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avapritinib

Generic: AVAPRITINIB·Brand: AYVAKIT, Ayvakit

avapritinib: developed by 1 company · 6 clinical trials tracked · 1 FDA decision.

avapritinib evidence translation

What we can confirm

1 FDA record · 6 tracked trials

What it means

Source-backed records connect avapritinib to BPMC; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could avapritinib → BPMC affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence3 indications · 0 study sponsors

Related indications

Solid Tumor, Unspecified, Child; Indolent Systemic Mastocytosis; Mastocytosis, SystemicSystemic mastocytosis (SM) including advanced SM (ASM, SM-AHN, MCL) and indolent SM; PDGFRA exon 18 mutant GISTIndolent Systemic Mastocytosis

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (2)
CompanyPhase
Blueprint Medicines Corp (BPMC)
Solid Tumor, Unspecified, Child; Indolent Systemic Mastocyto…
Phase 4
Blueprint Medicines Corp (BPMC)
Systemic mastocytosis (SM) including advanced SM (ASM, SM-AH…
Approved
Clinical Trials (6)

A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings

Recruiting

Primary endpoint: Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6

Observational80 targetStarted 2025-12Completion 2028-128 sites · GermanyBlueprint Medicines Corporation

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

NCT06748001·Phase 4·BPMC
Recruiting

Primary endpoint: Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation

Interventionalopen60 targetStarted 2024-11Completion 2027-127 sites · Belgium, Canada, Italy, Netherlands…Blueprint Medicines Corporation

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

NCT06327685·Phase 1
Recruiting

Primary endpoint: Recommended phase 2 dose (RP2D)

Interventionalopen34 targetStarted 2024-03Completion 2027-037 sites · United StatesH. Lee Moffitt Cancer Center and Research Institute

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

NCT04773782·Phase 1/Phase 2·BPMC
Completed

Primary endpoint: Determination of recommended Part 2 dose (Part 1)

Interventionalopen29 enrolledStarted 2022-02Completion 2025-1126 sites · Australia, Austria, Canada, France…Blueprint Medicines Corporation

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

NCT03731260·Phase 2·BPMC

Primary endpoint: Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM

InterventionalRandomizedquadruple251 targetStarted 2019-04Completion 2027-0649 sites · Belgium, Canada, Denmark, France…Blueprint Medicines Corporation

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

NCT03580655·Phase 2·BPMC
Completed

Primary endpoint: Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria

Interventionalopen107 enrolledStarted 2018-11Completion 2024-1232 sites · Austria, Canada, Denmark, France…Blueprint Medicines Corporation
FDA Decisions1 records
Approval1 INDICATIONS AND USAGE AYVAKIT is a kinase inhibitor indicated for: Gastrointestinal Stromal Tumor (GIST) the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. ( 1.1 , 2.2 ) Advanced Systemic Mastocytosis (AdvSM) the treatment of adult patients with AdvSM. AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hemato(BPMC)FDA ↗
NDA212608Type 1 - New Molecular EntityOrphan DrugPriorityOralTablet

Frequently asked questions

Who is developing avapritinib?

avapritinib is being developed by Blueprint Medicines Corp.

How many clinical trials involve avapritinib?

BioSniper tracks 6 clinical trials involving avapritinib.

What is avapritinib's latest FDA decision?

The most recent tracked FDA decision for avapritinib is Approval for 1 INDICATIONS AND USAGE AYVAKIT is a kinase inhibitor indicated for: Gastrointestinal Stromal Tumor (GIST) the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. ( 1.1 , 2.2 ) Advanced Systemic Mastocytosis (AdvSM) the treatment of adult patients with AdvSM. AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hemato, dated January 9, 2020.