据患者支持组织欧洲血友病联盟称，该患者于12月14日因中风后脑出血死亡。 该患者参与的长期研究涉及辉瑞一款血友病长效凝血因子疗法Hympavzi，该药物每周注射一次，去 年获得美国批准，用于治疗12岁及以上A型或B型血友病患者，通过靶向凝血蛋白来预防或减少出 血。 对于患者的死亡，辉瑞在一份声明中回应称："辉瑞与试验研究者和独立的外部数据监测委员会一 起，正在积极收集信息，以更好地了解围绕这一事件的复杂、多因素的情况。" 2025.12. 24 本文字数：739，阅读时长大约2分钟 作者 | 第一财经 钱童心 当地时间12月23日，辉瑞公司表示，一名参与了该公司血友病药物长期研究的患者在出现严重副作 用后死亡。辉瑞当天股价收盘下跌超过1%。 由于血友病患者负责调节凝血因子的基因存在缺陷，会导致患者在受伤或手术后出现自发性严重出 血。除了长效凝血因子疗法之外，近年来基因药物为血友病的治疗提供了另一种选择。 （注：我们会对线索进行核实。您的隐私将严格保密。） 推荐阅读 史上最大的IPO要来了？ 此前，辉瑞公司开发了一种针对中重度B型血友病成人患者治疗的一次性基因疗法Beqvez，并在美 国获得批准上市。然而，此类 ...

公司还表示，根据其目前的了解和迄今为止收集的总体临床数据，辉瑞预计患者的死亡事件不会对接受 该药物治疗的患者的安全性产生任何影响。 据患者支持组织欧洲血友病联盟称，该患者于12月14日因中风后脑出血死亡。 该患者参与的长期研究涉及辉瑞一款血友病长效凝血因子疗法Hympavzi，该药物每周注射一次，去年 获得美国批准，用于治疗12岁及以上A型或B型血友病患者，通过靶向凝血蛋白来预防或减少出血。 对于患者的死亡，辉瑞在一份声明中回应称："辉瑞与试验研究者和独立的外部数据监测委员会一起， 正在积极收集信息，以更好地了解围绕这一事件的复杂、多因素的情况。" 根据目前的了解和迄今为止收集的总体临床数据，辉瑞预计不会对接受该药物治疗的患者的安全性产生 任何影响。 当地时间12月23日，辉瑞公司表示，一名参与了该公司血友病药物长期研究的患者在出现严重副作用后 死亡。辉瑞当天股价收盘下跌超过1%。 由于血友病患者负责调节凝血因子的基因存在缺陷，会导致患者在受伤或手术后出现自发性严重出血。 除了长效凝血因子疗法之外，近年来基因药物为血友病的治疗提供了另一种选择。 此前，辉瑞公司开发了一种针对中重度B型血友病成人患者治疗的一次性 ...

Core Insights - Novo Nordisk has submitted applications to the FDA for the approval of icodec insulin for the treatment of type 2 diabetes and Mim8 for the treatment of hemophilia A [1] Group 1: Icodec Insulin - Icodec insulin is a long-acting insulin formulation designed based on oral insulin OI338, with a half-life of 196 hours [2] - The design modifications include replacing an 18C fatty acid with a 20C fatty acid to enhance binding affinity to human serum albumin, and substituting Tyr with His at position 16 of the B chain to reduce affinity to human insulin receptors [2] - The initial BLA submission in April 2023 included indications for both type 1 and type 2 diabetes, but the FDA advisory committee found insufficient data for type 1 diabetes, leading to a narrowed indication for type 2 diabetes in the resubmission [2] Group 2: Mim8 - Mim8 is a bispecific antibody developed using Genmab's DuoBody technology, designed to mimic the action of coagulation factor VIIIa (FVIIIa) and bridge factors IXa (FIXa) and X (FX) [3] - It exhibits approximately 15 times the coagulation activity compared to emicizumab [3] - Hemophilia A, affecting around 80%-85% of the estimated 1.125 million hemophilia patients globally, is caused by a deficiency or defect in FVIII [3] Group 3: Hemophilia Treatment Landscape - There are currently 46 approved hemophilia drugs globally, with only 8 being non-factor therapies [4] - Among these, several drugs have been approved specifically for hemophilia A patients, including concizumab, valoctocogene roxaparvovec, emicizumab, marstacimab, and fitusiran [4]

