Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market – By Treatment Type (Complement Inhibitors, Iron Therapy, Bone Marrow Transplantation, Immunosuppressant Drugs), Route of Administration, Age Group, End-use – Global Forecast, 2024 ­ 2032

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Size

Paroxysmal Nocturnal Hemoglobinuria Treatment Market size was valued at USD 3.9 billion in 2023 and is expected to grow at a CAGR of 11.2% between 2024 and 2032. Key factors driving the market's growth include the rising prevalence of PNH, which is anticipated to increase the demand for specialized diagnostic techniques. These advancements have led to earlier and more accurate diagnoses, resulting in a larger patient population requiring treatment.
 

For instance, according to a report published by the U.S. Census Bureau, the prevalence of PNH in the U.S. ranged from 12 to 13 cases per 1 million people, with an estimated incidence of 5.7 per 1 million person-years. Thus, the growing advancements in diagnostic techniques and increased awareness of PNH, combined with the availability of better treatment options, have increased the life expectancy of PNH patients, leading to sustained demand for long-term treatment solutions.
 

PNH is a rare blood disease that causes red blood cells to break apart, releasing hemoglobin. The release of hemoglobin causes many of the symptoms associated with PNH. Treatment focuses on managing these symptoms, preventing complications, and improving the quality of life for patients. Major treatment options include complement inhibitors, bone marrow transplantation, blood transfusions, iron supplements, folic acid, and various other approaches.      
 

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Trends

Ongoing innovation in the development of targeted therapies, such as complement inhibitors like eculizumab and ravulizumab, has revolutionized PNH treatment. These therapies effectively reduce hemolysis and improve patient outcomes, leading to higher adoption rates, which are anticipated to propel market growth. Moreover, leading pharmaceutical companies are heavily investing in the research and development of new drugs and therapies for PNH, resulting in a robust pipeline of potential treatments that contribute to market growth. For instance, in April 2024, Voydeya (danicopan) was approved in the U.S. as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with PNH. This approval aims to broaden treatment availability, complementing existing therapies for PNH and contributing to market growth.
 

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Analysis

Based on treatment type, the market is classified into complement inhibitors, iron therapy, immunosuppressant drugs, bone marrow transplantation, and other treatment types. The complement inhibitors segment dominated the market with a revenue of USD 1.4 billion in 2023.
 

• Complement inhibitors such as eculizumab and ravulizumab have shown great effectiveness in reducing hemolysis, preventing blood clots, and improving the quality of life for PNH patients. These strong clinical results are driving their increased use, thereby contributing to market growth.
   
• Ongoing research and development in complement inhibition have introduced next-generation inhibitors with better pharmacokinetics, less frequent dosing, and improved patient compliance, which boosts market growth.
   
• Increasing awareness among healthcare providers about the benefits of complement inhibitors in managing PNH has led to greater adoption of these therapies, driving market growth.
   

Based on the route of administration, the PNH treatment market is segmented into oral and parenteral. The parenteral segment dominated the market with a share of 58.5% in 2023.
 

• Intravenous parenteral administration delivers medication directly into the bloodstream, ensuring a quick onset of action, which is essential for managing acute PNH episodes.
   
• Treatments for PNH, such as eculizumab and ravulizumab, are monoclonal antibodies given through injections or infusions. These large biologic molecules cannot be taken orally because they are too unstable to be absorbed in the digestive system, fueling market growth for parenteral formulations.
   

Based on age group, the PNH treatment market is segmented into pediatric and adult. The adult segment dominated the market and is expected to reach USD 8.6 billion by 2032.
 

• PNH predominantly affects adults, with most diagnoses occurring in individuals between the ages of 30 and 50. The higher prevalence of PNH in this age group creates a larger patient base, driving demand for treatments specifically targeted at adults.
   
• Regulatory bodies such as the FDA and EMA have approved several therapies specifically for adult PNH patients. These approvals have facilitated the availability of effective treatments, encouraging their adoption among adult patients.
   

Based on end-use, the paroxysmal nocturnal hemoglobinuria treatment market is classified into hospitals, specialty clinics, homecare settings, and other end-users. The hospital segment is expected to exhibit a CAGR of 11.6% between 2024-2032.
 

• Hospitals, equipped with advanced diagnostic tools and treatment options, are essential for managing complex conditions like PNH. They often have specialized laboratories for genetic testing, flow cytometry, and other critical diagnostic techniques, ensuring accurate diagnosis and monitoring for PNH patients. As a result, hospitals are the preferred choice for PNH treatment.
   
• Hospitals are typically the first to adopt new therapies for PNH. With ongoing research and clinical trials, they have access to the latest treatments, such as complement inhibitors and other emerging options. This access to advanced treatments makes patients prefer hospital-based care, thereby fostering market growth.
   

