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.
- 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
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
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.
- 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- EU marketing authorisation application withdrawn 2020; no UK licence.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.
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2019 | Symptomatic tenosynovial giant cell tumour not amenable to surgery |
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Latest papers
topQuery 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.
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