Total: £ 56.28
Published Date: 2025-06-19 | Pages: 70 | Tables: 67 | Medical Care
The global market for PNH and aHUS was valued at US$ 2588 million in the year 2024 and is projected to reach a revised size of US$ 2377 million by 2031, growing at a CAGR of 2.4% during the forecast period.
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, 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.
The PNH and aHUS market size, estimations, and forecasts are provided in terms of and revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. This report segments the global PNH and aHUS market comprehensively. Regional market sizes, concerning products by Type, by Application, and by players, are also provided.
For a more in-depth understanding of the market, the report provides profiles of the competitive landscape, key competitors, and their respective market ranks. The report also discusses technological trends and new product developments.
The report will help the PNH and aHUS companies, new entrants, and industry chain related companies in this market with information on the revenues for the overall market and the sub-segments across the different segments, by company, by Type, by Application, and by regions.
Market Segmentation
By Company
AstraZeneca
Alexion Pharmaceutical
CJSC Generium
Segment by Type
Soliris
Ultomiris
Segment by Application
PNH
aHUS
By Region
North America
United States
Canada
Asia-Pacific
China
Japan
South Korea
Southeast Asia
India
Australia
Rest of Asia
Europe
Germany
France
U.K.
Italy
Russia
Nordic Countries
Rest of Europe
Latin America
Mexico
Brazil
Rest of Latin America
Middle East & Africa
Turkey
Saudi Arabia
UAE
Rest of MEA
Chapter Outline
Chapter 1: Introduces the report scope of the report, executive summary of different market segments (by Type, by Application, etc), including the market size of each market segment, future development potential, and so on. It offers a high-level view of the current state of the market and its likely evolution in the short to mid-term, and long term.
Chapter 2: Introduces executive summary of global market size, regional market size, this section also introduces the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by companies in the industry, and the analysis of relevant policies in the industry.
Chapter 3: Detailed analysis of PNH and aHUS company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.
Chapter 4: 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 5: 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 6, 7, 8, 9, 10: North America, Europe, Asia Pacific, Latin America, Middle East and Africa segment by country. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space, and capacity of each country in the world.
Chapter 11: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product sales, revenue, price, gross margin, product introduction, recent development, etc.
Chapter 12: The main points and conclusions of the report.
Research Methodology
The research methodology employed has been subjected by numerous procedures in order to guarantee the quality and accuracy of the data contained within the reports. The analysts are employed full-time and received more than six months training to satisfy the standard of our company. Our methodology can be divided into five stages:
★Stage 1 SECONDARY RESEARCH
The research team first collaborates with magazines, trade associations and administrative departments in the research field. The information provided by our internal documentation service is helpful for our further research. Our team has a wealth of experience and knowledge, and can effectively extract accurate information from existing resources.
★Stage 2 PRIMARY RESEARCH:INTERVIEWS WITH TRADE SOURCES
After the first stage, the research team conducts a large number of face-to-face or telephone interviews with representative companies working in the research field. Analysts are trying to have an opportunity to talk to leading companies and small companies in the field. Upstream suppliers, manufacturers, distributors, importers, installers, wholesalers and consumers were included in the interview. The data collected during the interview were then carefully examined and compared with the secondary study.
★Stage 3 ANALYSIS OF THE GATHERED DATA
The analysis team examines and synthesizes the data collected in the first two stages. In order to validate the data, a second round of interviews can be conducted.
★Stage 4 QUANTITATIVE DATA
The quantitative data such as market estimates, production and capacity of manufacturer, market forecasts and investment feasibility is provided by our company. The data is based on the estimates obtained during stage 3.
★Stage 5 QUALITY CONTROL
Before publishing, each report undergoes a rigorous review and editing process, which is done by the experience management team to ensure the reliability of the published data. Every analyst on the research team receives support and continuous training as part of our internal quality process.
