DelveInsight’s “Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology And Market Forecast – 2034” report delivers an in-depth understanding of the Paroxysmal Nocturnal Hemoglobinuria treatment market, historical and forecasted epidemiology, as well as market trends across the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The report comprehensively evaluates the current treatment landscape, emerging therapies, market share of individual treatments, and Paroxysmal Nocturnal Hemoglobinuria market forecasts through 2034, providing crucial insights for stakeholders in the Paroxysmal Nocturnal Hemoglobinuria therapeutic area.
According to DelveInsight’s analysis, the Paroxysmal Nocturnal Hemoglobinuria treatment market is projected to grow substantially during the forecast period (2025-2034), driven by enhanced understanding of disease mechanisms, complement pathway inhibition strategies, and personalized medicine approaches.
Market Size and Geographic Distribution
According to DelveInsight’s estimates, the Paroxysmal Nocturnal Hemoglobinuria Treatment Market Size in the 7MM was more than USD 1.3 billion in 2024, which is expected to increase by 2034 during the forecast period (2025–2034). The United States accounted for the highest Paroxysmal Nocturnal Hemoglobinuria Market Size among the 7MM, representing around 85% of the total market size, with more than USD 1 billion in 2024.
Among EU4 and the UK, the United Kingdom accounted for the highest paroxysmal nocturnal hemoglobinuria market size in 2024, followed by Germany, while Italy accounted for the lowest market share.
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Epidemiology Insights
The paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were approximately 16K cases in the 7MM in 2024, which will increase by 2034. In the 7MM, the highest number of Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were observed in the US, with around 8K cases in 2024.
DelveInsight’s gender-specific analysis revealed that in the US, females were more affected by paroxysmal nocturnal hemoglobinuria than males in 2024, with a 60% to 40% distribution. Furthermore, the highest number of age-specific cases was recorded for the 35-54 age group, followed by the age group of 55-74 years.
Among EU4 and the UK, the United Kingdom had the highest Paroxysmal Nocturnal Hemoglobinuria Diagnosed Prevalent Cases, accounting for more than 50% of cases among European countries in 2024.
Discover evolving trends in the Paroxysmal Nocturnal Hemoglobinuria patient pool forecasts @ Paroxysmal Nocturnal Hemoglobinuria Epidemiological Analysis.
Current Treatment Landscape
The paroxysmal nocturnal hemoglobinuria management includes complement inhibition therapy, with paroxysmal nocturnal hemoglobinuria drugs like SOLIRIS (Alexion Pharmaceuticals), ULTOMIRIS (AstraZeneca), and EMPAVELI (Apellis Pharmaceuticals) approved by the FDA and considered the gold standard. Treatment with a C5 inhibitor is the standard for paroxysmal nocturnal hemoglobinuria patients.
ULTOMIRIS (ravulizumab) by AstraZeneca (Alexion Pharmaceuticals) is a complement inhibitor designed to inhibit a specific aspect of the complement component of the immune system. The US FDA approved ULTOMIRIS in December 2018, followed by European Union authorization in July 2019, and Japan’s MHLW approval in June 2019.
EMPAVELI/ASPAVELI (pegcetacoplan) by Apellis Pharmaceuticals was the first paroxysmal nocturnal hemoglobinuria treatment bound to complement protein C3, representing a significant advancement in the treatment landscape. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan), VOYDEYA (danicopan), and PIASKY (crovalimab) aim to offer improved treatment with fewer side effects.
Emerging Pipeline Therapies
According to DelveInsight’s analysis, the paroxysmal nocturnal hemoglobinuria pipeline shows promising development with several novel treatments in late-stage clinical trials. Among the emerging therapies, Pozelimab appears to be the drug that can potentially transform the paroxysmal nocturnal hemoglobinuria market.
Pozelimab (REGN3918) + Cemdisiran by Regeneron Pharmaceuticals/Alnylam Pharmaceuticals is an investigational, fully human monoclonal antibody designed to block complement factor C5. In August 2019, Regeneron’s Pozelimab was granted Orphan Drug Designation by the FDA for paroxysmal nocturnal hemoglobinuria treatment. In December 2024, the company announced positive updated data from a Phase III study of pozelimab in combination with cemdisiran against ravulizumab in patients with Paroxysmal Nocturnal Hemoglobinuria.
