The paroxysmal nocturnal hemoglobinuria market is witnessing robust growth, driven by increasing disease awareness, improved diagnostics, and the expanding adoption of targeted therapies. The approval and uptake of complement inhibitors like eculizumab and ravulizumab have significantly transformed the treatment landscape. Emerging pipeline candidates, such as Pozelimab (REGN3918) + Cemdisiran, Zaltenibart, Ruxoprubart, and others, are expected to further fuel market expansion.

New York, USA, July 01, 2025 (GLOBE NEWSWIRE) -- Paroxysmal Nocturnal Hemoglobinuria Market Set for Robust Growth During the Forecast Period (2025–2034) Owing to the Advancements in Complement Inhibitor Therapies | DelveInsight 

The paroxysmal nocturnal hemoglobinuria market is witnessing robust growth, driven by increasing disease awareness, improved diagnostics, and the expanding adoption of targeted therapies. The approval and uptake of complement inhibitors like eculizumab and ravulizumab have significantly transformed the treatment landscape. Emerging pipeline candidates, such as Pozelimab (REGN3918) + Cemdisiran, Zaltenibart, Ruxoprubart, and others, are expected to further fuel market expansion.

DelveInsight's Paroxysmal Nocturnal Hemoglobinuria Market Insights report includes a comprehensive understanding of current treatment practices, emerging paroxysmal nocturnal hemoglobinuria drugs, market share of individual therapies, and current and forecasted paroxysmal nocturnal hemoglobinuria market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Takeaways from the Paroxysmal Nocturnal Hemoglobinuria Market Report

• According to DelveInsight's analysis, the total paroxysmal nocturnal hemoglobinuria market size was USD 1.3 billion in 2024 in the 7MM.
• The United States accounts for the largest market size of paroxysmal nocturnal hemoglobinuria, i.e., 85%, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan.
• In 2024, the PNH diagnosed prevalent cases were approximately 16,000 cases in the 7MM, which will increase by 2034.
• Prominent companies, including Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation, NovelMed Therapeutics, and others, are actively working on innovative paroxysmal nocturnal hemoglobinuria drugs. 
• Some of the key paroxysmal nocturnal hemoglobinuria therapies in the pipeline include Pozelimab (REGN3918) + Cemdisiran, Zaltenibart (OMS906), Ruxoprubart (NM8074), and others. These novel paroxysmal nocturnal hemoglobinuria therapies are anticipated to enter the paroxysmal nocturnal hemoglobinuria market in the forecast period and are expected to change the market.
• In May 2025, Otsuka and OPDC announced that the FDA had accepted the Biologics License Application for sibeprenlimab, an investigational antibody targeting APRIL, for the treatment of adults with IgA nephropathy (IgAN).
• In April 2025, Vera Therapeutics completed full enrollment in the pivotal ORIGIN Phase III trial evaluating atacicept in patients with IgAN.

Discover which paroxysmal nocturnal hemoglobinuria medications are expected to grab the market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report

Paroxysmal Nocturnal Hemoglobinuria Overview

Paroxysmal nocturnal hemoglobinuria is a rare, acquired condition that originates in pluripotent hematopoietic stem cells and can affect red blood cells, white blood cells, platelets, and, in some cases, endothelial cells. The disorder results from a somatic mutation in the X-linked PIG-A gene, which is essential for the synthesis of glycosylphosphatidylinositol anchors, molecules that tether specific proteins to the cell surface.

This mutation causes a lack of GPI anchors, leading to reduced expression of key complement-regulating proteins like decay-accelerating factor (DAF/CD55) and membrane inhibitor of reactive lysis (MIRL/CD59) on hematopoietic stem cells and their progeny. The absence of these protective proteins renders red blood cells vulnerable to destruction by the complement system, triggering complement-mediated intravascular hemolysis. This process releases free hemoglobin into the bloodstream, which binds to and depletes nitric oxide.

