Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Size, Share & Trends Analysis Report By Treatment (Medication, Stem Cell Transplant, Blood Transfusion), And Segment Forecasts, 2018 - 2025

Published: Oct, 2018 | Pages: 65 | Publisher: Grand View Research
Industry: Pharmaceutical | Report Format: Electronic (PDF)

The global paroxysmal nocturnal hemoglobinuria (PNH) treatment market size is expected to reach USD 5.8 billion by 2025 at a 11.2% CAGR during the forecast period, according to a new report by Grand View Research, Inc. Several factors such as rise in number of blood and bone marrow related disorders, increase in geriatric population, wider adoption of novel therapeutics, and emergence of biologics are driving the market.

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, chronic hematologic disorder. It is an ultra-rare disease with approximately 8,000 to 10,000 cases reported in North America and Europe. The life-threatening disease often manifests in three forms: thrombosis, persistent intravascular hemolysis, and pancytopenia. Although information on its prevalence is scarce, PNH is estimated to affect more than 20,000 people worldwide with an incidence of about 1.3 per million annually and a prevalence of around 16 per million. PNH affects men and women equally. Although physical symptoms can begin at any age, they usually develop between 30-40 years of age.

Novel treatments including monoclonal antibodies, recombinant proteins, and cyclic peptides are gaining penetration in developed markets. However, high cost of therapy with Soliris and other emerging pipeline drugs presents a barrier in adoption in developing regions. Blood transfusion and supportive care are anticipated to lose momentum during the forecast period due to adverse side effects and sub-optimal efficacy.

Further key findings from the report suggest:

• An estimated 5,000 - 6,000 people in U.S. suffer from PNH, with approximately 450 new cases being reported annually

• U.S. dominated the global PNH treatment market with more than 35% share in 2017 due to high cost of treatment in U.S., potential approval of a range of pipeline drugs, and adoption of novel therapeutics

• Treatment with medication dominated the PNH market with a share of more than 75% in 2017. Apart from drugs, the stem cell transplant segment is projected to witness double-digit growth through the forecast period

• Alexion, Akari, Apellis, Amgen, CinnaGen, Ra Pharmaceuticals, Alnylam, Achillion, Novartis, Roche, and Regeneron Pharmaceuticals are some of the key players in this market

• Currently, 16 products are under clinical evaluation for PNH treatment in various stages of development. Promising pipeline candidates such as ALXN1210, Coversin, APL2, and RA101495 offer significant commercial potential post regulatory approval.

 Table of Contents

Chapter 1 Report Scope
                 1.1 Treatment Scope
                 1.2 Country Scope
                 1.3 Estimates and Forecast Timeline
Chapter 2 Methodology
                 2.1 Research Methodology
                     2.1.1 Information procurement
                 2.2 Information or Data Analysis
                 2.3 Market Formulation & Validation
                 2.4 List of Sources
                 2.5 List of Abbreviations
Chapter 3 Objectives
                 3.1 Objective - 1: Understanding the market dynamics
                 3.2 Objective - 2: Understanding the trends & variables in the individual countries
                 3.3 Objective - 3: Understanding the attributes such as strategy framework
                 3.4 Objective - 4: Understanding the key service and application scopes
Chapter 4 Executive Summary
Chapter 5 Disease Primer and Epidemiology
                 5.1 Disease Primer
                 5.2 Epidemiology
                     5.2.1 Current Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)
                     5.2.2 Forecast Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)
                         5.2.2.1 U.S.
                         5.2.2.2 Japan
                         5.2.2.3 U.K.
                         5.2.2.4 Germany
                         5.2.2.5 France
                         5.2.2.6 Spain
                         5.2.2.7 Italy
                         5.2.2.8 RoW
Chapter 6 Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Overview
                 6.1 Market, by Treatment
                     6.1.1 Medication
                     6.1.2 Stem Cell Transplant (SCT)
                     6.1.3 Blood Transfusion
                 6.2 Market, by Country
                     6.2.1 U.S.
                     6.2.2 Japan
                     6.2.3 U.K.
                     6.2.4 Germany
                     6.2.5 France
                     6.2.6 Spain
                     6.2.7 Italy
                     6.2.8 RoW
                 6.3 Market Size & Forecast
                     6.3.1 Sales Performance
                         6.3.1.1 Sales Performance, by Treatment (2017 - 2025)
                         6.3.1.2 Sales Performance, by country (2017 - 2025)
                 6.4 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market: Drivers and Restraints
                 6.5 M & A, Deal Landscape (2014-2018 YTD)
                 6.6 SWOT
Chapter 7 Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market: Pipeline Intelligence
                 7.1 Pipeline Landscape
                     7.1.1 Leading Drugs in Development
                     7.1.2 Key R & D Trends
                 7.2 Promising Drug Candidates in Pipeline
                     7.2.1 Late Stage Pipeline Analysis
                     7.2.2 Profile of Disruptive Drugs
                 7.3 Global Pipeline Forecast
Chapter 8 Company Profiles
                 8.1 Alexion Pharmaceuticals
                     8.1.1 Company Overview
                     8.1.2 Current Product Portfolio- Major Products
                     8.1.3 Product Forecast Sales up to 2025
                     8.1.4 Strategic Initiatives
                         8.1.4.1 Key Company News Flow
                     8.1.5 SWOT
                 8.2 Akari Therapeutics
                     8.2.1 Company Overview
                     8.2.2 Current Product Portfolio- Major Products
                     8.2.3 Product Forecast Sales up to 2025
                     8.2.4 Strategic Initiatives
                         8.2.4.1 Key Company News Flow
                     8.2.5 SWOT
                 8.3 Apellis Pharmaceuticals
                     8.3.1 Company Overview
                     8.3.2 Current Product Portfolio- Major Products
                     8.3.3 Product Forecast Sales up to 2025
                     8.3.4 Strategic Initiatives
                         8.3.4.1 Key Company News Flow
                     8.3.5 SWOT
                 8.4 Ra Pharmaceuticals
                     8.4.1 Company Overview
                     8.4.2 Current Product Portfolio- Major Products
                     8.4.3 Strategic Initiatives
                         8.4.3.1 Key Company News Flow
                     8.4.4 SWOT
Chapter 9 Market Outlook
                 9.1 Winners and Losers
                 9.2 Emerging Companies
                 9.3 What the Future Holds

