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Mitotane

A relative of the insecticide DDT that is the only drug specific to adrenal cortex cancer; it needs blood-level monitoring and permanent steroid replacement.

Approved 1970. Adjuvant use for high-risk resected ACC (Terzolo 2007; ADIUVO 2023 negative for low risk), monotherapy for indolent advanced disease, and combined with EDP chemotherapy (FIRM-ACT). Therapeutic range 14-20 mg/L; toxicities include neurologic effects, GI intolerance, adrenal insufficiency (hydrocortisone replacement at higher doses), CYP3A4 induction affecting other drugs.

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Mitotane
PubChem
Modality
Adrenolytic agent (DDD derivative)
Mechanism
Metabolised in adrenocortical cells to an acyl chloride that binds mitochondrial proteins and inhibits steroidogenesis (SOAT1 inhibition causes ER stress); cytotoxic to adrenocortical tissue.
Brand / code
Lysodren
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).

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. Adrenocortical carcinoma is rare enough that most plans cover on diagnosis.

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
Only licensed drug for adrenocortical carcinoma; funded routinely without a TA.
NHS England
Routinely funded for the appraised indication (or via managed access)

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

Regulatory

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Approvals

RegionYearIndication
US1970Inoperable adrenocortical carcinoma (functional and non-functional)
EU2004Advanced adrenocortical carcinoma

Trials

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Recruiting now (live from ClinicalTrials.gov)

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

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Mitotane" OR ABSTRACT:"Mitotane" OR TITLE:"Lysodren" OR ABSTRACT:"Lysodren") 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 Mitotane, not a curated reading list.

Connected

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