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Ayvakit

(Avapritinib)

ApprovedSmall molecule

BBlueprint Medicines

Avapritinib is used to treat a rare type of stomach cancer called gastrointestinal stromal tumor (GIST) that has a specific genetic mutation. It works by blocking proteins that help cancer cells grow and spread.[2] Ayvakit is currently approved and in use across 10 indications[4].[1]

Ayvakit is one of 111 small molecule programs targeting Central Nervous System Cancer. It is currently in Approved.

10

Indications

22

Total Trials

11

Active Trials

110

Competitors

7

FDA Applications

NDA/BLA/ANDA regulatory filings

8

Patents

Pipeline

Status by Indication[4][1]

Indication

Central Nervous System CancerApproved

Systemic MastocytosisCompleted

Mast-Cell LeukemiaApproved

Malignant Solid TumorRecruiting

Relapsed or RefractoryRecruiting

AINNot yet recruiting

Induction TherapyRecruiting

AML, AdultRecruiting

Locally Advanced or Metastatic Malignant Solid TumorsRecruiting

Relapsed AMLRecruiting

Central Nervous System CancerApproved

Approved

Systemic MastocytosisPhase 2

Completed

Mast-Cell LeukemiaApproved

Approved

Malignant Solid TumorPhase 2

Recruiting

Relapsed or RefractoryPhase 2

Recruiting

AINPhase 2

Not yet recruiting

Induction TherapyPhase 2

Recruiting

AML, AdultPreclinical

Recruiting

Locally Advanced or Metastatic Malignant Solid TumorsPhase 2

Recruiting

Relapsed AMLPhase 2

Recruiting

Competition

Competitive Landscape

56 competitors in Relapsed or Refractory

#DrugCompanyPhaseTrials

Ayvakit

BBlueprint Medicines

Temodar

CCidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Decadron

AAspen Global Incorporated

Cytosar

Fludara

Dacogen

Sun Pharmaceutical Industries, Inc.

Oncovin

Fludara

Cytoxan

BBaxter Healthcare Corporation

Libtayo

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Recruiting

Locally Advanced or Metastatic Malignant Solid Tumors

NCT04771520

Q1 2027

Phase 1Recruiting

Study completion

NCT06327685

Phase 2Recruiting

Relapsed or Refractory

NCT06316960

Q2 2027

Phase 2Active, not recruiting

Indolent Systemic Mastocytosis

NCT03731260

Q4 2027

Phase 2Recruiting

Malignant Solid Tumor

NCT04116541

Phase 2Recruiting

Relapsed AML

NCT06783790

Clinical

Trials by Indication[1]

Systemic Mastocytosis2 trials

(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

Completed·BBlueprint Medicines·Phase 1·NCT02561988·86 enrolled

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

Completed·BBlueprint Medicines·Phase 2·NCT03580655·107 enrolled

Gastrointestinal Stromal Tumor2 trials

(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

Completed·BBlueprint Medicines·Phase 1·NCT02508532·250 enrolled

Safety and Efficacy of Avapritinib in Chinese Patients With Gastrointestinal Stromal Tumor (GIST) in the Real World

Completed·CCStone Pharmaceuticals··NCT05381753·61 enrolled

Locally Advanced2 trials

(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

Completed·BBlueprint Medicines·Phase 3·NCT03465722·476 enrolled

Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic GIST

Approved for marketing·BBlueprint Medicines··NCT03862885

Indolent Systemic Mastocytosis2 trials

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·BBlueprint Medicines··NCT07255638·80 enrolled

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

Active, not recruiting·BBlueprint Medicines·Phase 2·NCT03731260·251 enrolled

Induction Therapy1 trial

Clinical Study of Induction Therapy Options Based on Molecular Subtyping and MRD in Children and Adolescents With AML

Recruiting·—·Phase 2·NCT06221683·500 enrolled

12 more indications

Relapsed or Refractory1 trial

Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

Recruiting·—·Phase 2·NCT06316960·50 enrolled

AML1 trial

Avapritinib in CBF-AML With KIT Mutations

Unknown status·TThe First Affiliated Hospital of Soochow University·Phase 2·NCT05821738·50 enrolled

Relapsed AML1 trial

Avapritinib Combined With Azacitidine and Venetoclax in the Treatment of Relapsed AML After Allo-HSCT

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT06783790·20 enrolled

Interaction1 trial

A Drug-drug Interaction Study of Avapritinib and Midazolam

Completed·BBlueprint Medicines·Phase 1·NCT04908176·10 enrolled

ESS1 trial

Expanded Access Program for Avapritinib

No longer available·BBlueprint Medicines··NCT04714086

Patients With Solid Tumors1 trial

A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

Completed·BBlueprint Medicines·Phase 1/2·NCT04773782·29 enrolled

Locally Advanced or Metastatic Malignant Solid Tumors1 trial

Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT04771520·50 enrolled

Clinical Trial1 trial

Study for Patients Previously Treated in Avapritinib Clinical Trials

Completed·BBlueprint Medicines·Phase 4·NCT04825574·2 enrolled

AIN1 trial

Avapritinib Maintenance for AML With KIT Mutations

Not yet recruiting·RRuijin Hospital·Phase 2·NCT06765915·47 enrolled

AML, Adult1 trial

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

Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Preclinical·NCT07131059·537 enrolled

Malignant Solid Tumor1 trial

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.

Recruiting·CCentre Leon Berard·Phase 2·NCT04116541·455 enrolled

Unspecified2 trials

Avapritinib With Decitabine in Patients With SM-AHN

Recruiting·HH. Lee Moffitt Cancer Center and Research Institute·Phase 1·NCT06327685·34 enrolled

Avapritinib Rollover Study

Recruiting·BBlueprint Medicines·Phase 4·NCT06748001·60 enrolled

Regulatory

FDA Approval Timeline[5]

Jan 9, 2020

AYVAKIT

NDA NDA212608

Jun 16, 2021

AYVAKIT

NDA NDA212608

Jun 16, 2021

AYVAKIT

NDA NDA212608

Jun 16, 2021

AYVAKIT

NDA NDA212608

Mar 9, 2023

AYVAKIT

NDA NDA212608

May 22, 2023

AYVAKIT

NDA NDA212608

Nov 8, 2024

AYVAKIT

NDA NDA212608

Sources

References

[1]
ClinicalTrials.gov — 22 trial records for Avapritinib. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Ayvakit drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Avapritinib compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Avapritinib. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA212608). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Ayvakit. U.S. Food & Drug Administration.
[7]
PubMed — 1 publication indexed for Avapritinib. U.S. National Library of Medicine.
[8]
SEC EDGAR — Blueprint Medicines company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: KIT D816V Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: BBlueprint Medicines

Patent Landscape[6]

Patent Cliff

Oct 15, 2034

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9944651Oct 15, 2034

9200002Oct 15, 2034

11827642Oct 15, 2034

9994575Oct 15, 2034

11964980Apr 10, 2040

3 more patents

11999744Apr 10, 2040

12252494Apr 10, 2040

12060354Mar 8, 2042

Key Publications[7]

Avapritinib in Indolent Systemic Mastocytosis: PIONEER

N Engl J MedMar 16, 2023DOI: 10.1056/NEJMoa2212562

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