PUBLISHER: QYResearch | PRODUCT CODE: 1874353
PUBLISHER: QYResearch | PRODUCT CODE: 1874353
PNH and aHUS - Global Market Share and Ranking, Overall Sales and Demand Forecast 2025-2031
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
- 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-Pacific
- Europe
- Germany
- France
- U.K.
- Italy
- Netherlands
- 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, 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.
Table of Contents
1 Market Overview
- 1.1 PNH and aHUS Product Introduction
- 1.2 Global PNH and aHUS Market Size Forecast (2020-2031)
- 1.3 PNH and aHUS Market Trends & Drivers
- 1.3.1 PNH and aHUS Industry Trends
- 1.3.2 PNH and aHUS Market Drivers & Opportunity
- 1.3.3 PNH and aHUS Market Challenges
- 1.3.4 PNH and aHUS Market Restraints
- 1.4 Assumptions and Limitations
- 1.5 Study Objectives
- 1.6 Years Considered
2 Competitive Analysis by Company
- 2.1 Global PNH and aHUS Players Revenue Ranking (2024)
- 2.2 Global PNH and aHUS Revenue by Company (2020-2025)
- 2.3 Key Companies PNH and aHUS Manufacturing Base Distribution and Headquarters
- 2.4 Key Companies PNH and aHUS Product Offered
- 2.5 Key Companies Time to Begin Mass Production of PNH and aHUS
- 2.6 PNH and aHUS Market Competitive Analysis
- 2.6.1 PNH and aHUS Market Concentration Rate (2020-2025)
- 2.6.2 Global 5 and 10 Largest Companies by PNH and aHUS Revenue in 2024
- 2.6.3 Global Top Companies by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in PNH and aHUS as of 2024)
- 2.7 Mergers & Acquisitions, Expansion
3 Segmentation by Type
- 3.1 Introduction by Type
- 3.1.1 Soliris
- 3.1.2 Ultomiris
- 3.2 Global PNH and aHUS Sales Value by Type
- 3.2.1 Global PNH and aHUS Sales Value by Type (2020 VS 2024 VS 2031)
- 3.2.2 Global PNH and aHUS Sales Value, by Type (2020-2031)
- 3.2.3 Global PNH and aHUS Sales Value, by Type (%) (2020-2031)
4 Segmentation by Application
- 4.1 Introduction by Application
- 4.1.1 PNH
- 4.1.2 aHUS
- 4.2 Global PNH and aHUS Sales Value by Application
- 4.2.1 Global PNH and aHUS Sales Value by Application (2020 VS 2024 VS 2031)
- 4.2.2 Global PNH and aHUS Sales Value, by Application (2020-2031)
- 4.2.3 Global PNH and aHUS Sales Value, by Application (%) (2020-2031)
5 Segmentation by Region
- 5.1 Global PNH and aHUS Sales Value by Region
- 5.1.1 Global PNH and aHUS Sales Value by Region: 2020 VS 2024 VS 2031
- 5.1.2 Global PNH and aHUS Sales Value by Region (2020-2025)
- 5.1.3 Global PNH and aHUS Sales Value by Region (2026-2031)
- 5.1.4 Global PNH and aHUS Sales Value by Region (%), (2020-2031)
- 5.2 North America
- 5.2.1 North America PNH and aHUS Sales Value, 2020-2031
- 5.2.2 North America PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- 5.3 Europe
- 5.3.1 Europe PNH and aHUS Sales Value, 2020-2031
- 5.3.2 Europe PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- 5.4 Asia Pacific
- 5.4.1 Asia Pacific PNH and aHUS Sales Value, 2020-2031
- 5.4.2 Asia Pacific PNH and aHUS Sales Value by Region (%), 2024 VS 2031
- 5.5 South America
- 5.5.1 South America PNH and aHUS Sales Value, 2020-2031
- 5.5.2 South America PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- 5.6 Middle East & Africa
- 5.6.1 Middle East & Africa PNH and aHUS Sales Value, 2020-2031
- 5.6.2 Middle East & Africa PNH and aHUS Sales Value by Country (%), 2024 VS 2031
6 Segmentation by Key Countries/Regions
- 6.1 Key Countries/Regions PNH and aHUS Sales Value Growth Trends, 2020 VS 2024 VS 2031
- 6.2 Key Countries/Regions PNH and aHUS Sales Value, 2020-2031
- 6.3 United States
- 6.3.1 United States PNH and aHUS Sales Value, 2020-2031
- 6.3.2 United States PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.3.3 United States PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.4 Europe
- 6.4.1 Europe PNH and aHUS Sales Value, 2020-2031
- 6.4.2 Europe PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.4.3 Europe PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.5 China
- 6.5.1 China PNH and aHUS Sales Value, 2020-2031
- 6.5.2 China PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.5.3 China PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.6 Japan
- 6.6.1 Japan PNH and aHUS Sales Value, 2020-2031
- 6.6.2 Japan PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.6.3 Japan PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.7 South Korea
- 6.7.1 South Korea PNH and aHUS Sales Value, 2020-2031
- 6.7.2 South Korea PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.7.3 South Korea PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.8 Southeast Asia
- 6.8.1 Southeast Asia PNH and aHUS Sales Value, 2020-2031
