Last reviewed 2026-04-20 · How we verify

Lysodren (MITOTANE)

Lysodren works by inhibiting the production of cortisol and aldosterone in the adrenal cortex.

Mitotane (Lysodren), marketed by Esteve, is a unique therapeutic option for carcinoma in situ of the adrenal cortex, with a mechanism that specifically targets the inhibition of cortisol and aldosterone production. Its key strength lies in its distinct mechanism of action, which differentiates it from other same-class drugs such as asparaginase, altretamine, hydroxycarbamide, pentostatin, and masoprocol. The primary risk to Mitotane's market position is the expiration of its key composition patent in 2028, which could lead to increased competition from generics.

At a glance

Mechanism of action

Mitotane is an adrenal cytotoxic agent with an unknown mechanism of action. Mitotane modifies the peripheral metabolism of steroids and directly suppresses the adrenal cortex. reduction in 17-hydroxycorticosteroids in the absence of decreased corticosteroid concentrations and increased formation of 6--hydroxycortisol have been reported.

Approved indications

• Carcinoma in situ of adrenal cortex

Boxed warnings

• WARNING: ADRENAL CRISIS IN THE SETTING OF SHOCK, SEVERE TRAUMA OR INFECTION Patients treated with LYSODREN are at increased risk for developing adrenal crisis in the setting of shock, severe trauma or infection that may lead to death. If shock, severe trauma or infection occurs or develops, temporarily discontinue LYSODREN and administer exogenous steroids. Monitor patients closely for infections and instruct patients to contact their physician immediately if injury, infection, or any other concomitant illness occurs [see Dosage and Administration (2.3) and Warnings and Precautions (5.1)]. WARNING: ADRENAL CRISIS IN THE SETTING OF SHOCK, SEVERE TRAUMA OR INFECTION See full prescribing information for complete boxed warning. Patients treated with LYSODREN are at increased risk for developing adrenal crisis in the setting of shock, severe trauma or infection that may lead to death. If shock, severe trauma or infection occurs or develops, temporarily discontinue LYSODREN and administer exogenous steroids. Monitor patients closely for infections and instruct patients to contact their physician immediately if injury, infection, or any other concomitant illness occurs ( 2.3 , 5.1 ).

Common side effects

• Anorexia
• Nausea
• Vomiting
• Diarrhea
• Depression
• Dizziness
• Vertigo
• Rash
• Neutropenia
• Growth retardation
• Hypothyroidism
• Confusion

Key clinical trials

• Study of Preoperative Radiation Therapy in Participants With Resectable Recurrent Abdominal Adrenocortical Carcinoma (PHASE1)
• Mitotane With or Without Cisplatin and Etoposide After Surgery in Treating Patients With Stage I-III Adrenocortical Cancer With High Risk of Recurrence (PHASE3)
• Phase II Trial of Pembrolizumab Plus Lenvatinib in Advanced Adrenal Cortical Carcinoma (PHASE2)
• Dostarlimab for Locally Advanced or Metastatic Cancer (Non-colorectal/Non-endometrial) With Tumor dMMR/MSI (PHASE2)
• Cemiplimab as Maintenance Treatment for Advanced Adrenocortical Cancer (PHASE2)
• German Adrenocortical Carcinoma Registry
• Evaluation of Side Effects of Mitotane
• A Phase II Study to Evaluate the Efficacy and Safety of Pembrolizumab in Combination With Mitotane in Patients With Advanced Adrenocortical Carcinoma (PHASE2)

Primary sources

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