1 Report Overview
1.1 Study Scope
1.2 Market Analysis by Type
1.2.1 Global PNH and aHUS Market Size Growth Rate by Type: 2020 VS 2024 VS 2031
1.2.2 Soliris
1.2.3 Ultomiris
1.3 Market by Application
1.3.1 Global PNH and aHUS Market Growth by Application: 2020 VS 2024 VS 2031
1.3.2 PNH
1.3.3 aHUS
1.4 Assumptions and Limitations
1.5 Study Objectives
1.6 Years Considered
2 Global Growth Trends
2.1 Global PNH and aHUS Market Perspective (2020-2031)
2.2 Global PNH and aHUS Growth Trends by Region
2.2.1 Global PNH and aHUS Market Size by Region: 2020 VS 2024 VS 2031
2.2.2 PNH and aHUS Historic Market Size by Region (2020-2025)
2.2.3 PNH and aHUS Forecasted Market Size by Region (2026-2031)
2.3 PNH and aHUS Market Dynamics
2.3.1 PNH and aHUS Industry Trends
2.3.2 PNH and aHUS Market Drivers
2.3.3 PNH and aHUS Market Challenges
2.3.4 PNH and aHUS Market Restraints
3 Competition Landscape by Key Players
3.1 Global Top PNH and aHUS Players by Revenue
3.1.1 Global Top PNH and aHUS Players by Revenue (2020-2025)
3.1.2 Global PNH and aHUS Revenue Market Share by Players (2020-2025)
3.2 Global Top PNH and aHUS Players by Company Type and Market Share by Company Type (Tier 1, Tier 2, and Tier 3)
3.3 Global Key Players Ranking by PNH and aHUS Revenue
3.4 Global PNH and aHUS Market Concentration Ratio
3.4.1 Global PNH and aHUS Market Concentration Ratio (CR5 and HHI)
3.4.2 Global Top 10 and Top 5 Companies by PNH and aHUS Revenue in 2024
3.5 Global Key Players of PNH and aHUS Head office and Area Served
3.6 Global Key Players of PNH and aHUS, Product and Application
3.7 Global Key Players of PNH and aHUS, Date of Enter into This Industry
3.8 Mergers & Acquisitions, Expansion Plans
4 PNH and aHUS Breakdown Data by Type
4.1 Global PNH and aHUS Historic Market Size by Type (2020-2025)
4.2 Global PNH and aHUS Forecasted Market Size by Type (2026-2031)
5 PNH and aHUS Breakdown Data by Application
5.1 Global PNH and aHUS Historic Market Size by Application (2020-2025)
5.2 Global PNH and aHUS Forecasted Market Size by Application (2026-2031)
6 North America
6.1 North America PNH and aHUS Market Size (2020-2031)
6.2 North America PNH and aHUS Market Growth Rate by Country: 2020 VS 2024 VS 2031
6.3 North America PNH and aHUS Market Size by Country (2020-2025)
6.4 North America PNH and aHUS Market Size by Country (2026-2031)
6.5 United States
6.6 Canada
7 Europe
7.1 Europe PNH and aHUS Market Size (2020-2031)
7.2 Europe PNH and aHUS Market Growth Rate by Country: 2020 VS 2024 VS 2031
7.3 Europe PNH and aHUS Market Size by Country (2020-2025)
7.4 Europe PNH and aHUS Market Size by Country (2026-2031)
7.5 Germany
7.6 France
7.7 U.K.
7.8 Italy
7.9 Russia
7.10 Nordic Countries
8 Asia-Pacific
8.1 Asia-Pacific PNH and aHUS Market Size (2020-2031)
8.2 Asia-Pacific PNH and aHUS Market Growth Rate by Region: 2020 VS 2024 VS 2031
8.3 Asia-Pacific PNH and aHUS Market Size by Region (2020-2025)
8.4 Asia-Pacific PNH and aHUS Market Size by Region (2026-2031)
8.5 China
8.6 Japan
8.7 South Korea
8.8 Southeast Asia
8.9 India
8.10 Australia
9 Latin America
9.1 Latin America PNH and aHUS Market Size (2020-2031)
9.2 Latin America PNH and aHUS Market Growth Rate by Country: 2020 VS 2024 VS 2031
9.3 Latin America PNH and aHUS Market Size by Country (2020-2025)
9.4 Latin America PNH and aHUS Market Size by Country (2026-2031)
9.5 Mexico
9.6 Brazil
10 Middle East & Africa
10.1 Middle East & Africa PNH and aHUS Market Size (2020-2031)
10.2 Middle East & Africa PNH and aHUS Market Growth Rate by Country: 2020 VS 2024 VS 2031
10.3 Middle East & Africa PNH and aHUS Market Size by Country (2020-2025)
10.4 Middle East & Africa PNH and aHUS Market Size by Country (2026-2031)
10.5 Turkey
10.6 Saudi Arabia
10.7 UAE
11 Key Players Profiles
11.1 AstraZeneca
11.1.1 AstraZeneca Company Details
11.1.2 AstraZeneca Business Overview
11.1.3 AstraZeneca PNH and aHUS Introduction
11.1.4 AstraZeneca Revenue in PNH and aHUS Business (2020-2025)
11.1.5 AstraZeneca Recent Development
11.2 Alexion Pharmaceutical
11.2.1 Alexion Pharmaceutical Company Details
11.2.2 Alexion Pharmaceutical Business Overview
11.2.3 Alexion Pharmaceutical PNH and aHUS Introduction
11.2.4 Alexion Pharmaceutical Revenue in PNH and aHUS Business (2020-2025)
11.2.5 Alexion Pharmaceutical Recent Development
