FLT3 inhibitor · small molecule

gilteritinib

24 active trials across 5 of our biomarkers, targeting FLT3. 2 active approvals for a biomarker on marked.bio.

Also known as ASP2215, Xospata.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

Therapy	Biomarker	Tumour	United States	European Union
gilteritinib FDA, 2018 relapsed or refractory, FLT3 mutation FDA approval 28/11/2018; overall survival added to the label 29/05/2019. Companion test LeukoStrat CDx FLT3. Source EU (EMA), 2019 relapsed or refractory, FLT3 mutation EU marketing authorisation 24/10/2019. Source Xospata	FLT3 mutation (ITD/TKD)	AML	Approved 2018	Approved 2019
ADMIRAL vs salvage chemotherapy, 247 vs 124 patients CR+CRh 21% (first 138 patients)OS 9.3 months vs 5.6 months, HR 0.64 (0.49-0.83) Trial details and side effects Patients relapsed or refractory FLT3-mutated AML (NCT02421939) Most common side effects myalgia/arthralgia, raised liver enzymes, fatigue, fever, diarrhoea, dyspnoea, oedema, rash, pneumonia, nausea, stomatitis, cough Notes FDA approval summaries (2018 and 2019); side effects are those in 20% or more of patients. Source FDA approval summary

Biomarkers

Biomarker	Active trials	All trials	Furthest active phase
FLT3 mutation (ITD/TKD)	22	34	Phase 2/3
NPM1 mutation	3	3	Phase 1/2
ALK fusion	2	2	Phase 1
IDH1/2 mutation	2	3	Phase 2
EGFR exon 19 deletion / L858R	0	1	–

Trials

24 of 37 trials shown

FLT3-ITD Targeted Therapy in Fit AML Patients

Not yet recruiting Phase 2/3 European Organisation for Research and Treatment of Cancer - EORTC Started 2026Primary completion 2028-10 NCT07425808

gilteritinib FDA EUvenetoclaxazacitidineLocal standard of care (SOC)

FLT3-ITD mutation as per local IVDR-compliant testing.

FLT3 mutation (ITD/TKD)AML (Acute Myeloid Leukemia)
MRD-guided Maintenance Post-HCT: Gilteritini vs Sorafenib

Recruiting Phase 3 The First Affiliated Hospital of Soochow University Started 2026Primary completion 2028-05 NCT07463651

gilteritinib FDA EUsorafenib

…ogy, immunology, cytogenetics, and molecular biology (MICM) typing, and confirmed to have FLT3-ITD mutation;

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML), FLT3-ITD Mutation, Allogeneic Hematopoietic Cell Transplantation (HCT), Maintenance Therapy
VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML

Not yet recruiting Phase 3 Institute of Hematology & Blood Diseases Hospital Started 2026Primary completion 2029-02 NCT07407140

gilteritinib FDA EUazacitidinevenetoclaxcytarabinedaunorubicinidarubicinRe-induction TherapyConsolidation TherapyMaintenance Therapy

Documented FLT3-ITD or FLT3-TKD mutation by PCR or NGS

FLT3 mutation (ITD/TKD)AML, Adult
A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations

Recruiting Phase 3 Children's Oncology Group Started 2020Primary completion 2029-06 NCT04293562

Asparaginase Erwinia chrysanthemicytarabinedaunorubicinDexrazoxane Hydrochlorideetoposidegemtuzumab ozogamicingilteritinib FDA EUCPX-351methotrexatemitoxantroneTherapeutic Hydrocortisone

…to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia
A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy

Active, not recruiting Phase 3 Stichting Hemato-Oncologie voor Volwassenen Nederland Started 2019Primary completion 2025-10 NCT04027309

gilteritinib FDA EUmidostaurin FDA EU

… blasts-2 (EB2) defined according to WHO criteria (appendix A), with centrally documented FLT3 gene mutation (either TKD or ITD or both).

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts-2
Gilteritinib Plus VA Followed By Consolidation Chemotherapy in Newly Diagnosed FLT3-ITD+ AML

Recruiting Phase 2 First Affiliated Hospital of Zhejiang University Started 2026Primary completion 2028-12 NCT07259707

gilteritinib FDA EU

Newly diagnosed acute myeloid leukemia with FLT3-ITD mutation according to the European LeukemiaNet (ELN) 2022 diagnostic criteria (no VAF req…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, FLT3 Internal Tandem Duplication Positive, Intermediate Risk Acute Myeloid Leukemia, Fit Patients
Olutasidenib Combined With Co-targeted Therapy in Relapsed or Refractory IDH1-mutated Myeloid Malignancies Harboring Activated Signaling Pathway Mutations

Recruiting Phase 2 M.D. Anderson Cancer Center Started 2025Primary completion 2027-06 NCT07032727

olutasidenib FDAcladribinevenetoclaxgilteritinibRuxolitinibcytarabine

Participants must have a documented IDH1 mutation.

