Pexidartinib (TURALIO™, Daiichi Sankyo) was approved in 2019 as the only treatment for recurrent and inoperable tenosynovial giant cell tumor (TGCT) in the United States. It was approved for symptomatic TGCT patients associated with severe morbidity (unmanageable symptoms) and functional limitations where surgery would be extremely difficult or the risk, risk of recurrence or risk of complications associated with surgery is higher than the potential benefit (1). This drug is only available in the United States under a FDA Risk Evaluation and Mitigation Strategy (REMS) Program to reduce the severity and frequency of liver related side effects.
How does Turalio work?
Pexidartinib works by inhibiting CSF1 from signaling to TGCT and the surrounding cells. This disrupts the ability for the TGCT to recruit more cells, shrinking the tumor and halting the growth (2). Recently, it has been discovered that Turalio and other CSF1 inhibitors blocks CSF1 at the cell source and target the surrounding immune cells that are recruited (3). By blocking the tumor cells ability to communicate with the joint environment, the tumor shrinks and other associated symptoms such as pain and swelling are decreased (4,5). It is reported that patients' pain and symptoms continue to improve even after years of taking Turalio.
How do I talk to my doctor about turalio?
Turalio is part of a REMS program, meaning only certain providers can prescribe it. Consult with your oncologist or healthcare provider about Turalio and their REMS program. Providers who are part of the REMS program who can prescribe Turalio can be found here.
