Global Pompe disease Drugs Market Analysis


  • Type: Rare Disease Area
  • Published : February 2022
  • Region: Worldwide
  • Country: Other
  • Report ID : 5052

  • Format: PPT, PDF

Global Pompe disease Drugs Market: Segmented by Therapy type, molecule type, route of administration, and dosage formsGrowth, Market Size, Future Prospects & Competitive Analysis, 2020–2028

SKU: 5052 Categories: ,

Report Description of the Global Pompe disease Drugs Market

Research Scope and Assumption

  • The report provides the market value for the base year 2020 and a yearly forecast up to 2028 in terms of revenue (USD billion)
  • The key industry dynamics, regulatory scenario, reimbursement scenario, major market trends, and drug markets are evaluated to understand their impacts on the demand for the forecast period. The growth rates were estimated using correlation, regression, and time-series analysis
  • We have used the combination of top-down and bottom-up approach for market sizing, analyzing key regional markets, dynamics, and trends for various solutions, services, and end uses
  • All market estimates and forecasts have been validated through primary interviews with the Key Industry Players (KIPs) and secondary analysis
  • Inflation has not been accounted for in order to estimate and forecast the market
  • Numbers may not add up due to rounding off

Research Objective:

  • To analyze and forecast the market size of Global Pompe disease Drugs market
  • To classify and forecast Global Pompe disease Drugs market based on application, drugs, project type, and application
  • To identify drivers and challenges for Global Pompe disease Drugs market
  • To examine competitive developments such as mergers and acquisitions, agreements, collaborations and partnerships, etc., in Global Pompe disease Drugs market
  • To conduct pricing analysis for Global Pompe disease Drugs market
  • To identify and analyze the profile of leading players operating in Global Pompe disease Drugs market

Reason to buy the report:

  • Facilitate decision-making based on strong current and forecast data for Global Pompe disease drug market
  • Develop strategies based on the latest regulatory framework
  • Strategically analyze micro-markets with respect to individual growth trends, future prospects, and their contribution to the market
  • Analyze competitive developments such as expansions, investments, mergers & acquisitions, new product developments, and research & developments in the Global Pompe disease market
  • Analyze the opportunities in the market for stakeholders and draw a competitive landscape for market leaders
  • To strategically profile key players and comprehensively analyze their market shares and core competencies
  • We have technically sound team which do a deep dive research and also provide strategy-based consulting analysis

Global Pompe disease Drug Market Executive Summary

The global pompe disease market is expected to witness significant growth through the forecast period. Pompe disease (glycogenosis type II) is a rare disorder affecting approximately 1 in 40,000 people in the U.S. Factors such as continued uptake of long-term therapies, the launch of novel drugs, and high Drug cost are propelling the market growth.

Market Size and Key Findings

The Global Pompe diseaseDrugs market size is at around USD 1,277 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of 3.7% during the forecast period.

The Global Pompe disease market is expected to witness substantial growth during the forecast period. The market is expected to grow at a CAGR of 3.7 percent during forecast period. This can be attributed to the great investments by market players in the research and development of Pompe disease therapy. Another factor is the growing penetration of drugs during the forecast period, and the existence of a strong pipeline.

Market Dynamics

Market Growth Drivers Analysis

According to the National Organization for Rare Disorders, the estimated frequency occurrence of Pompe disease is approximately 1 in 40,000 people in the US. The increase in the prevalence of pompe disease is driving the Pompe Disease Market owing towards the increase of manufacturers focusing on expanding their pipeline to cater to the demand for Pompe disease Drug. Governments have taken initiatives for developing and manufacturing innovative products for the diagnosis & Drug of Pompe disease is the other major factor driving growth of the Pompe Disease market.

Increasing developments in the gene therapy and enzyme replacement therapies are the primary factors driving the growth of Pompe disease Market. However, inflated costs associated with the Pompe disease therapy could hinder the market growth opportunities. Moreover, research activities performed to find an effective Drug for the disease and the growth in the special regulatory drug designations for orphan drugs can further create the growth opportunities for Pompe disease market.

Market Restraints

The lack of cost-effective Drugs is restraining the growth of the market. The Drug is very expensive owing to the lack of FDA approved drugs which leads to the less affordability rate. The scarcity of low-cost Drug hampers the growth of the market owing towards the patients being uncured.

Competitive Landscape

Key Players

The Pompe Disease Market is moderately competitive and consists of several major players. Some of the companies which are currently dominating the market are Genzyme Corporation, Amicus Therapeutics, Valerion Therapeutics, Audentes Therapeutics, EpiVax, Inc, Oxyrane, and Sangamo Therapeutics.

Products in Pipeline

The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for the Drug of patients with Pompe disease (acid α-glucosidase deficiency). The target action date for the FDA decision is May 18, 2021.

Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of acid alpha-glucosidase (GAA) enzyme to muscle cells, and if approved, would offer a potential new standard of care for patients with Pompe disease.

Notable Recent Deals

Pharma companies are entering into multiple partnerships & collaborations in order to expand its offerings and enhance its capabilities to provide Pompe disease Drug solutions

Some Acquisition and Partnership deals are:

  • The biotech companies Shireand NanoMedSyn have launched a new research partnership to evaluate a potential Drug for lysosomal storage disorders including Pompe disease. NanoMedSyn has developed an enzyme replacement therapy using proprietary technology called AMFA that was shown in preclinical models to have promising biological activity. The companies will now explore its potential in the Drug of these types of disorders.

