Paroxysmal Nocturnal Hemoglobinuria (PNH) – Improved Therapies Are About to Launch


New Delhi, India -- (SBWIRE) -- 04/17/2018 -- Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired genetic disorder caused by a mutation in X chromosome gene PIGA (Phosphatidylinositol glycan class A), leading to the destruction of red blood cells, and subsequent release of hemoglobin into plasma. There are mainly three types of PNH cells, namely Normal (type I) stem cells, PNH (type II) stem cell, and PNH (type III) stem cells. Approximately one-half of the patients with PNH die from the disease within 10 years of diagnosis. It occurs equally in men and women, with the peak occurrence in the 30s and middle 50s.

According to the latest report of DelveInsight, "Paroxysmal Nocturnal Hemoglobinuria (PNH)– Competitive Landscape, Market Insights, Epidemiology and Market Forecast - 2027", The total diagnosed prevalent population of PNH in 7 major markets was 13,742 in 2016, and DelveInsight estimates that the prevalence of this disease will increase by 2027. The United States accounts for the highest prevalence of PNH, while Spain has the lowest prevalent population of PNH, as Spain recorded only 491 cases in 2016. Total treatable cases of PNH were 6,746 cases in 2016 and this number is expected to increase due to the launch of potential therapies in the market in near future. The worldwide market size for PNH was found to be USD 856 Million in 2016. As per DelveInsight's estimation, the global market size of PNH is expected to grow at a CAGR of 12% during 2015-2027.

Currently, Soliris (eculizumab) is the only approved drug in the market and provides a better quality of life to the patients by reducing the need of frequent blood transfusion. However, it has its own down sides such as higher dose requirement, painful mode of administration, risk of meningococcal infection, and hefty price tag. To overcome the drawbacks of existing treatment companies are developing therapies with novel MoA like RA101495 (Ra Pharmaceuticals), RO7112689 (Hoffmann-La Roche), Coversin (AKARI Therapeutics), ALN-CC5 (Alnylam Pharmaceuticals), ALXN1210 (Alexion Therapeutics) and APL-2 (Apellis Pharmaceuticals). APL-2 may enter the market by 2020, is expected to take 13% of 2027 market share owing its unique mechanism and convenient mode of administration.

The latest report has covered detailed description about disease background including Types, Clinical manifestations, Causes, Pathogenesis etc. Complete Pipeline drugs profiling covering each and every phase of development with clinical trials data. Total market size of PNH is provided covering the 7MM with country-wise market analysis to better understand the areas of improvement and growth opportunities in the market. Complete Epidemiology and patient population details are provided covering the 7MM to better understand the distribution of the given indication in 7MM. Key emerging therapy details are also provided to better understand the future market competition and plan the strategies accordingly.
Reasons to buy:
-The report will help in developing business strategies by understanding trends shaping and driving the PNH market.
-To understand the future market competition in the PNH market and Insight reviews of the key market drivers and barriers.
-Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
-In-depth understanding of the pipeline development and recent happenings will provide a competitive edge over competitors.

Companies Covered:
-Alexion Pharmaceuticals
-Akari Therapeutics
-Achillion Pharmaceuticals
-RA Pharmaceuticals
-Apellis Pharmaceuticals
-Alnylam Pharmaceuticals
And many others……………..

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Table of Contents:

1. Report Introduction

2. Paroxysmal Nocturnal Hemoglobinuria: Market Overview at a Glance

3. Disease Background and Overview

3.1. Introduction

3.2. Types of PNH

3.3. Clinical manifestations

3.4. Causes of PNH

3.5. Symptoms

3.6. Pathogenesis

3.7. Diagnosis

4. Epidemiology and Patient Population

4.1. Key Findings

4.1.1. Population and Forecast Parameters

4.1.2. Diagnosed Prevalent Cases of PNH in 7MM

4.2. United States

4.2.1. Diagnosed Prevalent Cases of PNH in the United States

4.2.2. Treatable Cases of PNH in the United States

4.3. EU5

4.3.1. United Kingdom Diagnosed Prevalent Cases of PNH in the United Kingdom Treatable Cases of PNH in the United Kingdom

