Drug

Avapritinib

Approved · EMA

Late-stage development

Also known as Ayvakit.

RxNorm2272107 ChEMBLCHEMBL4204794UNII513P80B4YJ

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

MegaMOST - A Multicenter, Open-label, Biology Driven, Phase II Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations /Characteristics in Advanced / Metastatic Tumors.

Clinical trial2026-07-08Results expected Q4 2026 · ClinicalTrials.gov

Phase 2 Study of Avapritinib in Patients With CKIT or PDGFRA Mutation-Positive Malignant Solid Tumors

Clinical trial2026-06-11Results expected Q4 2026 · ClinicalTrials.gov

MRD-positive AML: a Prospective, Single-arm, Multicenter Platform Clinical Study

Clinical trial2026-05-10Primary completion · ClinicalTrials.gov

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

Clinical trial2026-04-24Results posted · ClinicalTrials.gov

A Phase 1 Study of Avapritinib in Combination With Decitabine in Patients With Systemic Mastocytosis With an Associated Hematologic Neoplasm (SM-AHN)

Clinical trial2026-04-01Results expected Q1 2027 · ClinicalTrials.gov

A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed/Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation

Clinical trial2026-03-01Primary completion · ClinicalTrials.gov

An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study

Clinical trial2026-02-09Results expected Q4 2027 · ClinicalTrials.gov

Avapritinib Maintenance Following Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia With KIT Mutations

Clinical trial2026-02-01Primary completion · ClinicalTrials.gov

A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

Clinical trial2025-11-21Completed · ClinicalTrials.gov

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

Clinical trial2025-09-23Results expected Q2 2027 · ClinicalTrials.gov

Avapritinib in Relapsed Refractory or MRD-positive CBF-AML With KIT Mutations

Clinical trial2024-12-31Primary completion · ClinicalTrials.gov

Approval: Ayvakyt (EMA)

Regulatory2020-09-24EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Avapritinib

Aliases & brands

Ayvakit

RxNorm CUI

2272107

ChEMBL ID

CHEMBL4204794

UNII

513P80B4YJ

Regulatory jurisdictions

ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2020-09-24

Latest approval

2020-09-24

Authorities

EMA

Total events

emaEuropean Medicines Agency· 1 event

2020-09-24Approval

Approval: Ayvakyt (EMA)

Indication: Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic… Show full indication

Unresectable or metastatic gastrointestinal stromal tumour (GIST) AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation. Advanced systemic mastocytosis (AdvSM) AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL), after at least one systemic therapy. Indolent systemic mastocytosis (ISM) AYVAKYT is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment (see section 5.1).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.