Gaucher Disease Drugs Market Size, Share & Trends Analysis Report By Type, By Therapy (Enzyme Replacement Therapy, Substrate Replacement Therapy), By Major Markets, And Segment Forecasts, 2018 - 2025

Published: Oct, 2018 | Pages: 84 | Publisher: Grand View Research
Industry: Pharmaceutical | Report Format: Electronic (PDF)

The global gaucher disease (GD) drugs market size is expected to reach USD 1.71 billion by 2025, according to a new report by Grand View Research, Inc., exhibiting a CAGR of 1.1% during the forecast period. Growing prevalence of the disease and increasing research and development activities in the field are likely to help the market in gaining momentum over the coming years. With high unmet needs, there is a strong need for development of novel treatments with improved efficacy in neuronopathic complications, convenient dosage, and reduced cost.

Gaucher disease is a genetic metabolic disorder, caused by mutations in GBA gene. People with Gaucher disease produce low glucocerebrosidase (GCase), an enzyme required to degrade glucocerebroside. Excess accumulation of lipids in spleen, liver, kidney, brain, lungs, and bone marrow impedes the normal functioning of the organs.

In spite of being the most prevalent lysosomal storage disorder, Gaucher disease is a rare condition with an average prevalence of 1 in 100,000 individuals. However, certain ethnic races such as Ashkenazi Jews are more susceptible to this disorder. Ashkenazi Jews have an average disease prevalence of 1 in 500 individuals.

Currently, type 1 is the most common form of Gaucher disease. This condition is nonneuronopathic in nature, as it does not affect the central nervous system (CNS). Type 1 GD commanded more than 75.0% of the market in 2017. It is estimated to retain its position through 2025. Type 2 and type 3 are not as common as type 1.

On the basis of treatment, enzyme replacement therapy (ERT) was the leading revenue contributor in the market in 2017. The growth of the segment can be attributed to strong sales of Cerezyme and Vpriv. However, there has been an increase in inclination towards substrate replacement therapy (SRT), spearheaded by Cerdelga and Zavesca. ERT is mostly indicated for type 1 and type 3 (to address issues unrelated to the CNS), as replacement enzymes cannot penetrate blood brain barrier. Additionally, there is no effective treatment for type 2.

Further key findings from the report suggest:

• Type 2 is the rarest of the three forms of GD, with average mortality rate of less than two years

• Enzyme replacement therapy dominated the treatment landscape, headed by Sanofi Genzyme's Cerezyme. However, GD treatment is witnessing a shift towards substrate replacement therapy, which is anticipated to register a CAGR of over 7.5% during the forecast period

• The U.S. spearheaded the major markets, in terms of sales as well as disease burden. The country will continue to dominate the market through 2025, on account of widening patient base and easy accessibility to treatments

• Some of prominent companies operating in the market are Sanofi Genzyme, Shire, Pfizer, and Johnson & Johnson

• Sanofi Genzyme led the competitive space and is projected to continue holding prominent position in the market over the coming years, due to significant commercial performances of Cerezyme and Cerdelga

 Table of Contents

Chapter 1 Methodology and Scope
                 1.1 Research Methodology
                 1.2 Research Scope and Assumptions
                 1.3 List to Data Sources
                 1.4 List of Abbreviations
Chapter 2 Executive Summary
Chapter 3 Disease Primer and Epidemiology
                 3.1 Disease Primer
                     3.1.1 Types
                     3.1.2 Symptoms
                     3.1.3 Complications
                 3.2 Epidemiology
                 3.3 Current Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)
                 3.4 Forecast Prevalence and Incidence for Seven Major Markets (U.S., Japan, EU5)
Chapter 4 Global Gaucher Disease Drugs Market Overview
                 4.1 Introduction and Market Overview
                     4.1.1 Segmentation, by type
                         4.1.1.1 Type 1
                         4.1.1.2 Type 2
                         4.1.1.3 Type 3
                     4.1.2 Segmentation, by therapy
                         4.1.2.1 Enzyme replacement therapy
                         4.1.2.2 Substrate replacement therapy
                     4.1.3 Segmentation, by major market
                         4.1.3.1 U.S.
                         4.1.3.2 EU5
                         4.1.3.3 Japan
                     4.1.4 Market size and forecast
                     4.1.5 Sales performance
                     4.1.6 Market dynamics among leading brands
                 4.2 Patent Expiry Schedule
                 4.3 Drivers and Challenges
                 4.4 Deals Landscape (2013 - 2018)
                 4.5 Pricing and Reimbursement
                 4.6 Emerging Markets
                 4.7 SWOT Analysis
Chapter 5 Gaucher Disease Drugs Market: Pipeline Intelligence
                 5.1 Pipeline Landscape
                     5.1.1 Drugs under Development
                 5.2 Key R&D Trends
                     5.2.1 Biomarkers
                     5.2.2 DNA Mutation Analysis
                 5.3 Late-Stage Pipeline
                 5.4 Profile of Disruptive Drugs
                     5.4.1 GZ402671
Chapter 6 Company Profiles
                 6.1 Sanofi Genzyme
                     6.1.1 Company Overview
                     6.1.2 Current Product Portfolio
                     6.1.3 Product Forecast Sales
                     6.1.4 Company - key news flow
                     6.1.5 Pipeline View
                     6.1.6 Pipeline Forecast
                     6.1.7 Catalysts and Event Calendar
                     6.1.8 SWOT Analysis
                 6.2 Shire Therapeutics
                     6.2.1 Company Overview
                     6.2.2 Current Product Portfolio
                     6.2.3 Product Forecast Sales
                     6.2.4 Company - key news flow
                     6.2.5 Pipeline View
                     6.2.6 Pipeline Forecast
                     6.2.7 Catalysts and Events Calendar
                     6.2.8 SWOT Analysis
                 6.3 Pfizer
                     6.3.1 Company Overview
                     6.3.2 Current Product Portfolio
                     6.3.3 Product Forecast Sales
                     6.3.4 Company - key news flow
                     6.3.5 Pipeline View
                     6.3.6 Pipeline Forecast
                     6.3.7 Catalysts and Events Calendar
                     6.3.8 SWOT Analysis
                 6.4 Johnson & Johnson
                     6.4.1 Company Overview
                     6.4.2 Current Product Portfolio
                     6.4.3 Product Forecast Sales
                     6.4.4 Company - key news flow
                     6.4.5 Pipeline View
                     6.4.6 Pipeline Forecast
                     6.4.7 Catalysts and Event Calendar
                     6.4.8 SWOT Analysis
Chapter 7 Market Outlook
                 7.1 Winners and Losers
                 7.2 Emerging Companies
                 7.3 What the Future Holds

