这家罕见病药企终止在华业务，已上市的罕见病药物何去何从

Core Viewpoint - The sudden termination of operations by Ruikangdi (Beijing) Pharmaceutical Co., Ltd. highlights the commercial challenges faced by rare disease pharmaceutical companies in China, particularly in terms of market awareness and patient affordability [1][3][4]. Group 1: 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 Chinese patients [1]. - 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 [1]. Group 2: Market Challenges - The termination of Ruikangdi's operations raises questions about the future availability of the approved rare disease drugs in China, especially since there are currently no domestic generic versions available for the drugs phospho-ozagrel and long-acting injection of palmitoyl-β-naphthyl acetate [2][3]. - The estimated number of Cushing's syndrome patients in China is between 40,000 to 50,000, but only about 3,000 patients are likely to require medication, indicating a significant gap in treatment needs [2]. - The commercial difficulties faced by rare disease pharmaceutical companies are exacerbated by low awareness of rare diseases, diagnostic challenges, and insufficient coverage by health insurance and commercial insurance [3]. Group 3: Insights from Management - The general manager of Ruikangdi, Hu Maosheng, acknowledged the significant challenges in the rare disease sector, emphasizing the limited awareness among patients and doctors, which necessitates substantial investment in medical education and market cultivation [4]. - The high costs and time-consuming nature of educating the market about rare diseases pose additional hurdles, particularly as approximately 80% of rare diseases are genetic and many patients come from economically disadvantaged families [4].