Previously, PNH was diagnosed using tests like the Ham test or the sucrose lysis test, but these are now outdated. Today, flow cytometry (FCM) is the standard diagnostic method, identifying the characteristic absence of GPI-anchored proteins. This is done using monoclonal antibodies (e.g., anti-CD55, anti-CD59) or fluorescein-labeled proaerolysin (FLAER), which binds to the glycan portion of the GPI anchor. FLAER is particularly useful for detecting GPI deficiencies in nucleated cells, as it does not effectively stain red blood cells due to interference from glycophorin, a protein abundant on their surface that disrupts aerolysin binding.

Paroxysmal Nocturnal Hemoglobinuria Epidemiology Segmentation

The paroxysmal nocturnal hemoglobinuria epidemiology section provides insights into the historical and current paroxysmal nocturnal hemoglobinuria patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.

The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total PNH Diagnosed Prevalent Cases
• PNH Gender-specific Cases
• PNH Age Group-Specific Cases
• Total PNH Treated Cases 

Download the report to understand which factors are driving paroxysmal nocturnal hemoglobinuria epidemiology trends @ Paroxysmal Nocturnal Hemoglobinuria Treatment Algorithm

Paroxysmal Nocturnal Hemoglobinuria Treatment Market 

The conventional approach to treating PNH primarily relies on C5 inhibitors. Eculizumab (SOLIRIS) and ravulizumab (ULTOMIRIS) are FDA-approved monoclonal antibodies that target terminal complement activation by blocking C5, thereby preventing the generation of C5a and the membrane attack complex (C5b–9). Both therapies, delivered through intravenous infusion, bind to the same epitope on C5; however, ravulizumab has been engineered for a prolonged half-life. Developed by Alexion Pharmaceuticals, SOLIRIS was the first breakthrough in PNH therapy, later followed by ULTOMIRIS, marking significant progress in C5 inhibition.

While these treatments have been central to PNH care, the therapeutic landscape is evolving with the introduction of pegcetacoplan and the emergence of iptacopan from Novartis, offering promising new alternatives. Pegcetacoplan (EMPAVELI), the first approved C3 inhibitor, has received regulatory clearance from both the FDA and EMA and signifies a notable advancement by targeting an upstream component of the complement cascade. Additionally, FABHALTA (iptacopan), an oral Factor B inhibitor developed by Novartis, has recently been approved. It is being investigated for multiple complement-driven conditions, including IgA nephropathy, C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, and atypical hemolytic uremic syndrome (aHUS), further expanding its potential therapeutic impact.

Learn more about the paroxysmal nocturnal hemoglobinuria treatment options @ Paroxysmal Nocturnal Hemoglobinuria Treatment Guidelines

Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs and Companies

The Expected launch of potential therapies may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of PNH. Owing to the positive outcomes of several products during the developmental stage by key players such as Regeneron Pharmaceuticals (REGN3918), Omeros (Zaltenibart), NovelMed (Ruxoprubart), and others. The PNH market is expected to witness a significant positive shift in the forecast period of 2025-2034.

Pozelimab is an investigational, fully human monoclonal antibody developed to inhibit complement factor C5, thereby preventing hemolysis, the destruction of red blood cells, that leads to the clinical manifestations of paroxysmal nocturnal hemoglobinuria and other complement-mediated disorders. As an IgG4 antibody, it binds with high affinity to both wild-type and variant forms of C5, effectively blocking their activity. Pozelimab was created using Regeneron's VelocImmune platform, which employs genetically humanized mice to generate optimized fully human antibodies. The therapy is currently in Phase III clinical trials.

Zaltenibart (OMS906) is a leading monoclonal antibody that targets MASP-3, a key enzyme in activating the alternative complement pathway. It is in Phase II development for PNH and complement 3 glomerulopathy. In a Phase I trial involving healthy volunteers, the therapy was well tolerated with no significant safety concerns. OMS906 has received Orphan Drug Designation (ODD) from the FDA for PNH, and two open-label, multicenter trials are currently underway in adults with PNH.

