A life-saving drug that recently filled the treatment gap for a rare disease in China has announced its withdrawal from the market less than a year after its launch. For about 3,000 patients with Cushing’s syndrome, this is not just a corporate retreat but a direct risk of treatment interruption. The news quickly became a hot topic and drew widespread attention.
Recently, the Italian pharmaceutical company Recordati’s 100% owned Chinese subsidiary, Ricondi, has officially filed for deregistration, indicating a complete exit from the Chinese market. With Ricondi’s withdrawal, several rare disease drugs of the company will also cease supply to China, including the only oral medication, Ozoxisterone, used to treat Cushing’s syndrome in China.
Ozoxisterone was approved domestically in China in September 2024 and officially launched for commercialization in China in April 2025, becoming the only orally administered new drug approved in the country for treating adult Cushing’s syndrome, filling the domestic treatment gap for this rare disease.
According to a report by the Economic Daily, Ricondi’s exit from the market less than a year after its launch means that for Cushing’s syndrome patients, it’s like cutting off a “lifesaving drug.” If patients who need long-term medication are suddenly cut off, their average survival period is only about 5 years, with a 9.3 times higher risk of death compared to normal individuals.
The withdrawal of this rare disease drug from the Chinese market has sparked discussions. With the small scale of rare disease patients, high medication costs, and inadequate coverage by commercial insurance, the drugs cannot enter health insurance, meaning that even if the drugs are approved, they are challenging to sustainably market. Many analyses believe that the withdrawal of this rare disease drug is not accidental but closely related to its failure to enter the national medical insurance catalog.
A user on Weibo, “Storyteller,” commented that lifesaving drugs for rare diseases need strong support from the medical insurance fund. The high cost of lifesaving drugs for rare diseases, high research and development costs, and patients’ financial difficulties make it impossible for them to afford these drugs, resulting in the lack of such lifesaving medications. Reality is harsh.
“Dream Autumn Pharmacist,” the director of the Pharmacy Department at the Zhengzhou McClain Psychiatric Hospital, certified on Weibo, stated that Ricondi’s sudden market exit has put approximately 3,000 Cushing’s syndrome patients who rely on its medications to control their condition in a dilemma of lacking lifesaving drugs. The core reason for all this is that Ozoxisterone failed to enter the national medical insurance catalog in 2025. On December 11, 2025, just four days after the release of the medical insurance catalog for that year by the National Medical Insurance Bureau, a Cushing’s syndrome patient suddenly received a text message from the pharmacy informing her that the Ozoxisterone she was taking was about to exit the Chinese market.
The article mentioned that the annual cost of Ozoxisterone is about 200,000 RMB, exceeding the affordability of many households. With limited coverage by commercial health insurance (such as Huimin insurance), patients heavily rely on basic medical insurance. The inability to get medical insurance coverage means that this drug is still out of reach for most patients. Ricondi’s market exit serves as a mirror, reflecting the real challenges that rare disease medications face from research and development, market launch to accessibility for patients.
Patient groups are calling for the establishment of special funds or risk-sharing mechanisms, emphasizing that “every life should not be given up on.”
The Beijing News reported that positive results from multiple global clinical studies have fully verified the effectiveness and safety of Phospho-Ozoxisterone tablets. Two phase III clinical studies shared by Professor Xiao Hui Guo of the Endocrinology Department at Peking University First Hospital, covering 210 patients worldwide, showed that Phospho-Ozoxisterone tablets can rapidly and persistently reduce cortisol levels, with effects maintained for over 4 years, significantly improving the quality of life. Additionally, Chinese patients are more sensitive to Phospho-Ozoxisterone tablets, requiring about half the daily dose of European and American patients.
Professor Hui Juan Zhu of the Endocrinology Department at Peking Union Medical College Hospital pointed out that the ultimate goal of treating Cushing’s syndrome is not just achieving normal cortisol levels but, more importantly, achieving comprehensive physical and mental recovery for patients, enabling them to truly return to normal life.