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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.
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.
Inhibits colony-stimulating factor 1 receptor, depleting the CSF1-driven macrophage-rich tumour of tenosynovial giant cell tumour. Connects to CSF1R and KIT.
1.Pexidartinib slips into a pocket on CSF1R.
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). REMS-restricted for hepatotoxicity.
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. Symptomatic tenosynovial giant cell tumour not amenable to surgery.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE search: pexidartinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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| Region | Year | Indication |
|---|---|---|
| US | 2019 | Symptomatic tenosynovial giant cell tumour not amenable to surgery |
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Query for this drug: (TITLE:"Pexidartinib" OR ABSTRACT:"Pexidartinib" OR TITLE:"Turalio" OR ABSTRACT:"Turalio") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Pexidartinib, not a curated reading list.
Shares CSF1, CSF1R, Tenosynovial giant cell tumour (TGCT), Myeloid suppression: TAMs, MDSCs & don't-eat-me signals.
Shares Tenosynovial giant cell tumour (TGCT), Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
Shares CSF1, CSF1R, Small-molecule kinase inhibitors.
Shares KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
Shares KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.
Shares KIT, Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors.