The effect of Mitotane in the treatment of adrenocortical cancer has attracted much attention! Full analysis of efficacy and price information

The latest news in 2025, the field of rare tumors has once again attracted attention: According to the latest statistics from "Clinical Oncology News", adrenocortical cancer (Adrenocortical Although Carcinoma (ACC) has a low incidence rate, its 5 annual survival rate is less than 30% and has been listed by international cancer research institutions as an "orphan cancer" that requires key breakthroughs. In this difficult treatment battlefield, a drug named Mitotane (Mitotane) has once again entered the clinical frontline and has become the only oral drug approved for ACC systemic treatment, attracting much attention.

With the recent approval of the drug for marketing in mainland China, many patients and family members are highly concerned about its "how effective it is", "whether the price is affordable" and "whether generic drugs are available". This article will provide a comprehensive interpretation of mitotane’s pharmacological mechanism, efficacy data, clinical positioning and domestic and international prices to help patients make informed decisions.

1. Mitotane is popular: new hope for rare tumors, the only drug targeting ACCACC

Why is mitotane in the spotlight?

Mitotane (Mitotane) was actually produced by Bristol-Myers as early as the 60s of the last century Squibb was successfully developed by the company and was approved by the FDA in the 1970s. It was originally used to treat adrenocortical cancer (< span>ACC) - This is a rare but extremely aggressive malignant tumor of the endocrine system, with approximately 1-2 million new cases worldwide every year.

In 2023, the State Food and Drug Administration (NMPA) officially approved the marketing of mitotane’s original drug in mainland China, filling the gap in ACC oral drug treatment. This news triggered widespread discussion among doctors and patients. Because in the past few decades, ACCMost patients can only rely on chemotherapy and surgery. Mitotane is currently the only oral drug recommended by NCCN, ESMO and other guidelines for postoperative adjuvant treatment and maintenance treatment of advanced ACC.

2. Verification of efficacy: Mitotane can prolong survival and reduce the risk of recurrence

1. Clinical research support

The reason why mitotane is unique in the treatment ofACC is that it has a dual mechanism:

Cytotoxic effects: Mitotane selectively destroys adrenocortical cells and induces apoptosis.

Hormone suppression function: By reducing the synthesis of hormones such as cortisol and aldosterone, it can alleviate the symptoms of excessive hormones caused by tumors.

According to "Journal of Clinical Endocrinology & Metabolism》A pooledMeta analysis in 2024 pointed out that patients with ACC who received adjuvant mitotane after surgery Among them, the 3 year recurrence-free survival rate (RFS) reached 64%, which was significantly higher than the 42% of the unmedicated group.

Another European multicenter, real-world study (the ADIUVO trial) showed that in low-risk ACC postoperative patients, mitotane The 5 year disease-free survival rate of the 5 group was 76%, which was much higher than the 49% of the observation group. Especially in patients with a high risk of recurrence whose Ki67 index is higher than 10%, the efficacy of mitotane is more obvious.

2. Joint solutions are being explored

Currently, multiple international research centers are exploring options for combining mitotane with radiotherapy, immunotherapy and even targeted therapy. The Institute of Oncology of Milan, Italy, stated that the combination of mitotane and PD-1 inhibitors has shown synergistic anti-tumor effects in animal models, but clinical verification is still needed.

3. Side effects and coping strategies: Standardized monitoring can reduce risks

Despite its clear efficacy, the use of mitotane still needs to be strictly regulated. Its main side effects include:

Gastrointestinal reactions: such as nausea, vomiting, diarrhea;

Neurotoxicity: such as drowsiness, ataxia, and mental status changes;

Abnormal liver function: regular monitoring is requiredALT, AST;

Hormone suppression response: May induce a hypocortisol state.

Therefore, patients taking mitotane must strictly monitor the blood concentration (recommended target 10–20 μg/mL) under the guidance of a professional doctor, and cooperate with hormone replacement therapy (such as prednisone, hydrocortisone) to prevent the occurrence of adrenal crisis.

4. Disclosure of price information: original drugs are expensive, generic drugs are life-saving channels

Domestic original research drugs are on the market: the price is not yet clear

At present, the original drug of mitotane has been launched in mainland China, but it has not yet been included in the medical insurance catalog and is a self-paid drug. According to industry sources, since it has just been approved, some hospitals have not yet fully rolled it out, and the price may be in the negotiation stage.

It is recommended that patients consult the endocrinology department or oncology department of a tertiary hospital for actual prices and supply conditions at pharmacies or DTP pharmacies.

Reference prices for overseas drug purchases:

Generic drugs are basically the same as the original drugs in terms of ingredients and dosage, but there may be slight differences in production supervision, dissolution, etc. It is recommended to use them with the informed consent of a doctor, and to regularly review blood drug concentrations.

5. Mitotane purchasing methods and suggestions

1.Domestic purchase:

It has been approved to enter China, but the coverage is not yet wide.

Patients can consult local tertiary hospitals, specialty drug pharmacies, and oncology DTP pharmacies to obtain medicines or make an appointment for medicines.

2. Overseas purchasing or cross-border drug purchasing:

It can be purchased through regular overseas pharmacies.

It is recommended to give priority to legal channels with prescriptions and never buy online by yourself to avoid buying fake medicines.

3. Medication recommendations:

Carry out liver and kidney function, hormone levels, and CYP3A4metabolism assessment before use;

Monitor blood drug concentration regularly during medication, and review every4-6 weeks;

The initial dose is usually 2g/day, divided into 2-3 times, and gradually increased to 3-6g;

Taking it with a fatty meal can improve absorption.

6. Frequently Asked Questions for Patients (FAQ)

1. Is mitotane a targeted drug?

Strictly speaking, it is not a classic targeted drug, but a selective poison with targeting effects on adrenocortical cells. However, in ACC it functions similarly to "targeting" and is still the only recommended systemic oral treatment.

2. Can it be used for other tumors?

Currently it is only approved for ACC, but there are also studies trying to use it for Cushing's syndrome, congenital adrenal hyperplasia and other diseases, but it has not been widely promoted.

3. How long does it take for the medication to take effect?

The blood concentration of the drug needs to reach 10 μg/mL or above to exert the anti-cancer effect. It usually needs to be taken continuously for 1-2 months to take effect. In some patients, imaging lesions can be reduced after half a year.

4. Can the medication be stopped?

It needs to be decided under the guidance of a doctor and based on the tumor control situation, and the medication cannot be stopped on your own to avoid rapid recurrence of the tumor.

Conclusion

Adrenocortical cancer is a kind of“silent but dangerous” cancer. It is asymptomatic in the early stage and often metastasizes in the late stage. For this reason, oral drugs like mitotane, which have a clear mechanism and are supported by good efficacy data, are of great significance.

Although they are expensive, the emergence of generic drugs has brought a "lifeline" to patients with limited financial conditions. If it can be included in medical insurance or clinical assistance plans in the future, it will bring hope to more people.

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References:

1.NCCN Guidelines on Adrenocortical Carcinoma

2.Terzolo M, et al. Adjuvant mitotane treatment for adrenocortical carcinoma. N Engl J Med. 2007.

3.ClinicalTrials.gov: ADIUVO Trial - Adjuvant Mitotane in Low Risk ACC

4."Chinese Journal of Oncology": 2024Adrenocortical Cancer Clinical Diagnosis and Treatment Guidelines Update