此次申报上市，是基于一项在既往未接受过补体抑制剂治疗的PNH患者中进行的关键研究(HRS-5965- 301)。该研究是一项评估HRS-5965胶囊对比依库珠单抗用于治疗既往未接受过补体抑制剂治疗的PNH 患者的Ⅲ期临床研究，由中国医学科学院血液病医院张凤奎教授和中国医学科学院北京协和医院韩冰教 授担任主要研究者，全国共13家中心共同参与，共计76例患者入组用药。研究结果显示，HRS-5965胶 囊在提升PNH患者血红蛋白水平、避免输血、改善疲劳等方面均显着优于依库珠单抗，能明显提高患者 生活质量。 阵发性睡眠性血红蛋白尿症是一种后天获得性溶血性疾病，其特征为CD55(衰变加速因子，DAF)和 CD59(反应性溶解膜抑制物，MIRL)缺乏导致补体介导的血管内溶血，主要临床表现为血管内溶血、潜 在造血功能衰竭及血栓形成倾向[1]。因其发病率/患病率低，PNH已被纳入国家《第一批罕见病目录》 [2]。HRS5965胶囊是一种补体因子B抑制剂，可抑制补体介导的血管内外溶血反应，提升血红蛋白水 平。针对本适应症，国内外同靶点药物目前仅有诺华的盐酸伊普可泮胶囊(Fabhalta)获批上市，经查询 EvaluatePha ...

阵发性睡眠性血红蛋白尿症是一种后天获得性溶血性疾病，其特征为CD55(衰变加速因子，DAF)和 CD59(反应性溶解膜抑制物，MIRL)缺乏导致补体介导的血管内溶血，主要临床表现为血管内溶血、潜 在造血功能衰竭及血栓形成倾向。因其发病率/患病率低，PNH已被纳入国家《第一批罕见病目录》。 HRS5965胶囊是一种补体因子B抑制剂，可抑制补体介导的血管内外溶血反应，提升血红蛋白水平。针 对本适应症，国内外同靶点药物目前仅有诺华的盐酸伊普可泮胶囊(Fabhalta)获批上市，经查询 EvaluatePharma数据库，2024年该产品全球销售额约为1.29亿美元。截至目前，HRS-5965胶囊相关项目 累计研发投入约2.18亿元。 智通财经APP讯，恒瑞医药(01276)发布公告，近日，江苏恒瑞医药股份有限公司子公司成都盛迪医药有 限公司收到国家药品监督管理局下发的《受理通知书》，公司富马酸立康可泮(HRS-5965)胶囊的药品上 市许可申请获国家药监局受理。 此次申报上市，是基于一项在既往未接受过补体抑制剂治疗的PNH患者中进行的关键研究(HRS-5965- 301)。该研究是一项评估HRS-5965胶囊对比依 ...

（来源：市场星报） 历经儿童肾脏科及多学科团队52天的精心治疗与守护，患儿一步步渡过难关，病情稳步改善，最终康复 出院。 邓芳提醒家长：如果孩子出现不明原因的眼睑或下肢浮肿、尿色加深（如酱油色）、尿量减少、面色苍 白、乏力等情况，一定要及时就医，警惕急性肾功能不全及潜在罕见病的可能。早诊断、早治疗至关重 要。 星报讯 1月7日，记者获悉，近日，7岁男孩佑佑（化名）因"双眼睑、双腿浮肿4天"被紧急送至国家儿 童区域医疗中心、安徽省儿童医院（安徽省妇幼保健院）儿童肾脏科。 据佑佑妈妈回忆，孩子起初只是眼皮和腿有些肿，短短几天内情况急转直下，出现酱油色小便、尿量减 少、浑身乏力等症状。 入院后，儿童肾脏科团队迅速展开救治，一边监测生命体征，一边完成各项检查。结果提示，患儿血肌 酐、尿素氮显著升高，伴有肾小球源性血尿和蛋白尿，已发生急性肾功能不全，且病情进展极快。面对 持续恶化的肾功能，科室当机立断启动肾脏替代治疗，通过血液净化清除体内毒素、纠正内环境紊乱， 为后续诊断争取了宝贵时间。 待患儿情况稍稳定，团队为其进行了肾穿刺活检。病理结果显示：继发性IgA肾病，伴急性肾间质损伤 及肾小管坏死。在主任医师邓芳的指导下， ...

