Gaucher Disease Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2032

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Gaucher Disease Market Report Overview

The Gaucher Disease market across the 7MM (7MM: the US, France, Germany, Italy, Spain, the UK, and Japan) was valued $1.22 billion in 2022. A steady growth is anticipated at a CAGR of less than 1% from 2022 to 2032. The major driver for this growth will be improved diagnostic approaches, the launch of Sanofi’s SRT venglustat malate, as well as the introduction of eliglustat tartrate generics.

The Gaucher Disease market research report offers an overview of Gaucher Disease, including its epidemiology, symptoms, diagnosis, and disease management. The report offers key topics covered including strategic competitor assessment and market characterization to improve business strategies. In addition, the report’s data on unmet needs and clinical trial mapping will assist in identifying market gaps and making actionable predictions.

Gaucher Disease Market Outlook 2022-2032 ($ Billion)

Gaucher Disease Market Outlook 2022-2032 ($ Billion)

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Gaucher disease (GD) is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase (GCase) results in the accumulation of harmful quantities of lipids, specifically the lipid glucocerebroside, throughout the body, particularly in the bone marrow, spleen, and liver. GD can result in delays in growth and puberty in children, gynecological and obstetric problems, Parkinson’s disease, and cancers such as myeloma, leukemia, and lymphoma.

Market Size (2022) $1.22 billion
CAGR (2022-2032) <1%
Key Countries (7MM) ·        The US

·        5EU

·        Israel

Key Drug Classes ·        ERTs

·        SRTs

·        PCs

Key Sponsors ·        Sanofi

·        Takeda

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Gaucher Disease Market Dynamics

The sales of high-priced ERTs (Cerezyme, VPRIV, and Elelyso) is a significant driver for the GD market growth. However, ERTs are expected to lose small portions of patient share to similarly expensive therapies such as Cerdelga and venglustat malate (once launched). The non-adherence to ERTs is primarily owing to older patients being reluctant to switch therapies, combined with an increase in young patients opting for SRT.

The combination of improved physician awareness and decreased patient reluctance to become involved in diagnosis and treatment, especially in the Ashkenazi Jewish population are slightly contributing to the market growth. The other minor market growth factors are increased inclusion of GD in NBS panels and wider adoption of NBS.

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Gaucher Disease Market Segmentation by Country

The key countries in the Gaucher Disease market are the US, 5EU, and Israel. In 2022, the US dominated the total Gaucher Disease market share. The dominance of the US within the 7MM is due to its larger GD population and higher drug prices.

Gaucher Disease Market Analysis by Country, 2022 (%)

Gaucher Disease Market Analysis by Country, 2022 (%)

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Gaucher Disease Market Segmentation by Drug Classes

The key drugs in the Gaucher Disease market are ERTs, SRTs, and PCs. In 2022, ERTs was the leading drug class in the Gaucher Disease market. The current management of Type 1 GD relies on marketed ERT and SRT.

Gaucher Disease Market Analysis by Drug Classes, 2022 (%)

Gaucher Disease Market Analysis by Drug Classes, 2022 (%)

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Gaucher Disease Market Segmentation by Sponsors

The key sponsors in the Gaucher Disease market are Sanofi and Takeda.

Sanofi: Sanofi’s venglustat malate is expected to launch in the US and EU in Q2 2027. As a brain-penetrant molecule, venglustat may finally provide a solution to the lack of effective neuropathic therapies. Oral route mitigates lifestyle disruption, healthcare system resource expenditure, and infusion reactions caused by ERT. Despite ERT market dominance, the notable share held by marketed SRTs provides confidence for prescribing physicians.

Gaucher Disease Market Analysis by Sponsors

Gaucher Disease Market Analysis by Sponsors

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Segments Covered in this Report

Gaucher Disease Country Outlook ($ Billion, 2022-2032)

  • The US
  • 5EU
  • Israel

Gaucher Disease Drugs Outlook (%, 2022-2032)

  • ERTs
  • PCs
  • SRTs

Scope

  • Overview of Gaucher Disease, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized Gaucher Disease treatment market revenue, cost of therapy per patient, and treatment usage patterns forecast from 2022 to 2032.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications of these factors for the Gaucher Disease therapeutics market.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for Gaucher Disease treatment. The most promising candidates in Phase III and Phase IIb development are profiled.
  • Analysis of the current and future market competition in the global Gaucher Disease 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.

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.

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 sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

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

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 Us

Frequently asked questions

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