|Drug class||Kinase inhibitor|
|Main uses||Tenosynovial giant cell tumor (TGCT)|
|Side effects||Liver problems, loss of hair color, tiredness, low neutrophils, increased cholesterol, eye swelling, rash|
|Chemical and physical data|
|Molar mass||417.82 g·mol−1|
|3D model (JSmol)|
Pexidartinib, sold under the brand name Turalio, is a medication used to treat tenosynovial giant cell tumor (TGCT). It is used in cases which result in significant problems and cannot be treated by surgery. It is taken by mouth.
Common side effects include liver problems, loss of hair color, tiredness, low neutrophils, increased cholesterol, eye swelling, and rash. The liver problems can result in death. Use in pregnancy may harm the baby. It is a kinase inhibitor and works by blocking colony-stimulating factor-1 receptor (CSF-1R).
Pexidartinib was approved for medical use in the United States in 2019. It was refused approval in Europe in 2020 due to minimal benefits and concerns regarding side effects. It is not approved in the United Kingdom. In the United States it costs about 21,200 USD per month.
It is taken at a dose of 400 mg twice per day.
The approval of pexidartinib was based on the results of a trial of 120 subjects, 59 of whom received placebo. The primary efficacy endpoint was the overall response rate (ORR) analyzed after 25 weeks of treatment. The clinical trial demonstrated a statistically significant improvement in ORR in subjects who received pexidartinib, with an ORR of 38%, compared to no responses in subjects who received placebo. The complete response rate was 15% and the partial response rate was 23%. A total of 22 out of 23 responders who had been followed for a minimum of six months following the initial response maintained their response for six or more months, and a total of 13 out of 13 responders who had been followed for a minimum of 12 months following the initial response maintained their response for 12 or more months.
The U.S. Food and Drug Administration (FDA) granted the application for pexidartinib breakthrough therapy designation, orphan drug designation, and priority review designation. The FDA granted the approval of Turalio to Daiichi Sankyo.
Pexidartinib is available in the US only through the Turalio Risk Evaluation and Mitigation Strategy (REMS) Program. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.
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