Summary

Lysosomal Storage Disease Treatment Market Summary

The global lysosomal storage disease treatment market size was estimated at USD 4.54 billion in 2024 and is projected to reach USD 6.68 billion by 2033, growing at a CAGR of 4.54% from 2025 to 2033, driven by rising diagnostic capabilities, advancements in enzyme replacement and gene therapies, and increased awareness of rare genetic disorders. The lysosomal storage disease (LSD) treatment market is experiencing steady growth due to expanding research and development initiatives and rising awareness about these rare genetic conditions.

Significant R&D investments have enhanced therapeutic advancements, helping address unmet medical needs. A study published by the National Library of Medicine in July 2022 noted that LSDs, as a group, are more prevalent than previously assumed, with a collective incidence ranging from 1 in 5,000 to 1 in 8,000 individuals. It also highlighted the influence of ethnicity and geography on disease prevalence. For instance, Gaucher disease appears in 1 in 40,000 to 60,000 people in the general population, while the rate increases to 1 in 800 among Ashkenazi Jews. Similarly, Tay-Sachs disease and Niemann-Pick A are more common in this demographic, while the Finnish population has a higher frequency of aspartylglucosaminuria at 1 in 18,500. These rising prevalence rates underscore the growing need for effective treatments, fueling market expansion.

The introduction of innovative therapies is expected to strengthen the LSD treatment market further. Companies actively pursue novel drug development, supported by increasing global R&D spending. For example, in February 2021, Genethon initiated a gene therapy clinical trial targeting late-onset Pompe disease in collaboration with Spark Therapeutics, utilizing proprietary technologies. In parallel, awareness campaigns have gained momentum, playing a vital role in improving patient outcomes. In countries like the U.S. and the UK, awareness of Fabry disease has significantly increased through initiatives like Fabry Disease Awareness Month, observed every April, as promoted by the National Fabry Disease Foundation (NFDF), Fabry International Network, Fabry Australia, and the UK’s MPS Society.

Global Lysosomal Storage Disease Treatment Market Report Segmentation

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Grand View Research has segmented the global lysosomal storage disease treatment market report based on treatment type, disease type, and region:

• Treatment Type Outlook (Revenue, USD Million, 2021 - 2033)
• Enzyme Replacement Therapy
o Velaglucerase Alfa
o Alglucosidase Alfa (Myozyme/Lumizyme)
o Idursulfase (Elaprase)
o Imiglucerase (Cerezyme)
o Others
• Substrate Reduction Therapy
• Others
• Disease Type Outlook (Revenue, USD Million, 2021 - 2033)
• Gaucher Disease
• Mucopolysaccharidoses
• Pompe Disease
• Fabry Disease
• Others
• Regional Outlook (Revenue, USD Million, 2021 - 2033)
• North America
o U.S.
o Canada
o Mexico
• Europe
o UK
o Germany
o France
o Italy
o Spain
o Denmark
o Sweden
o Norway
• Asia Pacific
o Japan
o China
o India
o Australia
o South Korea
o Thailand
• Latin America
o Brazil
o Argentina
• Middle East & Africa
o South Africa
o Saudi Arabia
o UAE
o Kuwait

