这家罕见病药企终止在华业务

Core Viewpoint - Ruikangdi (Beijing) Pharmaceutical Co., Ltd. has announced the completion of its liquidation process and will terminate all business activities, ceasing to provide any products, services, or business support in the near future [2][6]. Company Overview - Ruikangdi was established in Beijing in 2021 as a subsidiary of the Italian pharmaceutical group Recordati, focusing on introducing rare disease medications to benefit patients in China [2][6]. - The company has received approval for three rare disease drugs in China, including treatments for methylmalonic acidemia, isovaleric acidemia, propionic acidemia, and hyperammonemia due to N-acetylglutamate synthase deficiency, as well as drugs for Cushing's syndrome and acromegaly [2][8]. Market Context - The sudden termination of Ruikangdi's operations raises questions about the future availability of the approved rare disease medications in China [2][8]. - In the field of Cushing's syndrome treatment, it is estimated that there are approximately 40,000 to 50,000 patients in China, with only about 3,000 requiring medication treatment [8]. - The market faces challenges due to low awareness of rare diseases, difficulties in diagnosis, and insufficient coverage by health insurance and commercial insurance, making commercialization difficult for rare disease pharmaceutical companies [8][9]. Challenges Faced - The company has acknowledged that the challenges in the rare disease sector are greater than initially anticipated, particularly regarding the limited awareness among patients and doctors [9]. - Significant resources are required for medical education and market cultivation, which are time-consuming and costly [9]. - Many rare disease patients come from economically disadvantaged families, making affordability a major challenge [9].