An evaluation of vimseltinib for treatment of tenosynovial giant cell tumors.

Introduction: Tenosynovial giant cell tumor (TGCT) is a rare soft tissue neoplasm with aggressive local growth. The disease is driven by excessive CSF1 expression in tumor cells, leading to increased recruitment of monocytes and macrophages, cytokine production, and tumor development. Targeted therapy against CSF1R is an effective treatment approach for unresectable, symptomatic TGCT. Vimseltinib, a novel, small-molecule tyrosine kinase inhibitor of CSF1R, has shown clinical efficacy in patients with TGCT.

Areas covered: This paper outlines the pathogenesis and therapeutic options for TGCT, along with a detailed profile of vimseltinib, including its mechanism of action, pharmacokinetics, efficacy and safety data from clinical studies. The efficacy and tolerability of vimseltinib are indirectly compared with previously known CSF1R inhibitors.

Expert opinion: In the MOTION study, the use of vimseltinib in patients with advanced TGCT resulted in a high objective response rate, substantial benefit in reducing clinical symptoms (such as pain and stiffness), and a favorable safety profile. Vimseltinib represents a promising new therapeutic option for patients with unresectable TGCT and is currently awaiting regulatory review by the FDA and EMA.