IDH1/2 mutationFLT3 mutation (ITD/TKD)Targeted Therapy, IDH1-Mutated Malignancies, Mutations
Gilteritinib in Combination With Venetoclax and Azacitidine for AML Patients With FLT3 Mutations Ineligible for Intensive Treatment

Recruiting Phase 2 Technische Universität Dresden Started 2025Primary completion 2029-04 NCT06696183

gilteritinib FDA EUvenetoclaxazacitidine

FLT3 mutation at initial diagnosis

FLT3 mutation (ITD/TKD)AML - Acute Myeloid Leukemia
Efficacy of Gilteritinib in Combination With FLAI as Induction Therapy of FLT3-positive Acute Myeloid Leukemia

Not yet recruiting Phase 2 Gruppo Italiano Malattie EMatologiche dell'Adulto Started 2025Primary completion 2028-08 NCT06667973

gilteritinib FDA EU

Efficacy of Gilteritinib in Combination With FLAI as Induction Therapy of FLT3-positive Acute Myeloid Leukemia

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, FLT3 Gene Mutation, Adult AML, Diagnosis
A Phase I/II Study of Gilteritinib and Momelotinib for Patients With Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia

Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2024Primary completion 2027-10 NCT06235801

gilteritinib FDA EUMomelotinib

a) Adults ≥18 years with relapsed/refractory FLT3-mutated AML.

FLT3 mutation (ITD/TKD)Myeloid Leukemia
Study Investigating the Efficacy and Safety of the Addition of Oral-azacitidine to Salvage Treatment by Gilteritinib in Subjects ≥18 Years of Age With Relapsed/Refractory FLT3-mutated Acute Myeloid Leukemia

Recruiting Phase 2 French Innovative Leukemia Organisation Started 2024Primary completion 2026-10 NCT06022003

azacitidinegilteritinib FDA EU

Presence of FLT3-mutation(s) at inclusion: in case of FLT3-ITD, the ITD/wt ratio must be \> 0.05

FLT3 mutation (ITD/TKD)AML, Adult, Refractory AML, Relapsed Adult AML, FLT3-TKD Mutation
Venetoclax and HMA Treatment of Older and Unfit Adults With FLT3 Mutated Acute Myeloid Leukemia (AML) (A MyeloMATCH Treatment Trial)

Recruiting Phase 2 National Cancer Institute (NCI) Started 2024Primary completion 2029-05 NCT06317649

azacitidinegilteritinib FDA EUvenetoclax

Patient must have the FLT3-ITD, D835, or I836del mutation based on MYELOMATCH Protocol

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia
A Phase 2 Study of Gemtuzumab Ozogamicin (GO)-Gilteritinib Combination in Adults With FLT3-ITD and/or FLT3-TKD Relapse/Refractory (R/R) AML

Active, not recruiting Phase 2 Centre Antoine Lacassagne Started 2023Primary completion 2028-07 NCT05199051

cytarabinegilteritinib FDA EUgemtuzumab ozogamicin

Presence of a FLT3-ITD mutation (allelic ratio ≥0.05 at last evaluation)\* or a FLT3 TKD mutation

FLT3 mutation (ITD/TKD)AML, Leukaemia (Acute Myeloid)
A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia

Active, not recruiting Phase 1/2 Astellas Started 2023Primary completion 2028-07 NCT05520567

gilteritinib FDA EUvenetoclaxazacitidine

Participant is positive for FMS-like tyrosine kinase 3 (FLT3) mutation (internal tandem duplication \[ITD\] and/or tyrosine kinase domain \[TKD\] \[D835/I836\] …

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML), FLT3-mutated Acute Myeloid Leukemia
A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)

Recruiting Phase 1/2 Sumitomo Pharma America Started 2022Primary completion 2027-06 NCT04988555

enzomenibazolesvenetoclaxgilteritinib FDA EUazacitidineIntensive chemotherapy with 7 + 3

Have MLLr or NPM1m AND any of the following FLT3 mutations: FLT3-ITD, FLT3-TKD/D835 or FLT3-TKD/I836.

