Description
Pompe Disease Treatment Market Analysis
Pompe disease treatment, Market Forecast till 2030
Market Overview
The global market for pompe disease treatment is expected to increase at a large over the forecast period of 2022-2030. This is a rare and fatal disorder that causes degeneration of brain cells, leading to tremors, seizures, and cognitive impairment. The treatment market for Pompe disease has been growing in recent years, with a prevalence of about 1 in 200,000 people in the USA and an annual incidence rate of about 1 case per 800,000 people. The treatment market includes both direct-to-consumer options such as stem cell transplantation, bone marrow transplantation, chemotherapy, and immunotherapy, as well as healthcare providers such as academic medical centers and private practices.
The market for Pompe disease treatment is expected to grow in the coming years due to the increasing prevalence of the disease, and the availability of new treatment options. The growing awareness about the disease and the availability of new therapies, such as enzyme replacement therapy (ERT), is also expected to drive the market growth.
ERT is the most commonly used treatment for Pompe disease and is considered to be the standard of care. However, there are also other treatment options available, such as gene therapy and substrate reduction therapy (SRT). These treatments are still in the early stages of development, but are showing promising results in clinical trials.
The Pompe disease treatment is expected to benefit from rising number of clinical trials for new therapies. This is expected to lead to the development of more effective treatments for the disease in the future.
Overall, the market for Pompe disease treatment is expected to grow in the coming years due to the increasing prevalence of the disease, the availability of new treatment options, and the growing awareness about the disease.
The market is also driven by the increasing number of patients being diagnosed with Pompe disease, and the growing number of clinical trials for new therapies. This is expected to lead to the development of more effective treatments for the disease in the future.
Segment Overview
The Type, treatment type, end user, and regional segmentation have been used to segment the market of the global Pompe Disease treatment market. The market has been segmented into three main categories based on the type of Pompe disease: Non-Classic Infantile-Onset Pompe Disease, Late-Onset Pompe Disease, and Classic Infantile-Onset Pompe Disease. The Therapy type segment has been split The Treatment Types section has been divided into Gene Therapy, Enzyme Replacement Therapy, and Others.urcated into Research and Academic Institutes, Hospitals and Clinics
Regional Analysis
North America is expected to dominate the market and hold the major share due to the presence of advanced healthcare infrastructure and the high prevalence of Pompe disease in the region. The availability of reimbursement policies, increased awareness about Pompe disease, and the presence of major market players in the region also contribute to the market growth in North America.
In Europe, countries such as Italy, Germany, and France are expected to show a significant progress due to the increasing prevalence of the disease and the availability of advanced healthcare infrastructure.
Major Players
Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), Audentes Therapeutics (US), Amicus Therapeutics, Inc (US), Sanofi (France), CENTOGENE AG (UK) are some of the affluent contenders holding sizeable pompe disease treatment market share.
COVID 19 Impacts
The pandemic caused by COVID-19 has affected the supply chain and the production of medical products for Pompe disease. The market may face disruption due to a lack of attendance of human labour at work and in factories. However, there is a silver lining. Just like any other market, advanced technology has taken place instead of human labour. Though it may need human interventions from time to time, it is indeed scarce. The government of various nations are running vaccine drives. The initial months of the COVID-19 pandemic saw a decline in production of the market. However, the Pompe Disease Treatment Market has paced up production.
TABLE OF CONTENTS
1 EXECUTIVE SUMMARY
2 MARKET INTRODUCTION
2.1 SCOPE OF THE STUDY 13
2.2 RESEARCH OBJECTIVE 13
2.3 MARKET STRUCTURE 13
2.4 ASSUMPTIONS & LIMITATIONS 14
3 RESEARCH METHODOLOGY
3.1 DATA MINING 15
3.2 SECONDARY RESEARCH 16
3.3 PRIMARY RESEARCH 17
3.4 BREAKDOWN OF PRIMARY RESPONDENTS 18
3.5 FORECASTING TECHNIQUES 19
3.6 RESEARCH METHODOLOGY FOR MARKET SIZE ESTIMATION 20
3.6.1 BOTTOM-UP APPROACH 20
3.6.2 TOP-DOWN APPROACH 21
3.7 DATA TRIANGULATION 21
