又一款罕见病药将退出中国市场，有患者称“吃掉100万”

Group 1 - The high cost of developing rare disease drugs, combined with a limited patient base and high pricing, creates a challenging environment for small to medium-sized enterprises compared to larger pharmaceutical companies [1][4] - Recordati's Chinese subsidiary, Ruikangdi, has officially filed for deregistration, marking its complete exit from the Chinese market, which will result in the discontinuation of several rare disease drugs, including the only oral medication for Cushing's syndrome, Osilodrostat [1][3] - Osilodrostat was approved in China in September 2024 and commercialized in April 2025, but its sales have been limited, with only about 50 patients purchasing the drug in domestic pharmacies [3][4] Group 2 - The average annual treatment cost for Osilodrostat in China is approximately 200,000 yuan, despite the drug being priced at 8,000 yuan per box, which is among the lowest in the world [3][4] - The drug was expected to be included in the national medical insurance directory, which would have reduced out-of-pocket expenses for patients to below 2,000 yuan per month, but it ultimately did not make it into the final directory [3][4] - The exit of Recordati from the Chinese market is not an isolated incident, as other companies like BioMarin have also withdrawn their rare disease treatments, highlighting the ongoing challenges faced by rare disease drug manufacturers in China [4]