FLT3 mutation (ITD/TKD)NPM1 mutationLeukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute, Multiple Myeloma, Myelodysplastic Syndromes
ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2021Primary completion 2028-01 NCT05010122

decitabinegilteritinib FDA EUvenetoclax

Phase I cohort: Adults \>= 18 years with relapsed/refractory FLT3-mutated AML or myelodysplastic syndrome (MDS) that is intermediate-2 or high-risk by the Internat…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2019Primary completion 2028-09 NCT04140487

azacitidinegilteritinib FDA EUvenetoclax

Phase I cohort: Adults ≥ 18 years with relapsed/refractory FLT3-mutated AML or CMML or other MDS/MPN that is intermediate-2 or high-risk by the International Pro…

FLT3 mutation (ITD/TKD)Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelomonocytic Leukemia, Recurrent Myelodysplastic/Myeloproliferative Neoplasm, Refractory Acute Myeloid Leukemia
A Study of Gilteritinib in Adults With Advanced ALK-positive Non-small Cell Lung Cancer (NSCLC)

Recruiting Phase 1 Astellas Started 2025Primary completion 2029-12 NCT07140016

gilteritinib

…tastatic non-small cell lung cancer (NSCLC) with a documented anaplastic lymphoma kinase (ALK) rearrangement and is not amenable to curative intent treatment.

ALK fusionNon-small Cell Lung Cancer (NSCLC), Anaplastic Lymphoma Kinase (ALK) Positive
SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation

Recruiting Phase 1 Uma Borate Started 2024Primary completion 2027-01 NCT06222580

gilteritinib FDA EUrevumenib

…afety and Efficacy of Dual Menin and FLT3 Inhibition in Patients With Relapsed/Refractory FLT3- Mutated Acute Myeloid Leukemia Containing a Concurrent MLL-Rearrangement or NPM1 Mutation: A Phas…

FLT3 mutation (ITD/TKD)NPM1 mutationAcute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With KMT2A Rearrangement, Acute Myeloid Leukemia With NPM1 Mutation, Recurrent Acute Myeloid Leukemia
Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Recruiting Phase 1 Kura Oncology Started 2024Primary completion 2026-12 NCT06001788

ziftomenibfludarabineidarubicincytarabinegilteritinib FDA EUGranulocyte colony-stimulating factor

Has a documented FLT3 mutation (cA-3 only).

FLT3 mutation (ITD/TKD)NPM1 mutationAML, AML With Mutated NPM1, Hematologic Malignancy, KMT2Ar
Gilteritinib for the Treatment of ALK NSCLC

Recruiting Phase 1 University of Michigan Rogel Cancer Center Started 2024Primary completion 2027-05 NCT06225427

gilteritinib

…nt Committee on Cancer \[AJCC\] 8th edition) non-small cell lung cancer with an oncogenic ALK fusion

ALK fusionLung Non-Small Cell Carcinoma, Stage IV Lung Cancer AJCC v8
A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML)

Active, not recruiting Phase 1 Memorial Sloan Kettering Cancer Center Started 2023Primary completion 2027-04 NCT05756777

gilteritinibivosidenib FDA EUenasidenib FDA

Patient has relapsed or refractory AML with dually mutant IDH2/FLT3, IDH1/FLT3 (ITD or TKD) , or other FLT3 mutation sensitive to gilteritinib.

IDH1/2 mutationFLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML)
Study of Iadademstat and Gilteritinib in Patients With R/R AML With FMS-like Tyrosine Kinase Mutation (FLT3 Mut+)

Active, not recruiting Phase 1 Oryzon Genomics Started 2022Primary completion 2026-01 NCT05546580

Iadademstatgilteritinib FDA EU

Patient must be positive for the following FLT3 mutations in bone marrow or PB: FLT3 internal tandem duplication (ITD), FLT3 tyrosine kinase domain…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory
Vyxeos Plus Gilteritinib in Relapsed or Refractory, FLT3-Mutated AML

Recruiting Phase 1 H. Lee Moffitt Cancer Center and Research Institute Started 2022Primary completion 2027-02 NCT05024552

gilteritinib FDA EUCPX-351daunorubicincytarabine

FLT3-ITD or FLT3-TKD mutated AML (non-M3) in 1st or greater relapse or refractory to at least one …

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia With FLT3/ITD Mutation
A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase 3 (FLT3) Mutation

Completed Phase 3 Astellas Started 2017 NCT03182244

gilteritinib FDA EUcytarabinemitoxantroneetoposidepegfilgrastimfludarabine

Participant is positive for FLT3 mutation in bone marrow or whole blood as determined by the central lab.

FLT3 mutation (ITD/TKD)AML With FLT3 Mutation
A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

Completed Phase 3 Astellas Started 2016 NCT02752035

gilteritinib FDA EUazacitidine

Subject is positive for FLT3 mutation (internal tandem duplication \[ITD\] or tyrosine kinase domain \[TKD\] \[D835/I836\] muta…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
A Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-like Tyrosine Kinase (FLT3) Mutation

Completed Phase 3 Astellas Started 2015 NCT02421939

gilteritinib FDA EUcytarabineazacitidinemitoxantroneetoposidepegfilgrastimfludarabineidarubicin

Participant is positive for FLT3 mutation in bone marrow or whole blood as determined by the central lab.