公布新增纳入目录的114种药品，是非小细胞肺癌、宫颈癌、罕见病的患者在等待的创新药、救命药。 比如说有一款药，打一针可维持半年有效的降血脂的效果，上市不久就能纳入医保，患者可以以非常合 理的价格控制血脂，这对患者来说，就是民生。 专题：新浪健康保险研究院 来源：中国医疗保险 2025年12月7日，国家医保局公布了2025年版《国家基本医疗保险、生育保险和工伤保险药品目录》和 首版《商业健康保险创新药品目录》。新公布的医保药品目录有什么新变化？又如何更好地满足大家的 用药需求呢？ 本次目录调整新增114种药品，其中50种为一类创新药，同时调出了29种临床没有供应或可被其他药物 更好替代的药品。本次调整后，目录内药品总数增至3253种，其中西药1857种、中成药1396种，肿瘤、 慢性病、精神疾病、罕见病、儿童用药等重点领域的保障水平得到明显提升。新版目录于2026年1月1日 在全国范围内正式实施。 2025年的药品目录调整与此前有哪些不同？ 而人们对于健康的不懈追求，一方面推动着药品日新月异的发展，同时也要求政策的调整，要跟得上行 业的变化。所以医保局成立以来，每年一次药品目录的调整，都是对民生需求的回应。 不 ...

炒股就看金麒麟分析师研报，权威，专业，及时，全面，助您挖掘潜力主题机会！ （来源：医学界） 转自：医学界 重症肌无力（MG）是一种由自身抗体介导、获得性的神经肌肉接头（NMJ）疾病，其核心特征是骨骼 肌无力和易疲劳性[1]。当肌无力症状超出眼外肌范围，累及到球部（如吞咽、构音）、四肢和躯干肌 肉时，即称为全身型重症肌无力（ MG），gMG约占所有MG病例的85%，是MG中最常见的亚型[2]。 此类患者在临床上常常面临哪些治疗困境？随着当前研究进展又有哪些破局之法？针对这些问题，医学 界有幸邀请到首都医科大学宣武医院笪宇威教授就gMG治疗困境、MG指南更新及gMG治疗新策略等方 面进行了深度分享。 起效慢、不良反应明显：传统治疗方案局限凸显靶向治疗需求 针对gMG治疗困境，笪宇威教授指出，尽管大部分患者在常规非特异性免疫治疗（激素和非激素类免 疫抑制剂）下可明显改善症状，但这些药物存在明显的局限性。首当其冲的是长期口服糖皮质激素所带 来的不良反应，激素治疗的并发症会进一步增加疾病治疗的负担，同时有10%~20%的患者对常规免疫 治疗药物反应有限，不能耐受药物不良反应或存在激素禁忌症的患者会陷入治疗困境，从而发展 ...

12月7日，国家医保局公布了2025年版《国家基本医疗保险、生育保险和工伤保险药品目录》和首版 《商业健康保险创新药品目录》。新公布的医保药品目录有什么新变化？又如何更好地满足大家的用药 需求呢？ 本次目录调整新增114种药品，其中50种为一类创新药，同时调出了29种临床没有供应或可被其他药物 更好替代的药品。本次调整后，目录内药品总数增至3253种，其中西药1857种、中成药1396种，肿瘤、 慢性病、精神疾病、罕见病、儿童用药等重点领域的保障水平得到明显提升。新版目录将于2026年1月1 日在全国范围内正式实施。 今年的药品目录调整与此前有哪些不同？ 公布新增纳入目录的114种药品，是非小细胞肺癌、宫颈癌、罕见病的患者在等待的创新药、救命药。 比如说今年有一款药，打一针可维持半年有效的降血脂的效果，上市不久就能纳入医保，患者可以以非 常合理的价格控制血脂，这对患者来说，就是民生。 而人们对于健康的不懈追求，一方面推动着药品日新月异的发展，同时也要求政策的调整，要跟得上行 业的变化。所以医保局成立以来，每年一次药品目录的调整，都是对民生需求的回应。 不断纳入新药 医保基金能承受吗？ 八年八次调整，每次都新增 ...

