Pompe Disease Opportunity Analysis and Forecasts to 2027 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Pompe
Disease: Opportunity Analysis and Forecasts to 2027” report has
been added to ResearchAndMarkets.com’s offering.

It is estimated that the 2017 sales for the PD market to be
approximately $684M across the 8 major markets (US, 5EU (France,
Germany, Italy, Spain, and the UK), Japan, and Brazil). The US market is
anticipated to grow the fastest of the three regions, recording a CAGR
of 4.9%, while the 5EU and Japan will each record CAGRs of 1.2% and
0.6%, respectively.

At the end of 2027, the US will contribute around 51.9% of global sales,
while the 5EU and Japan will account for 35.7% and 10.3% of global
sales, respectively. The higher sales numbers for the US can be
attributed to the higher price of pharmaceuticals and the greater
diagnosed prevalence of PD. By the end of the forecast period in 2027,
PD sales are expected to grow to $903M at a Compound Annual Growth Rate
(CAGR) of 2.8% over the 10-year period. The majority of sales in the 8MM
in 2027 will come from the US, which will represent 41.9% of the market.

An increase in the number of newly diagnosed cases of PD is forecasted,
and consequently in the number of treatable Pompe patients, as a result
of increasing awareness of PD among physicians. It is also expected that
because PD is a rare, life-threatening disease with debilitating
symptoms, ERT will continue to have a positive reimbursement status in
the 5EU and Japan, despite the high cost, and will continue to be
covered by insurance plans in the US.

The report “Pompe Disease: Opportunity Analysis and Forecasts to
2027”, answers the following key questions:

• ERT therapies are currently the cornerstone of PD treatment. Patients
  typically receive therapy from diagnosis to receive reduce glycogen
  accumulation. Nevertheless, ERT therapy cannot cross the blood brain
  barrier causing inevitable deterioration of the CNS resulting in
  death. Because of this, there are considerably high unmet needs within
  the indication. What are the main unmet needs in this market? Will the
  drugs under development fulfil the unmet needs of the PD market with
  novel mechanisms of action?
• The current late-stage PD pipeline encompasses one additional ERT
  therapy being development by the current market drug owners Sanofi
  Genzyme. Will the late-stage drug make a significant impact on the PD
  market? Will this late-stage developmental drug capture a share of the
  PD market? and why?

The report will enable you to:

• Develop and design your in-licensing and out-licensing strategies
  through a review of pipeline products and technologies, and by
  identifying the companies with the most robust pipeline. Additionally
  a list of acquisition targets included in the pipeline product company
  list.
• Develop business strategies by understanding the trends shaping and
  driving the global PD therapeutics market.
• Drive revenues by understanding the key trends, innovative products
  and technologies, market segments, and companies likely to impact the
  global PD therapeutics market in future.
• Formulate effective sales and marketing strategies by understanding
  the competitive landscape and by analysing the performance of various
  competitors.
• Identify emerging players with potentially strong product portfolios
  and create effective counter-strategies to gain a competitive
  advantage.
• Track drug sales in the global PD therapeutics market from 2017-2027.
• Organize your sales and marketing efforts by identifying the market
  categories and segments that present maximum opportunities for
  consolidations, investments and strategic partnerships.

Key Topics Covered:

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Pompe Disease: Executive Summary

2.1 Growth of the Pompe Disease Market Is Expected from 2017-2027

2.2 High Unmet Need for Earlier Pompe Disease Diagnosis

2.3 Pipeline Drugs for Pompe Disease Treatment

2.4 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.4.3 Diagnosed Incident Cases of Pompe Disease – Based on International
Registry Data

5.4.4 Diagnosed Incident Cases of Pompe Disease by Type – Based on
International Registry Data

5.4.5 Diagnosed Incident Cases of LOPD by Severity – Based on
International Registry Data

5.4.6 Diagnosed Incident Cases of IOPD by CRIM Status – Based on
International Registry Data

5.4.7 Diagnosed Prevalent Cases of Pompe Disease – Based on
International Registry Data

5.4.8 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.4.9 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.4.10 Forecast Adjusted for the Underestimation of International
Registry Data

5.5 Epidemiological Forecast for Pompe Disease (2017-2027) – Based on
International Registry Data

5.5.1 Diagnosed Incident Cases of Pompe Disease

5.5.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease

5.5.3 Diagnosed Incident Cases of Pompe Disease by Type

5.5.4 Diagnosed Incident Cases of LOPD by Severity

5.5.5 Diagnosed Incident Cases of IOPD by CRIM Status

5.5.6 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.5.7 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.6 Epidemiological Forecast for Pompe Disease (2017-2027) – Adjusted
for the Underestimation of International Registry Data

5.6.1 Diagnosed Incident Cases of Pompe Disease

5.6.2 Sex-Specific Diagnosed Incident Cases of Pompe Disease

5.6.3 Diagnosed Incident Cases of Pompe Disease by Type

5.6.4 Diagnosed Incident Cases of LOPD by Severity

5.6.5 Diagnosed Incident Cases of IOPD by CRIM Status

5.6.6 Diagnosed Prevalent Cases of Pompe Disease – Method-1

5.6.7 Diagnosed Prevalent Cases of Pompe Disease – Method-2

5.7 Discussion

5.7.1 Epidemiological Forecast Insight

5.7.2 Limitations of the Analysis

5.7.3 Strengths of the Analysis

6 Current Treatment Options

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Earlier Diagnosis

7.3 Therapies with Alternative MoA

7.4 Treatments with Improved Efficacy

7.5 Treatment with a Less Invasive Route of Administration

8 R&D Strategies

8.1 Overview

8.1.1 Novel Enzyme Replacement Therapies

8.1.2 Gene Therapy

8.2 Clinical Trials Design

8.2.1 Clinical Trial Treatment Periods

9 Pipeline Assessment

9.1 Overview

9.2 Other Drugs in Development

10 Pipeline Valuation Analysis

10.1 Clinical Benchmark of Key Pipeline Drugs

10.2 Commercial Benchmark of Key Pipeline Drugs

10.3 Competitive Assessment

10.4 Top-Line 10-Year Forecast

10.4.1 US

10.4.2 5EU

10.4.3 Japan

10.4.4 Brazil

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

11.3.1 Forecasting Methodology

11.3.2 Diagnosed Patients

11.3.3 Percent Drug-Treated Patients

11.3.4 Drugs Included in Each Therapeutic Class

11.3.5 Launch and Patent Expiry Dates

11.3.6 General Pricing Assumptions

11.3.7 Individual Drug Assumptions

11.4 Primary Research – KOLs Interviewed for This Report

11.4.1 KOLs

11.5 About the Authors

11.5.1 Analyst

11.5.2 Managing Analyst

11.5.3 Therapy Area Director

11.5.4 Epidemiologist

11.5.5 Managing Epidemiologist

11.5.6 Global Director of Therapy Analysis and Epidemiology

11.5.7 Global Head and EVP of Healthcare Operations and Strategy

Companies Mentioned

• Sanofi Genzyme
• Valerion Therapeutics
• Amicus Therapeutics
• Actus Therapeutics
• Audentes Therapeutics

For more information about this report visit https://www.researchandmarkets.com/r/4tgst8

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