Ruxoprubart is a next-generation humanized monoclonal antibody that specifically targets protein Bb of the alternative complement pathway, distinguishing it from agents like Iptacopan (FABHALTA) due to its lack of binding to Factor B. As a strong inhibitor of the Alternative Pathway, Ruxoprubart showed favorable safety and tolerability in a completed Phase I trial with healthy volunteers. It is now progressing to a Phase II multi-dose study in treatment-naïve PNH patients, representing the first anti-Bb biologic to be tested in this population. The FDA has authorized Phase II trials for multiple conditions, including C3G, atypical hemolytic uremic syndrome (aHUS), and ANCA-associated vasculitis (AAV)—all scheduled to occur in the U.S. Notably, in 2024, Ruxoprubart received FDA Orphan Drug Designation for the treatment of PNH.

The anticipated launch of these emerging paroxysmal nocturnal hemoglobinuria therapies are poised to transform the paroxysmal nocturnal hemoglobinuria market landscape in the coming years. As these cutting-edge paroxysmal nocturnal hemoglobinuria therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

To know more about new treatment for paroxysmal nocturnal hemoglobinuria, visit @ Paroxysmal Nocturnal Hemoglobinuria Management 

Paroxysmal Nocturnal Hemoglobinuria Market Dynamics

The paroxysmal nocturnal hemoglobinuria market dynamics are anticipated to change in the coming years. An improved understanding of the role of uncontrolled complement activation in PNH has significantly advanced its diagnosis and management over the years, driven by a strong industry presence from major pharmaceutical companies such as Alexion (AstraZeneca), Amgen, and Takeda, fostering a highly competitive and innovative treatment landscape. 

The introduction of the C5-targeting monoclonal antibody eculizumab has transformed the natural history of PNH in the past decade, creating opportunities for the development of new biomarkers to assess thrombotic risk. Meanwhile, the emergence of biosimilars like Bkemv and Epysqli, along with evolving pricing strategies and improved reimbursement frameworks, is enhancing affordability and accessibility, particularly in developing regions.

Furthermore, many potential therapies are being investigated for the treatment of paroxysmal nocturnal hemoglobinuria, and it is safe to predict that the treatment space will significantly impact the paroxysmal nocturnal hemoglobinuria market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate is expected to drive the growth of the paroxysmal nocturnal hemoglobinuria market in the 7MM.

However, several factors may impede the growth of the paroxysmal nocturnal hemoglobinuria market. Despite treatment with a C5 inhibitor, patients with conditions like PNH may still suffer from ongoing hemolysis, chronic anemia, transfusion dependence, fatigue, and diminished quality of life, compounded by limited physician awareness of the disease's varied and overlapping symptoms, the lack of extensive multicenter research on diagnostic efficiency, and growing market challenges posed by biosimilars such as Bkemv and Epysqli, which, while enhancing affordability, are intensifying pricing pressures and prompting strategic reassessments by pharmaceutical companies.

Moreover, paroxysmal nocturnal hemoglobinuria treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the paroxysmal nocturnal hemoglobinuria market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the paroxysmal nocturnal hemoglobinuria market growth.

Scope of the Paroxysmal Nocturnal Hemoglobinuria Market Report

• Paroxysmal Nocturnal Hemoglobinuria Therapeutic Assessment: Paroxysmal Nocturnal Hemoglobinuria current marketed and emerging therapies
• Paroxysmal Nocturnal Hemoglobinuria Market Dynamics: Conjoint Analysis of Emerging Paroxysmal Nocturnal Hemoglobinuria Drugs
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL's views, Analyst's views, Paroxysmal Nocturnal Hemoglobinuria Market Access and Reimbursement

Discover more about paroxysmal nocturnal hemoglobinuria drugs in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials

Table of Contents

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