深耕罕见病创新药“蓝海” 双核驱动迈向全球创新

Core Insights - The founder and CEO of Beihai Kangcheng, Xue Qun, emphasizes that the rare disease innovative drug market is a "blue ocean within a blue ocean," with significant unmet clinical needs in China [1] - The company has established a diverse product matrix with three approved products and seven in development, aiming to deepen its global innovation layout through a "dual-core drive" strategy [1][5] Group 1: Company Background and Development - Beihai Kangcheng has over 20 years of experience in the rare disease sector, with Xue Qun having spent a decade in the field before founding the company in 2012 [1] - The company initially focused on oncology due to financing challenges but shifted to rare diseases after significant policy changes in 2018, including the release of the first rare disease directory by the government [2] - The company’s three approved products fill multiple clinical gaps, including treatments for Mucopolysaccharidosis Type II and Alagille Syndrome, and the first domestically developed enzyme replacement therapy for Gaucher disease, expected to be approved by May 2025 [2][3] Group 2: Policy Support and Challenges - The high costs associated with rare disease drug development necessitate strong policy support, which has been crucial for overcoming industry challenges [3][4] - The successful launch of the core product, Gorenin, benefited from the national pilot policy for segmented production of biological products, which allows for innovative production methods [4] - Gorenin has also been included in China's first commercial insurance innovation drug directory, set to take effect in January 2026, which could enhance payment channels for innovative drugs [4] Group 3: Strategic Partnerships and Future Plans - Beihai Kangcheng is entering a 3.0 development phase, focusing on consolidating its leadership in the domestic rare disease market while expanding internationally [5][6] - The company has formed a strategic partnership with Baiyang Pharmaceutical to enhance its commercialization efforts, leveraging Baiyang's extensive sales network and financial resources [6][7] - Future plans include integrating rare disease diagnostics with genetic sequencing and patient education to create a synergistic ecosystem, enhancing patient awareness and treatment pathways [7]