{"entity":{"id":"mitotane","kind":"drug","name":"Mitotane","aka":[],"tldr":"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.","summary":"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.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mitotane","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mitotane"}],"tags":["gap-fill"],"related":[],"cancers":["adrenocortical","localised-adrenocortical-carcinoma","advanced-adrenocortical-carcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lysodren","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.","approvals":[{"region":"US","year":1970,"indication":"Inoperable adrenocortical carcinoma (functional and non-functional)"},{"region":"EU","year":2004,"indication":"Advanced adrenocortical carcinoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/mitotane/","neighbours":{"cancer":[{"id":"adrenocortical","kind":"cancer","name":"Adrenocortical carcinoma","route":"/cancers/adrenocortical/"},{"id":"advanced-adrenocortical-carcinoma","kind":"cancer","name":"Advanced and metastatic adrenocortical carcinoma (ENSAT stage IV or unresectable)","route":"/cancers/advanced-adrenocortical-carcinoma/"},{"id":"localised-adrenocortical-carcinoma","kind":"cancer","name":"Localised adrenocortical carcinoma (ENSAT stage I to III, resectable)","route":"/cancers/localised-adrenocortical-carcinoma/"}],"technology":[{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"}],"paper":[{"id":"paper-adiuvo-adjuvant-mitotane-low-grade-acc-terzolo-lancet-diabetes-endocrinol-2023","kind":"paper","name":"ADIUVO: adjuvant mitotane versus surveillance in low-grade, localised adrenocortical carcinoma","route":"/key-papers/paper-adiuvo-adjuvant-mitotane-low-grade-acc-terzolo-lancet-diabetes-endocrinol-2023/"},{"id":"paper-ese-ensat-adrenocortical-carcinoma-guideline-eur-j-endocrinol-2018","kind":"paper","name":"European Society of Endocrinology and ENSAT clinical practice guidelines on the management of adrenocortical carcinoma in adults","route":"/key-papers/paper-ese-ensat-adrenocortical-carcinoma-guideline-eur-j-endocrinol-2018/"},{"id":"paper-firm-act-edp-mitotane-adrenocortical-carcinoma-nejm-2012","kind":"paper","name":"FIRM-ACT: combination chemotherapy in advanced adrenocortical carcinoma","route":"/key-papers/paper-firm-act-edp-mitotane-adrenocortical-carcinoma-nejm-2012/"}],"term":[{"id":"adrenalectomy","kind":"term","name":"Adrenalectomy","route":"/terms/adrenalectomy/"}],"trial":[{"id":"adiuvo","kind":"trial","name":"ADIUVO","route":"/trials/adiuvo/"},{"id":"adiuvo-2","kind":"trial","name":"ADIUVO-2","route":"/trials/adiuvo-2/"}]}}