China Lysosomal Storage Disorders Therapeutics Market


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Asia
  • Country: China
  • Report ID : 1579

  • Format: PPT, PDF

China Lysosomal Storage Disorders Therapeutics Market: Segmented by Therapy (Enzyme Replacement Therapy, Substrate Reduction Therapy, Gene Therapy), Disease Type (Gaucher, Pompe, Fabry, Mucopolysaccharidosis), Route of Administration (Oral, Intravenous)- Size, Share, Growth, Trends, and Forecasts (2019 – 2028)

SKU: 1579 Categories: ,

Report Overview

China Lysosomal Storage Disorders 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 5.2% during the forecast period.

Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. Although each type of lysosomal storage disease is very rare, as a whole, the global incidence is about 1 in 5000-6000. Taking Shanghai as an example, about 200,000 newborns are born in the city each year, and the number of patients About 10-20.

Market Drivers

China is a growing market for lysosomal storage disorders due to an enormous population, increase in awareness of disease by the establishment of the Chinese Newborns Screening Cooperative Group for Lysosomal Storage Disease and improvement in R&D activities. Development of novel therapeutics and increase in global incidence (1 in 5000-6000) and prevalence rate leads to growth of the China lysosomal Storage Disorders drug market.

However, there are some restraining factors for the growth of lysosomal storage disorders therapeutics market in China such as high cost of treatment, limitations of medical insurance coverage and misdiagnosis of the disease (in 2019 61.9% of Gaucher disease patients have experienced misdiagnosis, and 27.9% of patients need 1-5 years to be diagnosed).

1. China Lysosomal Storage Disorders Therapeutics Market Overview………….
A. Market Size
2. Market Growth Drivers………………………………………………………………………
A. Increase in Awareness of Disease
B. Increase in Prevalence and Incidence Rate
C. Improvement in R&D Activities
D. Adoption of Novel Therapeutics
3. Major Types of Lysosomal Storage Disorders…………………………………………….
A. Gaucher Disease
B. Fabry Disease
C. Pompe Disease
D. Mucopolysaccharidosis Type I
4. Lysosomal Storage Disorders Therapeutics Market Segmentation…………
A. By Therapy
I. Enzyme Replacement Therapy
II. Substrate Reduction Therapy
III. Gene Therapy
B. By Disease Type
I. Gaucher Disease
II. Pompe Disease
III. Fabry Disease
IV. Mucopolysaccharidosis
C. By Route of Administration
I. Oral
II. Intravenous
5. Lysosomal Storage Disorders 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. Sanofi Company Overview, Product & Services, Strategies & Financials
B. Takeda Company Overview, Product & Services, Strategies & Financials
C. BioMarin 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 Lysosomal Storage Disorders Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapy:

On the basis of therapy, the China lysosomal storage disorders therapeutics market can be segmented into enzyme replacement therapy, substrate reduction therapy and gene therapy.

Enzyme replacement therapy (Velaglucerase Alfa, Taliglucerase Alfa, Agalsidase Beta, Laronidase, Imiglucerase) substrate reduction therapy (Eliglustat, Miglustat), and gene therapy. Enzyme replacement therapy delivers the missing enzyme through a vein (IV).

Substrate reduction therapy reduces the substance that is building up in the cells. For example, the drug Miglustat treats one form of Gaucher disease. Mostly gene therapy is used to correct visceral pathology in LSD.

By Disease Type:

On the basis of disease type, the China lysosomal storage disorders therapeutics market can be segmented into Gaucher disease, Pompe disease, Fabry disease and Mucopolysaccharidosis.

Gaucher disease (14%), Pompe disease (7%), Fabry disease (5%), Mucopolysaccharidosis (MPS). MPS is further segmented into MPS I – Hurler, Hurler Scheie and Scheie, MPS II – Hunter, MPS III – Sanfilippo, and others. A high incidence (1.24 per 100000) of Gaucher’s disease is attributable to its large market share.

Diseases such as Fabry, Pompe, and MPS are also gaining the market owing to rising awareness brought about by organizations such as Chinese Newborns Screening Cooperative Group for Lysosomal Storage Disease.

By Route of Administration:

On the basis of route of administration, the China lysosomal storage disorders therapeutics market can be segmented into oral and intravenous. Oral route of administration is usually the preferred method due to its convenience and pain-free administration. Oral medication is associated with a better quality of life, compared with infusion therapy (parenteral route of administration).

Major Players

Some of the key players in China lysosomal storage disorders therapeutics market are Sanofi; Takeda Pharmaceutical Co. Ltd; Genzyme; BioMarin Pharmaceuticals Inc; Amicus Therapeutics Inc.

Key players are adopting various strategies such as awareness campaigns, collaboration, and partnership. For instance, Sanofi started awareness programs, providing support to patients in China affected by lysosomal storage disorders.

WuXi Biologics and Amicus Sign Exclusive Manufacturing Partnership for Amicus’ Pompe biologic ATB200.

In order to better promote neonatal screening for lysosomal storage disease, the Shanghai Institute of Paediatrics and PerkinElmer Medical Diagnostic Products (Shanghai) Co., Ltd. signed a cooperation agreement. In this cooperation, the two parties will focus on conducting population data analysis of lysosomal storage diseases in China; jointly explore the neonatal screening system for lysosomal storage diseases, establish a closed-loop process of screening, diagnosis, and treatment; and strengthen rare Training of clinical and laboratory personnel for disease.

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