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Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Global Market Report 2025
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Paroxysmal nocturnal hemoglobinuria (PNH) treatment involves medical therapies aimed at managing this rare blood disorder, which is characterized by hemolysis (destruction of red blood cells), blood clots, and bone marrow dysfunction. PNH is an acquired hematologic disorder where red blood cell destruction occurs due to complement-mediated hemolysis, bone marrow dysfunction leading to cytopenias, and an increased risk of thrombosis. The primary focus of PNH management is to inhibit complement activation to prevent hemolysis, address bone marrow failure, and reduce thrombotic complications, ultimately improving patient outcomes and quality of life.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The main treatment options for PNH include complement C5 inhibitors, anti-complement component 5a receptor (C5aR) monoclonal antibodies, alternative pathway inhibitors, hematopoietic stem cell transplantation, and others. Complement C5 inhibitors are a class of targeted therapies that block the cleavage of complement component 5 (C5) in the immune system's complement cascade, preventing the formation of membrane attack complexes. This reduces complement-mediated hemolysis and inflammation in PNH and other complement-driven diseases. These therapies are administered through various routes, including intravenous, subcutaneous, and oral. The drugs are available in the market as approved, in the pipeline, and in investigational stages, and are used in settings such as hospitals, specialty clinics, homecare environments, and others.

The paroxysmal nocturnal hemoglobinuria (PNH) treatment market research report is one of a series of new reports from The Business Research Company that provides paroxysmal nocturnal hemoglobinuria (PNH) treatment market statistics, including paroxysmal nocturnal hemoglobinuria (PNH) treatment industry global market size, regional shares, competitors with a paroxysmal nocturnal hemoglobinuria (PNH) treatment market share, detailed paroxysmal nocturnal hemoglobinuria (PNH) treatment market segments, market trends and opportunities, and any further data you may need to thrive in the paroxysmal nocturnal hemoglobinuria (PNH) treatment industry. This paroxysmal nocturnal hemoglobinuria (PNH) treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The paroxysmal nocturnal hemoglobinuria (PNH) treatment market size has grown rapidly in recent years. It will grow from $5.35 billion in 2024 to $6.09 billion in 2025 at a compound annual growth rate (CAGR) of 13.8%. The growth observed in the historic period can be attributed to several factors, including the high prevalence of neurological disorders and chronic pain conditions, growing awareness of PNH, increasing recognition of rare hematological disorders, and a rise in regulatory approvals. Additionally, there has been a notable increase in research and development (R&D) efforts.

The paroxysmal nocturnal hemoglobinuria (PNH) treatment market size is expected to see rapid growth in the next few years. It will grow to $10.09 billion in 2029 at a compound annual growth rate (CAGR) of 13.5%. The growth projected for the forecast period can be attributed to several factors, including the rising prevalence of rare diseases, increased healthcare expenditure, and greater access to advanced therapies. Additionally, there is a growing investment in healthcare infrastructure, a rise in home-based testing and remote monitoring, and an increase in research and development activities. Key trends during this period include advancements in genetic testing and biomarkers, progress in therapies, the development of targeted treatments, innovations in biotechnology, and the creation of novel therapies.

The growing prevalence of hematologic disorders is expected to drive the expansion of the paroxysmal nocturnal hemoglobinuria (PNH) treatment market. Hematologic disorders encompass a wide range of medical conditions that affect the blood and blood-forming organs, including abnormalities in red blood cells, white blood cells, platelets, bone marrow, and the coagulation system. These conditions can lead to anemia, clotting disorders, or malignancies. The rising incidence of hematologic disorders is fueled by factors such as aging populations and genetic predispositions, with genetic mutations playing a crucial role in triggering abnormal blood cell production, leading to various hematologic diseases. PNH treatment plays a key role in managing these disorders by offering targeted therapies that address complement-mediated hemolysis, reduce thrombotic complications, and improve patient survival. For example, in July 2024, Cancer Research UK, a UK-based charity, reported that the number of new leukemia cases in the UK is expected to rise from around 12,400 between 2023-2025 to approximately 14,500 by 2038-2040. This increasing incidence of hematologic disorders is driving growth in the PNH treatment market.

