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Market Research Report

Pompe Disease: Opportunity Analysis and Forecasts to 2027

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Pompe Disease: Opportunity Analysis and Forecasts to 2027
Published: November 27, 2018 Content info: 86 Pages
Description

Pompe disease (PD) is a rare genetic disorder resulting in the accumulation of glycogen with lysosomes in various tissues of the body. It is caused by a multitude of different phenotypical mutations to the GAA gene which is responsible for the transcription of α-glucosidase. α-glucosidase is responsible for glycogen degradation through enzymatic cleavage. PD is characterized by two types: infantile-onset Pomp disease (IOPD) and late-onset Pompe disease (LOPD). IOPD tends to be caused a severe deficiency or entire absence of GAA, resulting in symptoms being apparent from birth. Sufferers of IOPD tend to not reach their second birthday. LOPD has a large spectrum of severity, with symptoms manifesting typically in early adulthood. Sufferers have known to live a full life span if diagnosed early and given enzyme replacement therapy (ERT) to slow the progression of the disease.

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?

Companies mentioned: Sanofi Genzyme, Valerion Therapeutics, Amicus Therapeutics, Actus Therapeutics, Audentes Therapeutics

Scope:

  • Overview of pulmonary arterial hypertension, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized PD therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2017 and forecast for ten years to 2027.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the PD therapeutics market.
  • Overview of pulmonary arterial hypertension, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized PD therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2017 and forecast for ten years to 2027.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the PD therapeutics market.

Reasons to buy:

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.
Table of Contents
Product Code: GDHC092POA

Table of Contents

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
  • 11.6. About GlobalData
  • 11.7. Contact Us
  • 11.8. Disclaimer

List of Tables

  • Table 1: Pompe Disease: Key Metrics in the 8MM 2017-2027
  • Table 2: Risk Factors for Pompe Disease
  • Table 3: Treatment Guidelines for Pompe Disease
  • Table 4: Comparison of Therapeutic Classes in Development for Pompe Disease, 2016-2027
  • Table 5: Drugs in Development for Pompe Disease, 2018
  • Table 6: Clinical Benchmark of Key Pipeline Drugs - PD Treatments
  • Table 7: Commercial Benchmark of Key Pipeline Drugs - Pompe Disease
  • Table 8: Key Events Impacting Sales for Pompe Disease, 2017-2027
  • Table 9: Pompe Disease Market - Drivers and Barriers, 2017-2027
  • Table 10: Key Historical and Projected Launch Dates for PD

List of Figures

  • Figure 1: Global Sales Forecast by Country for Pompe Disease in 2017 and 2027
  • Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Myozyme
  • Figure 3: Simultaneous Accumulation of Unfolded Proteins and Interventions
  • Figure 4: Classification of Pompe Disease
  • Figure 5: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease
  • Figure 6: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Pompe Disease
  • Figure 7: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of Pompe Disease by Type
  • Figure 8: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of LOPD by Severity
  • Figure 9: 8MM, Sources Used to Forecast the Diagnosed Incident Cases of IOPD by CRIM Status
  • Figure 10: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 11: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 12: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 13: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 14: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 15: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-1, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 16: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-2, N, Both Sexes, All Ages, 2017 (Forecast Based on International Registry Data)
  • Figure 17: 8MM, Diagnosed Incident Cases of Pompe Disease, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 18: 8MM, Sex-Specific Diagnosed Incident Cases of Pompe Disease, N, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 19: 8MM, Diagnosed Incident Cases of Pompe Disease by Type, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 20: 8MM, Diagnosed Incident Cases of LOPD by Severity, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 21: 8MM, Diagnosed Incident Cases of IOPD by CRIM Status, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 22: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-1, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 23: 8MM, Diagnosed Prevalent Cases of Pompe Disease - Method-2, N, Both Sexes, All Ages, 2017 (Forecast Adjusted for the Underestimation of International Registry Data)
  • Figure 24: Unmet Needs and Opportunities in Pompe Disease
  • Figure 25: Overview of the Development Pipeline in Pompe Disease
  • Figure 26: Key Phase III Trial for a Promising Novel ERT that GlobalData Expects be Licensed for Pompe Disease in the 8MM During the Forecast Period
  • Figure 27: Competitive Assessment of the Pipeline Drug Benchmarked Against the SOC, Myozyme
  • Figure 28: Global (8MM) Sales Forecast by Country for Pompe Disease in 2017 and 2027
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