OnCo
drugsProductNot mapped hereapproved

Pexidartinib

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.

ENLIVEN (Lancet 2019): 39% response at 25 weeks versus 0% placebo, with functional improvement. Approved 2019 under a REMS for mixed/cholestatic hepatotoxicity. Vimseltinib (2025) offers a safer alternative; EMA refused approval in 2020.

Loading structure…
|
Pexidartinib
PubChem
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.
Brand / code
Turalio
Medicare
Part D (self-administered)

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.

Commercial insurance
covered with prior authorisation

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.

Assistance programmes

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.

Not appraisedSearch NICE
Notes
EU marketing authorisation application withdrawn 2020; no UK licence.
NHS England
Not routinely funded

Sources: NICE search: pexidartinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

1top

Approvals

RegionYearIndication
US2019Symptomatic tenosynovial giant cell tumour not amenable to surgery

Trials

top
ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
14 studies1 recruiting12 Phase 22 Phase 3
Search “Pexidartinib” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Pexidartinib
intervention: Pexidartinib
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Latest papers

top
Latest papers · live from Europe PMC
Open in Europe PMC

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.

Connected

6top

Pages like this

not linked directly; found by shared links