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Gaucher Disease - Opportunity Assessment and Forecast

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    Report

  • 59 Pages
  • December 2023
  • Region: Global
  • GlobalData
  • ID: 5923843
The prevalence of GD is increasing at a steady rate, in line with population growth in the 7MM, at an AGR of 0.39%.

Currently, the marketed treatment regimen for Type 1 GD and Type 3 GD is dominated by ERTs and SRTs (Cerezyme, VPRIV, Elelyso, Cerdelga, Zavesca).

There are several unmet needs in the GD landscape, including the lack of effective therapies for treating the neuropathic manifestations presented by Type 2 GD and Type 3 GD patients.

The GD R&D pipeline offers some innovative products, including gene therapy and venglustat malate, which is expected to be the first brain-penetrant SRT.

Steady market growth is expected throughout the forecast period as the GD population slowly increases and diagnostic approaches improve.

Key Highlights

Report deliverables include a PowerPoint report and Excel-based forecast model.

Forecast includes 7MM: US, 5EU (France, Germany, Italy, Spain and the UK) and Israel.

Forecasts cover 2022-2032.

Scope

  • Overview of GD, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized GD therapeutics market revenue in 7MM (US, 5EU and Israel), annual cost of therapy and treatment usage pattern in 2022 and forecast for 10 years to 2032.
  • 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 assessing emerging trends and mechanisms of action under development for GD treatment. The most promising candidates in late-stage development are profiled.
  • Analysis of the current and future market competition in the 7MM GD therapeutics market. Insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide a qualitative analysis of its implications.

Reasons to Buy

  • 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.
  • 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 the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
  • 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

1. Preface
1.1. Contents
1.2. Abbreviations
1.3. Related Reports
2. Executive Summary
3. Disease Overview
3.1. Overview of Gaucher Disease
3.2. Gaucher Disease SWOT Analysis
3.3. Classification of Gaucher Disease
4. Epidemiology
4.1. Diagnosed Prevalent Cases of GD
4.2. Diagnosed Prevalent Cases of GD by Type
4.3. Sources and Methodology for GD Prevalence
4.4. Sources and Methodology for GD
5. Current Treatment Options
5.1. Treatment Paradigm
5.2. Current Treatment Options
5.3. Product Profile: Enzyme Replacement Therapy (Cerezyme, VPRIV, Elelyso)
5.4. Product Profile: Substrate Reduction Therapy (Zavesca, Cerdelga)
5.5. Current Treatment Options - KOL Perspective
6. Unmet Needs and Opportunities
6.1. Unmet Needs in Gaucher Disease
6.2. Significant Lack of Neuropathic Therapies
6.3. Marketed Therapies are Highly Expensive
6.4. Inconvenient Treatment Options
6.5. Early Diagnosis and Screening is Required
7. R&D Strategies
7.1. Trends in Deal-Making in Gaucher Disease
7.2. Trends in Clinical Trial Design in Gaucher Disease
8. Pipeline Assessment
8.1. Gaucher Disease Pipeline Overview
8.2. Late-Stage Pipeline Agents for Gaucher Disease
8.3. Product Profile: Sanofi’s Venglustat malate
8.4. Product Profile: Sanofi’s Eliglustat tartrate
8.5. Product Profile: SZMC-Takeda’s Ambroxol
8.6. Gaucher Disease Clinical Trials Overview
9. Market Outlook
9.1. Gaucher Disease Market Forecast
9.2. Market Drivers and Barriers
10. Appendix
10.1. Bibliography
10.2. About the Authors
11. Contact the Publisher

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Sanofi
  • Genzyme
  • Takeda
  • Pfizer
  • Actelion
  • Freeline Therapeutics
  • Prevail Therapeutics
  • Johnson & Johnson