At a CAGR 2.9% Gaucher Disease Market - Opportunity Analysis and Forecast to 2024
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About
Gaucher Disease Market
GlobalData estimated the 2014
sales for the Gaucher Disease (GD) market at approximately $875m
across the 7MM (US, France, Germany, Italy, Spain, UK, and Israel).
The US market contributed the majority of these sales, generating an
estimated $482m. By the end of the forecast period in 2024, GD sales
are expected to increase to $1.16 billion at a Compound Annual Growth
Rate (CAGR) of 2.9%. The majority of these sales will continue to
come from the US, which will represent approximately 56.2% of the GD
market in the 7MM in 2024. GlobalData forecasts an increase in the
number of diagnosed prevalent cases of GD, and therefore, an increase
in the treated GD population. Improved diagnosis will be driven by
increasing physician education and awareness of GD and its signs and
symptoms. Despite the anticipated increase in the number of diagnosed
prevalent cases of GD over the forecast period, it is expected that a
large portion of the GD population will remain undiagnosed. This is
due to continued late diagnosis and misdiagnosis of GD as a result of
the heterogeneous manifestations of the disease. The sparsely
populated GD pipeline, with no drugs in late-stage development, will
further hinder the growth of the GD market.
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Highlights
Key Questions Answered
Although the use of enzyme
replacement therapies (ERTs) and substrate reduction therapies (SRTs)
has significantly changed the natural history of GD, with a marked
decrease in morbidity, especially in Type 1 GD patients, GD market is
still marked by the presence of a number of unmet needs in current
treatments. What are the main unmet needs in this market? Will the
drugs under development fulfil the unmet needs of the GD market?
The late-stage GD pipeline is very
sparsely populated and there no drugs in late-stage development.
Nevertheless, the GD market will experience substantial growth. What
are the main drivers of this growth during 2014-2024? Which of the
marketed drugs will have the highest peak sales at the highest CAGR,
and why?
Key Findings
GlobalData forecasts an increase
in the number of diagnosed prevalent cases of GD, and therefore, an
increase in the treated GD population. Improved diagnosis will be
driven by increasing physician education and awareness of GD and its
signs and symptoms. This is largely attributable to Genzymes and
Shires continued efforts to raise awareness of rare diseases through
organized events and established websites, and by sponsoring patient
registries.
Genzymes recently approved oral
substrate reduction therapy (SRT), Cerdelga (eliglustat), is more
effective and safer than the other oral SRT, Zavesca (miglustat), and
therefore will be used more commonly as an oral alternative to IV ERT
in adult Type 1 GD patients. As Cerdelga is more expensive than the
other GD therapies, it is expected to drive the growth of the GD
market.
None of the therapies fulfilled
the greatest unmet need in GD treatment need for the drugs that can
treat the neurological manifestations of the disease. One of the main
R&D strategies in the GD space is the focus on the development of
new SRT formulations and pharmacological chaperones (PCs) that can
cross the blood brain barrier and thereby improve the neurological
manifestations of GD.
Scope
Overview of GD, including
epidemiology, etiology, pathophysiology, symptoms, diagnosis, and
treatment guidelines.
Annualized GD therapeutics market
revenue, annual cost of therapy and treatment usage pattern data from
2014 and forecast for ten years to 2024.
Key topics covered include
strategic competitor assessment, market characterization, unmet
needs, clinical trial mapping and implications for the GD
therapeutics market.
Pipeline analysis: comprehensive
data split across different phases, emerging novel trends under
development, and detailed analysis of late-stage pipeline drugs.
Analysis of the current and future
market competition in the global GD therapeutics market. Insightful
review of the key industry drivers, restraints and challenges. Each
trend is independently researched to provide qualitative analysis of
its implications.
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 GD therapeutics
market.
Drive revenues by understanding
the key trends, innovative products and technologies, market
segments, and companies likely to impact the GD 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 7MM GD
therapeutics market from 2014-2024.
Organize your sales and marketing
efforts by identifying the market categories and segments that
present maximum opportunities for consolidations, investments and
strategic partnerships.
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Table of Contents
1 Table of Contents
1 Table of Contents 10
1.1 List of Tables 14
1.2 List of Figures 17
2 Introduction 18
2.1 Catalyst 18
2.2 Related Reports 19
3 Disease Overview 20
3.1 Etiology and Pathophysiology
23
3.1.1 Etiology 23
3.1.2 Pathophysiology 26
3.2 Symptoms 27
3.3 Prognosis 31
3.4 Quality of Life 32
4 Epidemiology 33
4.1 Disease Background 33
4.2 Risk Factors and Comorbidities
34
4.3 Global Trends 35
4.3.1 US 35
4.3.2 5EU 36
4.3.3 Israel 36
4.4 Forecast Methodology 37
4.4.1 Sources Used 37
4.4.2 Sources Not Used 38
4.4.3 Forecast Assumptions and
Methods 39
4.5 Epidemiological Forecast for
Gaucher Disease (2014-2024) 40
4.5.1 Total Prevalent Cases of
Type 1 Gaucher Disease 40
4.5.2 Age-Specific Total Prevalent
Cases of Type 1 Gaucher Disease 42
4.5.3 Sex-Specific Total Prevalent
Cases of Type 1 Gaucher Disease 44
4.5.4 Age-Standardized Total
Prevalence of Type 1 Gaucher Disease 46
4.5.5 Total Prevalent Cases of
Type 2 Gaucher Disease 47
4.5.6 Sex-Specific Total Prevalent
Cases of Type 2 Gaucher Disease 50
4.5.7 Total Prevalent Cases of
Type 3 Gaucher Disease 50
4.5.8 Age-Specific Total Prevalent
Cases of Type 3 Gaucher Disease 52
4.5.9 Sex-Specific Total Prevalent
Cases of Type 3 Gaucher Disease 54
4.5.10 Age-Standardized Total
Prevalence of Type 3 Gaucher Disease 55
4.6 Discussion 56
4.6.1 Epidemiological Forecast
Insight 56
4.6.2 Limitations of the Analysis
57
4.6.3 Strengths of the Analysis 58
5 Current Treatment Options 59
5.1 Overview 59
5.1.1 Enzyme Replacement Therapy
59
5.1.2 Substrate Reduction Therapy
62
5.1.3 ERT Dosing Issues 64
5.1.4 Treatment Guidelines 70
5.2 Product Profiles 73
5.2.1 Enzyme Replacement Therapies
73
5.2.2 Substrate Reduction
Therapies 102
6 Unmet Needs Assessment and
Oppportunity Analysis 130
6.1 Overview 130
6.2 Treatment of the Neurological
Manifestations of Gaucher Disease 132
6.2.1 Unmet Needs 132
6.2.2 Gap Analysis 134
6.2.3 Opportunity 136
6.3 Earlier Gaucher Disease
Diagnosis and Disease Severity Assessment 138
6.3.1 Unmet Needs 138
6.3.2 Gap Analysis 145
6.3.3 Opportunity 149
6.4 Lower Cost of Gaucher Disease
Treatment 152
6.4.1 Unmet Needs 152
6.4.2 Gap Analysis 153
6.4.3 Opportunity 156
6.5 Improved Route of
Administration 158
6.5.1 Unmet Needs 158
6.5.2 Gap Analysis 159
6.5.3 Opportunity 161
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