In 2023, North America secured a substantial market share of 46.6% in the global PNH treatment market and is expected to dominate throughout the forecast period.
 

• The U.S. and Canada have favorable reimbursement policies for rare disease treatments, including PNH. These policies reduce the financial burden on patients, making expensive therapies more accessible. Insurance coverage for high-cost drugs like eculizumab (Soliris) and ravulizumab (Ultomiris) encourages patients to seek treatment.
   
• The U.S. FDA has implemented mechanisms like Orphan Drug Designation and Fast Track status to speed up the approval process for drugs treating rare diseases, including PNH. These regulatory incentives encourage pharmaceutical companies to invest in developing PNH therapies, leading to faster market growth.
   

Thus, the aforementioned factors contributed to North America's dominance in the paroxysmal nocturnal hemoglobinuria treatment market. The PNH treatment market in the U.S. is expected to exhibit a CAGR of 11.1% between 2024-2032.
 

• The high prevalence of PNH in the U.S. has increased the demand for effective treatments. Greater awareness among healthcare providers and patients has led to better diagnosis rates.
   
• Organizations like the Aplastic Anemia & MDS International Foundation (AA&MDSIF) and the National Organization for Rare Disorders (NORD) play a crucial role in raising awareness about PNH, advocating for patients, and supporting research. Their efforts increase demand for effective treatment options for PNH, thereby boosting market growth.
   

Germany is expected to experience the highest growth rate in the European market.
 

• Germany's Statutory Health Insurance (SHI) system boasts a robust healthcare reimbursement framework. This framework not only covers a wide array of innovative and costly treatments but also promotes the adoption of new therapies. As a result, treatments for PNH become accessible to a broader patient demographic, promoting market growth.
   
• The increase in key strategies adopted by local players in partnership with global pharmaceutical companies enables the early adoption of advanced PNH therapies. These collaborations introduce innovative treatments and help expand market growth.
   

The Asia-Pacific paroxysmal nocturnal hemoglobinuria treatment market is expected to grow at a significant CAGR of 11.7% during the forecast period.
 

• Countries in the Asia-Pacific region are improving healthcare infrastructure and implementing reforms to increase access to advanced therapies. For example, governments are adding treatments for rare diseases like PNH to national health insurance schemes, making these therapies more accessible to the public.
   
• Rising healthcare spending in emerging economies like India and China is boosting the demand for specialized treatments, including PNH. As disposable incomes grow and health insurance coverage expands, more people can afford PNH treatments, contributing to market growth.
   

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Share

The market is characterized by intense competition among key players aiming to expand their market presence. Leading companies are focusing on strategic initiatives such as mergers, acquisitions, and partnerships to strengthen their market positions. These efforts are aimed at enhancing their product and service offerings, expanding their geographic reach, and investing in advanced technologies. The market also witnesses the entry of new players, further intensifying the competitive landscape.
 

Paroxysmal Nocturnal Hemoglobinuria Treatment Market Companies

The company profile section includes both companies that have commercially available drugs in the market as well as those in the clinical phase of development. Prominent players operating in the market include:

• Amgen Inc.
• Apellis Pharmaceuticals, Inc.
• AstraZeneca Plc
• Children's National Hospital
• F. Hoffmann-La Roche Ltd.
• Memorial Sloan Kettering Cancer Center
• Novartis AG
• Pfizer, Inc.
• Regeneron Pharmaceuticals Inc.
• Teva Pharmaceuticals Industries Ltd.
• UCLA Health
• University of Rochester Medical Center
   

Paroxysmal Nocturnal Hemoglobinuria Treatment Industry News:

• In April 2024, Dawn Health and global pharmaceutical company Novartis announced the launch of Ekiva PNH, an innovative digital solution designed for people living with PNH. This helped the company expand its product offerings.
   
• In December 2023, Novartis received FDA approval for Fabhalta (iptacopan), offering superior hemoglobin improvement without the need for transfusions, as the first oral monotherapy for adults with PNH. This approval aimed to broaden treatment availability, complementing existing drug therapies for PNH.
   

The paroxysmal nocturnal hemoglobinuria treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2021 - 2032 for the following segments:

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Market, By Treatment Type

• Complement inhibitors
• Iron therapy
• Immunosuppressant drugs
• Bone marrow transplantation
• Other treatment types

Market, By Route of Administration

• Oral
• Parenteral

Market, By Age Group

• Pediatric
• Adult

Market, By End-use

• Hospitals
• Specialty clinics
• Homecare settings
• Other end-users

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Netherlands
  • Rest of Europe
• Asia Pacific
  • Japan
  • China
  • India
  • Australia
  • South Korea
  • Rest of Asia Pacific
• Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America
• Middle East and Africa
  • South Africa
  • Saudia Arabia
  • UAE
  • Rest of Middle East and Africa