Is it true that the reason Mitotane is banned in the country?

Mitotane (trade name Lysodren) is a specific inhibitory drug for the treatment of adrenocortical carcinoma (ACC) and related Cushing's syndrome. It has been approved by the U.S. FDA since 1970 ACC, Europe EMA also approved its indications. However, in China, it has not yet passed the local registration approval of the State Food and Drug Administration, so there is no relevant "sale ban" regulations, but it is in a situation of being unmarketed and not yet included in the national medical insurance.

Because ACC is a rare disease with very few patients in China. It is almost impossible to conduct large-scale II/III multi-center clinical trials, and the cost and difficulty of listing it are extremely high. To this end, medical institutions represented by Peking Union Medical College Hospital have tried to introduce mitotane to individual patients through the "overall imported drugs" system since 2019, and have formulated management procedures such as medication guidelines, informed consent, and concentration monitoring to ensure safe and reasonable use.

There is a rumor circulating in the marketThe saying that "mitotane is banned from sale by the state" is really a misunderstanding. China has banned the drug not because of the problem of the drug itself, but because ACC is a rare disease. The clinical path has not yet been established, registration information is lacking, and clinical trial challenges are huge. Therefore, it has not yet been marketed locally. However, it can still be used under compliance conditions through import approval and clinical exceptions. In other words, "unapproved" ≠ "state prohibited".

With the gradual improvement of rare disease policies, the state has gradually given preferential support to the introduction of orphan drugs, such as including"national urgent clinical use", "priority review", etc. Domestic medical institutions have established an ACC treatment expert group to form an expert consensus and concentration monitoring system. In the future, if preparation manufacturers apply for registration and get approval, Chinese patients will be able to obtain treatment opportunities more conveniently and with wider coverage. The current recommendations for ACC patients are: ① to be diagnosed in a tertiary A hospital; ② to communicate with oncology /endocrinology experts whether they can be imported as a whole; ③ to monitor blood gas concentrations and side effects according to the plan formulated by the experts.

Mitotan is not "banned" by the country due to safety issues but because of the rare disease that leads to high local marketing costs and lack of evidence. It is still used in compliance with the "whole import" system in practice. It is expected that future policies and rare disease drug registration channels will be smoother and more treatment options will be introduced.

Reference materials:https://www.drugs.com

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