List of Tables

Table 1 List of Abbreviation
Table 2 Clinical Features of Gaucher Disease
Table 3 Gaucher Disease Prevalence and Incidence - 2017
Table 4 Gaucher Disease Prevalence and Incidence Forecast, 2017A-2025E 
Table 5 Gaucher Disease Drugs Market Size and Forecast (in USD Million)
Table 6 Geographic Sales Performance, by Seven Major Markets (in USD Million)
Table 7 Gaucher Disease Drugs Market, by Type (in USD Million)
Table 8 Gaucher Disease Drugs Market, by Therapy (in USD Million)
Table 9 Gaucher Disease Drugs - Patent Expiry Schedule
Table 10 Gaucher Disease Deals Landscape (2013-2018)
Table 11 Gaucher Disease Product Pricing
Table 12 Gaucher Disease Drugs in Development
Table 13 Late-Stage Gaucher Disease Pipeline
Table 14 Pipeline Forecast - Global Gaucher Disease Drugs
Table 15 Profile of Disruptive Drug: GZ402671
Table 16 Product Portfolio: Cerezyme
Table 17 Product Portfolio: Cerdelga
Table 18 Cerezyme Product Forecast Sales 2017A-2025E (in USD Million)
Table 19 CerdelgaProduct Forecast Sales 2017A-2025E (in USD Million)
Table 20 Sanofi Genzyme Pipeline View
Table 21 Sanofi Genzyme Catalysts and Event Calendar
Table 22 Product Portfolio: Vpriv
Table 23 Vpriv Product Forecast Sales 2017A-2025E (in USD Million)
Table 24 Shire Pipeline View
Table 25 Shire Catalysts and Event Calendar
Table 26 Product Portfolio: Elelyso
Table 27 Elelyso Product Forecast Sales 2017A-2025E (in USD Million)
Table 28 Pfizer Pipeline View
Table 29 Pfizer Catalysts and Event Calendar
Table 30 Product Portfolio: Zavesca
Table 31 Zavesca Product Forecast Sales 2017A-2025E (in USD Million)
Table 32 Johnson & Johnson Pipeline View
Table 33 Johnson & Johnson Catalysts and Event Calendar

List of Figures

Fig. 1 Current Prevalence Across Seven Major Markets - 2017
Fig. 2 Current Incidence Across Seven Major Markets - 2017
Fig. 3 Market Segmentation and Scope
Fig. 4 Geographic Sales Performance by Seven Major Markets 2017
Fig. 5 Gaucher Disease Drugs Market, by Type (2017)
Fig. 6 Gaucher Disease Drugs Market, by Therapy, 2017A-2025E (in USD Million)
Fig. 7 Gaucher Disease Drugs Market Trend, by Therapy (2017)
Fig. 8 U.S. Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 9 U.S. Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 10 U.K. Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 11 U.K. Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 12 Germany Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 13 Germany Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 14 Spain Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 15 Spain Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 16 France Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 17 France Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 18 Italy Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 19 Italy Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 20 Japan Gaucher Disease Drugs Market, by Type (in USD Million)
Fig. 21 Japan Gaucher Disease Drugs Market, by Therapy (in USD Million)
Fig. 22 Gaucher Disease Drugs Market Share Distribution, by Company (2017A-2025E)
Fig. 23 Market Trends & Outlook
Fig. 24 Market Driver Relevance Analysis (Current & Future Impact)
Fig. 25 Market Restraint Relevance Analysis (Current & Future Impact)
Fig. 26 SWOT Analysis (Gaucher Disease Drugs Market)
Fig. 27 Pipeline Assets, by Phase
Fig. 28 SWOT Analysis (Sanofi Genzyme)
Fig. 29 SWOT Analysis (Shire Therapeutics)
Fig. 30 SWOT Analysis (Pfizer)
Fig. 31 SWOT Analysis (Johnson & Johnson)