Indian Pompe Disease Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Asia
  • Country: India
  • Report ID : 1283

  • Format: PPT, PDF

Indian Pompe Disease Market: Segmented By Therapy Type (Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Gene Therapy and Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, & Online Pharmacies) – Growth, Trends, and Forecasts (2019 – 2028)

SKU: 1283 Categories: ,

Report Overview

Indian Pompe Disease market size stood at USD XX Billion in 2019 and is projected to reach USD XX Billion by 2028, exhibiting a CAGR of xx% during the forecast period.

It is a genetic disorder in which complex sugar called glycogen builds up metabolism in the body’s cells. The disease results from the deficiency of an enzyme called Acid Alfa Glucosidase (GAA), which breaks downs complex sugars in the body. This buildup occurs in organs and tissues, especially in muscles, causing them to break down. The additional glycogen builds up in the patient and is stored in skeletal muscle, heart and other tissues, which causes the progressive indications of pompe disease. It causes muscle weakness and trouble breathing affecting the liver, heart (Cardiomegaly), and muscles that can lead to premature death in the new-born. Pompe disease is categorized into two broad categories as adult-onset pompe disease and infantile onset pompe disease. Both the parents pass on one strangely altered copy of the gene to their child. A parent with an altered copy of the gene is known as a carrier and the disease can affect both men and women irrespective of the gender.

Market Drivers

Increasing research activities preformed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs drive the market. Moreover, emerging immunotherapeutic approaches for Pompe disease offer lucrative opportunities for the expansion of the market. Furthermore, increasing development in the field of enzyme replacement therapies and gene therapy will also create a lucrative opportunity for the pompe disease market in the forecasted period as these are the therapies used in the treatment of this disorder. However, high treatment costs associated with the treatment of pompe diseases may hamper the market to a certain extent.

1. Indian Pompe Disease Market Overview………………………………………………….
A. Market Size
2. Market Growth Drivers………………………………………………………………….
A. Rise in Prevalence of Pompe Disease
B. Emerging Immunotherapeutic Approaches for Pompe Disease
3. Epidemiology of Pompe Disease ………………………………………………….
A. Age-specific and Sex-Specific Management for Pompe Disease
B. Medication Rates for Pompe Disease
4. Pompe Disease Market Segmentation……………………………………………………
A. Therapy Type
I. Enzyme Replacement Therapy (ERT)
II. Substrate Reduction Therapy (SRT)
III. Gene Therapy
IV. Others
B. Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Pompe Disease Major Drugs Market Share……………………………………
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Amicus Therapeutics Company overview, Product & Services, Strategies & Financials
B. Genzyme overview, Product & Services, Strategies & Financials
C. Valerion 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 Pompe Disease market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Therapy Type:

Based on therapy type the market is segmented into Enzyme Replacement Therapy (ERT), Substrate Reduction Therapy (SRT), Gene Therapy and Others. In 2019, Enzyme Replacement Therapy (ERT) segment dominated the Pompe Disease Market in terms of revenue is estimated to grow at a CAGR of 3.0%. Enzyme Replacement Therapy is used to increase the levels of GAA in the body and reduce the accumulation of glycogen inside cells. ERT is administered into the bloodstream and has been shown to reduce the abnormal thickening of the heart walls that is characteristic of Pompe disease. It also helps maintain muscle function and improve patients’ quality of life. Lumizyme (alglucosidase alfa) is an FDA approved drug for Enzyme Replacement Therapy abetting towards the market’s growth.

By Distribution Channel:

Based on distribution channel, the market for Pompe Disease can be segmented into hospital pharmacies, retail pharmacies, and online pharmacies. The hospital segment is expected to dominate the market throughout the forecast period. The increasing number of clinics is propelling the growth of the specialty clinics segment.

Major Players

India is expected to hold a significant market share in the global Pompe Disease Market due to rising launches of novel drugs, the presence of a large number of pharmaceutical companies, growing research and development expenditure, rising patient preference associated with advanced therapies, product launches, and technological advancements. Key players that operate in the market are Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.

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