Zaltenibart (OMS906) by Omeros Corporation is a humanized monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the alternative pathway of complement. This therapy has received Orphan Drug Designation and is currently in Phase II development.
Ruxoprubart (NM8074) by NovelMed Therapeutics is another promising therapy in Phase II development that has also received Orphan Drug Designation for PNH treatment.
Recent Advancements
BKEMV (eculizumab-aeeb), developed by Amgen, was approved in May 2024, is the first interchangeable biosimilar to SOLIRIS (eculizumab). It is approved for the same indications as Soliris, including paroxysmal nocturnal hemoglobinuria
PIASKY (crovalimab-akkz), a C5 inhibitor developed by Roche, received FDA approval in June 2024. It is indicated for adult and pediatric patients aged 13 and older, weighing at least 40 kg, with PNH. PIASKY is designed for subcutaneous self-administration monthly, following an initial intravenous loading dose and weekly subcutaneous loading doses.
EPYSQLI (eculizumab-aagh), another eculizumab biosimilar developed by Teva Pharmaceuticals, was approved by the FDA in July 2024. It is approved for the PNH treatment and atypical hemolytic uremic syndrome. Clinical studies have shown EPYSQLI to be highly similar to SOLIRIS in terms of safety, purity, and potency.
Discover recent advancements in the Paroxysmal Nocturnal Hemoglobinuria landscape @ Paroxysmal Nocturnal Hemoglobinuria Recent Developments.
Market Outlook and Future Prospects
The market growth is mainly driven by the rising awareness about the disease and supporting government policies, funds, approvals, and emerging research. The future approvals of paroxysmal nocturnal hemoglobinuria therapies are moving toward personalized patient-centered care with better options to reduce the frequency and self-administer medication.
Auto-injections of subcutaneous compounds and oral drugs will increase patient autonomy, and compliance will become critical. Substantial progress in the biology and paroxysmal nocturnal hemoglobinuria treatment has occurred over the past two decades, making PNH a model for progress in precision medicine.
However, there are still many unmet needs in the Paroxysmal Nocturnal Hemoglobinuria therapeutic landscape. Safety concerns are associated with current treatments, including the risk of meningococcal infections and the persistence of extravascular hemolysis in some individuals treated with complement inhibitors.
Looking ahead, the Paroxysmal Nocturnal Hemoglobinuria market is expected to witness continued innovation driven by enhanced understanding of disease mechanisms and the development of targeted therapies. The ongoing clinical trials hold promise in addressing the unmet need for approved therapies in Paroxysmal Nocturnal Hemoglobinuria treatment, ensuring better treatment options in the upcoming years.
Table of Contents
1. Key Insights
2. Executive Summary of Paroxysmal Nocturnal Hemoglobinuria
3. Competitive Intelligence Analysis for Paroxysmal Nocturnal Hemoglobinuria
4. Paroxysmal Nocturnal Hemoglobinuria Market Overview at a Glance
5. Paroxysmal Nocturnal Hemoglobinuria: Disease Background and Overview
6. Paroxysmal Nocturnal Hemoglobinuria Patient Journey
7. Paroxysmal Nocturnal Hemoglobinuria Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Paroxysmal Nocturnal Hemoglobinuria Unmet Needs
10. Key Endpoints of Paroxysmal Nocturnal Hemoglobinuria Treatment
11. Paroxysmal Nocturnal Hemoglobinuria Marketed Products
12. Paroxysmal Nocturnal Hemoglobinuria Emerging Therapies
13. Paroxysmal Nocturnal Hemoglobinuria: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Paroxysmal Nocturnal Hemoglobinuria
17. KOL Views
18. Paroxysmal Nocturnal Hemoglobinuria Market Drivers
19. Paroxysmal Nocturnal Hemoglobinuria Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight
Paroxysmal Nocturnal Hemoglobinuria pipeline insight provides comprehensive insights about the PNH pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the Paroxysmal Nocturnal Hemoglobinuria companies, including Hoffmann-La Roche, Apellis Pharmaceuticals, Regeneron Pharmaceuticals, Biocad, AKARI Therapeutics, Alexion Pharmaceuticals, Novartis Pharmaceuticals, Amgen, BioCryst Pharmaceuticals, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wuhan Createrna Science and Technology, CANbridge, Attune Pharmaceuticals, and RallyBio, among others.
About DelveInsight
DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.
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