Drug · Small Molecule

Pexidartinib

CI-DRUG-00000472Explore in graph →NCIt C88303 CHEMBL3813873 CIViC Approved

Regulatory

Approvals (3)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory3 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2022-10-14 (see label)	approved	Oct 14, 2022	openfda
US FDA	—	Efficacy supplement 2022-10-06 (see label)	approved	Oct 6, 2022	openfda
US FDA	—	TURALIO is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. TURALIO is a kinase inhibitor indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. ( 1 )	approved	Aug 2, 2019	openfda

Data updated 2 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	3	2	2	25	3	US	Aug 2, 2019	Oct 1, 2009
Leukemia	Phase2	Phase 2	1	1	0	2	0	—	—	Nov 21, 2011
Malignant Breast Neoplasm	Phase2	Phase 2	1	1	0	2	0	—	—	Mar 1, 2010
Hodgkin Lymphoma	Phase2	Phase 2	0	0	0	1	0	—	—

Curated evidence

Clinical evidence (14)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–14 of 14 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
FLT3 D839A1
Pexidartinib	Acute Myeloid Leukemia

Clinical trials

Trials with this intervention (25)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–25 of 25 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT02390752	Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...	recruiting	Phase 1	54	National Cancer Institute (NCI)NIH	1	Aug 24, 2026	clinicaltrials
NCT04488822	A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT

Molecule

Record

Kind: Small Molecule
Mechanism: Macrophage colony stimulating factor receptor inhibitor; Tyrosine-protein kinase receptor FLT3 inhibitor; Stem cell growth factor receptor inhibitor
Targets: 3 genes
UNII: YS6WAI3XN7
Aliases: 3-Pyridinemethanamine, N-(5-((5-chloro-1H-pyrrolo(2,3-b)pyridin-3-yl)methyl)-2-pyridinyl)-6-(trifluoromethyl)-, CML-261, Pexidartinib, PEXIDARTINIB (PLX3397), PEXIDARTINIBPLX-3397, PLX 3397, PLX-3397, PLX3397, TURALIO

Data updated 22 hours agoSource updated unknown

Identifiers

Codes (0)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

Data not yet available

No cross-reference code recorded yet beyond the record identifiers above.

Regulatory status is jurisdiction-specific and time-bound. Nothing on this page is a treatment recommendation.

FLT3 D839H1
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Resistance	2	submitted	EID8673 Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD. Proliferation studies showed that the FLT3-ITD D839H (IC50 = 8.6μM) confers relative resistance to PLX3397 i… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 D839H1

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Resistance

submitted

EID8673

Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD. Proliferation studies showed that the FLT3-ITD D839H (IC50 = 8.6μM) confers relative resistance to PLX3397 i… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

FLT3 D839N1
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Resistance	3	submitted	EID8672 Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD or FLT3-ITD/F691L. Proliferation and biochemical studies showed that the FLT3-ITD D839N (IC50 = 0.85μM) and F… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 D839N1

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Resistance

submitted

EID8672

Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD or FLT3-ITD/F691L. Proliferation and biochemical studies showed that the FLT3-ITD D839N (IC50 = 0.85μM) and F… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

FLT3 F691L4
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	3	submitted	EID8674 Deep sequencing revealed that no secondary F691L mutations were found in all 9 patients who achieved a partial or complete response on PLX3397 and who subsequently relapsed on the drug, suggesting thi… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Does Not Support Resistance	1	rejected	EID9716 Overall response rate was 21% in this clinical trial with Pexidartinib on relapsed or refractory AML patients with FLT3-ITD. Forty two out of the total 80 patients were tested for FLT3 KD mutations be… (full text at CIViC) PMID 32330242 · Smith et al., 2020 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Does Not Support Resistance	1	submitted	EID9717 Overall response rate was 21% in this clinical trial with Pexidartinib on relapsed or refractory AML patients with FLT3-ITD. Forty two out of the total 90 patients were tested for FLT3 KD mutations be… (full text at CIViC) PMID 32330242 · Smith et al., 2020 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID8664 Proliferation studies revealed that PLX3397 has the ability to inhibit the proliferation of Ba/F3 cells expressing FLT3-ITD/F691L (IC50 = 0.097μM). PLX3397 equally inhibited the proliferation of MOLM1… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 F691L4

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID8674

Deep sequencing revealed that no secondary F691L mutations were found in all 9 patients who achieved a partial or complete response on PLX3397 and who subsequently relapsed on the drug, suggesting thi… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

〃

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Does Not Support Resistance

rejected

EID9716

Overall response rate was 21% in this clinical trial with Pexidartinib on relapsed or refractory AML patients with FLT3-ITD. Forty two out of the total 80 patients were tested for FLT3 KD mutations be… (full text at CIViC)

PMID 32330242 · Smith et al., 2020 · Open in CIViC

civic

〃

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Does Not Support Resistance

submitted

EID9717

Overall response rate was 21% in this clinical trial with Pexidartinib on relapsed or refractory AML patients with FLT3-ITD. Forty two out of the total 90 patients were tested for FLT3 KD mutations be… (full text at CIViC)

