Global Pompe Disease Treatment Market Research Report Forecast to 2030

List of Tables

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.

Product Code: MRFR/HC/6761-CR

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