China Lysosomal Storage Disorders Therapeutics Market

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  • 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)

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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

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