2026 年 02 月 25 日 行业日报 看好/维持 医药 医药 太平洋医药日报（20260225）：酶替代疗法 Loargys 获 FDA 批准 ◼ 走势比较 (10%) (2%) 6% 14% 22% 30% 25/2/25 25/5/9 25/7/21 25/10/2 25/12/14 26/2/25 ◼ 子行业评级 | 化学制药 | 无评级 | | --- | --- | | 中药生产 | 无评级 | | 生物医药Ⅱ | 中性 | | 其 他 医 药 医 | 中性 | | 疗 | | 相关研究报告 <<太平洋医药日报（20260224）： Molbreevi 获 FDA 授予优先审评资 格>>--2026-02-25 <<太平洋医药日报（20260213）：安进 Inebilizumab 获欧盟批准>>--2026- 02-15 <<太平洋医药日报（20260212）：默沙 东 Keytruda 获 FDA 批准新适应症>>- -2026-02-15 证券分析师：周豫 电话： E-MAIL：zhouyua@tpyzq.com 分析师登记编号：S1190523060002 电话： E-MAIL：zha ...

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].

Core Insights - The company Beihai Kangcheng-B (01228) announced that its domestically developed enzyme replacement therapy CAN103 has been included in China's first version of the "Commercial Insurance Innovative Drug Directory," which will be implemented starting January 1, 2026 [1] Group 1: Product Development - CAN103 was approved for market launch on May 13, 2025, making it the first domestically developed enzyme replacement therapy for adolescents aged 12 and older, as well as adults with Type I and Type III Gaucher disease in China [1] - On July 2, 2025, CAN103 was officially prescribed at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine, marking its entry into clinical application [1] Group 2: Market Impact - The inclusion of CAN103 in the insurance directory is considered a milestone achievement for the company, as it ensures that Gaucher disease patients will have access to safe and effective domestic enzyme replacement therapy, providing broader and more comprehensive treatment coverage [1]

Core Viewpoint - The recent research on "AAV9-mediated gene therapy for infantile Pompe disease" led by Professor Feng Zhichun represents a significant breakthrough in treating this rare genetic disorder, potentially offering a "one-shot cure" solution for patients [1][5]. Group 1: Disease Overview - Pompe disease, also known as Glycogen Storage Disease Type II, is a rare autosomal recessive disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to glycogen accumulation in lysosomes, particularly affecting skeletal, cardiac, and smooth muscles [2][3]. - The infantile form of Pompe disease progresses rapidly, often resulting in heart failure and respiratory failure by the age of one if untreated [2]. Group 2: Current Treatment Limitations - Enzyme replacement therapy (ERT) has been the primary treatment since its introduction in 2006, significantly reducing mortality rates and improving quality of life for some patients; however, it does not penetrate the blood-brain barrier and has limitations in addressing central nervous system involvement [3][4]. - ERT requires bi-weekly administration and can be costly, with some patients developing antibodies that reduce treatment efficacy or cause allergic reactions [3]. Group 3: Gene Therapy Development - The newly developed GC301 injection, utilizing AAV9 as a vector to deliver a codon-optimized human GAA gene, aims to provide a long-lasting solution by correcting the genetic defect and restoring GAA enzyme synthesis [4][5]. - Initial trials with four infants showed promising results, with three achieving significant developmental milestones and improvements in cardiac function within a 52-week observation period [5]. Group 4: Safety and Future Directions - Preliminary findings indicate that GC301 is safe, with no severe immune-related adverse reactions reported; however, ongoing assessments are necessary to evaluate long-term safety and efficacy [6]. - Future research may explore the application of GC301 in late-onset Pompe disease and the development of personalized treatment plans based on different genotypes [7].

Core Viewpoint - The approval of domestic enzyme replacement therapy, Gorenin, for Gaucher disease patients in China is expected to significantly reduce treatment costs compared to imported alternatives [1][3][5]. Group 1: Product Overview - Gorenin is the first domestically developed enzyme replacement therapy for type I and III Gaucher disease patients aged 12 and above, fully replacing similar imported products [1][2]. - The drug has been approved for a comprehensive range of indications, covering both type I and III patients, including those who are intolerant or unresponsive to other treatments [2][5]. Group 2: Market Context - The annual treatment costs for imported therapies like imiglucerase can range from $100,000 to $300,000, with domestic adult patients potentially facing costs exceeding 1 million yuan [3][5]. - Approximately 3,000 Gaucher disease patients are reported in China, making it challenging for pharmaceutical companies to justify the investment in research and development for such a small patient population [5][6]. Group 3: Cost Management Strategies - WuXi Biologics, the CRDMO service provider, aims to control the development and production costs of rare disease drugs through innovative technology, allowing for competitive pricing [5][6]. - Gorenin is expected to reduce annual treatment costs for Gaucher disease patients by over 50% compared to imported drugs [3][5]. Group 4: Future Directions - The company plans to push for Gorenin's inclusion in medical insurance systems to ensure broader access for patients [6][7]. - There is a strategic focus on developing international markets for rare disease drugs, leveraging local clinical resources and competitive pricing strategies [7].