11.3 CJSC Generium
11.3.1 CJSC Generium Company Details
11.3.2 CJSC Generium Business Overview
11.3.3 CJSC Generium PNH and aHUS Introduction
11.3.4 CJSC Generium Revenue in PNH and aHUS Business (2020-2025)
11.3.5 CJSC Generium Recent Development
12 Analyst's Viewpoints/Conclusions
13 Appendix
13.1 Research Methodology
13.1.1 Methodology/Research Approach
13.1.1.1 Research Programs/Design
13.1.1.2 Market Size Estimation
13.1.1.3 Market Breakdown and Data Triangulation
13.1.2 Data Source
13.1.2.1 Secondary Sources
13.1.2.2 Primary Sources
13.2 Author Details
13.3 Disclaimer
List of Tables
Table 1. Global PNH and aHUS Market Size Growth Rate by Type (US$ Million): 2020 VS 2024 VS 2031
Table 2. Key Players of Soliris
Table 3. Key Players of Ultomiris
Table 4. Global PNH and aHUS Market Size Growth by Application (US$ Million): 2020 VS 2024 VS 2031
Table 5. Global PNH and aHUS Market Size by Region (US$ Million): 2020 VS 2024 VS 2031
Table 6. Global PNH and aHUS Market Size by Region (2020-2025) & (US$ Million)
Table 7. Global PNH and aHUS Market Share by Region (2020-2025)
Table 8. Global PNH and aHUS Forecasted Market Size by Region (2026-2031) & (US$ Million)
Table 9. Global PNH and aHUS Market Share by Region (2026-2031)
Table 10. PNH and aHUS Market Trends
Table 11. PNH and aHUS Market Drivers
Table 12. PNH and aHUS Market Challenges
Table 13. PNH and aHUS Market Restraints
Table 14. Global PNH and aHUS Revenue by Players (2020-2025) & (US$ Million)
Table 15. Global PNH and aHUS Market Share by Players (2020-2025)
Table 16. Global Top PNH and aHUS Players by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in PNH and aHUS as of 2024)
Table 17. Ranking of Global Top PNH and aHUS Companies by Revenue (US$ Million) in 2024
Table 18. Global 5 Largest Players Market Share by PNH and aHUS Revenue (CR5 and HHI) & (2020-2025)
Table 19. Global Key Players of PNH and aHUS, Headquarters and Area Served
Table 20. Global Key Players of PNH and aHUS, Product and Application
Table 21. Global Key Players of PNH and aHUS, Date of Enter into This Industry
Table 22. Mergers & Acquisitions, Expansion Plans
Table 23. Global PNH and aHUS Market Size by Type (2020-2025) & (US$ Million)
Table 24. Global PNH and aHUS Revenue Market Share by Type (2020-2025)
Table 25. Global PNH and aHUS Forecasted Market Size by Type (2026-2031) & (US$ Million)
Table 26. Global PNH and aHUS Revenue Market Share by Type (2026-2031)
Table 27. Global PNH and aHUS Market Size by Application (2020-2025) & (US$ Million)
Table 28. Global PNH and aHUS Revenue Market Share by Application (2020-2025)
Table 29. Global PNH and aHUS Forecasted Market Size by Application (2026-2031) & (US$ Million)
Table 30. Global PNH and aHUS Revenue Market Share by Application (2026-2031)
Table 31. North America PNH and aHUS Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031
Table 32. North America PNH and aHUS Market Size by Country (2020-2025) & (US$ Million)
Table 33. North America PNH and aHUS Market Size by Country (2026-2031) & (US$ Million)
Table 34. Europe PNH and aHUS Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031
Table 35. Europe PNH and aHUS Market Size by Country (2020-2025) & (US$ Million)
Table 36. Europe PNH and aHUS Market Size by Country (2026-2031) & (US$ Million)
Table 37. Asia-Pacific PNH and aHUS Market Size Growth Rate by Region (US$ Million): 2020 VS 2024 VS 2031
Table 38. Asia-Pacific PNH and aHUS Market Size by Region (2020-2025) & (US$ Million)
Table 39. Asia-Pacific PNH and aHUS Market Size by Region (2026-2031) & (US$ Million)
Table 40. Latin America PNH and aHUS Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031
Table 41. Latin America PNH and aHUS Market Size by Country (2020-2025) & (US$ Million)
Table 42. Latin America PNH and aHUS Market Size by Country (2026-2031) & (US$ Million)
Table 43. Middle East & Africa PNH and aHUS Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031
Table 44. Middle East & Africa PNH and aHUS Market Size by Country (2020-2025) & (US$ Million)
Table 45. Middle East & Africa PNH and aHUS Market Size by Country (2026-2031) & (US$ Million)
Table 46. AstraZeneca Company Details
Table 47. AstraZeneca Business Overview
Table 48. AstraZeneca PNH and aHUS Product
