Paroxysmal Nocturnal Hemoglobinuria (PNH) Market to Reach $0.84 Billion by 2034, Driven by Increasing Awareness and Novel Therapies

February 2025 – The global Paroxysmal Nocturnal Hemoglobinuria (PNH) market is on track for significant growth, with a projected market size of $0.84 billion by 2034, growing at a compound annual growth rate (CAGR) of 6.73% from 2025 to 2034. According to recent findings by MRFR, the PNH market is expected to see substantial growth, driven by rising awareness, an increasing prevalence of the disease, and the growing demand for novel therapeutic options.

Market Overview

PNH is a rare, life-threatening blood disorder characterized by the destruction of red blood cells. The market for PNH treatment is evolving rapidly, fueled by advancements in treatment options, including monoclonal antibodies, stem cell transplants, and innovative drug administration routes. The global market size was estimated at $0.44 billion in 2024 and is set to increase to $0.47 billion by 2025. The surge in novel therapies and personalized medicine are significant factors contributing to the market expansion.

Market Scope

The scope of the PNH market covers various treatment types, disease severities, patient demographics, and drug administration methods. Key treatment categories include eculizumab therapy, complement inhibitors (C5 inhibitors), stem cell transplant, and bone marrow transplant. The market is also segmented based on disease severity (mild, moderate, and severe PNH), patient age, gender, race/ethnicity, and drug administration routes (intravenous and subcutaneous). The global market is poised for growth due to the rising demand for effective and tailored treatment options.

Segmentation Insights

• Treatment Type: The eculizumab therapy segment is expected to dominate the market, with projections for growth from USD 0.24 billion in 2023 to USD 0.37 billion by 2032. Complement inhibitors are also gaining traction, alongside stem cell and bone marrow transplants.
• Disease Severity: The severe PNH segment holds the largest market share, contributing 60% of the global market revenue. The moderate PNH segment is expected to witness the highest growth rate due to the increasing availability of new therapies.
• Patient Demographics: The disease predominantly affects adults, especially those over 60 years old, with a higher incidence in males. Certain ethnic groups, like African Americans and Ashkenazi Jews, show higher PNH prevalence.
• Drug Administration Route: Subcutaneous (SC) administration is forecasted to experience rapid growth, offering patients greater convenience and flexibility compared to intravenous (IV) administration.

Regional Analysis

The PNH market is segmented into five key regions: North America, Europe, Asia Pacific, South America, and the Middle East and Africa (MEA). North America is currently the largest market, accounting for over 40% of global revenue in 2023. Europe follows closely, while the Asia Pacific region is expected to experience the fastest growth in the coming years, driven by increasing healthcare spending and disease awareness. South America and MEA are expected to grow steadily, as healthcare access and treatments continue to expand.

Key Drivers

• Increasing Prevalence: PNH affects approximately 1 in 100,000 to 1 in 500,000 people worldwide, with a higher prevalence in certain populations such as those of African descent.
• Demand for Novel Therapies: With limited current therapies, the market is shifting towards more effective, safer treatments that address unmet needs. Many new therapies are emerging from clinical pipelines, promising to revolutionize PNH care.
• Government Initiatives: Governments worldwide are increasingly investing in rare disease research and providing funding for PNH treatments, ensuring better access to therapies.

Key Players

The market is witnessing intense activity among major pharmaceutical companies, with several key players making significant contributions to the PNH therapeutic landscape. Leading companies in the market include:

• Alexion Pharmaceuticals, Inc. – Known for its eculizumab (Soliris), which has become a cornerstone treatment for PNH.
• Roche Holding AG – Developer of Heamlibra (emicizumab), another innovative therapeutic option.
• Amgen Inc., Ionis Pharmaceuticals, Inc., and Biogen Inc. – All focusing on advancing PNH therapies and securing a larger market share.

These companies are focused on expanding their global footprint, developing novel therapies, and enhancing treatment access, ensuring their leading positions in the PNH market.

Conclusion

The global PNH market is poised for remarkable growth, driven by the increasing prevalence of the disease, emerging treatment options, and ongoing research and development in the field. As the demand for personalized therapies and more effective treatments rises, the market is expected to experience accelerated growth, with key players striving to meet patient needs with innovative therapies and solutions.

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