Lysodren (mitotane)
An approved treatment for Adrenocortical Carcinoma.
The same compound appears under different names depending on the context. Here is how to identify Mitotane wherever you encounter it, plus the key facts at a glance.
- Generic name
- Mitotane
- Brand name
- Lysodren
- Drug class
- Adrenolytic small molecule
- Manufacturer
- Esteve Pharmaceuticals (formerly HRA Pharma Rare Diseases)
- How it's taken
- Mitotane is taken by mouth as tablets in three or four divided doses per day, starting at 2,000-6,000 mg daily.
The only drug specifically approved for adrenocortical carcinoma. Taken daily as a tablet, it directly attacks adrenal cancer cells while also reducing excess hormone production.
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Where Mitotane fits
The only FDA-approved drug specifically for adrenocortical carcinoma. FDA-approved for adrenocortical carcinoma that cannot be removed by surgery. Doctors also use it after surgery (adjuvant therapy) to try to prevent the cancer from coming back, but that use is off-label in the U.S.
How Mitotane works
Mitotane destroys cells of the adrenal gland (a small gland on top of the kidney). Exactly how it works is not known; it directly suppresses the adrenal cortex and changes how the body processes steroid hormones. Additionally, it blocks the production of hormones from the adrenal gland, helping reduce symptoms caused by excessive hormone production.
Mechanism: Adrenolytic agent that destroys adrenocortical cells and inhibits steroid synthesis
Side effects and safety
Lysodren has a boxed warning: it raises the risk of adrenal crisis during shock, severe injury, or infection, which can be fatal. If this happens, mitotane is stopped for a while and steroid hormones are given, so call your doctor right away about any injury, infection, or other illness. The drug can also cause adrenal insufficiency, so steroid replacement may be needed. Mitotane causes significant side effects in most patients. Gastrointestinal effects (nausea, vomiting, diarrhea) occur in about 80% of patients. Neurological effects include dizziness, vertigo, and loss of coordination in up to 40% of patients. Depression also occurs commonly. Levels above the therapeutic range can cause severe neurotoxicity. Lysodren can also cause liver injury, including liver failure, and low blood cell counts, so liver tests and blood counts are checked before starting, while the dose is adjusted, and as needed after that. It can harm an unborn baby: pregnancy status is checked before starting, and females who can become pregnant should use non-hormonal birth control during treatment and for as long as mitotane stays in the blood, because it can make hormonal birth control fail. The label also warns about ovarian cysts in premenopausal women, longer bleeding time before surgery or dental work, and changes in hormone test results.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Mitotane
Mitotane is taken by mouth as tablets in three or four divided doses per day, starting at 2,000-6,000 mg daily. The dose is gradually increased based on blood levels with a target of 14-20 mg/L, which typically takes 3-5 months to achieve. Tablets are swallowed whole with food, at a consistent time relative to meals; high-fat food increases absorption. Caregivers should wear gloves when handling the tablets.
Availability and cost
Only available as the brand-name product.
No generic is approved in the U.S.; Lysodren is the only FDA-approved mitotane product. The specialized indication and small manufacturing volumes keep costs notable.
Help paying for Lysodren
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Other support
Financial help may be available to lower Lysodren out-of-pocket costs; call 1-844-LYSODREN to see if you qualify.
The official page does not say who qualifies. Ask the program. · source - Insurance and case manager help
Walgreens Specialty Pharmacy (Lysodren's pharmacy) checks coverage and costs and connects patients to assistance programs. 1-800-320-2112.
The official page does not say who qualifies. Ask the program. · source
Good to know: The official site does not say what type of help is offered or who qualifies. The site cites ESTEVE for the US prescribing information, so the HRA Pharma manufacturer entry may be out of date.
Clinical trial results
Mitotane has been used for over 60 years and remains the only drug specifically approved by the FDA for inoperable adrenocortical carcinoma. It is also sometimes used after surgery (adjuvant therapy), but that use is off-label in the U.S.; the FDA label covers only adrenocortical carcinoma that cannot be removed by surgery.
