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市场调查报告书
商品编码
1874353
PNH 和 aHUS:全球市场份额和排名、总收入和需求预测(2025-2031 年)PNH and aHUS - Global Market Share and Ranking, Overall Sales and Demand Forecast 2025-2031 |
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2024 年全球 PNH 和 aHUS 市场规模估计为 25.88 亿美元,预计到 2031 年将调整至 23.77 亿美元,在预测期(2025-2031 年)内以 2.4% 的复合年增长率增长。
阵发性夜间血尿症(PNH) 和非典型溶血性尿毒症候群 (aHUS) 都是极为罕见的遗传性疾病。 PNH 患者的红血球破坏速度异常加快。这是一种后天性造血干细胞疾病,造血干细胞在骨髓中产生,最终分化为红血球、白血球和血小板。 PNH 患者的造血细胞有缺陷,产生异常红血球。这些异常细胞容易被补体系统过早破坏。 aHUS 是一种全身小血管内形成血栓的疾病。血栓性微血管病变 (TMA) 可导致心臟病发作、中风、肾衰竭甚至死亡。
阵发性夜间血尿症(PNH) 和非典型溶血性尿毒症候群 (aHUS) 的市场成长主要得益于人们对这些罕见且危及生命的血液和肾臟疾病的认识不断提高,以及诊断能力的进步。分子生物学和基因检测技术的进步使得早期诊断和更准确的诊断成为可能,从而能够及时启动治疗,显着改善了患者的预后。此外,新型标靶治疗(例如补体抑制剂Eculizumab和Ravulizumab)的引入和核准,透过提供有效的疾病管理方案,减少溶血、血栓事件和器官损伤,彻底改变了治疗模式。研发投入的增加,以及支持性的法规结构和孤儿药认定,也正在加速创新治疗方法的应用。此外,患者权益倡导和教育计画的增加提高了医疗专业人员对这些疾病的认识,也促进了市场成长。
儘管PNH和aHUS市场前景广阔,但仍面临许多挑战,限制了治疗方法的广泛应用。其中一个主要障碍是治疗费用高昂,尤其是补体抑制剂,这会给医疗系统和患者带来沉重的经济负担,尤其是在中低收入国家。高昂的费用限制了药物的普及和医疗覆盖,使患者无法获得救命药物。此外,这些疾病的罕见性导致患者群体较小,难以进行大规模临床试验,也难以收集足够的长期安全性和有效性数据。另一个挑战是诊断过程复杂,因为这些疾病的症状与其他血液系统疾病和肾臟疾病重迭,可能导致诊断延迟或漏诊。最后,许多病例需要终身治疗,这引发了人们对患者依从性、潜在副作用和抗药性发展的担忧,因此需要持续的监测和管理策略。
本报告旨在按地区/国家、类型和应用对全球 PNH 和 aHUS 市场进行全面分析,重点关注总收入、市场份额和主要企业的排名。
PNH 和 aHUS 的市场规模、估算和预测均以销售收入为指标,以 2024 年为基准年,并包含 2020 年至 2031 年的历史数据和预测数据。报告采用定量和定性分析相结合的方法,帮助读者制定 PNH 和 aHUS 的业务/成长策略,评估市场竞争,分析自身在当前市场中的地位,并做出明智的商业决策。
市场区隔
公司
按类型分類的细分市场
应用领域
按地区
The global market for PNH and aHUS was estimated to be worth US$ 2588 million in 2024 and is forecast to a readjusted size of US$ 2377 million by 2031 with a CAGR of 2.4% during the forecast period 2025-2031.
PNH and aHUS, both are extremely rare and genetic diseases. Due to PNH, destruction of red blood cells of a person occurs quite sooner than it should. It is an acquired hematopoietic stem cell disorder. Hematopoietic stem cells are developed in bone marrow and eventually turn into red blood cells, white blood cells and platelets. A person with PNH has some defected hematopoietic cells which create defective red blood cells. These defective cells are highly susceptible to premature destruction by the complement system. aHUS is a disorders in which blood clots are formed in small blood vessels throughout the body. TMA can lead to heart stroke, attack, kidney failure and death.
The market for Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) is primarily driven by increased awareness and improved diagnostic capabilities for these rare, life-threatening hematologic and renal disorders. Advances in molecular biology and genetic testing have enabled earlier and more accurate diagnosis, leading to timely treatment initiation, which significantly improves patient outcomes. Furthermore, the introduction and approval of novel targeted therapies, such as complement inhibitors (e.g., eculizumab and ravulizumab), have revolutionized the treatment landscape by offering effective disease management options that reduce hemolysis, thrombotic events, and organ damage. Growing investment in research and development, coupled with supportive regulatory frameworks and orphan drug designations, also accelerates the availability of innovative therapies. Additionally, increasing patient advocacy and education programs have raised disease recognition among healthcare professionals, contributing to market growth.
Despite the promising growth prospects, the PNH and aHUS market faces several challenges that limit broader access and adoption of therapies. One significant barrier is the high cost of treatment, especially with complement inhibitors, which can impose substantial financial burdens on healthcare systems and patients, particularly in low- and middle-income countries. This cost factor often restricts widespread accessibility and reimbursement, limiting patient access to life-saving drugs. Moreover, the rarity of these diseases results in a small patient population, which poses challenges for conducting large-scale clinical trials and hinders extensive data generation for long-term safety and efficacy. Another challenge is the complexity of disease diagnosis due to overlapping symptoms with other hematological or renal conditions, sometimes causing delayed or missed diagnosis. Lastly, the need for lifelong treatment in many cases raises concerns about patient adherence, potential side effects, and the development of resistance, necessitating continuous monitoring and management strategies.
This report aims to provide a comprehensive presentation of the global market for PNH and aHUS, focusing on the total sales revenue, key companies market share and ranking, together with an analysis of PNH and aHUS by region & country, by Type, and by Application.
The PNH and aHUS market size, estimations, and forecasts are provided in terms of sales revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. With both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding PNH and aHUS.
Market Segmentation
By Company
Segment by Type
Segment by Application
By Region
Chapter Outline
Chapter 1: Introduces the report scope of the report, global total market size. This chapter also provides the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.
Chapter 2: Detailed analysis of PNH and aHUS company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.
Chapter 3: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 4: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 5: Revenue of PNH and aHUS in regional level. It provides a quantitative analysis of the market size and development potential of each region and introduces the market development, future development prospects, market space, and market size of each country in the world.
Chapter 6: Revenue of PNH and aHUS in country level. It provides sigmate data by Type, and by Application for each country/region.
Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product revenue, gross margin, product introduction, recent development, etc.
Chapter 8: Analysis of industrial chain, including the upstream and downstream of the industry.
Chapter 9: Conclusion.