# Pexidartinib

Source: https://onco.cc/drugs/pexidartinib/  
OnCo record `pexidartinib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Pexidartinib is the first drug for tenosynovial giant cell tumour, a benign but destructive joint tumour; it is effective but has liver toxicity that requires a restricted programme.

## Summary

Pexidartinib is a small-molecule inhibitor of CSF1R, KIT and FLT3. In tenosynovial giant cell tumour, a benign but locally destructive joint tumour driven by CSF1 overproduction, blocking CSF1R depletes the macrophage-rich tumour mass. In ENLIVEN (Lancet 2019), 39% of patients responded at 25 weeks versus none on placebo, with improvements in joint function. The FDA approved it in 2019 for symptomatic disease not amenable to surgery, under a REMS programme because of mixed or cholestatic hepatotoxicity that requires liver monitoring, while the EMA refused approval in 2020 on the balance of benefit and liver risk. Vimseltinib, approved in 2025, offers a safer alternative in the same class. Pexidartinib was the first drug ever approved for this tumour and showed that a macrophage-driven tumour can be treated by starving it of macrophages.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill
- Brand: Turalio
- Modality: Small-molecule CSF1R/KIT/FLT3 inhibitor
- Mechanism: Inhibits colony-stimulating factor 1 receptor, depleting the CSF1-driven macrophage-rich tumour of tenosynovial giant cell tumour.
- Approvals: US 2019: Symptomatic tenosynovial giant cell tumour not amenable to surgery

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Pexidartinib
- ENLIVEN (Lancet 2019): https://doi.org/10.1016/S0140-6736(19)30764-0
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pexidartinib

## Connected records

- cancers: [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/), [Tenosynovial giant cell tumour (TGCT)](https://onco.cc/cancers/tenosynovial-giant-cell-tumour/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [CSF1](https://onco.cc/targets/csf1/), [CSF1R](https://onco.cc/targets/csf1r/), [KIT](https://onco.cc/targets/kit/)
- companies: [Daiichi Sankyo](https://onco.cc/companies/daiichi-sankyo/)
- trials: [A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT](https://onco.cc/trials/nct04488822/), [ENLIVEN](https://onco.cc/trials/enliven/)
- key papers: [Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial](https://onco.cc/key-papers/paper-tap-lancet/)
- pathways: [Myeloid suppression: TAMs, MDSCs & don't-eat-me signals](https://onco.cc/pathways/myeloid-suppression-axis/)

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