발작성 야간혈색소뇨증 치료 세계 시장 규모, 점유율, 동향 분석 보고서 : 치료법별, 지역별, 부문별(2025-2030년)

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Growth & Trends:

The global paroxysmal nocturnal hemoglobinuria (PNH) treatment market size is expected to reach USD 9.96 billion by 2030, registering a CAGR of 9.6% from 2025 to 2030, according to a new report by Grand View Research, Inc. Several factors such as rise in the number of blood and bone marrow related disorders, an increase in the geriatric population, wider adoption of novel therapeutics, and the emergence of biologics are driving the market.

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, chronic hematologic disorder. It is an ultra-rare disease with approximately 8,000 to 10,000 cases reported in North America and Europe. The life-threatening disease often manifests in three forms: thrombosis, persistent intravascular hemolysis, and pancytopenia. Although the information on its prevalence is scarce, PNH is estimated to affect more than 20,000 people worldwide with an incidence of about 1.3 per million annually and a prevalence of around 16 per million. PNH affects men and women equally. Although physical symptoms can begin at any age, they usually develop between 30-40 years of age.

Novel treatments including monoclonal antibodies, recombinant proteins, and cyclic peptides are gaining penetration in developed markets. However, the high cost of therapy with Soliris and other emerging pipeline drugs presents a barrier in adoption in developing regions. Blood transfusion and supportive care are anticipated to lose momentum during the forecast period due to adverse side effects and suboptimal efficacy.

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Report Highlights:

• An estimated 5,000 - 6,000 people in the U.S. suffer from PNH, with approximately 450 new cases being reported annually
• U.S. dominated the global PNH treatment market dominated with a significant revenue share in 2024 due to the high cost of treatment in the U.S., potential approval of a range of pipeline drugs, and adoption of novel therapeutics
• Treatment with medication dominated the PNH market with a share of more than 85.3% in 2024. Apart from drugs, the stem cell transplant segment is projected to witness double-digit growth through the forecast period
• Currently, 16 products are under clinical evaluation for PNH treatment in various stages of development. Promising pipeline candidates such as ALXN1210, Coversin, APL2, and RA101495 offer significant commercial potential post regulatory approval.

Table of Contents

Chapter 1. Methodology and Scope

• 1.1. Market Segmentation & Scope
• 1.2. Segment Definitions
  • 1.2.1. Treatment
• 1.3. Estimates and Forecast Timeline
• 1.4. Research Methodology
• 1.5. Information Procurement
  • 1.5.1. Purchased Database
  • 1.5.2. GVR's Internal Database
  • 1.5.3. Secondary Sources
  • 1.5.4. Primary Research
• 1.6. Information Analysis
  • 1.6.1. Data Analysis Models
• 1.7. Market Formulation & Data Visualization
• 1.8. Model Details
  • 1.8.1. Commodity Flow Analysis
• 1.9. List of Secondary Sources
• 1.10. Objectives

Chapter 2. Executive Summary

• 2.1. Market Snapshot
• 2.2. Segment Snapshot
• 2.3. Competitive Landscape Snapshot

Chapter 3. Paroxysmal Nocturnal Hemoglobinuria Treatment Market Variables, Trends, & Scope

• 3.1. Market Lineage Outlook
• 3.2. Market Dynamics
  • 3.2.1. Market Driver Analysis
  • 3.2.2. Market Restraint Analysis
• 3.3. Business Environment Analysis
  • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
    • 3.3.1.1. Supplier Power
    • 3.3.1.2. Buyer Power
    • 3.3.1.3. Substitution Threat
    • 3.3.1.4. Threat of New Entrants
    • 3.3.1.5. Competitive Rivalry
  • 3.3.2. PESTLE Analysis

Chapter 4. Paroxysmal Nocturnal Hemoglobinuria Treatment Market: Treatment Business Analysis

• 4.1. Treatment Market Share, 2024 & 2030
• 4.2. Treatment Segment Dashboard
• 4.3. Market Size & Forecasts and Trend Analysis, by Treatment, 2018 to 2030 (USD Billion)
• 4.4. Medication
  • 4.4.1. Medication Market, 2018 - 2030 (USD Billion)
• 4.5. Stem Cell Transplant
  • 4.5.1. Stem Cell Transplant Market, 2018 - 2030 (USD Billion)
• 4.6. Blood Transfusion
  • 4.6.1. Blood Transfusion Market, 2018 - 2030 (USD Billion)
• 4.7. Other Treatment Types
  • 4.7.1. Other Treatment Types Market, 2018 - 2030 (USD Billion)