Leading companies in the PNH treatment market are focusing on developing innovative solutions, such as oral Factor B inhibitors, which offer novel treatment options that enhance therapeutic effectiveness and patient convenience in managing PNH. An oral Factor B inhibitor is a targeted small-molecule drug that selectively inhibits Factor B in the alternative complement pathway, regulating immune system overactivation and reducing inflammation in complement-mediated diseases. For instance, in December 2023, Novartis AG, a Switzerland-based pharmaceutical company, announced the FDA approval of Fabhalta (iptacopan) as the first oral monotherapy for the treatment of PNH. This new Factor B inhibitor provides a more comprehensive approach to controlling complement-mediated hemolysis by targeting the alternative complement pathway earlier, improving hemoglobin levels, and reducing the need for blood transfusions. The approval of Fabhalta represents a significant advancement in PNH treatment, offering enhanced efficacy, better patient compliance, and a viable alternative to intravenous therapies, transforming the treatment landscape for this rare hematologic disorder.

In December 2024, The Max Foundation, a US-based nonprofit organization, announced a collaboration with Novartis AG to improve global health equity by expanding access to innovative treatments for underserved populations. This partnership aims to increase access to advanced PNH treatments in low-resource countries by leveraging strategic partnerships, strengthening healthcare infrastructure, providing essential medications at no cost, and fostering medical expertise through education and capacity-building initiatives. The goal is to ensure equitable healthcare access and improve patient outcomes worldwide. Novartis AG, a Switzerland-based pharmaceutical company, developed the oral monotherapy to manage PNH.

Major players in the paroxysmal nocturnal hemoglobinuria (pnh) treatment market are F. Hoffmann-La Roche Ltd, AstraZeneca PLC, Novartis AG, Amgen Inc., Mayo Clinic, Regeneron Pharmaceuticals, Cleveland Clinic Foundation, Johns Hopkins Medicine, Memorial Sloan Kettering Cancer Center, Stanford Health Care, University of California San Francisco (UCSF) Health, Emory Healthcare, Cedars-Sinai Medical Center, Duke University Health System, Mount Sinai Health System, Dana-Farber Cancer Institute, Swedish Orphan Biovitrum, Alnylam Pharmaceuticals, BioCryst Pharmaceuticals, Apellis Pharmaceuticals.

North America was the largest region in the paroxysmal nocturnal hemoglobinuria (PNH) treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in paroxysmal nocturnal hemoglobinuria (PNH) treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the paroxysmal nocturnal hemoglobinuria (PNH) treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The paroxysmal nocturnal hemoglobinuria (PNH) treatment market includes revenues earned by entities by providing services such as diagnostic testing services, genetic counseling, patient support programs, blood transfusion services, iron supplementation therapy, clinical trial services, and telemedicine consultations. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses on paroxysmal nocturnal hemoglobinuria (pnh) treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

Where is the largest and fastest growing market for paroxysmal nocturnal hemoglobinuria (pnh) treatment ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The paroxysmal nocturnal hemoglobinuria (pnh) treatment market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.

Scope

Table of Contents

1. Executive Summary

2. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Characteristics

3. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Trends And Strategies

4. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market

5. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Growth Analysis And Strategic Analysis Framework

6. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Segmentation

7. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Regional And Country Analysis

8. Asia-Pacific Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

9. China Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

10. India Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

11. Japan Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

12. Australia Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

13. Indonesia Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

14. South Korea Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

15. Western Europe Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

16. UK Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

17. Germany Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

18. France Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

19. Italy Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

20. Spain Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

21. Eastern Europe Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

22. Russia Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

23. North America Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

24. USA Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

25. Canada Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

26. South America Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

27. Brazil Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

28. Middle East Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

29. Africa Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

30. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Competitive Landscape And Company Profiles

31. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Other Major And Innovative Companies

32. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

34. Recent Developments In The Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

35. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market High Potential Countries, Segments and Strategies

36. Appendix

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