Mitotane

LYSODREN

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Pronunciation: MYE-toe-tayn

Mitotane ballicule: receptor binding, kinetics and half-life diagram — Mechanism of action: Mitotane ballicule

What it isAdrenolytic agent destroying adrenocortical cells; adrenocortical carcinoma and Cushing syndrome.

Why this oneThe only drug that is directly cytotoxic to the adrenal cortex.

What limits itCauses adrenal insufficiency by design, so glucocorticoid replacement at higher-than-usual doses is mandatory. Neurotoxicity; it induces CYP3A4.

Mitotane molecular structure (skeletal formula) — Molecular structure: Mitotane

Mitotane prescription history

Animated chart of estimated U.S. prescriptions per year, Oral anticancer meds (1953–2026). Mitotane enters in 1970 (estimated).

FDA-approved for

adrenocortical carcinoma (1970)

Off-label

Cushing syndrome from adrenal causes

Classes and tags

Clinical profile

Therapeutic safety burden3 / 4

Overdose danger2 / 4

Weight-gain liability0 / 4

Sedation liability2 / 4

QT / Torsades0 / 4

Direct muscarinic antagonism0 / 4

Embryofetal risk3 / 4

Legacy pregnancy categoryD

Defining liabilityMyelosuppression and agent-specific hepatic, cardiac, pulmonary, neurologic or reproductive toxicity require oncology monitoring.

FDA label

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BOXED WARNING Adrenal crisis in the setting of shock, severe trauma, or infection may lead to death; withhold and administer exogenous steroids.

FDA dosing	Population	Start	Target	Max
Adrenocortical carcinoma	Adult	2000–6000 mg/day in 3–4 divided doses	Plasma 14–20 mg/L

Renal Not recommended in severe impairment; in mild–moderate, monitor plasma levels frequently and adjust.

Hepatic Not recommended in severe impairment; in mild–moderate, monitor plasma levels frequently and adjust.

Titrate to plasma level 14–20 mg/L; neurotoxicity more likely >20 mg/L; levels may persist for weeks after stopping due to adipose accumulation.

Instructions

Swallow tablets whole; do not crush, chew, or split.
Take with food; keep timing relative to meals consistent (high-fat food enhances absorption).
Caregivers should wear disposable gloves when handling tablets.
Withhold before planned surgery and during shock, trauma, or infection; provide steroids.

Available strengths

tab 500 mg

Cautions

Adrenal insufficiency and adrenal crisis
CNS toxicity (sedation, lethargy, vertigo)
Ovarian macrocysts in premenopausal women
Hepatotoxicity
Hematologic toxicity (leukopenia, anemia, thrombocytopenia)
Prolonged bleeding time
Embryo-fetal toxicity

Adverse reactions

Anorexia
Nausea
Vomiting
Diarrhea
Dizziness/vertigo
Rash
Hypercholesterolemia
Hypertriglyceridemia
Hypothyroidism
Decreased free testosterone (males)

Pregnancy Can cause fetal harm; preterm births and early pregnancy loss reported.

Lactation Excreted in human milk; do not breastfeed during treatment and while plasma levels remain detectable.

Cost

Mitotane (LYSODREN)

Strength		#/mo	generic $/unit	CostPlus
500 mg	tab	per unit		not offered

NADAC is a voluntary CMS survey of what retail community pharmacies pay for the drugs they stock. Mitotane is dispensed outside retail pharmacies, so it rarely appears on surveyed invoices.

Generic entryNo US generic

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Self-check

Why is glucocorticoid replacement mandatory with mitotane?
It causes adrenal insufficiency by design.
Does mitotane inhibit or induce CYP3A4?
Induces it.