Paroxysmal Nocturnal Hemoglobinuria Treatment Market Gains Strength from Research Grants

The global Paroxysmal Nocturnal Hemoglobinuria Treatment Market was valued at USD 5.75 billion in 2024. Projections indicate growth to USD 9.96 billion by 2030, corresponding to a compound annual growth rate (CAGR) of 9.6% during 2025-2030. The increasing reported incidence of PNH is largely attributed to greater disease awareness and enhanced diagnostic capabilities.

Advances in treatment are evident: targeted therapies—especially complement inhibitors like eculizumab and ravulizumab—play a central role in reducing hemolysis and its complications. Next-generation treatments are being developed with improved safety profiles and more convenient administration schedules. On the diagnostic front, improvements such as flow cytometry, genetic testing, and biomarkers are increasing both accuracy and early detection, allowing treatments to begin sooner and improving patient outcomes.

Key Market Trends & Insights

• In 2024, North America held the largest share of revenue, accounting for 40.4% of the global PNH treatment market.
• Across regions, Asia Pacific is forecast to show the fastest growth, with a projected CAGR of 10.3% over the forecast period.
• When categorised by treatment type, medication dominated in 2024, contributing 85.3% of the total revenue.

Market Size & Forecast

• 2024 market size: USD 5.75 billion.
• Forecast for 2030: USD 9.96 billion.
• Growth rate: CAGR of 9.6% between 2025 and 2030.
• Regionally, North America was largest in 2024
• Asia Pacific is expected to grow the fastest.

With increasing numbers of identified PNH cases—thanks to better awareness and diagnostic improvements—demand for treatments is set to rise. Novel medication therapies and expanding acceptance of stem cell transplantation are important drivers. Efforts to improve diagnosis and treatment timing are expected to enhance disease management and support market growth.

Order a free sample PDF of the Paroxysmal Nocturnal Hemoglobinuria Treatment Market Intelligence Study, published by Grand View Research.

These firms are leveraging strategic initiatives such as mergers, acquisitions, and launching new products to deepen their presence and respond to changing healthcare needs. For example, Alexion provides Soliris (eculizumab) as the first approved therapy for PNH, along with ULTOMIRIS (ravulizumab) for less frequent dosing, and a new oral add-on therapy, Voydeya (danicopan), aimed at addressing extravascular hemolysis in patients already on complement inhibitors. Apellis is known for EMPAVELI (pegcetacoplan), the first targeted complement C3 inhibitor approved for adults with PNH, whether treatment-naïve or switching from C5 inhibitors. It also offers support through a program that assists patients with insurance and education.

Key Companies List

• Alexion Pharmaceuticals, Inc.
• Apellis Pharmaceuticals
• Novartis AG
• Hoffmann-La Roche Ltd
• Genentech, Inc.
• BioCryst Pharmaceuticals, Inc.
• Regeneron Pharmaceuticals Inc.
• Sanofi
• Pfizer Inc.
• Amgen Inc.

Conclusion

Over the period from 2025 to 2030, the PNH treatment market is positioned for strong growth, nearly doubling in size from USD 5.75 billion to USD 9.96 billion. This expansion is driven by enhanced diagnostics, increased disease recognition, and the growing availability of advanced medication and treatment options. While North America presently maintains the bulk of revenues, Asia Pacific is expected to lead in growth rate. Key pharmaceutical companies are innovating rapidly—both in therapies and delivery methods—to meet unmet needs and improve patient experience.

Explore Horizon Databook – The world’s most expansive market intelligence platform developed by Grand View Research.