DelveInsight’s “Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Paroxysmal Nocturnal Hemoglobinuria, historical and forecasted epidemiology as well as the Paroxysmal Nocturnal Hemoglobinuria market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
To Know in detail about the Paroxysmal Nocturnal Hemoglobinuria market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Paroxysmal Nocturnal Hemoglobinuria Market Forecast
Some of the key facts of the Paroxysmal Nocturnal Hemoglobinuria Market Report:
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The Paroxysmal Nocturnal Hemoglobinuria market size was valued ~USD 1,141 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034).
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In September 2025, Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a clinical-stage company specializing in small interfering RNA (siRNA) therapeutics, announced that the National Medical Products Administration of China (NMPA) has granted Investigational New Drug (IND) approval for a Phase II clinical trial of BW-40202. This investigational siRNA therapy targets complement factor B (CFB) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other complement-related diseases. The trial is set to begin in January 2026. BW-40202 is also undergoing a Phase I study as a potential treatment for IgA Nephropathy (IgAN), with Phase I/IIa IND approval from the NMPA in China expected in June 2025.
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In June 2025, Novartis has reported positive outcomes from the APPULSE-PNH Phase 3B trial, which evaluated the safety and effectiveness of iptacopan (Fabhalta), a twice-daily oral monotherapy, in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had hemoglobin (Hb) levels of ≥10 g/dL and transitioned from anti-C5 treatments such as eculizumab or ravulizumab.
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In May 2025, NovelMed announced encouraging 12-week interim findings from its ongoing multi-dose Phase II study of Ruxoprubart, an innovative complement-targeting immunotherapy, in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH). The interim results show that Ruxoprubart, used as a monotherapy, was safe, well-tolerated, and successfully met all primary efficacy goals. The treatment led to transfusion avoidance, higher hemoglobin levels, lower LDH levels, and an increase in PNH clone size—key indicators of better disease management and improved quality of life for individuals with PNH.
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In December 2024, Novartis has announced positive topline results from the multicenter Phase IIIB APPULSE-PNH trial evaluating oral Fabhalta (iptacopan) for adults with paroxysmal nocturnal hemoglobinuria (PNH). Participants in the study transitioned from anti-C5 therapies and had a baseline hemoglobin (Hb) level of at least 10g/dL following treatment with eculizumab or ravulizumab.
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In December 2024, Omeros Corporation (Nasdaq: OMER) announced that two posters on zaltenibart (OMS906), its investigational MASP-3 inhibitor targeting the alternative complement pathway, were presented yesterday at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego. The posters, focused on zaltenibart's potential in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder, highlighted positive Phase 2 clinical data and clinical pharmacology analyses supporting dose selection for the upcoming Phase 3 program. Enrollment for the Phase 3 trials in PNH is expected to begin in early 2025.
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In 2023, the US market size for Paroxysmal Nocturnal Hemoglobinuria therapeutics was around USD 845.6 million and is expected to grow with the introduction of emerging treatments.
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In 2023, the total market size for Paroxysmal Nocturnal Hemoglobinuria in the EU4 and the UK was estimated at approximately USD 480.1 million, accounting for nearly 34% of the total market revenue across the 7MM.
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In 2023, the UK had the largest market share among the EU4 and the UK, generating approximately USD 230.2 million, followed by Germany with around USD 125.9 million.
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In 2023, Japan's market size for Paroxysmal Nocturnal Hemoglobinuria therapeutics was around USD 88.5 million, with expectations of growth throughout the forecast period (2024-2034).
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Projections indicate that OMS906 is anticipated to generate around USD 303.7 million across the 7MM by 2034.
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DelveInsight projects that approximately 12,485 diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria were recorded across the 7MM in 2023, with this figure anticipated to increase by 2034.
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In 2023, the US recorded the highest number of diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria among the 7MM, totaling around 6,127 cases.
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Among the EU4 and the UK, the highest number of diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria in 2023 was reported in the UK, with approximately 2,596 cases, followed by Germany with around 1,359 cases. Italy had the lowest prevalence, with roughly 250 cases.
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In 2023, Japan had around 944 diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria, with this number projected to increase by 2034.
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In 2023, an estimated 5,799 males and 6,686 females were diagnosed with Paroxysmal Nocturnal Hemoglobinuria across the 7MM.
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The pipeline of emerging treatments for Paroxysmal Nocturnal Hemoglobinuria includes pozelimab, OMS906, and NM8074 (ruxoprubart), among other potential therapies.
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Key Paroxysmal Nocturnal Hemoglobinuria Companies: Hoffmann-La Roche (Chugai Pharmaceutical), AstraZeneca (Alexion Pharmaceuticals), Apellis Pharmaceuticals, Swedish Orphan Biovitrum, Regeneron, Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation, BioCryst Pharmaceuticals, Novartis, Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, Regeneron Pharma, Kira Pharma, NovelMed Therapeutics, and others
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Key Paroxysmal Nocturnal Hemoglobinuria Therapies: PIASKY (Crovalimab), SOLIRIS (eculizumab), EMPAVELI/ASPAVELI (pegcetacoplan), Pozelimab (REGN3918), Zaltenibart (OMS906), Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906 study drug, HRS-5965 tablets, MY008211A tablets, Iptacopan, Pozelimab, KP104, NM8074, Danicopan, Pegcetacoplan, and others
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The Paroxysmal Nocturnal Hemoglobinuria based on gender analyzed that the majority of cases of PNH are female as compared to male
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The Paroxysmal Nocturnal Hemoglobinuria market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Paroxysmal Nocturnal Hemoglobinuria pipeline products will significantly revolutionize the Paroxysmal Nocturnal Hemoglobinuria market dynamics.
Paroxysmal Nocturnal Hemoglobinuria Overview
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and acquired hematologic disorder characterized by the abnormal breakdown of red blood cells (hemolysis), blood clots (thrombosis), and impaired bone marrow function. It is caused by a mutation in the PIGA gene, which leads to the deficiency of proteins that anchor certain cell surface proteins (such as CD55 and CD59) that protect red blood cells from destruction by the body's immune system.
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Paroxysmal Nocturnal Hemoglobinuria Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Paroxysmal Nocturnal Hemoglobinuria Epidemiology Segmentation:
The Paroxysmal Nocturnal Hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
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Total Prevalence of Paroxysmal Nocturnal Hemoglobinuria
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Prevalent Cases of Paroxysmal Nocturnal Hemoglobinuria by severity
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Gender-specific Prevalence of Paroxysmal Nocturnal Hemoglobinuria
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Diagnosed Cases of Episodic and Chronic Paroxysmal Nocturnal Hemoglobinuria
Download the report to understand which factors are driving Paroxysmal Nocturnal Hemoglobinuria epidemiology trends @ Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast
Paroxysmal Nocturnal Hemoglobinuria Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Paroxysmal Nocturnal Hemoglobinuria market or expected to get launched during the study period. The analysis covers Paroxysmal Nocturnal Hemoglobinuria market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Paroxysmal Nocturnal Hemoglobinuria Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Paroxysmal Nocturnal Hemoglobinuria Therapies and Key Companies
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PIASKY (Crovalimab): Hoffmann-La Roche (Chugai Pharmaceutical)
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SOLIRIS (eculizumab): AstraZeneca (Alexion Pharmaceuticals)
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EMPAVELI/ASPAVELI (pegcetacoplan): Apellis Pharmaceuticals/Swedish Orphan Biovitrum
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Pozelimab (REGN3918): Regeneron Pharmaceuticals/Alnylam Pharmaceuticals
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Zaltenibart (OMS906): Omeros Corporation
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Eculizumab: Alexion Pharmaceuticals
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BCX9930: BioCryst Pharmaceuticals
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Pegcetacoplan: Apellis Pharmaceuticals, Inc.
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Crovalimab: Hoffmann-La Roche
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OMS906 study drug: Omeros Corporation
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HRS-5965 tablets: Chengdu Suncadia Medicine Co., Ltd.
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MY008211A tablets: Wuhan Createrna Science
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Iptacopan: Novartis
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Pozelimab: Regeneron Pharma
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KP104: Kira Pharma
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NM8074: NovelMed Therapeutics
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Danicopan: Alexion Pharmaceuticals, Inc.
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Pegcetacoplan: Apellis Pharma
Discover more about therapies set to grab major Paroxysmal Nocturnal Hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Treatment Market
Paroxysmal Nocturnal Hemoglobinuria Market Strengths
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Despite receiving treatment with a C5 inhibitor, patients may still experience continuous hemolysis, persistent chronic anemia, need for continued blood transfusions, fatigue, and poor health-related quality of life
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The physicians are not generally aware of the broad spectrum of signs and symptoms of the disease, which are often similar to those of other diseases, and vary from one patient to another
Paroxysmal Nocturnal Hemoglobinuria Market Opportunities
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The C5 targeting monoclonal antibody eculizumab has changed the natural history of PNH in the last 10 years. This is a great opportunity to develop new biomarkers to assess thrombotic risk in PNH patients
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There is a potential for companies to price their drugs at a premium price if their therapies provide an edge over the current therapies
Scope of the Paroxysmal Nocturnal Hemoglobinuria Market Report
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Study Period: 2020–2034
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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
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Key Paroxysmal Nocturnal Hemoglobinuria Companies: Hoffmann-La Roche (Chugai Pharmaceutical), AstraZeneca (Alexion Pharmaceuticals), Apellis Pharmaceuticals, Swedish Orphan Biovitrum, Regeneron, Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation, BioCryst Pharmaceuticals, Novartis, Chengdu Suncadia Medicine Co., Ltd., Wuhan Createrna Science, Regeneron Pharma, Kira Pharma, NovelMed Therapeutics, and others
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Key Paroxysmal Nocturnal Hemoglobinuria Therapies: PIASKY (Crovalimab), SOLIRIS (eculizumab), EMPAVELI/ASPAVELI (pegcetacoplan), Pozelimab (REGN3918), Zaltenibart (OMS906), Eculizumab, BCX9930, Pegcetacoplan, Crovalimab, OMS906 study drug, HRS-5965 tablets, MY008211A tablets, Iptacopan, Pozelimab, KP104, NM8074, Danicopan, Pegcetacoplan, and others
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Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment: Paroxysmal Nocturnal Hemoglobinuria current marketed and Paroxysmal Nocturnal Hemoglobinuria emerging therapies
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Paroxysmal Nocturnal Hemoglobinuria Market Dynamics: Paroxysmal Nocturnal Hemoglobinuria market drivers and Paroxysmal Nocturnal Hemoglobinuria market barriers
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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
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Paroxysmal Nocturnal Hemoglobinuria Unmet Needs, KOL’s views, Analyst’s views, Paroxysmal Nocturnal Hemoglobinuria Market Access and Reimbursement
To know more about Paroxysmal Nocturnal Hemoglobinuria companies working in the treatment market, visit @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials and Therapeutic Assessment
Table of Contents
1. Paroxysmal Nocturnal Hemoglobinuria Market Report Introduction
2. Executive Summary for Paroxysmal Nocturnal Hemoglobinuria
3. SWOT analysis of Paroxysmal Nocturnal Hemoglobinuria
4. Paroxysmal Nocturnal Hemoglobinuria Patient Share (%) Overview at a Glance
5. Paroxysmal Nocturnal Hemoglobinuria Market Overview at a Glance
6. Paroxysmal Nocturnal Hemoglobinuria Disease Background and Overview
7. Paroxysmal Nocturnal Hemoglobinuria Epidemiology and Patient Population
8. Country-Specific Patient Population of Paroxysmal Nocturnal Hemoglobinuria
9. Paroxysmal Nocturnal Hemoglobinuria Current Treatment and Medical Practices
10. Paroxysmal Nocturnal Hemoglobinuria Unmet Needs
11. Paroxysmal Nocturnal Hemoglobinuria Emerging Therapies
12. Paroxysmal Nocturnal Hemoglobinuria Market Outlook
13. Country-Wise Paroxysmal Nocturnal Hemoglobinuria Market Analysis (2020–2034)
14. Paroxysmal Nocturnal Hemoglobinuria Market Access and Reimbursement of Therapies
15. Paroxysmal Nocturnal Hemoglobinuria Market Drivers
16. Paroxysmal Nocturnal Hemoglobinuria Market Barriers
17. Paroxysmal Nocturnal Hemoglobinuria Appendix
18. Paroxysmal Nocturnal Hemoglobinuria Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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