Pexidartinib (Turalio)

Mechanism of action

From the NCI Drug Dictionary: A small-molecule receptor tyrosine kinase (RTK) inhibitor of proto-oncogene receptor tyrosine kinase (KIT), colony-stimulating factor-1 receptor (CSF1R) and FMS-like tyrosine kinase 3 (FLT3), with antineoplastic activity. Upon oral administration, pexidartinib targets, binds to and inhibits phosphorylation of KIT, CSF1R and FLT3 harboring an internal tandem duplication (ITD) mutation. This results in the inhibition of tumor cell proliferation.

Toxicity management

• Link to REMS

Diseases for which it is established (work in progress)

• Tenosynovial giant cell tumor

History of changes in FDA indication

• 2019-08-02: Approved for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery. (Based on ENLIVEN)

Also known as

• Code name: PLX-3397
• Brand name: Turalio