Paroxysmal Nocturnal Hemoglobinuria Market

Industry Outlook

The paroxysmal nocturnal hemoglobinuria (PNH) market was valued at $2,341.4 million in 2018 and is expected to reach $6,203.0 million by 2027, with a compounded annual growth rate (CAGR) of 11.7% projected from 2019 to 2027.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 have 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 a strong drug pipeline during the forecast period.

Market Synopsis

Excellent drug safety and efficacy reported during clinical trial studies and a strong product pipeline during the forecast period will increase the popularity of monoclonal antibodies 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 from paroxysmal nocturnal hemoglobinuria (PNH). However, this treatment module is associated with a 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 that 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 a strong product pipeline during the forecast period will increase the popularity of monoclonal antibodies as the first-line therapy for PNH.

The presence of major players and an affordable reimbursement scenario together drive the market’s growth in North America.

North America presently represents 35% of the market and is spearheading the regional segment of the paroxysmal nocturnal hemoglobinuria (PNH) market. As per the research cited and brought forward by the National Institute of Health (NIH), approximately 5,000 people suffer from the disease in the United States. Affordable reimbursement scenarios and the presence of major players such as Amgen, Inc., Alexion Pharmaceuticals, Roche, Regeneron Pharmaceuticals, etc., together drive the market’s growth in the North American region. Europe has a 28% market growth rate and lags behind North America owing to its high prevalence rate. According to the statistics provided by the National Organization for Rare Disorders (NORD), the prevalence rate of PNH is 15.9 per million people in Europe. The 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 rising public health awareness related to PNH and developing healthcare infrastructure to provide affordable treatment pertaining to PNH.

Periods of History and Forecast

This research report presents the analysis of each segment from 2017 to 2027, considering 2018 as the base year for the research. The compound annual growth rate (CAGR) for each of the respective segments is calculated for the forecast period from 2019 to 2027.

Report Scope by Segments

The market is segmented by treatment and by geography.

Pharmaceutical giants competing to achieve a strong foothold in the paroxysmal nocturnal hemoglobinuria market are Amgen, Inc.; Achillion Pharmaceuticals; Alexion Pharmaceuticals, Inc.; Akari Therapeutics; Apellis Pharmaceuticals, Inc.; CinnaGen; F. Hoffmann-La Roche Ltd.; Novartis AG; Ra Pharmaceuticals; and Regeneron Pharmaceuticals.

Key questions are answered in this report.

• Which drug molecules are currently being studied in clinical trials and appear to be promising drug candidates during the forecast period?
• What is the competitive scenario currently present among major players providing treatment options for PNH?
• What is the disease etiology associated with paroxysmal nocturnal hemoglobinuria (PNH)?
• Why is eculizumab the most popular drug for the treatment of PNH?
• What is the market performance of PNH in both developed and developing regions worldwide?

Chapter 1. Preface
1.1. Report Scope and Description
1.1.1. Purpose of the Report
1.1.2. Target Audience
1.1.3. USP and Key Treatment
1.2. Research Scope
1.3. Research Methodology
1.3.1. Phase I-Secondary Research
1.3.2. Phase II-Primary Research
1.3.3. Approach Adopted
1.3.4. Top-down Approach
1.3.5. Bottom-up Approach
1.3.6. Phase III-Expert Panel Review
1.3.7. Assumptions
1.4. Market Segmentation

Chapter 2. Executive Summary
2.1. Global PNH Market Portraiture
2.2. Global PNH Market, by Treatment, 2018 (US$ Mn)
2.3. Global PNH Market, by Geography, 2018 (US$ Mn)

Chapter 3. Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: Dynamics and Future Outlook
3.1. Market Overview
3.2. Drivers
3.3. Challenges
3.4. Opportunities
3.5. Attractive Investment Proposition, by Geography, 2018
3.6. Competitive Analysis: Global PNH Market, by Key Players, 2018

Chapter 4. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market, by Treatment
4.1. Overview
4.2. Blood Transfusion
4.3. Medication
4.4. Stem Cell Transplant
4.5. Pipeline Analysis
4.5.1. Phase III Drug
4.5.1.1. ALXN1210
4.5.1.2. APL-2
4.5.1.3. rVA576
4.5.1.4. ABP 959
4.5.2. Tabular Representation of Phase I and II Drug Pipeline