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Table of Contents

Table of Contents

Chapter 1. Methodology and Scope
1.1. Market Segmentation & Scope
1.2. Segment Definitions
1.2.1. Treatment Type
1.2.2. Disease Type
1.2.3. Regional
1.3. Estimates and Forecast Timeline
1.4. Research Methodology
1.5. Information Procurement
1.5.1. Purchased Database
1.5.2. GVR’s Internal Database
1.5.3. Secondary Sources
1.5.4. Primary Research
1.6. Information Analysis
1.6.1. Data Analysis Models
1.7. Market Formulation & Data Visualization
1.8. Model Details
1.8.1. Commodity Flow Analysis
1.9. List of Secondary Sources
1.10. Objectives
Chapter 2. Executive Summary
2.1. Market Snapshot
2.2. Segment Snapshot
2.3. Competitive Landscape Snapshot
Chapter 3. Lysosomal Storage Disease (LSDs) Treatment Market Variables, Trends, & Scope
3.1. Market Lineage Outlook
3.2. Market Dynamics
3.2.1. Market Driver Analysis
3.2.2. Market Restraint Analysis
3.3. Business Environment Analysis
3.3.1. Industry Analysis - Porter’s Five Forces Analysis
3.3.1.1. Supplier Power
3.3.1.2. Buyer Power
3.3.1.3. Substitution Threat
3.3.1.4. Threat of New Entrants
3.3.1.5. Competitive Rivalry
3.3.2. PESTLE Analysis
3.3.3. Pipeline Analysis
3.3.4. Patent Expiry Analysis
3.3.5. Pricing Analysis
Chapter 4. Lysosomal Storage Disease (LSDs) Treatment Market: Treatment Type Business Analysis
4.1. Treatment Type Market Share, 2025 & 2033
4.2. Treatment Type Segment Dashboard
4.3. Market Size & Forecasts and Trend Analysis, by Treatment Type, 2021 to 2033 (USD Million)
4.4. Enzyme Replacement Therapy
4.4.1. Enzyme Replacement Therapy Market, 2021 - 2033 (USD Million)
4.4.2. Velaglucerase Alfa
4.4.2.1. Velaglucerase Alfa Market, 2021 - 2033 (USD Million)
4.4.3. Alglucosidase Alfa (Myozyme/Lumizyme)
4.4.3.1. Alglucosidase Alfa (Myozyme/Lumizyme) Market, 2021 - 2033 (USD Million)
4.4.4. Idursulfase (Elaprase)
4.4.4.1. Idursulfase (Elaprase) Market, 2021 - 2033 (USD Million)
4.4.5. Imiglucerase (Cerezyme)
4.4.5.1. Imiglucerase (Cerezyme) Market, 2021 - 2033 (USD Million)
4.4.6. Others
4.4.6.1. Others Market, 2021 - 2033 (USD Million)
4.5. Substrate Reduction Therapy
4.5.1. Substrate Reduction Therapy Market, 2021 - 2033 (USD Million)
4.5.2. Others Market, 2021 - 2033 (USD Million)
Chapter 5. Lysosomal Storage Disease (LSDs) Treatment Market: Disease Type Business Analysis
5.1. Disease Type Market Share, 2025 & 2033
5.2. Disease Type Segment Dashboard
5.3. Market Size & Forecasts and Trend Analysis, by Disease Type, 2021 to 2033 (USD Million)
5.4. Gaucher Disease
5.4.1. Gaucher Disease Market, 2021 - 2033 (USD Million)
5.5. Mucopolysaccharidoses
5.5.1. Mucopolysaccharidoses Market, 2021 - 2033 (USD Million)
5.6. Pompe Disease
5.6.1. Pompe Disease Market, 2021 - 2033 (USD Million)
5.7. Fabry Disease
5.7.1. Fabry Disease Market, 2021 - 2033 (USD Million)
5.8. Others
5.8.1. Others Market, 2021 - 2033 (USD Million)
Chapter 6. Lysosomal Storage Disease (LSDs) Treatment Market: Regional Estimates & Trend Analysis
6.1. Regional Market Share Analysis, 2025 & 2033
6.2. Regional Market Dashboard
6.3. Market Size & Forecasts Trend Analysis, 2021 to 2033:
6.4. North America
6.4.1. North America Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, By Country, 2021 - 2033 (USD Million)
6.4.2. U.S.
6.4.2.1. Key Country Dynamics
6.4.2.2. Target Disease Type Prevalence
6.4.2.3. Regulatory Framework
