DelveInsight’s “Paroxysmal Nocturnal Hemoglobinuria Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Paroxysmal Nocturnal Hemoglobinuria market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Paroxysmal Nocturnal Hemoglobinuria drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Paroxysmal Nocturnal Hemoglobinuria treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Paroxysmal Nocturnal Hemoglobinuria: An Overview
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological disorder that results in episodic intravascular hemolysis, hemoglobinuria, hemolysis, and venous thrombosis. PNH is caused by somatic mutations in PIGA in one or more HSC clones. The gene product of PIGA has required for the biosynthesis of glycosylphosphatidylinositol (GPI) anchors; thus, PIGA mutations lead to a deficiency of GPI-anchored proteins, such as complement decay-accelerating factor (also known as CD55) and CD59 glycoprotein (CD59), which are both complement inhibitors. Erythrocytes lacking these proteins are exquisitely sensitive to complement‐mediated intravascular hemolysis. PNH may occur de novo, but it is also associated with aplastic anemia and evolve into aplastic anemia after an indeterminate amount of time.
Paroxysmal Nocturnal Hemoglobinuria Market Key Facts
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The market size of Paroxysmal Nocturnal Hemoglobinuria in the 7MM was observed to be USD 1123 million in 2021.
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In 2021, the US has the largest Paroxysmal Nocturnal Hemoglobinuria market size of USD 703 million among the 7MM countries.
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Among the EU4 countries, Germany had the highest market size for Paroxysmal Nocturnal Hemoglobinuria in 2021, i.e., USD 90 million. The lowest market size was estimated in Italy with USD 17 million in 2021.
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In Japan, the market size for Paroxysmal Nocturnal Hemoglobinuria was observed to be USD 63 million in 2021.
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In 2021, the total cases of Paroxysmal Nocturnal Hemoglobinuria in the 7MM countries were 12,358 and these cases are anticipated to increase during the study period. The US accounted for 6,043 cases of Paroxysmal Nocturnal Hemoglobinuria in 2021.
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Among the EU4 countries, Germany had the highest number of cases of Paroxysmal Nocturnal Hemoglobinuria in 2021, i.e., 1,355 cases, followed by France which had 851 cases in 2021. On the other hand, Italy had the lowest number of cases of Paroxysmal Nocturnal Hemoglobinuria, i.e. 250 cases in 2021.
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The United Kingdom accounted for 2,569 cases of Paroxysmal Nocturnal Hemoglobinuria in 2021.
Paroxysmal Nocturnal Hemoglobinuria Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Paroxysmal Nocturnal Hemoglobinuria pipeline therapies. It also thoroughly assesses the Paroxysmal Nocturnal Hemoglobinuria market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the market trend for each marketed Paroxysmal Nocturnal Hemoglobinuria drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Paroxysmal Nocturnal Hemoglobinuria Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Paroxysmal Nocturnal Hemoglobinuria epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Paroxysmal Nocturnal Hemoglobinuria epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Paroxysmal Nocturnal Hemoglobinuria Epidemiology, Segmented as –
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Total Diagnosed Prevalence of PNH in the 7MM [2019–2032]
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Gender-specific Prevalence of PNH in the 7MM [2019–2032]
Paroxysmal Nocturnal Hemoglobinuria Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Paroxysmal Nocturnal Hemoglobinuria market or expected to be launched during the study period. The analysis covers the Paroxysmal Nocturnal Hemoglobinuria market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Paroxysmal Nocturnal Hemoglobinuria drugs based on their sale and 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 Paroxysmal Nocturnal Hemoglobinuria 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.
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Paroxysmal Nocturnal Hemoglobinuria Therapeutics Analysis
Primarily, PNH treatment was mostly supportive. Patients were given a blood transfusion, iron supplementation, and erythropoietin stimulating agents for recurrent hemolysis and anemia, and given anti-thrombosis prophylaxis to prevent thrombosis. An allogeneic bone marrow transplant was offered in case of bone marrow complications, which is curative. Complement-mediated hemolysis and chronic dysregulation of the alternative complement pathway are the main convicts of PNH.
Several major pharma and biotech companies are developing therapies for Paroxysmal Nocturnal Haemoglobinuria. Currently, Hoffman-La-Roche is leading the therapeutics market with its Paroxysmal Nocturnal Haemoglobinuria drug candidates in the most advanced stage of clinical development.
“June 09, 2023, Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced that positive results from the global Phase III COMMODORE 1 and 2 studies, evaluating the efficacy and safety of crovalimab, an investigational, novel anti-C5 recycling monoclonal antibody, compared to eculizumab, a current standard of care in paroxysmal nocturnal hemoglobinuria (PNH), were presented at the European Hematology Association (EHA) Hybrid Congress, taking place in Frankfurt, Germany on June 8-11, 2023.”
Paroxysmal Nocturnal Hemoglobinuria Companies Actively Working in the Therapeutics Market Include
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AKARI Therapeutics
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Alexion Pharmaceuticals
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Alnylam Pharmaceuticals
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Amgen
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Apellis Pharmaceuticals
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Arrowhead Pharmaceuticals
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Attune Pharmaceuticals
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Biocad
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BioCryst Pharmaceuticals
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CANbridge
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Hoffmann-La Roche
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MorphoSys
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Novartis
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Novartis Pharmaceuticals
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Ra Pharmaceuticals
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RallyBio
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Regeneron Pharmaceuticals
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Wuhan Createrna Science and Technology
And Many Others
Emerging and Marketed Paroxysmal Nocturnal Hemoglobinuria Therapies Covered in the Report Include:
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Crovalimab: Hoffman-La-Roche
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Pozelimab: Regeneron Pharmaceuticals
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Iptacopan: Novartis
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Danicopan: Alexion Pharmaceuticals
And Many Others
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Paroxysmal Nocturnal Hemoglobinuria Competitive Intelligence Analysis
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 Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Paroxysmal Nocturnal Hemoglobinuria 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 Therapies
12. Paroxysmal Nocturnal Hemoglobinuria Emerging Drugs and Latest Therapeutic Advances
13. Paroxysmal Nocturnal Hemoglobinuria Seven Major Market Analysis
14. Attribute Analysis
15. Paroxysmal Nocturnal Hemoglobinuria Market Outlook (In US, EU5, and Japan)
16. Paroxysmal Nocturnal Hemoglobinuria Companies Active in the Market
17. Paroxysmal Nocturnal Hemoglobinuria Access and Reimbursement Overview
18. KOL Views on the Paroxysmal Nocturnal Hemoglobinuria Market
19. Paroxysmal Nocturnal Hemoglobinuria Market Drivers
20. Paroxysmal Nocturnal Hemoglobinuria Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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