avapritinib

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ID	`DRUG-AVAPRITINIB`
Tipo	Fármaco
Alegais	AyvakitAyvakytАваприніб
Estado	reviewed 2026-04-27 \| pending_clinical_signoff
Doenças	`DIS-GIST` `DIS-MASTOCYTOSIS`
Fontes	`SRC-NCCN-GIST-2025` `SRC-NCCN-SM-2025`

Fatos sobre as drogas

Classe	Inibidor seletivo do tipo I KIT/PDGFRA (D816V- e D842V-ativo)
Mecanismo	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.
Dose típica	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).
Ucrânia registada	Falso
NSZU reembolsado	Falso
Ucrânia verificada pela última vez	2026-04-27

Avisos

Hemorragia intracraniana / microsangues cerebrais — relacionada à dose; monitorar a RM basal + em tratamento em SM avançado

Notas

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).

Usado por

Esquema terapêutico

REG-AVAPRITINIB-ADVSM-1L - Avapritinib em monoterapia (mastocitose sistémica avançada, 1L; KIT D816V+)
REG-AVAPRITINIB-GIST-1L - Avapritinib em monoterapia (GIST avançado/metástatico 1L; PDGFRA D842V)