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Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Report 2018: Analysis & Forecasts 2017-2025 By Medication, Stem Cell Transplant & Blood Transfusion -

The "Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market Size, Share & Trends Analysis Report By Treatment (Medication, Stem Cell Transplant, Blood Transfusion), And Segment Forecasts, 2018 - 2025" report has been added to's offering.

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.

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.

Key Topics Covered:

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

2.2 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) U.S. Japan U.K. Germany France Spain Italy 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

6.3 Market Size & Forecast

6.3.1 Sales Performance Sales Performance, by Treatment (2017 - 2025) 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

  • Alexion Pharmaceuticals
  • Akari Therapeutics
  • Apellis Pharmaceuticals
  • Ra Pharmaceuticals

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Last Updated: 20-Nov-2018