Pexidartinib

Pexidartinib is a prescription medicine used to treat adult patients with tenosynovial giant cell tumor (TGCT) that cannot be removed by surgery or has spread to other parts of the body. Pexidartinib is a kinase inhibitor, which works by blocking certain proteins in the body that may be involved in the growth and spread of TGCT. It is available in tablet form and taken twice daily.

Pexidartinib, sold under the brand name Turalio, is a medication used to treat adults with a specific type of tumor called tenosynovial giant cell tumor (TGCT). Here's a breakdown of its key aspects:

• Purpose: Treats symptomatic tenosynovial giant cell tumors (TGCT) in adults. These are tumors that cause significant problems or limit function, and cannot be effectively improved with surgery [1, 2].
• How it works: Pexidartinib is a type of drug called a kinase inhibitor. It works by blocking a specific protein called the colony-stimulating factor 1 receptor (CSF-1R) which plays a role in the growth and survival of TGCT cells [2, 3].
• Dosage: It is taken orally as capsules, typically twice a day on an empty stomach [1].
• Important Safety Information: Pexidartinib can cause serious side effects, particularly liver problems. Regular blood tests are needed to monitor liver function while taking this medication [1, 2].

Here are some additional details about Pexidartinib:

• First FDA-approved systemic treatment for TGCT: Prior to pexidartinib, treatment options for TGCT were mainly surgical procedures. Pexidartinib offers a non-surgical approach for patients who cannot undergo surgery or for whom surgery is not effective [2].
• Approval process: The medication was approved by the FDA in August 2019 based on a clinical trial that showed it to be effective in shrinking tumors and improving symptoms in patients with TGCT [1].