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市場調查報告書
商品編碼
1874353
PNH 和 aHUS:全球市場佔有率和排名、總收入和需求預測(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 市場規模估計為 25.88 億美元,預計到 2031 年將調整至 23.77 億美元,在預測期(2025-2031 年)內以 2.4% 的複合年成長率成長。
陣發性夜間血尿症(PNH) 和非典型溶血性尿毒症候群 (aHUS) 都是極為罕見的遺傳性疾病。 PNH 患者的紅血球破壞速度異常加快。這是一種後天性造血幹細胞疾病,造血幹細胞在骨髓中產生,最終分化為紅血球、白血球和血小板。 PNH 患者的造血細胞有缺陷,產生異常紅血球。這些異常細胞容易被補體系統過早破壞。 aHUS 是一種全身小血管內形成血栓的疾病。血栓性微血管病變 (TMA) 可導致心臟病發作、中風、腎衰竭甚至死亡。
陣發性夜間血尿症(PNH) 和非典型溶血性尿毒症候群 (aHUS) 的市場成長主要得益於人們對這些罕見且危及生命的血液和腎臟疾病的認知不斷提高,以及診斷能力的進步。分子生物學和基因檢測技術的進步使得早期診斷和更準確的診斷成為可能,從而能夠及時啟動治療,顯著改善了患者的預後。此外,新型標靶治療(例如補體抑制劑Eculizumab和Ravulizumab)的引入和核准,透過提供有效的疾病管理方案,減少溶血、血栓事件和器官損傷,徹底改變了治療模式。研發投入的增加,以及支持性的法規結構和孤兒藥認定,也正在加速創新治療方法的應用。此外,患者權益倡導和教育計畫的增加提高了醫療專業人員對這些疾病的認知,也促進了市場成長。
儘管PNH和aHUS市場前景廣闊,但仍面臨許多挑戰,限制了治療方法的廣泛應用。其中一個主要障礙是治療費用高昂,尤其是補體抑制劑,這會給醫療系統和患者帶來沉重的經濟負擔,尤其是在中低收入國家。高昂的費用限制了藥物的普及和醫療覆蓋,使患者無法獲得救命藥物。此外,這些疾病的罕見性導致患者群體較小,難以進行大規模臨床試驗,也難以收集足夠的長期安全性和有效性數據。另一個挑戰是診斷過程複雜,因為這些疾病的症狀與其他血液系統疾病和腎臟疾病重疊,可能導致診斷延遲或漏診。最後,許多病例需要終身治療,這引發了人們對患者依從性、潛在副作用和抗藥性發展的擔憂,因此需要持續的監測和管理策略。
本報告旨在按地區/國家、類型和應用對全球 PNH 和 aHUS 市場進行全面分析,重點關注總收入、市場佔有率和主要企業的排名。
PNH 和 aHUS 的市場規模、估算和預測均以銷售收入為指標,以 2024 年為基準年,並包含 2020 年至 2031 年的歷史數據和預測數據。報告採用定量和定性分析相結合的方法,幫助讀者制定 PNH 和 aHUS 的業務/成長策略,評估市場競爭,分析自身在當前市場中的地位,並做出明智的商業決策。
市場區隔
公司
按類型分類的細分市場
應用領域
按地區
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.