Pexidartinib (TURALIO™) is an orally administered small molecule tyrosine kinase 
inhibitor with selective activity against the colony-stimulating factor 1 (CSF1) 
receptor, KIT proto-oncogene receptor tyrosine kinase (KIT) and FMS-like 
tyrosine kinase 3 harboring an internal tandem duplication mutation (FLT3-ITD). 
In August 2019, the US FDA approved pexidartinib capsules 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. This approval was based on positive results from the phase III 
ENLIVEN trial. Pexidartinib is being investigated in various malignancies as 
monotherapy or combination therapy. This article summarizes the milestones in 
the development of pexidartinib leading to its first approval for TGCT.