AYVAKIT (avapritinib)
Ayvakit treats adults with gastrointestinal stromal tumors (GIST) that are metastatic or cannot be surgically removed, specifically when the tumor carries a PDGFRA exon 18 mutation. It also helps patients with various forms of systemic mastocytosis, including indolent cases and advanced conditions such as mast cell leukemia or aggressive systemic mastocytosis. This medication is used to manage these diseases by targeting specific genetic drivers, though it is not recommended for patients with low platelet counts.
How AYVAKIT Works
This medication works by inhibiting specific tyrosine kinases, such as KIT and PDGFRA, which can become mutated and cause receptors to stay constantly active. This constitutive activation leads to the uncontrolled growth of tumor cells and mast cells. By blocking these targets, the drug prevents cell signaling and helps stop the proliferation of diseased cells.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2020-01-09
- Patent Cliff
- 2042
- Revenue
- $186M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- TABLET
AYVAKIT Approval History
What AYVAKIT Treats
6 indicationsAYVAKIT is approved for 6 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Gastrointestinal Stromal Tumor
- Advanced Systemic Mastocytosis
- Aggressive Systemic Mastocytosis
- Systemic Mastocytosis with Associated Hematological Neoplasm
- Mast Cell Leukemia
- Indolent Systemic Mastocytosis
AYVAKIT Target & Pathway
ProTarget
AYVAKIT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in AYVAKIT's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications AYVAKIT treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to AYVAKIT
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
15 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04116541 MegaMOST | ET19-073 (MegaMOST) 2023-510567-35-00, 2019-001494-88 | Ph 2 | recruiting | A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors. |
| NCT03580655 results posted | BLU-285-2202 2017-004836-13 | Ph 2 | completed | (PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis |
| NCT06327685 | MCC-22318 | Ph 1 | recruiting | Avapritinib With Decitabine in Patients With SM-AHN |
| NCT04771520 | 2020-0439 NCI-2021-00702, 2020-0439 | Ph 2 | recruiting | Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors |
| NCT06748001 | BLU-285-2404 | Ph 4 | recruiting | Avapritinib Rollover Study |
| NCT04773782 | BLU-285-3101 | Ph 1, Ph 2 | completed | A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling |
| NCT03731260 | BLU-285-2203 2018-000588-99 | Ph 2 | active not recruiting | (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis |
| NCT06765915 | RJ-BMT-011 | Ph 2 | not yet recruiting | Avapritinib Maintenance for AML With KIT Mutations |
| NCT04908176 | BLU-285-1107 | Ph 1 | completed | A Drug-drug Interaction Study of Avapritinib and Midazolam |
| NCT04825574 results posted | BLU-285-1408 | Ph 4 | completed | Study for Patients Previously Treated in Avapritinib Clinical Trials |
| NCT06316960 | AVACBFKIT | Ph 2 | recruiting | Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation |
| NCT06221683 GMCAII | GMCAII | Ph 2 | recruiting | Clinical Study of Induction Therapy Options Based on Molecular Subtyping and MRD in Children and Adolescents With AML |
| NCT02561988 | BLU-285-2101 2015-001661-12 | Ph 1 | completed | (EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies |
| NCT03465722 results posted | BLU-285-1303 | Ph 3 | completed | (VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST |
| NCT02508532 results posted | BLU-285-1101 | Ph 1 | completed | (NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
AYVAKIT FDA Label Details
Indications & Usage
FDA Label (PDF)AYVAKIT is indicated for the treatment of Gastrointestinal Stromal Tumor; Advanced Systemic Mastocytosis; Aggressive Systemic Mastocytosis; Systemic Mastocytosis with Associated Hematological Neoplasm; Mast Cell Leukemia; Indolent Systemic Mastocytosis.
AYVAKIT Patents & Exclusivity
Patents (8 active)
Exclusivity
Pro Intelligence Preview
Deep insights for AYVAKIT
Revenue Insights
- • Q4-2025: $186M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2042
- • 195 active patents
Trial Analysis
- • 15 total trials
- • Stage: Growth
Competitive Landscape
- • 3 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment