Industry Outlook

The paroxysmal nocturnal hemoglobinuria (PNH) is currently valued at US$ 3,163.2 Mn in 2021 and is set to reach US$ 8,380.2 Mn by 2033 projecting stunning growth at a compounded annual growth rate (CAGR) of 11.7% during the forecast period from 2025 to 2033. PNH is a rare disease wherein red blood cells are reported to burst prematurely. It has a global incidence rate of 1.3 cases per million people. The high treatment cost and mortality rate associated with stem cell transplant procedures has increased the popularity of monoclonal antibodies as the most effective treatment molecule for the treatment of PNH. Presently, 16 drug molecules are being investigated under different stages of clinical trial studies which suggests strong drug pipeline during the forecast period. 

"Excellent drug safety and efficacy reported during clinical trial studies and strong product pipeline during the forecast period increase the popularity of monoclonal antibody as first-line therapy for PNH"

According to the research findings brought forward by the International Bone Marrow Transplant Registry, hematopoietic stem cell transplantation employing allogeneic donors is the only curative option for patients suffering with paroxysmal nocturnal hemoglobinuria (PNH). However, this treatment module is associated with high mortality rate of 40 to 50%. Eculizumab (Soliris) developed by Alexion Pharmaceuticals, Inc., since its approval in 2007, has revolutionized the treatment regimen for PNH. It is the only licensed drug approved by the USFDA and EMA for the treatment of hemolysis associated with paroxysmal nocturnal hemoglobinuria (PNH). It is basically an anti complement monoclonal antibody which specifically targets the C5 human complement protein and inactivates it thereby increasing the survival rate of PNH patients. Excellent drug safety and efficacy reported in clinical studies and strong product pipeline during the forecast period will increase the popularity of monoclonal antibody as the first line therapy for PNH. 

"Presence of major players and affordable reimbursement scenario together drive the market growth in North America"

North America presently represents 35% market share and is spearheading the regional segment paroxysmal nocturnal hemoglobinuria (PNH) market. As per the research citing brought forward by National Institute of Health (NIH), approximately 5,000 people suffer with the disease in the United States. Affordable reimbursement scenario and presence of major players such as Amgen, Inc., Alexion Pharmaceuticals, Roche, Regeneron Pharmaceuticals etc. together drive the market growth in North America region. Europe holds 28% market growth and trails behind North America primarily due to high prevalence rate. According to the statistics provided by National Organization for Rare Disorders (NORD), the prevalence rate of PNH is 15.9 per million people in Europe. European Medical Agency has classified PNH as an orphan disease and has provided fast track designation and orphan drug status for novel drugs developed for its treatment. Asia Pacific accounts for 14.5% market growth owing to the rising public health awareness related to PNH and developing healthcare infrastructure to provide affordable treatment pertaining to PNH.

Historical & Forecast Period

This study report represents analysis of each segment from 2023 to 2033 considering 2024 as the base year. Compounded Annual Growth Rate (CAGR) for each of the respective segments estimated for the forecast period of 2025 to 2033.

The current report comprises of quantitative market estimations for each micro market for every geographical region and qualitative market analysis such as micro and macro environment analysis, market trends, competitive intelligence, segment analysis, porters five force model, top winning strategies, top investment markets, emerging trends and technological analysis, case studies, strategic conclusions and recommendations and other key market insights.

Research Methodology

The complete research study was conducted in three phases, namely: secondary research, primary research, and expert panel review. key data point that enables the estimation of Paroxysmal Nocturnal Hemoglobinuria market are as follows:

• Research and development budgets of manufacturers and government spending
• Revenues of key companies in the market segment
• Number of end users and consumption volume, price and value.
• Geographical revenues generate by countries considered in the report
• Micro and macro environment factors that are currently influencing the Paroxysmal Nocturnal Hemoglobinuria market and their expected impact during the forecast period.

Market forecast was performed through proprietary software that analyzes various qualitative and quantitative factors. Growth rate and CAGR were estimated through intensive secondary and primary research. Data triangulation across various data points provides accuracy across various analyzed market segments in the report. Application of both top down and bottom-up approach for validation of market estimation assures logical, methodical and mathematical consistency of the quantitative data.

Key questions answered in this report

• What are the key micro and macro environmental factors that are impacting the growth of Paroxysmal Nocturnal Hemoglobinuria market?
• What are the key investment pockets with respect to product segments and geographies currently and during the forecast period?
• Estimated forecast and market projections up to 2033.
• Which segment accounts for the fastest CAGR during the forecast period?
• Which market segment holds a larger market share and why?
• Are low and middle-income economies investing in the Paroxysmal Nocturnal Hemoglobinuria market?
• Which is the largest regional market for Paroxysmal Nocturnal Hemoglobinuria market?
• What are the market trends and dynamics in emerging markets such as Asia Pacific, Latin America, and Middle East & Africa?
• Which are the key trends driving Paroxysmal Nocturnal Hemoglobinuria market growth?
• Who are the key competitors and what are their key strategies to enhance their market presence in the Paroxysmal Nocturnal Hemoglobinuria market worldwide?