Summary

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.

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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
10.2 Author Details
10.3 Disclaimer