Is Pexidartinib on the market?

Turalio's Pexidartinib (Pexidartinib) is a kinase inhibitor indicated for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) that is associated with significant morbidity or functional limitations and is not amenable to surgical improvement. Pexidartinib blocks the activity of colony-stimulating factor-1 receptor (CSF-1R). In August 2019, it was approved by the U.S. Food and Drug Administration (FDA) for the treatment of giant cell tumor of the tendon sheath (GC-TS) . Pesidartinib is only available in the United States through the Turalio Risk Evaluation and Mitigation Strategy (REMS) program, and the FDA considers it a first-in-class drug.

The approval of pexidartinib was based on the results of a multicenter international clinical trial of120 subjects, 59 of whom received placebo. The primary efficacy endpoint was overall response rate (ORR) analyzed after 25 weeks of treatment. Clinical trials showed that subjects who received pexidartinib had a statistically significant improvement in ORR, with an ORR of 38%, a complete response rate of 15%, and a partial response rate of 23%, compared with subjects who received placebo who had no response. A total of 22 of 23 responders who were followed for at least 6 months after first remission maintained remission for 6 months or longer, and a total of 13 of 13 responders who were followed for at least 12 months after first remission maintained remission for 12 months or longer. Side effects of pexidartinib are increased lactate dehydrogenase, increased aspartate aminotransferase, changes in hair color, increased alanine aminotransferase, and increased cholesterol.

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.