The global paroxysmal nocturnal hemoglobinuria (PNH) market accounted for USD 4.01 billion in 2023 and is expected to reach at USD 14.68 billion by 2034 with a CAGR of 12.53% during the forecast period 2024-2034. The market will grow as a result of factors including rising PNH prevalence, rising awareness, and diagnosis, improvements in drug research and development, and supportive regulatory environments.
The market is growing due to the growing pipeline of innovative PNH treatment medicines, such as gene therapy techniques and complement inhibitors. These cutting-edge medicines give patients and healthcare professionals more options by replacing or supplementing current therapy. For instance, Novartis presented a thorough analysis of the Phase III APPOINT-PNH study, which examined the effects of investigational oral monotherapy (iptacopan) in individuals with paroxysmal nocturnal hemoglobinuria (PNH) who had not previously taken complement inhibitors, including anti-C5 therapies.
By treatment, the medication segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 and is predicted to grow at the fastest CAGR during the forecast period owing to the rapidly expanding pipeline of innovative therapies and the increasing adoption of targeted treatment approaches, driven by advancements in precision medicine and growing awareness among healthcare professionals and patients. For instance, Kira Pharmaceuticals' application for an investigational new drug (IND) was approved by the Chinese National Medical Products Administration (NMPA) in October 2022.
By drugs, the eculizumab segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the established efficacy, widespread adoption, and extensive clinical experience, solidifying its position as the cornerstone therapy for managing PNH. For instance, Novartis showcased the safety and effectiveness of Fabhalta (iptacopan) in adults with PNH in January 2023 by presenting the 48-week findings from the Phase III APPLY-PNH trial. Additionally, the ravulizumab segment is predicted to grow at the fastest CAGR during the forecast period owing to the growing acceptance of its extended dosing interval, improved patient convenience, and increasing preference for next-generation complement inhibitors in the management of PNH.
By route of administration, the parenteral segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the convenience of administration, ensuring consistent drug delivery and effective disease management, particularly for patients requiring regular treatment monitoring and adherence. For instance, in December 2022, Apellis Pharmaceuticals reported encouraging results from their Phase III clinical trial (NCT04344747), which assessed pegcetacoplan as a treatment for PNH. Additionally, the oral segment is predicted to grow at the fastest CAGR during the forecast period owing to the advancements in oral treatment options, offering improved patient convenience, compliance, and quality of life compared to traditional parenteral therapies.
By end-user, the hospitals segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the prevalence of severe cases requiring specialized care, advanced diagnostic facilities, and access to comprehensive treatment options available in hospital settings. For instance, in August 2023, Novartis and Regeneron worked together to develop pozelimab (REGN3918) as a therapy for PNH. Additionally, the homecare segment is predicted to grow at the fastest CAGR during the forecast period owing to the increasing trend towards patient-centric care, rising preference for home-based treatment options, and advancements in technology facilitating remote monitoring and administration of therapies in the comfort of patients' homes.
North American region is anticipated to have the highest revenue share during the forecast period owing to the presence of well-established healthcare infrastructure, high adoption rates of advanced therapies, favorable reimbursement policies, and robust research and development activities focused on addressing unmet needs in PNH management. Additionally, the Asia Pacific region is predicted to grow at the fastest CAGR during the forecast period owing to the rapidly expanding healthcare infrastructure, increasing awareness about PNH, rising healthcare expenditure, and growing initiatives to improve access to innovative treatments across the region. For instance, in February 2023, UCB acquired Ra Pharmaceuticals. Ra Pharma's PNH pipeline development may be impacted by UCB's purchase.
This extensive research report concentrates on the size and projections of the global and regional markets from 2023 to 2034.
The market is growing due to the growing pipeline of innovative PNH treatment medicines, such as gene therapy techniques and complement inhibitors. These cutting-edge medicines give patients and healthcare professionals more options by replacing or supplementing current therapy. For instance, Novartis presented a thorough analysis of the Phase III APPOINT-PNH study, which examined the effects of investigational oral monotherapy (iptacopan) in individuals with paroxysmal nocturnal hemoglobinuria (PNH) who had not previously taken complement inhibitors, including anti-C5 therapies.
By treatment, the medication segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 and is predicted to grow at the fastest CAGR during the forecast period owing to the rapidly expanding pipeline of innovative therapies and the increasing adoption of targeted treatment approaches, driven by advancements in precision medicine and growing awareness among healthcare professionals and patients. For instance, Kira Pharmaceuticals' application for an investigational new drug (IND) was approved by the Chinese National Medical Products Administration (NMPA) in October 2022.
By drugs, the eculizumab segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the established efficacy, widespread adoption, and extensive clinical experience, solidifying its position as the cornerstone therapy for managing PNH. For instance, Novartis showcased the safety and effectiveness of Fabhalta (iptacopan) in adults with PNH in January 2023 by presenting the 48-week findings from the Phase III APPLY-PNH trial. Additionally, the ravulizumab segment is predicted to grow at the fastest CAGR during the forecast period owing to the growing acceptance of its extended dosing interval, improved patient convenience, and increasing preference for next-generation complement inhibitors in the management of PNH.
