Drug intelligence / Profile preview

pexidartinib

Development stage
Approved
Lead developer
Daiichi Sankyo
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Irreversible Covalent Inhibitors → Covalent Small Molecules → Small Molecules
Administration
Oral
01

Overview

Pexidartinib is a small molecule tyrosine kinase inhibitor used for the treatment of tenosynovial giant cell tumor (TGCT), also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), in adults when surgical resection is not likely to improve outcomes. It acts primarily by inhibiting colony-stimulating factor 1 receptor (CSF1R), as well as c-Kit and fms-like tyrosine kinase 3 (FLT3). By blocking these kinases, pexidartinib disrupts signaling pathways that drive abnormal proliferation and accumulation of cells in the synovium, leading to reduction in tumor size and improvement in symptoms. Pexidartinib was developed by Daiichi Sankyo and received FDA approval in August 2019 as the first systemic therapy for TGCT not amenable to surgery[2][5][9].

Brand names
Turalio
Other names
pexidartinib hydrochloride
02

Targets

KIT (c-KIT proto-oncogene receptor tyrosine kinase)FLT3 (Fms related receptor tyrosine kinase 3)CSF1R (Macrophage colony-stimulating factor receptor)

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