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Drug

Gilteritinib

Approved · EMA
ClassTyrosine-protein kinase receptor FLT3 inhibitorEmerging researchLate-stage development

Also known as Xospata.

RxNorm2105806ChEMBLCHEMBL3301622ATCL01EX13UNII66D92MGC8M
1
Research papers
32
Active clinical trials
2
Protein targets
1
Regulatory events
Latest activity
beta

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

Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia

Clinical trial2026-08-01Primary completion · ClinicalTrials.gov

Phase 3 Open-label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

Clinical trial2026-07-16Results posted · ClinicalTrials.gov

A Randomized Phase II Study of Venetoclax and HMA-Based Therapies for the Treatment of Older and Unfit Adults With Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia (AML): A MyeloMATCH Treatment Trial

Clinical trial2026-07-10Results expected Q2 2029 · ClinicalTrials.gov

Master Screening and Reassessment Protocol (MSRP) for the NCI MyeloMATCH Clinical Trials

Clinical trial2026-07-10Results expected Q2 2029 · ClinicalTrials.gov

A Phase 1b Study of Gilteritinib in Participants With Locally Advanced or Metastatic NSCLC With ALK Rearrangement After Prior Treatment With an ALK Inhibitor

Clinical trial2026-06-26Results expected Q4 2029 · ClinicalTrials.gov

Phase I Study of Gilteritinib for ALK Positive Non-Small Cell Lung Cancer

Clinical trial2026-06-26Results expected Q2 2027 · ClinicalTrials.gov

Randomized Trial of Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia (AML)

Clinical trial2026-06-23Results posted · ClinicalTrials.gov

A Phase 1b Multi-center Study of the FLT3 Inhibitor Gilteritinib in Combination With the IDH1 Inhibitor Ivosidenib or the IDH2 Inhibitor Enasidenib for Patients With Relapsed or Refractory Acute Myeloid Leukemia Who Have Co-occurring FLT3/IDH1 or FLT3/IDH2 Mutations

Clinical trial2026-06-22Results expected Q2 2027 · ClinicalTrials.gov

Non Interventional Study Aiming to Assess the Immunological Impact of a Post Cell Therapy Treatment With FLT3 Inhibitors on the Phenotype, the Metabolism, the Function of T-cells and Monocytes and Macrophages

Clinical trial2026-06-22Results expected Q3 2028 · ClinicalTrials.gov

A Phase I/II Study of ASTX727, Venetoclax, and Gilteritinib for Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome With an Activating FLT3 Mutation

Clinical trial2026-06-11Results expected Q1 2028 · ClinicalTrials.gov

Approval: Xospata (EMA)

Regulatory2019-10-24EMA

Gilteritinib or Chemotherapy for Relapsed or Refractory <i>FLT3</i>-Mutated AML.

Research2019-10-01The New England journal of medicine

Profile

Identifiers & mechanism

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

Canonical name
Gilteritinib
Aliases & brands
Xospata
RxNorm CUI
2105806
ChEMBL ID
CHEMBL3301622
ATC codes
L01EX13
UNII
66D92MGC8M
Primary mechanism
Tyrosine-protein kinase receptor FLT3 inhibitor
Regulatory jurisdictions
ema

Pharmacology & targets

2 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Receptor-type tyrosine-protein kinase FLT3
FLT3 · P36888
INHIBITORTyrosine-protein kinase receptor FLT3 inhibitor
Tyrosine-protein kinase receptor UFO
AXL · P30530
INHIBITORTyrosine-protein kinase receptor UFO inhibitor

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
2019-10-24
Latest approval
2019-10-24
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2019-10-24Approval
Approval: Xospata (EMA)

Indication: Xospata is indicated as monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation.

Evidence ↗

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

Clinical trials

53 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
53
registered trials across all phases
LATEST COMPLETION 2025
32
Active studies
26
Recruiting
10
Late-stage (III+)
14
Completed
7
Discontinued
PHASE DISTRIBUTIONn = 53
Phase 116Phase 1 / 215Phase 212Phase 2 / 31Phase 37Phase N / A2

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3, Multicenter, Open-label, Randomized, 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 (AML) or Myelodysplastic Syndromes With Excess Blasts-2 (MDS-EB2) With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML / AMLSG 28-18)

Active, not recruitingPH IIIStichting Hemato-Oncologie voor Volwassenen Nederlandn=7772033

Gilteritinib in Combination With Azacitidine and Venetoclax Compared to Induction Chemotherapy "7+3" in Combination With a FLT3-inhibitor in Fit, Newly Diagnosed, FLT3-ITD Mutated Adult AML Patients: a Randomized Trial of the EORTC Leukemia Group and GIMEMA.

Not yet recruitingPH II/IIIEuropean Organisation for Research and Treatment of Cancer - EORTCn=2302032

Post-transplant Optimization Based on Ultra-high Sensitivity MRD Detection: A Prospective, Randomized Controlled Study Comparing Gilteritinib Versus Sorafenib as Post-transplant Maintenance Therapy in FLT3-ITD Mutation-positive Acute Myeloid Leukemia Patients

RecruitingPH IIIThe First Affiliated Hospital of Soochow Universityn=5942030

Phase 3 Open-label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

Active, not recruitingPH IIIAstellas Pharma Incn=2762027

A Phase 3 Open-Label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

CompletedPH IIIAstellas Pharma Global Development, Inc.n=3712025

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 3 Open-Label, Multicenter, Randomized Study of ASP2215 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

CompletedPH IIIAstellas Pharma Global Development, Inc.n=3712025

A Phase 3 Multicenter, Open-label, Randomized Study of ASP2215 (Gilteritinib), Combination of ASP2215 Plus Azacitidine and Azacitidine Alone in the Treatment of Newly Diagnosed Acute Myeloid Leukemia With FLT3 Mutation in Patients Not Eligible for Intensive Induction Chemotherapy

CompletedPH IIIAstellas Pharma Global Development, Inc.n=1832024

A Phase 1/2 Study of ASP2215 in Combination With Induction and Consolidation Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia

CompletedPH I/IIAstellas Pharma Incn=972024

A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of the FLT3 Inhibitor Gilteritinib (ASP2215) Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FLT3/ITD AML in First Complete Remission

CompletedPH IIAstellas Pharma Global Development, Inc.n=982024

A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Trial of the FLT3 Inhibitor Gilteritinib Administered as Maintenance Therapy Following Allogeneic Transplant for Patients With FLT3/ITD AML

CompletedPH IIIAstellas Pharma Global Development, Inc.n=3562023

A Phase 1 Open-label Study to Investigate the Effect of Severe Renal Impairment on the Pharmacokinetics, Safety and Tolerability of Gilteritinib Compared to Healthy Participants With Normal Renal Function

CompletedPH IAstellas Pharma Global Development, Inc.n=172022

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

Gilteritinib or Chemotherapy for Relapsed or Refractory <i>FLT3</i>-Mutated AML.

The New England journal of medicine · 2019 · 966 cites
Recent papers

Gilteritinib or Chemotherapy for Relapsed or Refractory <i>FLT3</i>-Mutated AML.

The New England journal of medicine · 2019 · 966 cites
Major research themes2
Mutation1Salvage Therapy1
Journals, researchers & institutions
Top journals
  • The New England journal of medicine1
Leading researchers
  • Baer MR1
  • Bahceci E1
  • Berman E1
  • Chou WC1
  • Ciceri F1
  • Cortes JE1
  • Di Stasi A1
  • Erba HP1
Leading institutions
free-text, unnormalised
  • From the Abramson Cancer Center1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.