- 6.8.2 Southeast Asia PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.8.3 Southeast Asia PNH and aHUS Sales Value by Application, 2024 VS 2031
- 6.9 India
- 6.9.1 India PNH and aHUS Sales Value, 2020-2031
- 6.9.2 India PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- 6.9.3 India PNH and aHUS Sales Value by Application, 2024 VS 2031
7 Company Profiles
- 7.1 AstraZeneca
- 7.1.1 AstraZeneca Profile
- 7.1.2 AstraZeneca Main Business
- 7.1.3 AstraZeneca PNH and aHUS Products, Services and Solutions
- 7.1.4 AstraZeneca PNH and aHUS Revenue (US$ Million) & (2020-2025)
- 7.1.5 AstraZeneca Recent Developments
- 7.2 Alexion Pharmaceutical
- 7.2.1 Alexion Pharmaceutical Profile
- 7.2.2 Alexion Pharmaceutical Main Business
- 7.2.3 Alexion Pharmaceutical PNH and aHUS Products, Services and Solutions
- 7.2.4 Alexion Pharmaceutical PNH and aHUS Revenue (US$ Million) & (2020-2025)
- 7.2.5 Alexion Pharmaceutical Recent Developments
- 7.3 CJSC Generium
- 7.3.1 CJSC Generium Profile
- 7.3.2 CJSC Generium Main Business
- 7.3.3 CJSC Generium PNH and aHUS Products, Services and Solutions
- 7.3.4 CJSC Generium PNH and aHUS Revenue (US$ Million) & (2020-2025)
- 7.3.5 CJSC Generium Recent Developments
8 Industry Chain Analysis
- 8.1 PNH and aHUS Industrial Chain
- 8.2 PNH and aHUS Upstream Analysis
- 8.2.1 Key Raw Materials
- 8.2.2 Raw Materials Key Suppliers
- 8.2.3 Manufacturing Cost Structure
- 8.3 Midstream Analysis
- 8.4 Downstream Analysis (Customers Analysis)
- 8.5 Sales Model and Sales Channels
- 8.5.1 PNH and aHUS Sales Model
- 8.5.2 Sales Channel
- 8.5.3 PNH and aHUS Distributors
9 Research Findings and Conclusion
10 Appendix
- 10.1 Research Methodology
- 10.1.1 Methodology/Research Approach
- 10.1.1.1 Research Programs/Design
- 10.1.1.2 Market Size Estimation
- 10.1.1.3 Market Breakdown and Data Triangulation
- 10.1.2 Data Source
- 10.1.2.1 Secondary Sources
- 10.1.2.2 Primary Sources
- 10.1.1 Methodology/Research Approach
- 10.2 Author Details
- 10.3 Disclaimer
List of Tables
- Table 1. PNH and aHUS Market Trends
- Table 2. PNH and aHUS Market Drivers & Opportunity
- Table 3. PNH and aHUS Market Challenges
- Table 4. PNH and aHUS Market Restraints
- Table 5. Global PNH and aHUS Revenue by Company (2020-2025) & (US$ Million)
- Table 6. Global PNH and aHUS Revenue Market Share by Company (2020-2025)
- Table 7. Key Companies PNH and aHUS Manufacturing Base Distribution and Headquarters
- Table 8. Key Companies PNH and aHUS Product Type
- Table 9. Key Companies Time to Begin Mass Production of PNH and aHUS
- Table 10. Global PNH and aHUS Companies Market Concentration Ratio (CR5 and HHI)
- Table 11. Global Top Companies by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in PNH and aHUS as of 2024)
- Table 12. Mergers & Acquisitions, Expansion Plans
- Table 13. Global PNH and aHUS Sales Value by Type: 2020 VS 2024 VS 2031 (US$ Million)
- Table 14. Global PNH and aHUS Sales Value by Type (2020-2025) & (US$ Million)
- Table 15. Global PNH and aHUS Sales Value by Type (2026-2031) & (US$ Million)
- Table 16. Global PNH and aHUS Sales Market Share in Value by Type (2020-2025)
- Table 17. Global PNH and aHUS Sales Market Share in Value by Type (2026-2031)
- Table 18. Global PNH and aHUS Sales Value by Application: 2020 VS 2024 VS 2031 (US$ Million)
- Table 19. Global PNH and aHUS Sales Value by Application (2020-2025) & (US$ Million)
- Table 20. Global PNH and aHUS Sales Value by Application (2026-2031) & (US$ Million)
- Table 21. Global PNH and aHUS Sales Market Share in Value by Application (2020-2025)
- Table 22. Global PNH and aHUS Sales Market Share in Value by Application (2026-2031)
- Table 23. Global PNH and aHUS Sales Value by Region, (2020 VS 2024 VS 2031) & (US$ Million)
- Table 24. Global PNH and aHUS Sales Value by Region (2020-2025) & (US$ Million)
- Table 25. Global PNH and aHUS Sales Value by Region (2026-2031) & (US$ Million)
- Table 26. Global PNH and aHUS Sales Value by Region (2020-2025) & (%)
- Table 27. Global PNH and aHUS Sales Value by Region (2026-2031) & (%)
- Table 28. Key Countries/Regions PNH and aHUS Sales Value Growth Trends, (US$ Million): 2020 VS 2024 VS 2031
- Table 29. Key Countries/Regions PNH and aHUS Sales Value, (2020-2025) & (US$ Million)
- Table 30. Key Countries/Regions PNH and aHUS Sales Value, (2026-2031) & (US$ Million)
- Table 31. AstraZeneca Basic Information List
- Table 32. AstraZeneca Description and Business Overview
- Table 33. AstraZeneca PNH and aHUS Products, Services and Solutions
- Table 34. Revenue (US$ Million) in PNH and aHUS Business of AstraZeneca (2020-2025)
- Table 35. AstraZeneca Recent Developments