Core Insights - The article highlights the dual challenges faced by rare disease patients, including difficulties in diagnosis and lack of effective treatments, with over 90% of known rare diseases lacking any therapeutic options [1][2] - China is evolving from a participant to a "breakthrough engine" in the global rare disease research landscape, leveraging its unique advantages in clinical trial efficiency, technological innovation, and disease data accumulation [1][3] Diagnosis Challenges - The number of known rare diseases has increased from 7,000 to over 10,000 due to advancements in gene sequencing, yet approximately 90% still lack treatment [2] - 80% of rare diseases are genetic and often manifest in childhood, making pediatric rare disease drug development a pressing and complex task [2] - Variations in the definition of rare diseases across countries complicate research efforts, with China lacking a clear regulatory list of rare diseases [2] China's Role in Rare Disease Research - China is recognized for its leading clinical trial speed and quality, with many Phase III trials involving Chinese participants [3] - The country is at the forefront of technological innovations in cell and gene therapies, which can accelerate local research and provide global insights [3] - China's healthcare system allows for efficient disease data collection, crucial for understanding disease progression and treatment endpoints [3] Collaborations and Partnerships - AstraZeneca has established collaborations with top Chinese hospitals to create disease excellence centers focusing on rare kidney and neurological diseases [4] - The company has engaged in acquisitions and partnerships with 14 Chinese biotech firms to enhance its rare disease research capabilities [5] Strategic Focus on Rare Diseases - AstraZeneca's commitment to rare disease research is driven by the potential for transformative therapies that meet significant unmet needs, despite the high costs and small patient populations [5][6] - The company has developed drugs that can significantly reduce mortality rates in conditions like hemolytic uremic syndrome, emphasizing the clinical value of these treatments [6] Pipeline and Future Outlook - AstraZeneca has seven approved rare disease drugs and ten ongoing Phase III clinical trials across various therapeutic areas [6] - The year 2025 is anticipated to be significant for the release of numerous rare disease research outcomes [7] Patient-Centric Approach - AstraZeneca involves patients and caregivers early in the research process to ensure that clinical trials address the most critical disease improvement metrics [8] - The company collaborates with global patient associations to enhance patient engagement and communication with regulatory bodies [8] Accessibility and Affordability - AstraZeneca has three innovative rare disease drugs approved in China, with some included in the national medical insurance catalog, improving patient access [9] - The company advocates for the establishment of rare disease-specific funding to alleviate patient financial burdens, recognizing that insurance alone cannot cover all costs [9] - Optimism is expressed regarding the commercial prospects in China as policies improve and more rare disease drugs are included in insurance coverage [9]

Core Insights - The article highlights the dual challenges faced by rare disease patients, including difficulties in diagnosis and lack of effective treatments, with over 90% of known rare diseases lacking any therapeutic options [2][3] - China is evolving from a participant in global rare disease research to a "breakthrough engine," leveraging its unique advantages in clinical trial efficiency, technological innovation, and disease data accumulation [2][4] Rare Disease Landscape - There are over 10,000 known rare diseases, with 90% lacking effective treatments and 80% being genetic, often manifesting in childhood, making pediatric rare disease drug development urgent yet complex [3][4] - The definition of rare diseases varies significantly across countries, complicating research and development efforts, particularly in China, where a clear regulatory list of rare diseases is still absent [3][4] China's Role in Rare Disease Research - China is recognized for its leading clinical trial speed and quality, with many Phase III trials involving Chinese participants, addressing the recruitment challenges faced in rare disease studies [4][5] - The country is at the forefront of technological innovations in cell and gene therapies, which not only accelerate local research but also provide innovative ideas for global applications [5][6] - China's healthcare system allows for efficient collection of disease registry data, crucial for understanding disease progression and setting treatment endpoints [5][6] AstraZeneca's Strategy in Rare Diseases - AstraZeneca has established partnerships with 14 Chinese biopharmaceutical companies to enhance its rare disease research, focusing on unmet clinical needs and differentiated project advantages [6][7] - The company emphasizes the importance of developing transformative therapies for rare diseases, which can create significant clinical value and support sustainable business models [7][8] Clinical Developments and Future Outlook - AstraZeneca has received approval for seven rare disease drugs globally and has ten ongoing Phase III clinical and regulatory review projects across various therapeutic areas [8][9] - The year 2025 is anticipated to be significant for AstraZeneca's rare disease research, with key Phase III study results expected to be released [9][10] - AstraZeneca is actively building a diagnostic and treatment ecosystem in China, having established 138 rare disease treatment centers to ensure standardized care [9][10] Patient Engagement and Accessibility - The company involves patients and caregivers early in the research process to ensure that clinical trials address the most critical disease improvement indicators [10] - AstraZeneca is advocating for the inclusion of rare disease drugs in national insurance programs to enhance accessibility for patients, while also calling for the establishment of special funds to alleviate patient financial burdens [10]

