Background: Tenosynovial giant cell tumor refers to a group of rarely occurring tumors that are
formed in the joints, which are characterized by pain, swelling, and limitation of movement of the joint. Surgery
is the main treatment strategy, but the tumor is likely to recur, especially in pigmented villonodular synovitis,
which is the diffuse-type giant cell tumor. Pexidartinib was approved in August 2019 by the Food and Drug
Administration (FDA) with a brand name TURALIO as the first systemic approved therapy for patients having
Tenosynovial Giant Cell Tumors (TGCT).
Objective: In this review, different aspects pertaining to pexidartinib have been summarized, including the
pathophysiology of TGCT, chemistry, pharmacokinetics and pharmacodynamics of pexidartinib. Special
attention is given to various reported clinical trials of pexidartinib.
Methods: A comprehensive literature search was conducted in the relevant databases to identify studies
published in this field during recent years.
Conclusion: Pexidartinib acts by inhibiting the Colony-Stimulating Factor (CSF1)/CSF1 receptor pathway,
which leads to the inhibition of the cell lines proliferation and promotes the autophosphorylation process of the
ligand-induced CSF1 receptor. Pexidartinib emerged as a potential drug candidate for the treatment of TGCT.