발작성 야간 혈색소뇨증 치료 시장, 기회, 성장 촉진요인, 산업 동향 분석 및 예측(2024-2032년)

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market was valued at USD 3.9 billion in 2023 and is projected to indicate a CAGR of 11.2% from 2024-2032. The rising prevalence of PNH is a primary driver of this market growth, leading to heightened demand for specialized diagnostic techniques. These advancements have facilitated earlier and more precise diagnoses, expanding the patient population in need of treatment. Consequently, as diagnostic techniques advance and awareness of PNH grows, coupled with improved treatment options, the life expectancy of PNH patients has risen. This uptick in life expectancy has resulted in a sustained demand for long-term treatment solutions, further propelling market growth.

The overall paroxysmal nocturnal hemoglobinuria (PNH) treatment industry is classified based on treatment type, route of administration, age group, end-use, and region.

The global PNH treatment market categorizes treatments into complement inhibitors, iron therapy, immunosuppressants, bone marrow transplants, and other methods. In 2023, the complement inhibitors segment led the market, generating revenues of USD 1.4 billion. Complement inhibitors, including eculizumab and ravulizumab, have proven effective in curbing hemolysis, thwarting blood clots, and enhancing the quality of life for PNH patients. These compelling clinical outcomes are fueling their rising adoption, thus bolstering market growth. Research advancements in complement inhibition have birthed next-gen inhibitors, boasting superior less frequent dosing, pharmacokinetics, and heightened patient compliance, all of which amplify market growth.

The global PNH treatment market distinguishes between oral and parenteral routes of administration. In 2023, the parenteral route commanded a dominant 58.5% market share. Intravenous administration ensures medications enter the bloodstream instantly, a crucial factor for addressing acute PNH episodes. Monoclonal antibodies like eculizumab and ravulizumab, administered via injections or infusions, are biologics too large and unstable for oral absorption, underscoring the market's tilt towards parenteral formulations.

North America, with a commanding 46.6% share in 2023, is poised to maintain its dominance in the global PNH treatment market. Favorable reimbursement policies in the U.S. and Canada for rare disease treatments, including PNH, alleviate financial burdens on patients. Such policies enhance accessibility to costly therapies. Insurance backing for premium drugs like eculizumab (Soliris) and ravulizumab (Ultomiris) further motivates patients to pursue treatment. Regulatory mechanisms like Fast Track status and Orphan Drug Designation by the U.S. FDA expedite approvals for rare disease drugs, including those for PNH. Such incentives attract pharmaceutical investments in PNH therapy development, accelerating market growth.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360º synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Growing prevalence of PNH
    • 3.2.1.2 Improved healthcare infrastructure
    • 3.2.1.3 Advancement in treatment options
    • 3.2.1.4 Increasing R&D activities to introduce new therapies for PNH treatment
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory approvals
    • 3.2.2.2 High cost of treatment
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Future market trends
• 3.6 Porter's analysis
• 3.7 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company matrix analysis
• 4.3 Competitive analysis of major market players
• 4.4 Competitive positioning matrix
• 4.5 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Million)

• 5.1 Key trends
• 5.2 Complement inhibitors
• 5.3 Iron therapy
• 5.4 Immunosuppressants drugs
• 5.5 Bone marrow transplantation
• 5.6 Other treatment types

Chapter 6 Market Estimates and Forecast, By Route of administration, 2021 - 2032 ($ Million)

• 6.1 Key trends
• 6.2 Oral
• 6.3 Parenteral

Chapter 7 Market Estimates and Forecast, By Age Group, 2021 - 2032 ($ Million)

• 7.1 Key trends
• 7.2 Pediatric
• 7.3 Adult

Chapter 8 Market Estimates and Forecast, By End-use, 2021 - 2032 ($ Million)

• 8.1 Key trends
• 8.2 Hospitals
• 8.3 Specialty clinics
• 8.4 Homecare settings
• 8.5 Other end-users

Chapter 9 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Million)

• 9.1 Key trends
• 9.2 North America
  • 9.2.1 U.S.
  • 9.2.2 Canada
• 9.3 Europe
  • 9.3.1 Germany
  • 9.3.2 UK
  • 9.3.3 France
  • 9.3.4 Spain
  • 9.3.5 Italy
  • 9.3.6 Netherlands
  • 9.3.7 Rest of Europe
• 9.4 Asia Pacific
  • 9.4.1 Japan
  • 9.4.2 China
  • 9.4.3 India
  • 9.4.4 Australia
  • 9.4.5 South Korea
  • 9.4.6 Rest of Asia Pacific
• 9.5 Latin America
  • 9.5.1 Brazil
  • 9.5.2 Mexico
  • 9.5.3 Rest of Latin America
• 9.6 Middle East and Africa
  • 9.6.1 South Africa
  • 9.6.2 Saudi Arabia
  • 9.6.3 UAE
  • 9.6.4 Rest of Middle East and Africa

Chapter 10 Company Profiles

• 10.1 Amgen Inc.
• 10.2 Apellis Pharmaceuticals, Inc.
• 10.3 AstraZeneca Plc
• 10.4 Children's National Hospital
• 10.5 F. Hoffmann-La Roche Ltd.
• 10.6 Memorial Sloan Kettering Cancer Center
• 10.7 Novartis AG
• 10.8 Pfizer, Inc.
• 10.9 Regeneron Pharmaceuticals Inc.
• 10.10 Teva Pharmaceuticals Industries Ltd.
• 10.11 UCLA Health
• 10.12 University of Rochester Medical Center