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阿斯利康Gianluca Pirozzi博士:中国成全球罕见病研发“突破引擎” 高价值药物有望构建商业闭环
Mei Ri Jing Ji Xin Wen· 2025-09-29 14:08
"作为一名医生,同时也是罕见病患儿的父亲,我深知这个群体面临双重困境,既难以确诊,又无药可 治。"日前,阿斯利康全球高级副总裁Gianluca Pirozzi博士在接受《每日经济新闻》记者专访时如是袒 露心声。 统计数据显示,目前已知罕见病超1万种,但90%缺乏有效治疗手段,80%为遗传性疾病且多在儿童期 发病,平均诊断周期长达4.8年。这一严峻现状正在被全球医药巨头与中国市场深度绑定所改变。 首先是临床试验速度与质量领先。Gianluca Pirozzi告诉记者,在阿斯利康的大部分三期临床试验中,中 国都是首个参与方,且患者入组速度全球领先。"中国拥有规模庞大的临床研究中心,能将分散在各地 的患者有效聚集,这解决了罕见病临床试验最大的难题,患者招募。"更重要的是,中国中心提供的数 据质量与全球标准完全接轨,"速度与质量的双重优势,让中国成为药物开发的加速器"。 其次是前沿技术创新与转化领先。在Gianluca Pirozzi看来,中国在细胞治疗、基因治疗、IIT临床研究 (研究者发起的临床研究)等领域已走在全球前列。"这些技术突破不仅能加速本土研发,更能为全球 提供创新思路。"他解释道,技术创新与数据积累形 ...
专访阿斯利康全球高级副总裁Gianluca Pirozzi:中国正成为全球罕见病研发“突破引擎” 高价值药物有望构建商业闭环
Mei Ri Jing Ji Xin Wen· 2025-09-26 04:55
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]
专访阿斯利康全球高级副总裁Gianluca Pirozzi:中国正成为全球罕见病研发“突破引擎”,高价值药物有望构建商业闭环
Mei Ri Jing Ji Xin Wen· 2025-09-26 04:47
每经记者|甄素静 每经编辑|陈俊杰 Gianluca Pirozzi博士表示,全球罕见病诊疗面临诊断难、用药贵的痛点,中国凭借临床试验入组效率、前沿技术创新及疾病数据积累的独特优势,已从全球 研发版图的重要参与者升级为"突破引擎",而针对高致死率疾病的高价值药物策略,正破解罕见病研发的商业可持续性难题。 罕见病定义差异进一步加剧了研发挑战。Gianluca Pirozzi介绍,不同国家和地区对罕见病的认定标准存在显著差异,中国目前尚未出台监管机构明确的罕见 病清单,这对制药企业研发方向选择带来了一定难度。这种差异与政策衔接问题,在世界罕见病保障体系中普遍存在,从国家部门间到地方之间均有体现。 受访者供图 以渐冻症为例,Gianluca Pirozzi指出,这类疾病至今缺乏有效治疗药物,成为医药研发领域的"硬骨头"。 "作为一名医生,同时也是罕见病患儿的父亲,我深知这个群体面临双重困境,既难以确诊,又无药可治。"日前,阿斯利康全球高级副总裁Gianluca Pirozzi 博士在接受《每日经济新闻》记者专访时袒露心声。 万种疾病九成无药,诊断困境凸显医疗缺口 "患者群体分散、疾病机制认知不足、缺乏合适的动物模型 ...
补体研究助力罕见病治疗 有望让更多患者获益
Zhong Guo Jing Ji Wang· 2025-08-28 09:20
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]
中央发文兜底帮扶低收入群体,如何减轻重症肌无力患者负担
Nan Fang Du Shi Bao· 2025-06-17 13:25
Core Viewpoint - The article highlights the challenges faced by patients with myasthenia gravis, a rare autoimmune disease, including misdiagnosis, treatment difficulties, and the need for better healthcare support and awareness [1][4][11]. Group 1: Patient Challenges - Patients with myasthenia gravis often experience symptoms such as sudden weakness, drooping eyelids, and difficulty swallowing, leading to misdiagnosis as psychological issues or other conditions [3][4]. - The prevalence of myasthenia gravis is approximately 7.4 per million people, translating to around 650,000 patients in China due to the large population [4][7]. - Many patients report being misdiagnosed for years, which delays appropriate treatment and exacerbates their condition [3][4]. Group 2: Treatment and Healthcare Support - Current treatments for myasthenia gravis include cholinesterase inhibitors, corticosteroids, and immunosuppressants, but many essential medications are not covered by insurance, leading to high out-of-pocket costs [7][8]. - The new national medical insurance drug list, effective January 1, 2024, includes two medications for myasthenia gravis, but patients still face significant financial burdens for ongoing and crisis treatments [7][8]. - Experts suggest that myasthenia gravis should be included in outpatient chronic disease categories to improve reimbursement rates and access to necessary medications [8][9]. Group 3: Psychological and Social Aspects - The psychological impact of myasthenia gravis is significant, with many patients facing discrimination and social stigma due to visible symptoms like drooping eyelids [11]. - A study indicated that 17.1% of surveyed patients experienced discrimination affecting their work and study opportunities, highlighting the need for greater societal awareness and support [11]. - Mental health support is emphasized as a crucial component of treatment, alongside physical health interventions [11].
股价日内逆市拉升20cm,荣昌生物(09995)何以成为“资金宠儿”?
智通财经网· 2025-04-09 12:15
Core Viewpoint - The recent external environment has intensified, impacting the pharmaceutical sector, with the Hang Seng Healthcare Index experiencing significant fluctuations due to news of potential U.S. tariffs on drugs [1] Company Summary - Rongchang Biopharmaceuticals (09995) demonstrated resilience by surging over 15% within half an hour of trading despite initial market negativity, recovering from a maximum drop of 10.23% [2] - The stock's performance was driven by the announcement of promising results for Telitacicept (RC18) in treating generalized myasthenia gravis (gMG), showing significant clinical improvements compared to placebo [5][6] - Telitacicept treatment led to a 5.74-point reduction in MG-ADL scores and an 8.66-point reduction in QMG scores after 24 weeks, with 98.1% of patients showing significant improvement [5][7] - The safety profile of Telitacicept was comparable to placebo, with a lower incidence of infection-related adverse events [7] Industry Summary - The current market for gMG treatments is competitive, with existing therapies primarily developed by multinational corporations (MNCs) targeting B-cell pathways and complement C5 inhibitors [7] - The global market for gMG is projected to reach $7.24 billion by 2030, with approximately 1.2 million patients worldwide, including 220,000 in China [8] - Recent policy initiatives in China are favoring the development of innovative drugs, with a notable increase in the number of domestic innovative drugs approved from 51 in 2019 to 93 in 2024 [10] - Chinese companies are leading in antibody-drug conjugate (ADC) innovation, holding a significant share of the global pipeline and demonstrating a concentrated focus on key therapeutic targets [11] - The trend towards valuing "source innovation" and "true innovation" in drug development is expected to accelerate the revaluation of companies with strong innovative pipelines in the current market [12]