China Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Asia
  • Country: China
  • Report ID : 1575

  • Format: PPT, PDF

China Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market: Segmented by Therapy (Medication, Stem Cell Therapy, Blood Transfusion Therapy), Diagnostic Tests (CBC, LDH, Bone Marrow Examination, Flow Cytometry), Distribution Channel (Hospital, Retail and Online Pharmacies)- Size, Share, Growth, Trends, and Forecasts (2019 – 2028)

SKU: 1575 Categories: ,

Report Overview

China Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics market size stood at around USD xx billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of 9.2% during the forecast period.

PNH is a chronic, progressive, debilitating, and life-threatening ultra-rare blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). The average age of onset in the early 30s. China’s Incidence rate is 2.7/100,000 persons and the prevalence rate varies between provinces (0.8 to 1.4/100,000 persons) in China.

Market Drivers

The China is growing market for paroxysmal nocturnal hemoglobinuria (PNH) disease due to rise in a number of blood and bone marrow related disorders and increase in geriatric population (12.57% in 2019 in China) are the key factors which drive the market growth of PNH treatment market. In addition to these, the emergence of biologics, increase in China’s incidence rate (2.7/100,000 persons), increase in China’s prevalence rate (0.8 to 1.4/100,000 persons) and adoption of novel therapeutics are further pushing the market growth.

However, adverse effects associated with currently available treatments, increase in the cost of medical equipment, specifically surgical equipment required for stem cell transformation are some factors that restrain market growth of PNH.

1. China Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market Overview….
A. Market Size
2. Market Growth Drivers………………………………………………………………………
A. Rise In a Number of Blood and Bone Marrow Related Disorders
B. Increase in Geriatric Population
C. Emergence of Biologics
D. Adoption of Novel Therapeutics
3. Types of Paroxysmal Nocturnal Hemoglobinuria (PNH)………………………………
A. Classical PNH
B. Subclinical PNH
C. PNH In the Context of Other Primary Bone Marrow Disorders
4. Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market Segmentation
A. By Therapy
I. Medication Therapy
II. Stem Cell Therapy
III. Blood Transfusion Therapy
B. By Diagnostic Tests
III. Bone Marrow Examination
IV. Flow Cytometry
C. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Paroxysmal Nocturnal Hemoglobinuria (PNH) Major Products Market Share……
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape………………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………………
A. Alexion Company Overview, Product & Services, Strategies & Financials
B. Samsung Bioepis Company Overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………….
A. Policy Changes and Reimbursement Scenario
9. Factors Driving Future Growth……………………………………………………………
A. New Trends and Development of Paroxysmal Nocturnal Hemoglobinuria (PNH) Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapy:

On the basis of therapy, the China paroxysmal nocturnal hemoglobinuria (PNH) therapeutics market can be segmented into medication therapy, stem cell therapy and blood transfusion therapy.

Medication therapy includes Eculizumab (Soliris), Pegcetacoplan, Ravulizumab. The mainstay of PNH treatment is the drug Eculizumab (Soliris). It is a humanized monoclonal antibody that binds to proteins in the blood that can destroy red blood cells. The drug reduces the risk of thrombosis and can improve quality of life in PNH patients.

Stem cell therapy may be appropriate for patients who do not respond to Eculizumab or for those who have severe reductions in red blood cells, white blood cells and platelets.

By Diagnosis Test:

On the basis of diagnostic tests, the China paroxysmal nocturnal hemoglobinuria (PNH) therapeutics market can be divided into complete blood count test (CBC), lactate dehydrogenase test (LDH), bilirubin test, bone marrow examination, urine test for hemosiderin and flow cytometry.

Currently, diagnosis of PNH is with flow cytometry, which is used to determine the absence of specific RBC or white blood cell cell surface proteins (CD59 and CD55). This test is highly sensitive and specific. Flow cytometry has become the gold standard for making the diagnosis.

Bone marrow examination is indicated when bone marrow transplantation is being considered and to rule out other disorders when pancytopenia is present. Cytogenetic analysis of bone marrow samples is recommended to help identify underlying disease processes associated with PNH.

By Distribution Channel:

On the basis of distribution channel, the China paroxysmal nocturnal hemoglobinuria (PNH) therapeutics market can be segmented into retail, online and hospital pharmacies. During the forecast period (2019-2028) hospital pharmacies is the key distribution channel segment of the paroxysmal nocturnal hemoglobinuria (PNH) therapeutics market. Growth of the hospital pharmacies segment can be attributed to increasing incidence of paroxysmal nocturnal hemoglobinuria (PNH) and rising number of admissions of patients with the disease. Clinical examination and supporting laboratory investigations for the diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) are best possible in hospitals.

Major Players

Some of the key players in China Paroxysmal Nocturnal Hemoglobinuria (PNH) therapeutics market are Alexion Pharmaceuticals, Inc; Samsung Bioepis Co. Ltd; AffaMed Therapeutics; Amgen Inc; Novartis AG; CANbridge Pharmaceuticals, Inc.

Key players are adopting various strategies such as licensing agreement, collaboration and partnerships. For instance, Samsung Bioepis Co. Ltd. entered in China by licensing agreement with C-Bridge Capital.

Samsung Bioepis Co. Ltd. and AffaMed Therapeutics announced that the China National Medical Products Administration (NMPA) has approved the Clinical Trial Application (CTA) for SB12, also referred to as AMT904 in China – a biosimilar candidate referencing Soliris (eculizumab). SB12 (also referred to as AMT904) is an eculizumab biosimilar candidate for the treatment of paroxysmal nocturnal hemoglobinuria.

Iptacopan- Pipeline drug of Novartis for paroxysmal nocturnal hemoglobinuria.

CANbridge is developing CAN106 in conjunction with WuXi Biologics for treatment of paroxysmal nocturnal hemoglobinuria.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

  • Statistical Databases
  • Company Websites/Annual Reports
  • Trade Publications
  • Online Databases
  • Published Research Reports
  • Whitepapers
  • Press Releases of Key Market Players

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

Insights: Our reports deliver actionable insights backed with supporting facts and figures to assist you in achieving exemplary growth. Our in-depth analyses are interspersed with relevant insights and statistics to offer an executive-level view of a given market. The description helps in correlating many minor factors affecting the market and its impact to the different segments within the market.
Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

To request a free sample copy this report, please complete the form below.

    We value your inquiry and offer free customization with every report to fulfil your exact research needs.

    This website is secure and your personal details are safe.

    This report addresses

    • Market intelligence to enable effective decision making
    • Growth opportunities and trend analysis
    • Competitive benchmarking
    • Historical data & forecasts
    • Regional opportunities

    Need more?

    • Speak to our analyst to know how this research was put together
    • Add more countries or regions as part of customisation
    • Understand how this report can have direct impact on your revenue

    Get a custom report based on your requirements