Development history
Mitotane was approved by the FDA on July 8, 1970, making it one of the oldest cancer drugs still in use. The drug's origins trace back to observations of adrenal damage in animals exposed to DDT-related compounds in the 1950s-1960s. It was chemically derived from DDT metabolites.
Explore Adrenocortical Carcinoma trials
Other Adrenocortical Carcinoma treatments
Common questions about Mitotane
▸What is Mitotane (Lysodren)?
The only drug specifically approved for adrenocortical carcinoma. Taken daily as a tablet, it directly attacks adrenal cancer cells while also reducing excess hormone production.
▸How does Mitotane work?
Mitotane destroys cells of the adrenal gland (a small gland on top of the kidney). Exactly how it works is not known; it directly suppresses the adrenal cortex and changes how the body processes steroid hormones. Additionally, it blocks the production of hormones from the adrenal gland, helping reduce symptoms caused by excessive hormone production.
▸What are the side effects of Mitotane?
Lysodren has a boxed warning: it raises the risk of adrenal crisis during shock, severe injury, or infection, which can be fatal. If this happens, mitotane is stopped for a while and steroid hormones are given, so call your doctor right away about any injury, infection, or other illness. The drug can also cause adrenal insufficiency, so steroid replacement may be needed. Mitotane causes significant side effects in most patients. Gastrointestinal effects (nausea, vomiting, diarrhea) occur in about 80% of patients. Neurological effects include dizziness, vertigo, and loss of coordination in up to 40% of patients. Depression also occurs commonly. Levels above the therapeutic range can cause severe neurotoxicity. Lysodren can also cause liver injury, including liver failure, and low blood cell counts, so liver tests and blood counts are checked before starting, while the dose is adjusted, and as needed after that. It can harm an unborn baby: pregnancy status is checked before starting, and females who can become pregnant should use non-hormonal birth control during treatment and for as long as mitotane stays in the blood, because it can make hormonal birth control fail. The label also warns about ovarian cysts in premenopausal women, longer bleeding time before surgery or dental work, and changes in hormone test results.
▸How is Mitotane taken?
Mitotane is taken by mouth as tablets in three or four divided doses per day, starting at 2,000-6,000 mg daily. The dose is gradually increased based on blood levels with a target of 14-20 mg/L, which typically takes 3-5 months to achieve. Tablets are swallowed whole with food, at a consistent time relative to meals; high-fat food increases absorption. Caregivers should wear gloves when handling the tablets.
▸Is Mitotane FDA approved?
Yes, Mitotane (Lysodren) is FDA approved (1970) for the treatment of Adrenocortical Carcinoma.
▸How old is mitotane and why is it still used?
Mitotane was approved by the FDA in 1970, making it one of the oldest cancer drugs still in use. It remains the only drug specifically approved for adrenocortical carcinoma. Despite significant side effects, no newer drug has replaced it for this ultra-rare cancer.
▸What is the origin of mitotane?
Mitotane's origins trace back to observations of adrenal damage in animals exposed to DDT-related compounds in the 1950s-1960s. It was chemically derived from DDT metabolites and repurposed as a cancer treatment targeting adrenal cells.
▸Why does mitotane need to be taken with high-fat meals?
Mitotane is a highly lipophilic (fat-soluble) drug, and taking it with high-fat meals significantly enhances its absorption from the gut. Without fat, blood levels may remain subtherapeutic.
▸What blood level monitoring is required?
Target blood levels are 14-20 mg/L, which typically takes 3-5 months to achieve. Levels above the therapeutic range can cause severe neurotoxicity, so regular blood level monitoring is essential during dose titration.
▸What are the most challenging side effects?
Gastrointestinal effects (nausea, vomiting, diarrhea) occur in about 80% of patients. Neurological effects including dizziness, vertigo, and coordination problems affect up to 40%. Depression is also common. These side effects limit the dose many patients can tolerate.
▸Is generic mitotane available?
No. In the U.S., mitotane is sold only as the brand Lysodren; the FDA has not approved a generic version. Call 1-844-LYSODREN to ask about financial help.