Core Insights - The article highlights the dual challenges faced by rare disease patients, including difficulties in diagnosis and lack of effective treatments, with over 90% of known rare diseases having no available therapies [1][3] - China is evolving from a participant to a "breakthrough engine" in the global rare disease research landscape, leveraging its unique advantages in clinical trial efficiency, technological innovation, and disease data accumulation [1][4] Group 1: Rare Disease Landscape - There are over 10,000 known rare diseases, with 90% lacking effective treatments and 80% being genetic, often manifesting in childhood, leading to urgent and complex pediatric drug development needs [3][4] - The definition of rare diseases varies significantly across countries, complicating research and development efforts, particularly in China, which currently lacks a clear regulatory list of rare diseases [3][4] - The challenges in rare disease research include dispersed patient populations, insufficient understanding of disease mechanisms, and a lack of suitable animal models and biomarkers [3][4] Group 2: China's Role in Rare Disease Research - China has become a leader in clinical trial speed and quality, with many of AstraZeneca's Phase III trials involving rapid patient recruitment from its extensive clinical research centers [4][5] - The country is at the forefront of technological innovations in cell and gene therapies, which not only accelerate local research but also provide innovative ideas for global applications [5][6] - China's healthcare system allows for efficient collection of disease registry data, crucial for understanding disease progression and setting treatment endpoints [5][6] Group 3: AstraZeneca's Strategy and Collaborations - AstraZeneca has engaged in numerous collaborations with Chinese biopharmaceutical companies, focusing on unmet clinical needs and differentiated project advantages [6][7] - The company emphasizes the importance of developing transformative therapies for rare diseases, which can create significant clinical value and support sustainable business models [7][8] - AstraZeneca has approved seven rare disease drugs globally and has ten ongoing Phase III clinical and regulatory review projects across various therapeutic areas [7][8] Group 4: Future Outlook and Patient Engagement - AstraZeneca anticipates a significant release of rare disease research results in 2025, with ongoing studies in hemophilia and other conditions involving Chinese patients [8][9] - The company is actively building a diagnostic and treatment ecosystem in China, having established 138 rare disease treatment centers to ensure standardized care [8][9] - AstraZeneca involves patients and caregivers early in the research process to better understand disease improvement metrics and streamline clinical trial participation [9] Group 5: Access and Affordability Challenges - AstraZeneca has three innovative rare disease drugs approved in China, with some included in the national medical insurance catalog, enhancing patient access [9] - The company advocates for the establishment of special funds for rare diseases to alleviate patient financial burdens, recognizing that insurance alone cannot cover all high-value innovative drugs [9] - AstraZeneca remains optimistic about the commercial prospects in China, anticipating that improved policies and payment systems will unlock market potential [9]

Group 1 - The complement system is a major component of innate immunity, and its rapid development is transforming clinical practices in various fields such as hematology, neurology, and nephrology [1] - Significant advancements in the understanding and treatment of rare diseases like Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), generalized Myasthenia Gravis (gMG), and Neuromyelitis Optica Spectrum Disorders (NMOSD) have been made, with breakthrough therapies included in medical insurance [1] - Several complement inhibitors have been approved for use in China, with multiple projects in the research phase, indicating a growing application of these treatments in more diseases [1] Group 2 - AstraZeneca's acquisition of Alexion for $39 billion at the end of 2020 marked its entry into the rare disease sector, with its core product, eculizumab, being the first approved C5 complement inhibitor globally [2] - AstraZeneca plans to advance nearly 10 new products or indications in the rare disease field in China over the next five years, focusing on making medications accessible to all patients in need [2] - The company suggests the establishment of a rare disease special fund by the government to alleviate patient financial burdens and proposes leveraging international experiences to broaden funding channels for long-term medication support [2]