Table 49. AstraZeneca Revenue in PNH and aHUS Business (2020-2025) & (US$ Million)
Table 50. AstraZeneca Recent Development
Table 51. Alexion Pharmaceutical Company Details
Table 52. Alexion Pharmaceutical Business Overview
Table 53. Alexion Pharmaceutical PNH and aHUS Product
Table 54. Alexion Pharmaceutical Revenue in PNH and aHUS Business (2020-2025) & (US$ Million)
Table 55. Alexion Pharmaceutical Recent Development
Table 56. CJSC Generium Company Details
Table 57. CJSC Generium Business Overview
Table 58. CJSC Generium PNH and aHUS Product
Table 59. CJSC Generium Revenue in PNH and aHUS Business (2020-2025) & (US$ Million)
Table 60. CJSC Generium Recent Development
Table 61. Research Programs/Design for This Report
Table 62. Key Data Information from Secondary Sources
Table 63. Key Data Information from Primary Sources
Table 64. Authors List of This Report
List of Figures
Figure 1. PNH and aHUS Picture
Figure 2. Global PNH and aHUS Market Size Comparison by Type (2020-2031) & (US$ Million)
Figure 3. Global PNH and aHUS Market Share by Type: 2024 VS 2031
Figure 4. Soliris Features
Figure 5. Ultomiris Features
Figure 6. Global PNH and aHUS Market Size by Application (2020-2031) & (US$ Million)
Figure 7. Global PNH and aHUS Market Share by Application: 2024 VS 2031
Figure 8. PNH Case Studies
Figure 9. aHUS Case Studies
Figure 10. PNH and aHUS Report Years Considered
Figure 11. Global PNH and aHUS Market Size (US$ Million), Year-over-Year: 2020-2031
Figure 12. Global PNH and aHUS Market Size, (US$ Million), 2020 VS 2024 VS 2031
Figure 13. Global PNH and aHUS Market Share by Region: 2024 VS 2031
Figure 14. Global PNH and aHUS Market Share by Players in 2024
Figure 15. Global PNH and aHUS Market Share by Company Type (Tier 1, Tier 2, and Tier 3)
Figure 16. The Top 10 and 5 Players Market Share by PNH and aHUS Revenue in 2024
Figure 17. North America PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 18. North America PNH and aHUS Market Share by Country (2020-2031)
Figure 19. United States PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 20. Canada PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 21. Europe PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 22. Europe PNH and aHUS Market Share by Country (2020-2031)
Figure 23. Germany PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 24. France PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 25. U.K. PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 26. Italy PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 27. Russia PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 28. Nordic Countries PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 29. Asia-Pacific PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 30. Asia-Pacific PNH and aHUS Market Share by Region (2020-2031)
Figure 31. China PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 32. Japan PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 33. South Korea PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 34. Southeast Asia PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 35. India PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 36. Australia PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 37. Latin America PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 38. Latin America PNH and aHUS Market Share by Country (2020-2031)
Figure 39. Mexico PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 40. Brazil PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 41. Middle East & Africa PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 42. Middle East & Africa PNH and aHUS Market Share by Country (2020-2031)
Figure 43. Turkey PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 44. Saudi Arabia PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 45. UAE PNH and aHUS Market Size YoY Growth (2020-2031) & (US$ Million)
Figure 46. AstraZeneca Revenue Growth Rate in PNH and aHUS Business (2020-2025)
Figure 47. Alexion Pharmaceutical Revenue Growth Rate in PNH and aHUS Business (2020-2025)
Figure 48. CJSC Generium Revenue Growth Rate in PNH and aHUS Business (2020-2025)
Figure 49. Bottom-up and Top-down Approaches for This Report
Figure 50. Data Triangulation
Figure 51. Key Executives Interviewed