Healthcare Policies and Regulatory Landscape

Pricing and Reimbursement scenario of pompe disease drug

Reimbursement for pompe disease Drug can be granted either under the commitment of assessing clinical performance by means of relevant health outcomes – “conditional reimbursement linked to performance” and “coverage with evidence development” – or by stemming the budget impact (financially based schemes) – “price-volume” and “per patient utilisation caps”. These reimbursement mechanisms have been categorised as managed entry agreements (MEAs) or market access agreements.

1. Report Description of the Global Pompe disease Drug Market
1.1 Research Scope and Assumption
1.2 Objective of the study
1.3 Research Methodology
1.4 Reason to buy the report
2. Global Pompe disease Drug Market Executive Summary
2.1 Global Pompe Disease Drug Market – Industry Snapshot & key buying criteria, 2020-2028
2.2 Market Size, Growth Prospects and Key findings
3. Market Dynamics of Global Pompe disease Drug Market
3.1 Market Growth Drivers Analysis
3.1.1 Increase In The Prevalence of Pompe Disease
3.1.2 Increasing developments in the gene therapy and enzyme replacement therapies
3.2 Market Restraints Analysis
3.2.1 High Cost of Drug
3.2.2 Lack of Regulatory Standards
3.2.3 Rising Patient Cliff
4. Global Pompe disease Drug Market Segmentation
4.1 By therapy type
4.1.1 Enzyme Replacement Therapy
4.1.2 Substrate Reduction Therapy
4.2 By molecule type
4.2.1 Biologics
4.2.2 Small Molecules
4.3 By route of administration
4.3.1 Oral
4.3.2 Parenterial
4.4 By the dosage form
4.4.1 Solid
4.4.2 Liquid
4.5 By Geography
4.5.1 North America
4.5.2 Europe
4.5.3 Asia Pacific
4.5.4 Middle East and Africa
4.5.5 South America
5. Global Pompe disease Major Products Market Share
5.1 Market Analysis, Insights and Forecast – By Revenue
b6.1 Major Top Market Players
6.2 Products in Pipeline
6.3 R&D Initiatives in Global Pompe disease Market
6.4 Notable recent deals in Global Pompe disease Drugs Market
6.4.1 Joint Ventures
6.4.2 Partnerships
6.4.3 Mergers & Acquisitions
6.4.4 Strategic divestments
7. Key Company Profiles
7.1 Market Research Future Overview
7.2 Fortune Business Insights Overview
Product & Services, Strategies & Financials
7.3 Market Watch Overview
Product & Services, Strategies & Financials
7.4 Mordor Intelligence Overview
Product & Services, Strategies & Financials
7.5 Industry ARC Overview
Product & Services, Strategies & Financials
7.6 Allied Market Research Overview
Product & Services, Strategies & Financials
7.7 Biospace Overview
Product & Services, Strategies & Financials
7.9 Amicus Therapeutics Inc. Overview
Product & Services, Strategies & Financials
7.10 The insights Partners Overview
Product & Services, Strategies & Financials
7.11 Cipla Limited Company Overview
Product & Services, Strategies & Financials
7.12 Delveinsights
Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape
8.1 Pricing and Reimbursement scenario of Pompe disease drug
8.2 Government Initiatives / Intervention programs
9. Factors Driving Future Growth
9.1 New Trends and Development of Global Pompe disease drugs market
9.2 Future Opportunities
10. Strategic Recommendations

Global Pompe disease Drug Market Segmentation

This research report categorizes the Pompe Disease to forecast the revenues and analyze the trends in each of the following sub-markets:

ByTherapy Type:

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

  • Enzyme Replacement Therapy (ERT)
  • Substrate Replacement Therapy (SRT)
  • Chaperone- Advance Replacement Therapy (CART)
  • Supportive Care

By Molecule Type:

  • Pompe disease market Drug segmented by moleucle which is Biologics and small molecules

By Dosage Form Type:

  • Pompe disease market Drug is segmented by dosage forms in the market which is in the Solid and Liquid forms.

By Route of Administration

The market is segmented based on the route of administration, the Pompe disease Drug market is segmented into oral and Parental. In 2020, the oral drug delivery segment accounted for the largest share of the pharmaceutical drug delivery market.

By Geography

Based on Geography the global pompe disease drugs market is segmented into North

America, Europe, Asia-Pacific and the Rest of the World

  • North America
    • US
    • Canada
    • Mexico
  • Europe
    • Germany
    • France
    • UK
    • Italy
    • Spain
    • Rest of Europe
  • Asia-Pacific
    • Japan
    • China
    • India
    • Australia
    • South Korea
    • Rest of APAC
    • Brazil
    • Saudi Arabia
    • South Africa
    • Rest Of LAMEA

Regional Analysis:

North America dominated global pompe disease market. Increasing prevalence of pompe disease in the region and increasing healthcare expenditure are some of the major factors driving the growth of the pompe disease Drug market. According to US national library of Medicine National Institute of Health, the prevelance of pompe disease is 1 in 28,000 in United States. The Asia Pacific is anticipated to grow significant CAGR due to increasing awareness among patients regarding rare diseases and improving healthcare facilities in Asian countries.

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.

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