把天价药打下来，这个海归救了无数家庭

Core Viewpoint - The article discusses the journey of Beihai Kangcheng and its founder Xue Qun, highlighting the company's resilience in the face of stock price declines and its recent success with the launch of the innovative drug for Gaucher disease, which aims to make treatment more accessible for patients [2][4][6]. Company Overview - Beihai Kangcheng's stock price fell from 12.18 HKD at its IPO in December 2021 to a low of 0.095 HKD in January 2025, resulting in a market value loss of over 99% [4]. - The company has successfully developed and launched its first self-researched drug, Velaglucerase beta injection (Gorainin), which received approval in May 2025 [6][23]. Market Context - There are approximately 3,000 patients with Gaucher disease in China, a rare genetic disorder that has high treatment costs, making it unaffordable for many families [4][10]. - The annual treatment cost for imported drugs ranges from 1.5 million to 2.5 million RMB, leading to a situation where many patients are unable to access necessary medications [4][11]. Drug Development and Approval - Gorainin is expected to reduce annual treatment costs by at least 50%, making it more accessible to patients [6][10]. - In December 2025, Gorainin was included in China's first commercial health insurance innovation drug directory, marking a significant milestone for rare disease treatment in the country [9][10]. Financial Implications - The inclusion of Gorainin in the commercial insurance directory allows for a sustainable business model, where patients will only need to pay a small portion of the treatment costs (20%-50%) after insurance reimbursement [10][12]. - If 10% of the 3,000 Gaucher disease patients in China use Gorainin, the company can achieve sustainable operations with an estimated annual treatment cost of around 700,000 RMB [12][14]. Industry Challenges and Opportunities - The article emphasizes the need for a long-term payment mechanism for rare disease treatments, as many existing drugs still have high out-of-pocket costs for patients [12][14]. - The rare disease market in China is projected to grow significantly, with estimates suggesting it could reach 25.9 billion USD by 2030, representing about 7% of the global market [19]. Strategic Vision - Xue Qun views rare diseases as a critical area for innovation in biomedicine, with the potential to pave the way for treatments for more common diseases [16][20]. - The company aims to create a platform for developing multiple rare disease drugs, leveraging enzyme replacement therapy (ERT) technology to reduce costs and improve accessibility [20][21]. Conclusion - Beihai Kangcheng's journey reflects the challenges and potential of the rare disease market in China, with a focus on innovation, accessibility, and sustainable business practices [23][31].