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Pexidartinib: first approved systemic therapy for patients with tenosynovial giant cell tumor

This article reviewed pexidartinib in the treatment of tenosynovial giant cell tumor (TGCT). It is the first US FDA approved treatment for this indication. Results from the Phase III ENLIVEN study provided the evidence for this approval and data from clinical studies demonstrates the efficacy of pexidartinib, including tumor response and improved range of motion. In summary, pexidartinib represents significant advancement in the field and a valuable treatment option for selected patients with TGCT.

The latest review article published in Future Oncology discusses the use of pexidartinib in the treatment of TGCT.


Pexidartinib is an orally administered small molecule tyrosine kinase inhibitor. Phase III ENLIVEN study results provided clinical evidence for USA FDA approval for treatment of adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Recommended dosage is 400 mg orally twice daily on an empty stomach. Long-term follow-up in pooled analyses showed increased response rates compared to those observed in ENLIVEN. Patients on pexidartinib also experience meaningful improvements in range of motion. Side effects associated with pexidartinib are generally manageable; however, there is a risk of potentially life-threatening mixed or cholestatic hepatotoxicity and pexidartinib has a Risk Evaluation and Mitigation Strategy program to ensure appropriate monitoring.

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