3.8 VALIDATION 21
4 MARKET DYNAMICS
4.1 OVERVIEW 22
4.2 DRIVERS 23
4.2.1 INCREASING GOVERNMENT INITIATIVES 23
4.2.2 RISING R&D ACTIVITIES BY MANUFACTURERS 23
4.3 RESTRAINTS 24
4.3.1 HIGH COST OF TREATMENT 24
4.4 OPPORTUNITIES 25
4.4.1 EMERGENCE OF IMMUNOTHERAPEUTIC APPROACHES 25
4.5 CHALLENGES 25
5 MARKET FACTOR ANALYSIS
5.1 VALUE CHAIN ANALYSIS 26
5.1.1 R&D AND DESIGNING 27
5.1.2 MANUFACTURING 27
5.1.3 DISTRIBUTION & SALES 27
5.1.4 POST-SALES SERVICES 27
5.2 PORTER’S FIVE FORCES MODEL 28
5.2.1 BARGAINING POWER OF SUPPLIERS 29
5.2.2 BARGAINING POWER OF BUYERS 29
5.2.3 THREAT OF NEW ENTRANTS 29
5.2.4 THREAT OF SUBSTITUTES 29
5.2.5 INTENSITY OF RIVALRY 29
6 POMPE DISEASE TREATMENT MARKET, BY TYPE
6.1 OVERVIEW 30
6.2 LATE-ONSET POMPE DISEASE 31
6.3 CLASSIC INFANTILE-ONSET POMPE DISEASE 31
6.4 NON-CLASSIC INFANTILE-ONSET POMPE DISEASE 32
7 POMPE DISEASE TREATMENT MARKET, BY THERAPY
7.1 OVERVIEW 33
7.2 ENZYME REPLACEMENT THERAPY (ERT) 34
7.3 GENE THERAPY 34
7.4 OTHERS 34
8 POMPE DISEASE TREATMENT MARKET, BY END USER
8.1 OVERVIEW 35
8.2 HOSPITALS & CLINICS 36
8.3 RESEARCH & ACADEMIC INSTITUTES 36
9 GLOBAL POMPE DISEASE TREATMENT MARKET, BY REGION
9.1 OVERVIEW 37
9.2 AMERICAS 39
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.2.1 NORTH AMERICA 40
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.2.1.1 US 42
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.2.1.2 CANADA 43
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.2.2 SOUTH AMERICA 44
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3 EUROPE 46
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.1 GERMANY 48
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.2 FRANCE 49
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.3 UK 50
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.4 ITALY 51
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.5 SPAIN 52
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.3.6 REST OF EUROPE 53
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4 ASIA-PACIFIC 54
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.1 CHINA 56
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.2 JAPAN 57
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.3 INDIA 58
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.4 SOUTH KOREA 59
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.5 AUSTRALIA 60
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.4.6 REST OF ASIA-PACIFIC 61
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.5 MIDDLE EAST & AFRICA 62
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.5.1 MIDDLE EAST 63
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
9.5.2 AFRICA 64
POMPE DISEASE TREATMENT MARKET, BY TYPE
POMPE DISEASE TREATMENT MARKET, BY THERAPY
POMPE DISEASE TREATMENT MARKET, BY END USER
10 COMPETITIVE LANDSCAPE
10.1 OVERVIEW 66
10.2 MAJOR PLAYERS IN THE GLOBAL POMPE DISEASE TREATMENT MARKET 66
10.3 COMPETITIVE BENCHMARKING 67
10.4 COMPETITOR DASHBOARD 68
10.5 MAJOR GROWTH STRATEGY IN THE GLOBAL POMPE DISEASE TREATMENT MARKET 69
10.6 THE LEADING PLAYER IN TERMS OF NUMBER OF DEVELOPMENTS IN GLOBAL POMPE DISEASE TREATMENT
MARKET 69
10.7 KEY DEVELOPMENTS & GROWTH STRATEGIES 70
10.7.1 EXPANSION 70
10.7.2 ANNOUNCEMENT 71
10.7.3 PATENT 71
10.7.4 DEVELOPMENTS 71
10.7.5 DEFINITIVE AGREEMENT 71
11 COMPANY PROFILES
11.1 AMICUS THERAPEUTICS, INC 72
11.1.1 COMPANY OVERVIEW 72
11.1.2 FINANCIAL OVERVIEW 72
11.1.3 PRODUCTS/SERVICES OFFERED 72
11.1.4 KEY DEVELOPMENTS 73
11.1.5 SWOT ANALYSIS 73
11.1.6 KEY STRATEGIES 73
11.2 SANOFI 74
11.2.1 COMPANY OVERVIEW 74
11.2.2 FINANCIAL OVERVIEW 74
11.2.3 PRODUCTS/SERVICES OFFERED 75
11.2.4 KEY DEVELOPMENTS 75
11.2.5 SWOT ANALYSIS 76
11.2.6 KEY STRATEGIES 76
11.3 AUDENTES THERAPEUTICS 77
11.3.1 COMPANY OVERVIEW 77
11.3.2 FINANCIAL OVERVIEW 77
11.3.3 PRODUCTS/SERVICES OFFERED 77
11.3.4 KEY DEVELOPMENTS 78
11.3.5 SWOT ANALYSIS 78
11.3.6 KEY STRATEGIES 78
11.4 OXYRANE 79
11.4.1 COMPANY OVERVIEW 79
11.4.2 FINANCIAL OVERVIEW 79
11.4.3 PRODUCTS/SERVICES OFFERED 79
11.4.4 KEY DEVELOPMENTS 79
11.4.5 SWOT ANALYSIS 80
11.4.6 KEY STRATEGIES 80
11.5 VALERION THERAPEUTICS 81
11.5.1 COMPANY OVERVIEW 81
11.5.2 FINANCIAL OVERVIEW 81
11.5.3 PRODUCTS/SERVICES OFFERED 81
11.5.4 KEY DEVELOPMENTS 81
11.5.5 SWOT ANALYSIS 82
11.5.6 KEY STRATEGIES 82
11.6 AVROBIO, INC. 83
11.6.1 COMPANY OVERVIEW 83
11.6.2 FINANCIAL OVERVIEW 83
11.6.3 PRODUCTS/SERVICES OFFERED 84
11.6.4 KEY DEVELOPMENTS 84
11.6.5 SWOT ANALYSIS 84
11.6.6 KEY STRATEGIES 84
11.7 CENTOGENE AG 85
11.7.1 COMPANY OVERVIEW 85
11.7.2 FINANCIAL OVERVIEW 85
11.7.3 PRODUCTS/SERVICES OFFERED 85
11.7.4 KEY DEVELOPMENTS 85
11.7.5 SWOT ANALYSIS 86
11.7.6 KEY STRATEGIES 86
12 APPENDIX
12.1 REFERENCES 87
12.2 RELATED REPORTS 87
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