List of Tables

Table 1 List of Secondary Sources
Table 2 List of Abbreviation
Table 3 Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence and Incidence, 2017 - 2025
Table 4 Sales Performance, by Treatment (in $ Million)
Table 5 Sales Performance, by Country (in $ Million)
Table 6 Paroxysmal Nocturnal Hemoglobinuria (PNH) R & D Pipeline - Drugs in Development
Table 7 R & D Pipeline Overview
Table 8 Late Stage Pipeline
Table 9 Product Profile: Soliris (eculizumab)
Table 10 Product Profile: ALXN1210 (ravulizumab)
Table 11 Product Profile: Coversin (nomacopan)
Table 12 Product Profile: APL2
Table 13 Product Profile: RA101495
Table 14 Product Profile: Cemdisiran (ALN-CC5)
Table 15 Product Profile: ACH-4471
Table 16 Product Profile: LFG316 (tesidolumab)
Table 17 Product Profile: RO7112689 (SKY59)
Table 18 Global Pipeline Forecast
Table 19 Product Portfolio: Soliris (eculizumab)
Table 20 Soliris (eculizumab) Sales Forecast, 2017- 2025 (in $ Million)
Table 21 Product Portfolio: ALXN1210 (ravulizumab)
Table 22 ALXN1210 (ravulizumab) Sales Forecast, 2017- 2025 (in $ Million)
Table 23 Product Portfolio: Coversin (nomacopan)
Table 24 Coversin (nomacopan) Sales Forecast, 2017- 2025 (in $ Million)
Table 25 Product Portfolio: APL2
Table 26 APL2 Sales Forecast, 2017- 2025 (in $ Million)
Table 27 Product Portfolio: RA101495
Table 28 RA101495 Sales Forecast, 2017- 2025 (in $ Million)

List of Figures

Fig. 1 Market research process
Fig. 2 Information procurement
Fig. 3 Primary research pattern
Fig. 4 Market research approaches
Fig. 5 Value chain based sizing & forecasting
Fig. 6 QFD modelling for market share assessment
Fig. 7 Market formulation & validation
Fig. 8 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Segmentation
Fig. 9 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Outlook: Key Takeaways
Fig. 10 Current Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence Across Seven Major Markets - 2017
Fig. 11 Current Paroxysmal Nocturnal Hemoglobinuria (PNH) Incidence Across Seven Major Markets - 2017
Fig. 12 U.S. Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 13 Japan Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 14 U.K. Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 15 Germany Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 16 France Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 17 Spain Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 18 Italy Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025) 
Fig. 19 RoW Forecast Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence (2017 - 2025)
Fig. 20 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Share, by Treatment (2017 & 2025)
Fig. 21 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Share, by Country (2017 & 2025)
Fig. 22 U.S. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 23 Japan Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 24 U.K. Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 25 Germany Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 26 France Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 27 Spain Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 28 Italy Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 29 RoW Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market, $ Million (2017 - 2025)
Fig. 30 Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Size & Forecast, $ Million (2017 - 2025)
Fig. 31 Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market SWOT
Fig. 32 SWOT Analysis (Alexion Pharmaceuticals)
Fig. 33 SWOT Analysis (Akari Therapeutics)
Fig. 34 SWOT Analysis (Apellis Pharmaceuticals)
Fig. 35 SWOT Analysis (Ra Pharmaceuticals)