Chapter 5. Paroxysmal Nocturnal Hemoglobinuria Treatment Market: Regional Estimates & Trend Analysis

• 5.1. Regional Market Share Analysis, 2024 & 2030
• 5.2. Regional Market Dashboard
• 5.3. Market Size, & Forecasts Trend Analysis, 2018 to 2030:
• 5.4. North America
  • 5.4.1. North America Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 5.4.2. U.S.
    • 5.4.2.1. Key Country Dynamics
    • 5.4.2.2. Regulatory Framework
    • 5.4.2.3. Competitive Insights
    • 5.4.2.4. U.S. Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.4.3. Canada
    • 5.4.3.1. Key Country Dynamics
    • 5.4.3.2. Regulatory Framework
    • 5.4.3.3. Competitive Insights
    • 5.4.3.4. Canada Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.4.4. Mexico
    • 5.4.4.1. Key Country Dynamics
    • 5.4.4.2. Regulatory Framework
    • 5.4.4.3. Competitive Insights
    • 5.4.4.4. Mexico Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 5.5. Europe
  • 5.5.1. Europe Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 5.5.2. UK
    • 5.5.2.1. Key Country Dynamics
    • 5.5.2.2. Regulatory Framework
    • 5.5.2.3. Competitive Insights
    • 5.5.2.4. UK Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.3. Germany
    • 5.5.3.1. Key Country Dynamics
    • 5.5.3.2. Regulatory Framework
    • 5.5.3.3. Competitive Insights
    • 5.5.3.4. Germany Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.4. France
    • 5.5.4.1. Key Country Dynamics
    • 5.5.4.2. Regulatory Framework
    • 5.5.4.3. Competitive Insights
    • 5.5.4.4. France Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.5. Italy
    • 5.5.5.1. Key Country Dynamics
    • 5.5.5.2. Regulatory Framework
    • 5.5.5.3. Competitive Insights
    • 5.5.5.4. Italy Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.6. Spain
    • 5.5.6.1. Key Country Dynamics
    • 5.5.6.2. Regulatory Framework
    • 5.5.6.3. Competitive Insights
    • 5.5.6.4. Spain Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.7. Norway
    • 5.5.7.1. Key Country Dynamics
    • 5.5.7.2. Regulatory Framework
    • 5.5.7.3. Competitive Insights
    • 5.5.7.4. Norway Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.8. Denmark
    • 5.5.8.1. Key Country Dynamics
    • 5.5.8.2. Regulatory Framework
    • 5.5.8.3. Competitive Insights
    • 5.5.8.4. Denmark Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.5.9. Sweden
    • 5.5.9.1. Key Country Dynamics
    • 5.5.9.2. Regulatory Framework
    • 5.5.9.3. Competitive Insights
    • 5.5.9.4. Sweden Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
• 5.6. Asia Pacific
  • 5.6.1. Asia Pacific Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.2. Japan
    • 5.6.2.1. Key Country Dynamics
    • 5.6.2.2. Regulatory Framework
    • 5.6.2.3. Competitive Insights
    • 5.6.2.4. Japan Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.3. China
    • 5.6.3.1. Key Country Dynamics
    • 5.6.3.2. Regulatory Framework
    • 5.6.3.3. Competitive Insights
    • 5.6.3.4. China Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.4. India
    • 5.6.4.1. Key Country Dynamics
    • 5.6.4.2. Regulatory Framework
    • 5.6.4.3. Competitive Insights
    • 5.6.4.4. India Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.5. South Korea
    • 5.6.5.1. Key Country Dynamics
    • 5.6.5.2. Regulatory Framework
    • 5.6.5.3. Competitive Insights
    • 5.6.5.4. South Korea Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.6. Australia
    • 5.6.6.1. Key Country Dynamics
    • 5.6.6.2. Regulatory Framework
    • 5.6.6.3. Competitive Insights
    • 5.6.6.4. Australia Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.6.7. Thailand
    • 5.6.7.1. Key Country Dynamics
    • 5.6.7.2. Regulatory Framework
    • 5.6.7.3. Competitive Insights
    • 5.6.7.4. Thailand Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 5.7. Latin America
  • 5.7.1. Latin America Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 5.7.2. Brazil
    • 5.7.2.1. Key Country Dynamics
    • 5.7.2.2. Regulatory Framework
    • 5.7.2.3. Competitive Insights
    • 5.7.2.4. Brazil Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.7.3. Argentina
    • 5.7.3.1. Key Country Dynamics
    • 5.7.3.2. Regulatory Framework
    • 5.7.3.3. Competitive Insights
    • 5.7.3.4. Argentina Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
• 5.8. MEA
  • 5.8.1. MEA Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, by Country, 2018 - 2030 (USD Billion)
  • 5.8.2. South Africa
    • 5.8.2.1. Key Country Dynamics
    • 5.8.2.2. Regulatory Framework
    • 5.8.2.3. Competitive Insights
    • 5.8.2.4. South Africa Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.8.3. Saudi Arabia
    • 5.8.3.1. Key Country Dynamics
    • 5.8.3.2. Regulatory Framework
    • 5.8.3.3. Competitive Insights
    • 5.8.3.4. Saudi Arabia Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.8.4. UAE
    • 5.8.4.1. Key Country Dynamics
    • 5.8.4.2. Regulatory Framework
    • 5.8.4.3. Competitive Insights
    • 5.8.4.4. UAE Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)
  • 5.8.5. Kuwait
    • 5.8.5.1. Key Country Dynamics
    • 5.8.5.2. Regulatory Framework
    • 5.8.5.3. Competitive Insights
    • 5.8.5.4. Kuwait Paroxysmal Nocturnal Hemoglobinuria Treatment Market Estimates and Forecasts, 2018 - 2030 (USD Billion)

