Paroxysmal Nocturnal Hemoglobinuria Treatment Market To Reach $9.96 Billion By 2030

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.

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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.

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Segmentation

Grand View Research has segmented the global paroxysmal nocturnal hemoglobinuria treatment market report based on treatment, and region:

Paroxysmal Nocturnal Hemoglobinuria Treatment Outlook (Revenue, USD Billion, 2018 - 2030)

• Medication

• Stem Cell Transplant

• Blood Transfusion

• Other Treatment Types

Paroxysmal Nocturnal Hemoglobinuria Treatment Regional Outlook (Revenue, USD Billion, 2018 - 2030)

• North America

  • U.S.       

  • Canada

  • Mexico

• Europe

  • UK

  • Germany

  • France 

  • Italy

  • Spain

  • Denmark

  • Sweden

  • Norway

• Asia Pacific

  • Japan

  • China

  • India

  • Australia

  • South Korea

  • Thailand

• Latin America

  • Brazil

  • Argentina

• Middle East & Africa

  • South Africa

  • Saudi Arabia

  • UAE

  • Kuwait 

List of Key Players in the Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market

• Alexion Pharmaceuticals, Inc.

• Apellis Pharmaceuticals

• Novartis AG

•  F. Hoffmann-La Roche Ltd

• Genentech, Inc.

• BioCryst Pharmaceuticals, Inc.

• Regeneron Pharmaceuticals Inc.

• Sanofi

• Pfizer Inc.

• Amgen Inc.