Pexidartinib trials in the country

Pexidartinib is a new type of targeted therapy used to treat a rare tumor called synovial sarcoma (TGCT), also known as tenosynovial giant cell tumor. At present, the clinical trial situation of pesidartinib in China is not very clear. Because pexidartinib is a newer drug, its clinical trials and studies are typically conducted globally. These trials are usually organized by pharmaceutical companies or medical research institutions and require relevant regulatory approval processes to be conducted domestically.

The approval of pexidartinib by the U.S. Food and Drug Administration (FDA) was based on the durable overall response rate (ORR) observed in an international, multicenter, randomized (1:1), double-blind, placebo-controlled trial that enrolled 120 patients with giant cell tumor of the tenosynovium who were not suitable for surgical resection. An independent review committee determined the ORR (Response Evaluation Criteria in Solid Tumors 1.1) at week 25. After 25 weeks of treatment, the ORR was 38% (95% confidence interval: 27, 50), the complete response rate was 15%, and the partial response rate was 23%. Patients receiving placebo had no response (p<0.0001). Twenty-two of 23 patients were followed for at least 6 months after first remission and remained in remission for ≥6 months. Additionally, 13 of 13 responding patients were followed for at least 12 months after initial response, with responses maintained for ≥12 months.

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.