FLT3 mutation (ITD/TKD)Leukemia, Acute Myeloid (AML)
A Study of Anti-Cancer Therapies Targeting the MAPK Pathway in Patients With Hematologic Malignancies

Withdrawn Phase 1/2 Erasca Started 2022 NCT05279859

ERAS-007ERAS-601gilteritinib FDA EU

Positive for FLT3 mutation in bone marrow or whole blood.

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia
A Study to Evaluate Lanraplenib (LANRA) in Combination With Gilteritinib in Participants With FLT3-mutated Relapsed or Refractory Acute Myeloid Leukemia (AML)

Terminated Phase 1/2 Kronos Bio Started 2022 NCT05028751

Lanraplenibgilteritinib FDA EU

FMS-like tyrosine kinase 3 (FLT3)-mutated disease documented in a local reference laboratory at the time of consideration for enrol…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia

Completed Phase 2 PrECOG Started 2019 NCT03836209

gilteritinib FDA EUmidostaurin FDA EUdaunorubicincytarabine

Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia
A Study of ASP2215 (Gilteritinib) Combined With Atezolizumab in Patients With Relapsed or Treatment Refractory FMS-like Tyrosine Kinase (FLT3) Mutated Acute Myeloid Leukemia (AML)

Completed Phase 1/2 Astellas Started 2019 NCT03730012

gilteritinib FDA EUatezolizumab

Subject is positive for FLT3 mutation in bone marrow or blood after completion of the subject's last interventional treatment.

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation
A Study of ASP2215 in Combination With Erlotinib in Subjects With Epidermal Growth Factor Receptor (EGFR) Activating Mutation-Positive (EGFRm+) Advanced Non-Small-Cell Lung Cancer (NSCLC) Who Have Acquired Resistance to an EGFR Tyrosine Kinase Inhibitor (TKI)

Terminated Phase 1/2 Astellas Started 2015 NCT02495233

gilteritiniberlotinib FDA EU

Participant had a documented exon 19 deletion or exon 21 L858R EGFR activating mutation.

EGFR exon 19 deletion / L858RNon-Small-Cell Lung Cancer
A Study of ASP2215 in Combination With Induction and Consolidation Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia.

Completed Phase 1/2 Astellas Started 2015 NCT02310321

gilteritinib FDA EUidarubicincytarabine

Subject is positive for FLT3-ITD and/or TKD mutation in bone marrow or whole blood as determined by the central lab.

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia, FLT3-mutated Acute Myeloid Leukemia
GM-CLAG in Relapsed/Refractory FLT3-mutated AML

Withdrawn Phase 1 Ayman H Qasrawi Started 2023 NCT05330377

gilteritinib FDA EUcladribinecytarabinepegfilgrastimmitoxantrone

Evidence of FLT3-ITD or FLT3-TKD mutations as detected by polymerase chain reaction and/or next-generation seq…

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia
Glasdegib-Based Treatment Combinations for the Treatment of Patients With Relapsed Acute Myeloid Leukemia Who Have Undergone Hematopoietic Cell Transplantation

Withdrawn Phase 1 City of Hope Medical Center Started 2022 NCT04655391

Bosutinib Monohydratedecitabineenasidenib FDAgilteritinibGlasdegib Maleateivosidenib FDA EUvenetoclax

CHAPTER 4: Patients with a confirmed susceptible IDH1 mutation (R132)

IDH1/2 mutationFLT3 mutation (ITD/TKD)Recurrent Acute Myeloid Leukemia
A Safety and Efficacy Study of CC-90009 Combinations in Subjects With Acute Myeloid Leukemia

Terminated Phase 1 Bristol Myers Squibb Started 2020 NCT04336982

CC-90009venetoclaxazacitidinegilteritinib FDA EU

Fms-like tyrosine kinase 3 (FLT3) mutation positive.

FLT3 mutation (ITD/TKD)Leukemia, Myeloid, Acute
A Study to Assess Safety and Efficacy of Venetoclax in Combination With Gilteritinib in Participants With Relapsed/Refractory Acute Myeloid Leukemia

Completed Phase 1 AbbVie Started 2018 NCT03625505

venetoclaxgilteritinib FDA EU

…icipants enrolling into the Expansion Cohort only: a documented FMS-like Tyrosine Kinase (FLT3) mutation in bone marrow or peripheral blood, as described in the protocol.

FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML)

Approvals and pivotal results

Biomarkers

Sponsors

Trials