Core Viewpoint - The article highlights the challenges faced by hemophilia patients in accessing necessary medication due to tightening insurance reimbursement policies and the stigma associated with their condition, which has led to increased difficulties in obtaining treatment and maintaining a normal life [8][12][36]. Group 1: Patient Experiences - Patients like Xiang Huan experience significant pain and difficulty in accessing treatment, often spending hours in hospitals to obtain necessary medications [4][10]. - The tightening of reimbursement policies has made it increasingly difficult for patients to receive their required doses of clotting factors, leading to a cycle of emergency treatments rather than preventive care [8][9]. - Many patients report that they are now required to provide extensive documentation and proof of their condition to obtain medication, which was not previously necessary [10][12]. Group 2: Policy Changes and Impacts - Recent changes in insurance policies have restricted the reimbursement of clotting factors to only when patients are actively bleeding, which is a shift from previous practices that allowed for more flexible access [8][9]. - In regions like Zhejiang, stricter regulations have been implemented, resulting in significant savings for insurance funds but at the cost of patient access to necessary treatments [12][16]. - The article notes that the average treatment cost for hemophilia patients is around 276,000 yuan per year, with some patients needing up to 700,000 yuan to achieve international treatment standards [36][37]. Group 3: Stigma and Fraud Concerns - The article discusses how recent fraud cases involving hemophilia patients have led to increased scrutiny and stigma against the entire patient community, affecting their relationships with healthcare providers [17][18]. - Patients feel that they are being unfairly labeled as potential fraudsters, which has changed the dynamics of their interactions with medical professionals [17][20]. - The fear of being accused of fraud has made it more challenging for patients to advocate for their treatment needs, further complicating their healthcare journey [18][20]. Group 4: Economic Pressures - The financial burden of hemophilia treatment is significant, with many patients relying on insurance and charity to cover costs, leading to a precarious situation where any changes in policy can have dire consequences [36][42]. - The article emphasizes that the high costs of treatment and the uneven distribution of insurance coverage across regions exacerbate the difficulties faced by patients, particularly in less economically developed areas [37][38]. - Patients often resort to participating in clinical trials as a means to access necessary medications, highlighting the desperation and lack of options available to them [32][33].

Summary of the Conference Call for Shuyou Shen Company Overview - Shuyou Shen focuses on infectious diseases, respiratory and critical care, autoimmune diseases, and neurological disorders [2][6] - The company has faced revenue pressure due to declining sales of its main products, Sutai Sheng and Shuyou Qing, but has a rich pipeline of innovative products that present growth potential [2][6] Key Products and Market Potential - **Bomeipide Enzyme Alpha**: A new therapy for hemophilia, showing superior hemostatic efficiency, safety, and onset speed compared to existing therapies. It is expected to become a best-in-class option [2][7][9] - Clinical data indicates a hemostatic rate and onset speed better than existing treatments [2][9] - Anticipated peak sales could exceed 2 billion RMB, with its market application already accepted [2][9] - **C5A Pathway Drugs (001 and 1,002)**: Targeting ANCA-associated vasculitis (AAV) and acute respiratory distress syndrome (ARDS) [2][5][10] - 1,002 is an upgraded version of 001, with promising clinical trial progress [2][5] - The ARDS market is significant, with approximately 3 million new patients annually and high mortality rates [4][16] - The global market for ARDS treatments is largely unmet, presenting a blue ocean opportunity [4][16] Financial Performance and R&D Progress - The company has seen a decline in revenue since 2022 due to decreased sales of existing products and increased R&D expenses [6] - Despite financial pressures, the extensive R&D pipeline, including Bomeipide Enzyme Alpha and C5A pathway drugs, is expected to drive future growth [6][17] Competitive Landscape - The global hemophilia treatment market is approximately 30 billion RMB, with major competitors including recombinant factor VII, PCC, and emicizumab [3][8] - Bomeipide Enzyme Alpha is positioned as a first-in-class product with a unique mechanism, potentially capturing a significant market share due to its advantages over existing therapies [3][9][12] Commercialization Strategies - Bomeipide Enzyme Alpha's commercialization strategy may involve high pricing to target reimbursable patients or competitive pricing to expand market share [4][12] - The overseas market for hemophilia treatments is estimated at nearly 3 billion USD, with potential for market share capture through licensing [12][14] Clinical Trial Insights - The clinical trial data for 1,002 in ARDS has shown excellent results, indicating a strong potential for market entry [5][15] - The company is also exploring multiple indications for its products, enhancing its R&D pipeline diversity [17] Investor Concerns - Investors are particularly interested in the development of 1,002 and its potential patient population size, with expectations that it will serve a broader range of patients than initially thought [18][19] Conclusion - Shuyou Shen is positioned in a promising market with innovative products that address significant unmet medical needs. The company's strategic focus on R&D and potential market capture strategies could lead to substantial growth in the coming years [2][4][6]