- Table 36. Alexion Pharmaceutical Basic Information List
- Table 37. Alexion Pharmaceutical Description and Business Overview
- Table 38. Alexion Pharmaceutical PNH and aHUS Products, Services and Solutions
- Table 39. Revenue (US$ Million) in PNH and aHUS Business of Alexion Pharmaceutical (2020-2025)
- Table 40. Alexion Pharmaceutical Recent Developments
- Table 41. CJSC Generium Basic Information List
- Table 42. CJSC Generium Description and Business Overview
- Table 43. CJSC Generium PNH and aHUS Products, Services and Solutions
- Table 44. Revenue (US$ Million) in PNH and aHUS Business of CJSC Generium (2020-2025)
- Table 45. CJSC Generium Recent Developments
- Table 46. Key Raw Materials Lists
- Table 47. Raw Materials Key Suppliers Lists
- Table 48. PNH and aHUS Downstream Customers
- Table 49. PNH and aHUS Distributors List
- Table 50. Research Programs/Design for This Report
- Table 51. Key Data Information from Secondary Sources
- Table 52. Key Data Information from Primary Sources
List of Figures
- Figure 1. PNH and aHUS Product Picture
- Figure 2. Global PNH and aHUS Sales Value, 2020 VS 2024 VS 2031 (US$ Million)
- Figure 3. Global PNH and aHUS Sales Value (2020-2031) & (US$ Million)
- Figure 4. PNH and aHUS Report Years Considered
- Figure 5. Global PNH and aHUS Players Revenue Ranking (2024) & (US$ Million)
- Figure 6. The 5 and 10 Largest Companies in the World: Market Share by PNH and aHUS Revenue in 2024
- Figure 7. PNH and aHUS Market Share by Company Type (Tier 1, Tier 2, and Tier 3): 2020 VS 2024
- Figure 8. Soliris Picture
- Figure 9. Ultomiris Picture
- Figure 10. Global PNH and aHUS Sales Value by Type (2020 VS 2024 VS 2031) & (US$ Million)
- Figure 11. Global PNH and aHUS Sales Value Market Share by Type, 2024 & 2031
- Figure 12. Product Picture of PNH
- Figure 13. Product Picture of aHUS
- Figure 14. Global PNH and aHUS Sales Value by Application (2020 VS 2024 VS 2031) & (US$ Million)
- Figure 15. Global PNH and aHUS Sales Value Market Share by Application, 2024 & 2031
- Figure 16. North America PNH and aHUS Sales Value (2020-2031) & (US$ Million)
- Figure 17. North America PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- Figure 18. Europe PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 19. Europe PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- Figure 20. Asia Pacific PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 21. Asia Pacific PNH and aHUS Sales Value by Region (%), 2024 VS 2031
- Figure 22. South America PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 23. South America PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- Figure 24. Middle East & Africa PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 25. Middle East & Africa PNH and aHUS Sales Value by Country (%), 2024 VS 2031
- Figure 26. Key Countries/Regions PNH and aHUS Sales Value (%), (2020-2031)
- Figure 27. United States PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 28. United States PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 29. United States PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 30. Europe PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 31. Europe PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 32. Europe PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 33. China PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 34. China PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 35. China PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 36. Japan PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 37. Japan PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 38. Japan PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 39. South Korea PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 40. South Korea PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 41. South Korea PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 42. Southeast Asia PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 43. Southeast Asia PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 44. Southeast Asia PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 45. India PNH and aHUS Sales Value, (2020-2031) & (US$ Million)
- Figure 46. India PNH and aHUS Sales Value by Type (%), 2024 VS 2031
- Figure 47. India PNH and aHUS Sales Value by Application (%), 2024 VS 2031
- Figure 48. PNH and aHUS Industrial Chain
- Figure 49. PNH and aHUS Manufacturing Cost Structure
- Figure 50. Channels of Distribution (Direct Sales, and Distribution)
- Figure 51. Bottom-up and Top-down Approaches for This Report
- Figure 52. Data Triangulation
- Figure 53. Key Executives Interviewed
