Summary

The 7 major paroxysmal nocturnal hemoglobinuria markets reached a value of US$ 2.9 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 6.9 Billion by 2034, exhibiting a growth rate (CAGR) of 8.29% during 2024-2034.

The paroxysmal nocturnal hemoglobinuria market has been comprehensively analyzed in IMARC's new report titled "Paroxysmal Nocturnal Hemoglobinuria Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Paroxysmal nocturnal hemoglobinuria (PNH) refers to a rare and acquired hematological disorder characterized by the abnormal breakdown of red blood cells (hemolysis) and the presence of hemoglobin in the urine (hemoglobinuria). Some of the common symptoms associated with the ailment are fatigue, weakness, shortness of breath, pale skin, etc. PNH patients are also at an increased risk of thrombosis, which can result in pain, swelling, and organ damage. Various additional indications may include abdominal pain, fatigue, weakness, and, in advanced cases, bone marrow failure, that can lead to decreased production of other blood cells and a higher susceptibility to infections and bleeding problems. The diagnosis of paroxysmal nocturnal hemoglobinuria typically involves a combination of clinical evaluation, laboratory procedures, and specialized testing. Initially, a healthcare professional will review the patient's medical history as well as symptoms and conduct a physical examination. Numerous laboratory tests, such as a complete blood count, may reveal signs of hemolysis, including a low red blood cell count and elevated levels of lactate dehydrogenase (LDH). Flow cytometry is a key diagnostic test for PNH, as it detects the absence or reduced expression of certain GPI-anchored proteins on blood cells. Additionally, further genetic testing may be done to identify the specific mutation in the PIGA gene.

The rising cases of mutations in genes that affect the production of glycosylphosphatidylinositol (GPI) anchors, which are responsible for attaching various proteins to the surface of blood cells, are primarily driving the paroxysmal nocturnal hemoglobinuria market. Apart from this, the widespread adoption of numerous complement inhibitors, including eculizumab and ravulizumab, for reducing intravascular hemolysis, thereby leading to an increase in hemoglobin levels and a decrease in the need for blood transfusions, is also acting as another significant growth-inducing factor. Moreover, several key players are making extensive investments in R&D activities to develop small molecule inhibitors of the complement system, which could provide a simpler, more convenient treatment option compared to injectables. This, in turn, is further creating a positive outlook for the market. Additionally, the emerging popularity of multiparametric flow cytometry, since it offers enhanced sensitivity, specificity, quantification, subtyping, disease monitoring, and treatment guidance in the diagnosis as well as management of the ailment, is expected to drive the paroxysmal nocturnal hemoglobinuria market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the paroxysmal nocturnal hemoglobinuria market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for paroxysmal nocturnal hemoglobinuria and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the paroxysmal nocturnal hemoglobinuria market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the paroxysmal nocturnal hemoglobinuria market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the paroxysmal nocturnal hemoglobinuria market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current paroxysmal nocturnal hemoglobinuria marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the paroxysmal nocturnal hemoglobinuria market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the paroxysmal nocturnal hemoglobinuria market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the paroxysmal nocturnal hemoglobinuria market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the number of prevalent cases (2018-2034) of paroxysmal nocturnal hemoglobinuria across the seven major markets?
What is the number of prevalent cases (2018-2034) of paroxysmal nocturnal hemoglobinuria by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of paroxysmal nocturnal hemoglobinuria by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with paroxysmal nocturnal hemoglobinuria across the seven major markets?
What is the size of the paroxysmal nocturnal hemoglobinuria patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of paroxysmal nocturnal hemoglobinuria?
What will be the growth rate of patients across the seven major markets?

Paroxysmal Nocturnal Hemoglobinuria: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for paroxysmal nocturnal hemoglobinuria drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the paroxysmal nocturnal hemoglobinuria market?
What are the key regulatory events related to the paroxysmal nocturnal hemoglobinuria market?
What is the structure of clinical trial landscape by status related to the paroxysmal nocturnal hemoglobinuria market?
What is the structure of clinical trial landscape by phase related to the paroxysmal nocturnal hemoglobinuria market?
What is the structure of clinical trial landscape by route of administration related to the paroxysmal nocturnal hemoglobinuria market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Paroxysmal Nocturnal Hemoglobinuria - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023) and Forecast (2024-2034)
4.4 Market Overview (2018-2023) and Forecast (2024-2034)
4.5 Competitive Intelligence

5 Paroxysmal Nocturnal Hemoglobinuria - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Paroxysmal Nocturnal Hemoglobinuria - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)

8 Paroxysmal Nocturnal Hemoglobinuria - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Paroxysmal Nocturnal Hemoglobinuria - Unmet Needs

10 Paroxysmal Nocturnal Hemoglobinuria - Key Endpoints of Treatment

11 Paroxysmal Nocturnal Hemoglobinuria - Marketed Products
11.1 List of Paroxysmal Nocturnal Hemoglobinuria Marketed Drugs Across the Top 7 Markets
11.1.1 Empaveli (Pegcetacoplan) - Apellis Pharmaceuticals/Swedish Orphan Biovitrum
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
11.1.2 Soliris (Eculizumab) - Alexion AstraZencea Rare Disease
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Ultomiris (Ravulizumab) - Alexion AstraZencea Rare Disease
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets

Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 Paroxysmal Nocturnal Hemoglobinuria - Pipeline Drugs
12.1 List of Paroxysmal Nocturnal Hemoglobinuria Pipeline Drugs Across the Top 7 Markets
12.1.1 Crovalimab - Chugai Pharmaceutical/Roche
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 Danicopan - Achillion Pharmaceuticals
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 Iptacopan - Novartis Pharmaceuticals
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Pozelimab - Regeneron Pharmaceuticals
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 NM8074 - NovelMed Therapeutics
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.

13. Paroxysmal Nocturnal Hemoglobinuria - Attribute Analysis of Key Marketed and Pipeline Drugs

14. Paroxysmal Nocturnal Hemoglobinuria – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events

15 Paroxysmal Nocturnal Hemoglobinuria - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023)
15.2.2.2 Market Forecast by Therapies (2024-2034)
15.3 Market Scenario - United States
15.3.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023)
15.3.2.2 Market Forecast by Therapies (2024-2034)
15.3.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023)
15.4.2.2 Market Forecast by Therapies (2024-2034)
15.4.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023)
15.5.2.2 Market Forecast by Therapies (2024-2034)
15.5.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023)
15.6.2.2 Market Forecast by Therapies (2024-2034)
15.6.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023)
15.7.2.2 Market Forecast by Therapies (2024-2034)
15.7.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023)
15.8.2.2 Market Forecast by Therapies (2024-2034)
15.8.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Paroxysmal Nocturnal Hemoglobinuria - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Paroxysmal Nocturnal Hemoglobinuria - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023)
15.9.2.2 Market Forecast by Therapies (2024-2034)
15.9.3 Paroxysmal Nocturnal Hemoglobinuria - Access and Reimbursement Overview

16 Paroxysmal Nocturnal Hemoglobinuria - Recent Events and Inputs From Key Opinion Leaders

17 Paroxysmal Nocturnal Hemoglobinuria Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Paroxysmal Nocturnal Hemoglobinuria Market – Strategic Recommendations

19 Appendix