Efficacy of Pexidartinib

Pexidartinib is an oral, small-molecule tyrosine kinase inhibitor that selectively inhibits CSF1R as well as c-Kit receptor tyrosine kinase (KIT) and fms-like tyrosine kinase 3 internal tandem duplication (FLT 3-ITD). Multiple studies have demonstrated the efficacy of pexidartinib as an effective systemic therapy for advanced tenosynovial giant cell tumor (TGCT) in settings where surgery is deemed unduly morbid or clinically unfeasible. Pesidartinib may also be used to reduce initial tumor burden to facilitate surgery. Treatment of tenosynovial giant cell tumor depends on whether the tumor is localized or spreading, the patient's treatment history, and the patient's suitability for various treatment modalities.

Results from the ENLIVEN Phase III study provide clinical evidence for U.S. Food and Drug Administration (FDA) approval for the treatment of adult patients with symptomatic giant cell tumors of the tendon sheath that are associated with severe morbidity or functional limitations that cannot be improved by surgery. The recommended dose is 400 mg twice daily on an empty stomach. Long-term follow-up in the pooled analysis showed an increased response rate compared with that observed in ENLIVEN. Patients taking pesidartinib also experienced meaningful improvements in range of motion. Side effects associated with pesidartinib are generally manageable; however, there is a risk of potentially life-threatening mixed or cholestatic hepatotoxicity, and pesidartinib has a Risk Evaluation and Mitigation Strategy (REMS) program to ensure appropriate monitoring.

Pesidartinib is not marketed in the country. There is less information on the price and other related information of pexidartinib after it is launched overseas. For more drug information and specific prices, please consult the medical consultant.