Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market

By Drugs;

Approved Drugs [Eculizumab (Soliris) and Ravulizumab (Ultomiris)] and Pipeline Drugs [APL-2, Nomacopan (Coversin), ACH-4471 and Others]

By Treatment Type;

Complement Inhibitors, Iron Therapy, Bone Marrow Transplantation and Immunosuppressant Drugs

By Route Of Administration;

Injection/Infusion and Oral

By Distribution Channel;

Hospital Pharmacies, Retail Pharmacies and Online Pharmacies

By Geography;

North America, Europe, Asia Pacific, Middle East & Africa and Latin America - Report Timeline (2021 - 2031)
Report ID: Rn129389833 Published Date: August, 2025

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market Overview

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market (USD Million)

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market was valued at USD 4,543.12 million in the year 2024. The size of this market is expected to increase to USD 9,231.84 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 10.7%.


Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market

*Market size in USD million

CAGR 10.7 %


Study Period2025 - 2031
Base Year2024
CAGR (%)10.7 %
Market Size (2024)USD 4,543.12 Million
Market Size (2031)USD 9,231.84 Million
Market ConcentrationLow
Report Pages390
4,543.12
2024
9,231.84
2031

Major Players

  • Alexion Pharmaceuticals, Inc
  • Apellis Pharmaceuticals
  • Ra Pharmaceuticals
  • Novartis AG
  • Alnylam Pharmaceuticals
  • Roche Holdings AG
  • Pfizer Inc
  • Sanofi Genzyme
  • Bristol Myers Squibb
  • Amgen Inc

Market Concentration

Consolidated - Market dominated by 1 - 5 major players

Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market

Fragmented - Highly competitive market without dominant players


The Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market is advancing steadily with increasing diagnosis rates and the adoption of innovative treatment protocols. Driven by awareness campaigns and healthcare improvements, the market has seen a growth momentum exceeding 15% in therapy uptake. This trend is reinforced by the shift toward personalized and precision medicine.

Technological Advancements
Progress in monoclonal antibody therapies has redefined the therapeutic landscape, providing longer-lasting disease control. These therapies have led to a boost of nearly 20% in clinical efficacy. With new treatment modalities minimizing transfusion dependency, the sector is evolving rapidly toward safer, more convenient solutions.

Regulatory and Clinical Developments
Recent drug approvals under orphan designation have significantly impacted market dynamics. The number of patients involved in clinical studies has grown by more than 12%, expediting the introduction of new therapies. Streamlined regulatory frameworks have further contributed to shorter development cycles and broader access.

Future Outlook
Research into novel complement pathway inhibitors and oral therapies is expected to transform the treatment landscape. The industry anticipates a price drop of up to 10% due to biosimilar competition. Continuous innovation and global expansion of patient access programs will be key in shaping long-term market progress.

  1. Introduction
    1. Research Objectives and Assumptions
    2. Research Methodology
    3. Abbreviations
  2. Market Definition & Study Scope
  3. Executive Summary
    1. Market Snapshot, By Drugs
    2. Market Snapshot, By Treatment Type
    3. Market Snapshot, By Route of Administration
    4. Market Snapshot, By Distribution Channel
    5. Market Snapshot, By Region
  4. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market Dynamics
    1. Drivers, Restraints and Opportunities
      1. Drivers
        1. Innovation in treatment options
        2. Growing patient awareness and education
        3. Increasing healthcare expenditure globally
        4. Advancements in diagnostic technologies
      2. Restraints
        1. High cost of treatment
        2. Limited availability of approved therapies
        3. Regulatory challenges in some regions
        4. Potential adverse effects of therapies
      3. Opportunities
        1. Collaborative research and development efforts
        2. Personalized medicine approaches
        3. Development of novel therapeutics
    2. PEST Analysis
      1. Political Analysis
      2. Economic Analysis
      3. Social Analysis
      4. Technological Analysis
    3. Porter's Analysis
      1. Bargaining Power of Suppliers
      2. Bargaining Power of Buyers
      3. Threat of Substitutes
      4. Threat of New Entrants
      5. Competitive Rivalry
  5. Market Segmentation
    1. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market, By Drugs, 2021 - 2031 (USD Million)
      1. Approved Drugs
        1. Eculizumab (Soliris)
        2. Ravulizumab (Ultomiris)
      2. Pipeline Drugs
        1. APL-2
        2. Nomacopan (Coversin)
        3. ACH-4471
      3. Others
    2. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market, By Treatment Type, 2021 - 2031 (USD Million)

      1. Complement Inhibitors

      2. Iron Therapy

      3. Bone Marrow Transplantation

      4. Immunosuppressant Drugs

    3. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market, By Route of Administration, 2021 - 2031 (USD Million)
      1. Injection/Infusion
      2. Oral
    4. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market, By Distribution Channel, 2021 - 2031 (USD Million)
      1. Hospital Pharmacies
      2. Retail Pharmacies
      3. Online Pharmacies
    5. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market, By Geography, 2021 - 2031 (USD Million)
      1. North America
        1. United States
        2. Canada
      2. Europe
        1. Germany
        2. United Kingdom
        3. France
        4. Italy
        5. Spain
        6. Nordic
        7. Benelux
        8. Rest of Europe
      3. Asia Pacific
        1. Japan
        2. China
        3. India
        4. Australia & New Zealand
        5. South Korea
        6. ASEAN (Association of South East Asian Countries)
        7. Rest of Asia Pacific
      4. Middle East & Africa
        1. GCC
        2. Israel
        3. South Africa
        4. Rest of Middle East & Africa
      5. Latin America
        1. Brazil
        2. Mexico
        3. Argentina
        4. Rest of Latin America
  6. Competitive Landscape
    1. Company Profiles
      1. Alexion Pharmaceuticals, Inc
      2. Apellis Pharmaceuticals
      3. Ra Pharmaceuticals
      4. Novartis AG
      5. Alnylam Pharmaceuticals
      6. Roche Holdings AG
      7. Pfizer Inc
      8. Sanofi Genzyme
      9. Bristol Myers Squibb
      10. Amgen Inc
  7. Analyst Views
  8. Future Outlook of the Market