6.4.2.4. Reimbursement Framework
6.4.2.5. U.S. Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.4.3. Canada
6.4.3.1. Key Country Dynamics
6.4.3.2. Target Disease Type Prevalence
6.4.3.3. Regulatory Framework
6.4.3.4. Reimbursement Framework
6.4.3.5. U.S. Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.4.4. Mexico
6.4.4.1. Key Country Dynamics
6.4.4.2. Target Disease Type Prevalence
6.4.4.3. Regulatory Framework
6.4.4.4. Reimbursement Framework
6.4.4.5. Mexico Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5. Europe
6.5.1. Europe Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.2. UK
6.5.2.1. Key Country Dynamics
6.5.2.2. Target Disease Type Prevalence
6.5.2.3. Regulatory Framework
6.5.2.4. Reimbursement Framework
6.5.2.5. Uk Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.3. Germany
6.5.3.1. Key Country Dynamics
6.5.3.2. Target Disease Type Prevalence
6.5.3.3. Regulatory Framework
6.5.3.4. Reimbursement Framework
6.5.3.5. Germany Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.4. France
6.5.4.1. Key Country Dynamics
6.5.4.2. Target Disease Type Prevalence
6.5.4.3. Regulatory Framework
6.5.4.4. Reimbursement Framework
6.5.4.5. France Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.5. Italy
6.5.5.1. Key Country Dynamics
6.5.5.2. Target Disease Type Prevalence
6.5.5.3. Regulatory Framework
6.5.5.4. Reimbursement Framework
6.5.5.5. Italy Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.6. Spain
6.5.6.1. Key Country Dynamics
6.5.6.2. Target Disease Type Prevalence
6.5.6.3. Regulatory Framework
6.5.6.4. Reimbursement Framework
6.5.6.5. Spain Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.7. Denmark
6.5.7.1. Key Country Dynamics
6.5.7.2. Target Disease Type Prevalence
6.5.7.3. Regulatory Framework
6.5.7.4. Reimbursement Framework
6.5.7.5. Denmark Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.8. Sweden
6.5.8.1. Key Country Dynamics
6.5.8.2. Target Disease Type Prevalence
6.5.8.3. Regulatory Framework
6.5.8.4. Reimbursement Framework
6.5.8.5. Sweden Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.5.9. Norway
6.5.9.1. Key Country Dynamics
6.5.9.2. Target Disease Type Prevalence
6.5.9.3. Regulatory Framework
6.5.9.4. Reimbursement Framework
6.5.9.5. Norway Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6. Asia Pacific
6.6.1. Asia Pacific Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.2. Japan
6.6.2.1. Key Country Dynamics
6.6.2.2. Target Disease Type Prevalence
6.6.2.3. Regulatory Framework
6.6.2.4. Reimbursement Framework
6.6.2.5. Japan Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.3. China
6.6.3.1. Key Country Dynamics
6.6.3.2. Target Disease Type Prevalence
6.6.3.3. Regulatory Framework
6.6.3.4. Reimbursement Framework
6.6.3.5. China Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.4. India
6.6.4.1. Key Country Dynamics
6.6.4.2. Target Disease Type Prevalence
6.6.4.3. Regulatory Framework
6.6.4.4. Reimbursement Framework
6.6.4.5. India Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.5. Australia
6.6.5.1. Key Country Dynamics
6.6.5.2. Target Disease Type Prevalence
6.6.5.3. Regulatory Framework
6.6.5.4. Reimbursement Framework
6.6.5.5. Australia Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.6. South Korea
6.6.6.1. Key Country Dynamics
6.6.6.2. Target Disease Type Prevalence
6.6.6.3. Regulatory Framework
6.6.6.4. Reimbursement Framework
