又一款罕见病药将退出中国市场，有患者称“吃掉100万”

Core Viewpoint - Recordati's subsidiary in China, Ruikangdi, has officially filed for deregistration, marking its complete exit from the Chinese market, which will result in the discontinuation of several rare disease medications, including the only oral drug for Cushing's syndrome, Osilodrostat [3][5]. Group 1: Company Actions - Ruikangdi's exit signifies a significant loss for Cushing's syndrome patients, as it is considered a "lifesaving drug" [5]. - Osilodrostat was approved in China in September 2024 and commercialized in April 2025, but has been available since 2021 as part of a pilot program [5]. - Recordati had high sales expectations for Osilodrostat in China, where it was priced at approximately 8,000 yuan per box, with annual treatment costs averaging around 200,000 yuan [5]. Group 2: Market Challenges - The drug's potential inclusion in the national medical insurance directory was anticipated, which could have reduced out-of-pocket costs for patients to below 2,000 yuan per month, but it ultimately was not included [5]. - The high cost of rare disease drug development and the limited patient population make pricing and reimbursement a significant challenge, especially in China where commercial insurance is still developing [6]. - Only about 50 patients in China are estimated to have purchased Osilodrostat, highlighting the drug's limited market penetration due to affordability issues [6]. Group 3: Industry Context - Recordati's exit is not an isolated incident; BioMarin also withdrew a rare disease treatment from the Chinese market in 2024, leaving patients without available options [7]. - The challenges faced by rare disease pharmaceutical companies include lengthy and costly market education and the financial burden on patients, many of whom struggle economically [7]. - Even drugs that are included in insurance plans can face supply issues, as seen with the drug Nitisinone, which experienced shortages despite being on the market for two years [7].