Chapter 6. Competitive Landscape

• 6.1. Participant Overview
• 6.2. Company Market Position Analysis
• 6.3. Company Categorization
• 6.4. Strategy Mapping
• 6.5. Company Profiles/Listing
  • 6.5.1. Alexion Pharmaceuticals, Inc.
    • 6.5.1.1. Overview
    • 6.5.1.2. Financial Performance
    • 6.5.1.3. Treatment Benchmarking
    • 6.5.1.4. Strategic Initiatives
  • 6.5.2. Apellis Pharmaceuticals
    • 6.5.2.1. Overview
    • 6.5.2.2. Financial Performance
    • 6.5.2.3. Treatment Benchmarking
    • 6.5.2.4. Strategic Initiatives
  • 6.5.3. Novartis AG
    • 6.5.3.1. Overview
    • 6.5.3.2. Financial Performance
    • 6.5.3.3. Treatment Benchmarking
    • 6.5.3.4. Strategic Initiatives
  • 6.5.4. F. Hoffmann-La Roche Ltd
    • 6.5.4.1. Overview
    • 6.5.4.2. Financial Performance
    • 6.5.4.3. Treatment Benchmarking
    • 6.5.4.4. Strategic Initiatives
  • 6.5.5. Genentech, Inc.
    • 6.5.5.1. Overview
    • 6.5.5.2. Financial Performance
    • 6.5.5.3. Treatment Benchmarking
    • 6.5.5.4. Strategic Initiatives
  • 6.5.6. BioCryst Pharmaceuticals, Inc.
    • 6.5.6.1. Overview
    • 6.5.6.2. Financial Performance
    • 6.5.6.3. Treatment Benchmarking
    • 6.5.6.4. Strategic Initiatives
  • 6.5.7. Regeneron Pharmaceuticals Inc.
    • 6.5.7.1. Overview
    • 6.5.7.2. Financial Performance
    • 6.5.7.3. Treatment Benchmarking
    • 6.5.7.4. Strategic Initiatives
  • 6.5.8. Sanofi
    • 6.5.8.1. Overview
    • 6.5.8.2. Financial Performance
    • 6.5.8.3. Treatment Benchmarking
    • 6.5.8.4. Strategic Initiatives
  • 6.5.9. Pfizer Inc.
    • 6.5.9.1. Overview
    • 6.5.9.2. Financial Performance
    • 6.5.9.3. Treatment Benchmarking
    • 6.5.9.4. Strategic Initiatives
  • 6.5.10. Amgen Inc.
    • 6.5.10.1. Overview
    • 6.5.10.2. Financial Performance
    • 6.5.10.3. Treatment Benchmarking
    • 6.5.10.4. Strategic Initiatives