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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Related
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