By route of administration, the parenteral segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the convenience of administration, ensuring consistent drug delivery and effective disease management, particularly for patients requiring regular treatment monitoring and adherence. For instance, in December 2022, Apellis Pharmaceuticals reported encouraging results from their Phase III clinical trial (NCT04344747), which assessed pegcetacoplan as a treatment for PNH. Additionally, the oral segment is predicted to grow at the fastest CAGR during the forecast period owing to the advancements in oral treatment options, offering improved patient convenience, compliance, and quality of life compared to traditional parenteral therapies.
By end-user, the hospitals segment accounted for the highest revenue-grossing segment in the global paroxysmal nocturnal hemoglobinuria (PNH) market in 2023 owing to the prevalence of severe cases requiring specialized care, advanced diagnostic facilities, and access to comprehensive treatment options available in hospital settings. For instance, in August 2023, Novartis and Regeneron worked together to develop pozelimab (REGN3918) as a therapy for PNH. Additionally, the homecare segment is predicted to grow at the fastest CAGR during the forecast period owing to the increasing trend towards patient-centric care, rising preference for home-based treatment options, and advancements in technology facilitating remote monitoring and administration of therapies in the comfort of patients' homes.
North American region is anticipated to have the highest revenue share during the forecast period owing to the presence of well-established healthcare infrastructure, high adoption rates of advanced therapies, favorable reimbursement policies, and robust research and development activities focused on addressing unmet needs in PNH management. Additionally, the Asia Pacific region is predicted to grow at the fastest CAGR during the forecast period owing to the rapidly expanding healthcare infrastructure, increasing awareness about PNH, rising healthcare expenditure, and growing initiatives to improve access to innovative treatments across the region. For instance, in February 2023, UCB acquired Ra Pharmaceuticals. Ra Pharma's PNH pipeline development may be impacted by UCB's purchase.
This extensive research report concentrates on the size and projections of the global and regional markets from 2023 to 2034.
Report Scope:
- Base Year: 2023
- Forecast Period: 2024-2034
- Study Coverage
- Market Forecast by Treatment, Drug, Route of Administration, and End-User
- Market Forecast for 5 Regions and 17+ Countries
- North America (U.S. and Canada)
- Europe (Germany, France, UK, Spain, Italy, Russia, Rest of Europe)
- Asia Pacific (China, Japan, India, Australia, South Korea, Rest of APAC)
- Latin America (Brazil, Mexico, Argentina, Rest of LATAM)
- MEA (South Africa, GCC, Rest of MEA)
- Exhaustive Company Profiles of the Top 10+ Major Market Players
Segmentation: Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Report 2023 - 2034
Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Analysis & Forecast by Treatment 2023 - 2034 (Revenue USD Bn)
- Medication
- Supplements
- Blood Transfusion
- Bone Marrow Transplant
Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Analysis & Forecast by Drugs 2023 - 2034 (Revenue USD Bn)
- Eculizumab
- Ravulizumab
- Others
Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Analysis & Forecast by Route of Administration 2023 - 2034 (Revenue USD Bn)
- Oral
- Parenteral
Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Analysis & Forecast by End-User 2023 - 2034 (Revenue USD Bn)
- Hospitals
- Homecare
- Specialty Clinics
- Others
Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Analysis & Forecast by Region 2023 - 2034 (Revenue USD Bn)
- North America
- U.S.
- Canada
- Europe
- Germany
- France
- UK
- Spain
- Italy
- Rest of Europe
- Asia Pacific
- China
- Japan
- India
- Australia
- South Korea
- Rest of APAC
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of LATAM
- Middle East & Africa
- South Africa
- GCC
- Rest of MEA
Table of Contents
1. Research Methodology
2. Introduction
4. Market Environment Analysis
5. Market Dynamics
7. Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: Treatment Estimates & Trend Analysis
8. Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: Drugs Estimates & Trend Analysis
9. Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: Route of Administration Estimates & Trend Analysis
10. Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: End-user Estimates & Trend Analysis
11. Regional Market Analysis
12. North America Paroxysmal Nocturnal Hemoglobinuria (PNH) Market
13. Europe Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market
14. Asia Pacific Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market
15. Latin America Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market
16. MEA Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market
17. Competitor Analysis
18. Company Profiles
Companies Mentioned
- Alexion Pharmaceuticals
- Apellis Pharmaceuticals
- Ra Pharmaceuticals (acquired by UCB)
- Novartis AG
- Sanofi Genzyme
- Regeneron Pharmaceuticals
- Roche Holdings AG
- Pfizer Inc.
- Bristol Myers Squibb
- AstraZeneca plc
- Takeda Pharmaceutical Company Limited
- GlaxoSmithKline plc
- Johnson & Johnson
- CSL Behring
- Achillion Pharmaceuticals