PMID 32330242 · Smith et al., 2020 · Open in CIViC

civic

〃

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID8664

Proliferation studies revealed that PLX3397 has the ability to inhibit the proliferation of Ba/F3 cells expressing FLT3-ITD/F691L (IC50 = 0.097μM). PLX3397 equally inhibited the proliferation of MOLM1… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

FLT3 ITD & D839G1
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Resistance	3	submitted	EID8669 Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD or FLT3-ITD/F691L. Proliferation and biochemical studies showed that the FLT3-ITD D839G (IC50 =4.1μM) and FLT… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 ITD & D839G1

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Resistance

submitted

EID8669

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

FLT3 ITD & N841K1
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Resistance	3	submitted	EID9936 Using a saturation mutagenesis screen, resistant clones harboring N841K were identified in a Ba/F3 proliferation assay when exposed to PLX3397 on a FLT3-ITD + F691L background (Figure 3B). The N841K m… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 ITD & N841K1

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Resistance

submitted

EID9936

Using a saturation mutagenesis screen, resistant clones harboring N841K were identified in a Ba/F3 proliferation assay when exposed to PLX3397 on a FLT3-ITD + F691L background (Figure 3B). The N841K m… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

FLT3 M664I1
Pexidartinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Resistance	3	submitted	EID8667 Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD or FLT3-ITD/F691L. Proliferation and biochemical studies showed that the FLT3-ITD M664I (IC50 =0.61μM) and FL… (full text at CIViC) PMID 25847190 · Smith et al., 2015 · Open in CIViC	civic

FLT3 M664I1

Pexidartinib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Resistance

submitted

EID8667

Mutations were identified by in vitro saturation mutagenesis on the background of FLT3-ITD or FLT3-ITD/F691L. Proliferation and biochemical studies showed that the FLT3-ITD M664I (IC50 =0.61μM) and FL… (full text at CIViC)

PMID 25847190 · Smith et al., 2015 · Open in CIViC

civic

CSF1R Expression3
Pexidartinib	GlioblastomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID8132 A comparison of tyrosine kinase inhibitors that target glioma tumour cells directly or the tumour microenvironment were tested on a PDGF-B-driven glioma genetically engineered mouse model (PDG). PDG m… (full text at CIViC) PMID 28759044 · Yan et al., 2017 · Open in CIViC	civic
Dovitinib + PexidartinibCombination	GlioblastomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID8134 A comparison of tyrosine kinase inhibitors that target glioma tumour cells directly or the tumour microenvironment were tested on a PDGF-B-driven glioma genetically engineered mouse model (PDG). The C… (full text at CIViC) PMID 28759044 · Yan et al., 2017 · Open in CIViC	civic
Pexidartinib + VatalanibCombination	GlioblastomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID8133 A comparison of tyrosine kinase inhibitors that target glioma tumour cells directly or the tumour microenvironment were tested on a PDGF-B-driven glioma genetically engineered mouse model (PDG). The C… (full text at CIViC) PMID 28759044 · Yan et al., 2017 · Open in CIViC	civic

CSF1R Expression3

Pexidartinib

GlioblastomaCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID8132

A comparison of tyrosine kinase inhibitors that target glioma tumour cells directly or the tumour microenvironment were tested on a PDGF-B-driven glioma genetically engineered mouse model (PDG). PDG m… (full text at CIViC)

PMID 28759044 · Yan et al., 2017 · Open in CIViC

civic

Dovitinib + PexidartinibCombination

GlioblastomaCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID8134

PMID 28759044 · Yan et al., 2017 · Open in CIViC

civic

Pexidartinib + VatalanibCombination

GlioblastomaCURATED_BROADER

Predictive

Supports Sensitivity Response

submitted

EID8133

PMID 28759044 · Yan et al., 2017 · Open in CIViC

civic

CSF1 Overexpression1
Pexidartinib	Tenosynovial Giant Cell Tumor	Predictive	B	Supports Sensitivity Response	3	submitted	EID1969 23 patients were enrolled in the phase 2 extension study. The chosen phase 2 dose of PLX3397 was 1000 mg per day. 12/23 patients with tenosynovial giant-cell tumors had a partial response and 7 patien… (full text at CIViC) PMID 26222558 · Tap et al., 2015 · Open in CIViC	civic

CSF1 Overexpression1

Pexidartinib

Tenosynovial Giant Cell Tumor

Predictive

Supports Sensitivity Response

submitted

EID1969

23 patients were enrolled in the phase 2 extension study. The chosen phase 2 dose of PLX3397 was 1000 mg per day. 12/23 patients with tenosynovial giant-cell tumors had a partial response and 7 patien… (full text at CIViC)

PMID 26222558 · Tap et al., 2015 · Open in CIViC

civic