Chapter 5. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market, by Geography
5.1. Overview
5.2. North America PNH Market Analysis, 2017 – 2027
5.2.1. North America PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
5.2.2. North America PNH Market, by Country, 2017 – 2027 (US$ Mn)
5.2.2.1. U.S.
5.2.2.2. Canada
5.3. Europe PNH Market Analysis, 2017– 2027
5.3.1. Europe PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
5.3.2. Europe PNH Market, by Country/Region, 2017 – 2027 (US$ Mn)
5.3.2.1. U.K.
5.3.2.2. Germany
5.3.2.3. Rest of Europe
5.4. Asia Pacific PNH Market Analysis, 2017 – 2027
5.4.1. Asia Pacific PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
5.4.2. Asia Pacific PNH Market, by Country/Region, 2017 – 2027 (US$ Mn)
5.4.2.1. China
5.4.2.2. Japan
5.4.2.3. Rest of Asia Pacific
5.5. Latin America PNH Market Analysis, 2017 – 2027
5.5.1. Latin America PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
5.5.2. Latin America PNH Market, by Country/Region, 2017 – 2027 (US$ Mn)
5.5.2.1. Brazil
5.5.2.2. Mexico
5.5.2.3. Rest of Latin America
5.6. Middle East & Africa PNH Market Analysis, 2017 – 2027
5.6.1. MEA PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
5.6.2. MEA PNH Market, by Region, 2017– 2027 (US$ Mn)
5.6.2.1. GCC
5.6.2.2. Rest of MEA

Chapter 6. Company Profiles
6.1. Amgen, Inc.
6.1.1. Business Description
6.1.2. Financial Information (Subject to data availability)
6.1.3. Product Portfolio
6.1.4. News Coverage
6.2. Achillion Pharmaceuticals
6.3. Alexion Pharmaceutcials, Inc.
6.4. Akari Therapeutics
6.5. Apellis Pharmaceutcials, Inc.
6.6. CinnaGen
6.7. F.Hoffman-La Roche Ltd.
6.8. Novartis AG
6.9. Ra Pharmaceutcials
6.10. Regeneron Pharmaceuticals

List of Figures

FIG. 1 Paroxysmal Nocturnal Hemoglobinuria (PNH) Market: Research Methodology
FIG. 2 PNH: Market Segmentation
FIG. 3 Global PNH Market, by Treatment, 2018 (US$ Mn)
FIG. 4 Global PNH Market, by Geography, 2018 (US$ Mn)
FIG. 5 Attractive Investment Proposition: by Geography, 2018
FIG. 6 Competitive Analysis: Global PNH Market, by Key Players, 2018
FIG. 7 Global Blood Transfusion Market for PNH, 2017 – 2027 (US$ Mn)
FIG. 8 Global Medication Market for PNH, 2017 – 2027 (US$ Mn)
FIG. 9 Global Stem Cell Transplant Market for PNH, 2017 – 2027 (US$ Mn)
FIG. 10 U.S. PNH Market, 2017 – 2027 (US$ Mn)
FIG. 11 Canada PNH Market, 2017 – 2027 (US$ Mn)
FIG. 12 U.K. PNH Market, 2017 – 2027 (US$ Mn)
FIG. 13 Germany PNH Market, 2017 – 2027 (US$ Mn)
FIG. 14 Rest of Europe PNH Market, 2017 – 2027 (US$ Mn)
FIG. 15 China PNH Market, 2017– 2027 (US$ Mn)
FIG. 16 Japan PNH Market, 2017 – 2027 (US$ Mn)
FIG. 17 Rest of Asia Pacific PNH Market, 2017 – 2027 (US$ Mn)
FIG. 18 Brazil PNH Market, 2017 – 2027 (US$ Mn)
FIG. 19 Mexico PNH Market, 2017 – 2027 (US$ Mn)
FIG. 20 Rest of Latin America PNH Market, 2017 – 2027 (US$ Mn)
FIG. 21 GCC PNH Market, 2017 – 2027 (US$ Mn)
FIG. 22 Rest of Middle East and Africa PNH Market, 2017 – 2027 (US$ Mn)

List of Tables

TABLE 1 Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Market Portraiture
TABLE 2 Global PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
TABLE 3 Global PNH Market, by Geography, 2017 – 2027 (US$ Mn)
TABLE 4 North America PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
TABLE 5 North America PNH Market, by Country, 2017 – 2027 (US$ Mn)
TABLE 6 Europe PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
TABLE 7 Europe PNH Market, by Country/Region, 2017– 2027 (US$ Mn)
TABLE 8 Asia Pacific PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
TABLE 9 Asia Pacific PNH Market, by Country/Region, 2017 – 2027 (US$ Mn)
TABLE 10 Latin America PNH Market, by Treatment, 2017 – 2027 (US$ Mn)
TABLE 11 Latin America PNH Market, by Country/Region, 2017 – 2027 (US$ Mn)
TABLE 12 Middle East and Africa PNH Market, by Treatment, 2017– 2027 (US$ Mn)
TABLE 13 Middle East and Africa PNH Market, by Region, 2017 – 2027 (US$ Mn)
TABLE 14 Amgen, Inc.: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 15 Achillion Pharmaceuticals: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 16 Alexion Pharmaceuticals, Inc.: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 17 Akari Therapeutics: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 18 Apellis Pharmaceuticals, Inc.: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 19 CinnaGen: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 20 F.Hoffman-La Roche Ltd.: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 21 Novartis AG: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 22 Ra Pharmaceuticals: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)
TABLE 23 Regeneron Pharmaceuticals: Company Snapshot (Business Description; Financial Information; Product Portfolio; Key Developments)