Back to discover
Drug

Pexidartinib

In clinical development
ClassMacrophage colony stimulating factor receptor inhibitorEmerging researchLate-stage developmentSteady momentum

Also known as Turalio.

RxNorm2183102ChEMBLCHEMBL3813873ATCL01EX15UNII6783M2LV5X
4
Research papers
1
Active clinical trials
3
Protein targets
Latest activity
beta

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

Multicenter, Single Arm Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor

Clinical trial2026-06-26Results posted · ClinicalTrials.gov

A Phase 2, Multicenter, Two-Part, Open-Label Study of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor in Japan

Clinical trial2026-06-24Results posted · ClinicalTrials.gov

Brain-wide microglia replacement using a nonconditioning strategy ameliorates pathology in mouse models of neurological disorders.

Research2025-04-30Science translational medicine

A Phase 1b and 2a Study to Assess Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of CGT9486 as a Single Agent and in Combination With PLX3397 or Sunitinib (Sutent®) in Patients With Advanced Solid Tumors and Patients With Locally Advanced, Unresectable, or Metastatic Gastrointestinal Stromal Tumor (GIST) Who Have Been Previously Treated With Imatinib Mesylate/KIT-Directed Tyrosine Kinase Inhibitor (TKI) Therapy

Clinical trial2025-02-14Results posted · ClinicalTrials.gov

A Phase 4, Multicenter Study to Evaluate Discontinuation and Re-Treatment in Subjects With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

Clinical trial2024-12-30Results posted · ClinicalTrials.gov

Distinguishing the effects of systemic CSF1R inhibition by PLX3397 on microglia and peripheral immune cells.

Research2023-10-21Journal of neuroinflammation

A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath

Clinical trial2022-05-11Results posted · ClinicalTrials.gov

A Phase 1 Study of Single Agent Pexidartinib in Asian Subjects With Advanced Solid Tumors

Clinical trial2022-04-25Results posted · ClinicalTrials.gov

An Open-label, Single-dose Study to Assess the Pharmacokinetics of Pexidartinib in Subjects With Moderate Hepatic Impairment Compared to Healthy Subjects

Clinical trial2021-06-22Results posted · ClinicalTrials.gov

An Open-label, Single Sequence, Crossover Drug-drug Interaction Study Assessing the Effect of Pexidartinib on the Pharmacokinetics of CYP3A4 and CYP2C9 Substrates in Patients

Clinical trial2021-05-14Results posted · ClinicalTrials.gov

Microglial activation contributes to cognitive impairments in rotenone-induced mouse Parkinson's disease model.

Research2021-01-05Journal of neuroinflammation

Microglia drive APOE-dependent neurodegeneration in a tauopathy mouse model.

Research2019-10-10The Journal of experimental medicine

Profile

Identifiers & mechanism

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

Canonical name
Pexidartinib
Aliases & brands
Turalio
RxNorm CUI
2183102
ChEMBL ID
CHEMBL3813873
ATC codes
L01EX15
UNII
6783M2LV5X
Primary mechanism
Macrophage colony stimulating factor receptor inhibitor

Pharmacology & targets

3 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Macrophage colony-stimulating factor 1 receptor
CSF1R · P07333
INHIBITORMacrophage colony stimulating factor receptor inhibitor
Receptor-type tyrosine-protein kinase FLT3
FLT3 · P36888
INHIBITORTyrosine-protein kinase receptor FLT3 inhibitor
Mast/stem cell growth factor receptor Kit
KIT · P10721
INHIBITORStem cell growth factor receptor inhibitor

Clinical trials

11 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
11
registered trials across all phases
LATEST COMPLETION 2026
1
Active studies
3
Late-stage (III+)
10
Completed
PHASE DISTRIBUTIONn = 11
Early Phase 11Phase 15Phase 1 / 21Phase 21Phase 32Phase 41

Recent completions

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

A Phase 2, Multicenter, Two-Part, Open-Label Study of Pexidartinib in Adult Subjects With Tenosynovial Giant Cell Tumor in Japan

CompletedPH IIDaiichi Sankyo Co., Ltd.n=92026

A Phase 4, Multicenter Study to Evaluate Discontinuation and Re-Treatment in Subjects With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

CompletedPH IVDaiichi Sankyon=322023

A Phase 1 Study of Single Agent Pexidartinib in Asian Subjects With Advanced Solid Tumors

CompletedPH IDaiichi Sankyo Co., Ltd.n=122021

A Double-blind, Randomized, Placebo-controlled Phase 3 Study of Orally Administered PLX3397 in Subjects With Pigmented Villonodular Synovitis or Giant Cell Tumor of the Tendon Sheath

CompletedPH IIIDaiichi Sankyon=1202021

A Phase I Study of Binimetinib in Combination With Pexidartinib in Patients With Advanced Gastrointestinal Stromal Tumor (GIST)

CompletedPH IMemorial Sloan Kettering Cancer Centern=32021

An Open-label, Single Sequence, Crossover Drug-drug Interaction Study Assessing the Effect of Pexidartinib on the Pharmacokinetics of CYP3A4 and CYP2C9 Substrates in Patients

CompletedPH IDaiichi Sankyon=322021

Research activity

4 papers

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

Publications over time
20192025
Major research themes4
Microglia2Brain1Lipopolysaccharides1Nervous System Diseases1
Journals, researchers & institutions
Top journals
  • Journal of neuroinflammation2
  • Science translational medicine1
  • The Journal of experimental medicine1
Leading researchers
  • Li S2
  • Bu G1
  • Cao-Dao VD1
  • Chen D1
  • Chen S1
  • Chen X1
  • Chen XF1
  • Coburn MA1
Leading institutions
free-text, unnormalised
  • Center for Brain Immunology and Glia1
  • Division of Life Science and State Key Laboratory of Molecular Neuroscience1
  • Duke University School of Medicine1
  • First Affiliated Hospital of Xiamen University1
  • Hope Center for Neurological Disorders1
  • National-Local Joint Engineering Research Center for Drug-Research and Development (R&D) of Neurodegenerative Diseases1

Related diseases

1 conditions

Regulator-approved indications and conditions co-mentioned with this drug across the research literature. Number shows shared papers.

Active research areas
Disease Models, Animal2

Related drugs

3 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Minocycline

Studied across 3 of the same disease areas as Pexidartinib in the shared literature.

3 shared diseases1 shared paper
Rotenone

Studied across 3 of the same disease areas as Pexidartinib in the shared literature.

3 shared diseases1 shared paper
Insecticides

Studied across 2 of the same disease areas as Pexidartinib in the shared literature.

2 shared diseases1 shared paper
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