6.6.6.5. South Korea Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.6.7. Thailand
6.6.7.1. Key Country Dynamics
6.6.7.2. Target Disease Type Prevalence
6.6.7.3. Regulatory Framework
6.6.7.4. Reimbursement Framework
6.6.7.5. Thailand Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.7. Latin America
6.7.1. Latin America Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.7.2. Brazil
6.7.2.1. Key Country Dynamics
6.7.2.2. Target Disease Type Prevalence
6.7.2.3. Regulatory Framework
6.7.2.4. Reimbursement Framework
6.7.2.5. Japan Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.7.3. Argentina
6.7.3.1. Key Country Dynamics
6.7.3.2. Target Disease Type Prevalence
6.7.3.3. Regulatory Framework
6.7.3.4. Reimbursement Framework
6.7.3.5. China Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.8. Middle East and Africa
6.8.1. Middle East and Africa Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2017 - 2033 (USD Million)
6.8.2. South Africa
6.8.2.1. Key Country Dynamics
6.8.2.2. Target Disease Type Prevalence
6.8.2.3. Regulatory Framework
6.8.2.4. Reimbursement Framework
6.8.2.5. South Africa Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.8.3. Saudi Arabia
6.8.3.1. Key Country Dynamics
6.8.3.2. Target Disease Type Prevalence
6.8.3.3. Regulatory Framework
6.8.3.4. Reimbursement Framework
6.8.3.5. Saudi Arabia Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.8.4. UAE
6.8.4.1. Key Country Dynamics
6.8.4.2. Target Disease Type Prevalence
6.8.4.3. Regulatory Framework
6.8.4.4. Reimbursement Framework
6.8.4.5. UAE Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
6.8.5. Kuwait
6.8.5.1. Key Country Dynamics
6.8.5.2. Target Disease Type Prevalence
6.8.5.3. Regulatory Framework
6.8.5.4. Reimbursement Framework
6.8.5.5. Kuwait Lysosomal Storage Disease (LSDs) Treatment Market Estimates and Forecasts, 2021 - 2033 (USD Million)
Chapter 7. Competitive Landscape
7.1. Participant Overview
7.2. Company Market Position Analysis
7.3. Company Categorization
7.4. Strategy Mapping
7.5. Company Profiles/Listing
7.5.1. Pfizer Inc
7.5.1.1. Overview
7.5.1.2. Financial Performance
7.5.1.3. Treatment Type Benchmarking
7.5.1.4. Strategic Initiatives
7.5.2. Takeda Pharmaceutical Company Limited (Shire Plc)
7.5.2.1. Overview
7.5.2.2. Financial Performance
7.5.2.3. Treatment Type Benchmarking
7.5.2.4. Strategic Initiatives
7.5.3. Sanofi (Genzyme Corporation)
7.5.3.1. Overview
7.5.3.2. Financial Performance
7.5.3.3. Treatment Type Benchmarking
7.5.3.4. Strategic Initiatives
7.5.4. BioMarin
7.5.4.1. Overview
7.5.4.2. Financial Performance
7.5.4.3. Treatment Type Benchmarking
7.5.4.4. Strategic Initiatives
7.5.5. Johnson & Johnson (Actelion Pharmaceuticals Ltd)
7.5.5.1. Overview
7.5.5.2. Financial Performance
7.5.5.3. Treatment Type Benchmarking
7.5.5.4. Strategic Initiatives
7.5.6. Amicus Therapeutics, Inc
7.5.6.1. Overview
7.5.6.2. Financial Performance
7.5.6.3. Treatment Type Benchmarking
7.5.6.4. Strategic Initiatives
7.5.7. Alexion Pharmaceuticals, Inc
7.5.7.1. Overview
7.5.7.2. Financial Performance
7.5.7.3. Treatment Type Benchmarking
7.5.7.4. Strategic Initiatives
7.5.8. Sigilon Therapeutics, Inc
7.5.8.1. Overview
7.5.8.2. Financial Performance
7.5.8.3. Treatment Type Benchmarking
7.5.8.4. Strategic Initiatives
7.5.9. Orphazyme A/S
7.5.9.1. Overview
7.5.9.2. Financial Performance
7.5.9.3. Treatment Type Benchmarking
7.5.9.4. Strategic Initiatives