4.3.2. France Diagnosed Prevalent Cases of PNH in France Treatable Cases of PNH in France

4.3.3. Spain Diagnosed Prevalent Cases of PNH in Spain Treatable Cases of PNH in Spain

4.3.4. Italy Diagnosed Prevalent Cases of PNH in Italy Treatable Cases of PNH in Italy

4.3.5. Germany Diagnosed Prevalent Cases of PNH in Germany Treatable Cases of PNH in Germany

4.4. Japan

4.4.1. Diagnosed Prevalent Cases of PNH in Japan

4.4.2. Treatable Cases of PNH in Japan

5. Current Treatment and Medical Practices

5.1. Treatment Algorithm

5.2. Therapy areas for PNH

5.3. US Association Current Recommendations for PNH (Guidelines)

5.4. Europe Association Current Recommendations for PNH

5.5. Japan Association Current Recommendations for PNH

6. Unmet needs

7. Marketed Drugs List

8. Marketed Drugs

8.1. Soliris: Alexion Pharmaceuticals Inc.

8.1.1. Drug Description

8.1.2. Mechanism of Action

8.1.3. Regulatory Milestones

8.1.4. Advantages & Disadvantages

8.1.5. Safety and Efficacy

8.1.6. Product Profile

9. Clinical Products [Phase III to Phase I]

9.1. Late Phase Drugs Analysis

9.2. Clinical Products: Drugs Chapters

9.2.1. Ravulizumab (ALXN1210): Alexion Pharmaceuticals Inc.

9.2.2. Regulatory Milestones

9.2.3. Other development activities

9.2.4. Clinical Development

9.2.5. Ongoing Trials Information

9.2.6. Safety and Efficacy

9.2.7. Advantages and Disadvantages

9.2.8. Product Profile

9.2.3. Coversin: Akari Therapeutics Plc.

9.2.3. Regulatory Milestones

9.2.4. Other development activities

9.2.5. Clinical Development

9.2.6. Ongoing Trials Information

9.2.7. Safety and Efficacy

9.2.8. Advantages and Disadvantages

9.2.9. Product Profile

9.2.4. ACH-0144471: Achillion Pharmaceuticals, Inc.

9.2.5. Regulatory Milestones

9.2.6. Other development activities

9.2.7. Clinical Development

9.2.8. Ongoing Trials Information

9.2.9. Safety and Efficacy

9.2.10. Advantages and Disadvantages

9.2.11. Product Profile

9.2.5. RA101495: RA Pharmaceuticals Inc.

9.2.5. Regulatory Milestones

9.2.6. Clinical Development

9.2.7. Ongoing Trials Information

9.2.8. Safety and Efficacy

9.2.9. Advantages and Disadvantages

9.2.10. Product Profile

9.2.6. APL-2: Apellis Pharmaceuticals, Inc.

9.2.7. Regulatory Milestones

9.2.8. Other development activities

9.2.9. Clinical Development

9.2.10. Ongoing Trials Information

9.2.11. Safety and Efficacy

9.2.12. Advantages and Disadvantages

9.2.13. Product Profile

9.2.7. ALN-CC5: Alnylam Pharmaceuticals Inc.

9.2.7. Other development activities

9.2.8. Clinical Development

9.2.9. Ongoing Trials Information

9.2.10. Safety and Efficacy

9.2.11. Advantages and Disadvantages

9.2.12. Product Profile

9.2.8. RO7112689: F. Hoffman-La Roche Ltd.

9.2.9. Other development activities

9.2.10. Clinical Development

9.2.11. Ongoing Trials Information

9.2.12. Safety and Efficacy

9.2.13. Advantages and Disadvantages

9.2.14. Product Profile

9.3. Non-Clinical Products

9.2.15. OMS906: Omeros Corporation

9.2.16. Drug Description

9.2.17. Pre-Clinical results

9.2.18. Other Development Activities

9.2.19. Product Profile

9.3.2. To be continued in the report...

9. Therapeutic Assessment

9.3. Assessment by Monotherapy Products

9.4. Assessment by Combination Products

9.5. Assessment by Route of Administration

9.6. Assessment by Stage and Route of Administration

9.7. Assessment by Molecule Type

9.8. Assessment by Stage and Molecule Type

9.9. Assessment by Domain

9.10. Assessment by Stage and Domain

10. PNH: Market Analysis

10.3. Key Findings

10.4. 7MM Market Size of Paroxysmal Nocturnal Hemoglobinuria

10.4.1. Region-Wise Market size of PNH

10.4.2. Total Market Size of PNH in 7MM by Therapies

10.4.3. Patient Uptake of PNH in 7MM by Therapies

10.5. United States

10.5.1. Total Market Size of PNH in the United States

10.5.2. Total Market Size of PNH in United States by Therapies

10.5.3. Therapy-Based Market Size of PNH-US

10.6. EU5

10.6.1. Total Market Size of PNH in EU5

10.6.2. Total Market Size of PNH in EU5 by Therapies

10.6.3. Therapy-Based Market Size of PNH-EU5

10.6.4. Italy Total Market Size of PNH in Italy Therapy-Based Market Size of PNH-Italy

10.6.5. Germany Total Market Size of PNH in Germany Therapy-Based Market Size of PNH-Germany

10.6.6. France Total Market Size of PNH in France Therapy-Based Market Size of PNH-France

10.6.7. Spain Total Market Size of PNH in Spain Therapy-Based Market Size of PNH-Spain

10.6.8. United Kingdom Total Market Size of PNH in the United Kingdom Therapy-Based Market Size of PNH- United Kingdom

10.7. Japan

10.7.1. Total Market Size of PNH in Japan

10.7.2. Total Market Size of PNH in Japan by Therapies

10.7.3. Therapy-Based Market Size of PNH-Japan

11. Market drivers

12. Market barriers

13. Appendix

14. Report Methodology

15. Sources Used

16. Consulting Services

17. Disclaimer

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