Table of Content

1. Executive Summary
   1.1. Global Enzyme Replacement Therapy Market Snapshot, 2024 and 2031
   1.2. Market Opportunity Assessment, 2024 - 2031, US$ Bn
   1.3. Key Market Trends
   1.4. Future Market Projections
   1.5. Premium Market Insights
   1.6. Industry Developments and Key Market Events
   1.7. PMR Analysis and Recommendations
2. Market Overview
   2.1. Market Scope and Definition
   2.2. Market Dynamics
       2.2.1. Drivers
       2.2.2. Restraints
       2.2.3. Opportunity
       2.2.4. Challenges
       2.2.5. Key Trends
   2.3. Macro-Economic Factors
       2.3.1. Global Sectorial Outlook
       2.3.2. Global GDP Growth Outlook
       2.3.3. Global Healthcare Spending Outlook
   2.4. COVID-19 Impact Analysis
   2.5. Forecast Factors - Relevance and Impact
3. Value Added Insights
   3.1. Therapeutic Conditions Adoption Analysis
   3.2. Technology Assessment
   3.3. Regulatory Landscape
   3.4. Value Chain Analysis
   3.5. Key Deals and Mergers
   3.6. PESTLE Analysis
   3.7. Porter’s Five Force Analysis
4. Price Trend Analysis, 2019 - 2031
   4.1. Key Highlights
   4.2. Key Factors Impacting Product Prices
   4.3. Pricing Analysis, By Therapeutic Conditions
   4.4. Regional Prices and Product Preferences
5. Global Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   5.1. Key Highlights
       5.1.1. Market Volume (Units) Projections
       5.1.2. Market Size (US$ Bn) and Volume (Units) and Y-o-Y Growth
       5.1.3. Absolute $ Opportunity
   5.2. Market Size (US$ Bn) and Volume (Units) Analysis and Forecast
       5.2.1. Historical Market Size (US$ Bn) and Volume (Units) Analysis, 2019-2023
       5.2.2. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, 2023-2031
   5.3. Global Enzyme Replacement Therapy Market Outlook: Therapeutic Conditions
       5.3.1. Introduction / Key Findings
       5.3.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Therapeutic Conditions, 2019 - 2023
       5.3.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
           5.3.3.1. Fabry Disease
           5.3.3.2. Gaucher Disease
           5.3.3.3. Mucopolysaccharidosis (MPS)
               5.3.3.3.1. MPS I (Hurler Syndrome)
               5.3.3.3.2. MPS II (Hunter Syndrome)
               5.3.3.3.3. MPS IVA (Morquio Syndrome, Type A)
               5.3.3.3.4. MPS VI (Maroteaux-Lamy Syndrome)
               5.3.3.3.5. MPS VII (Sly Syndrome)
           5.3.3.4. Pompe Disease
           5.3.3.5. Lysosomal Acid Lipase Deficiency
           5.3.3.6. Others
   5.4. Market Attractiveness Analysis: Therapeutic Conditions
   5.5. Global Enzyme Replacement Therapy Market Outlook: Route of Administration
       5.5.1. Introduction / Key Findings
       5.5.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Route of Administration, 2019 - 2023
       5.5.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
           5.5.3.1. Oral
           5.5.3.2. Injectable
   5.6. Market Attractiveness Analysis: Route of Administration
   5.7. Global Enzyme Replacement Therapy Market Outlook: Distribution Channel
       5.7.1. Introduction / Key Findings
       5.7.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Distribution Channel, 2019 - 2023
       5.7.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
           5.7.3.1. Hospital Pharmacies
           5.7.3.2. Specialty Treatment Pharmacies
           5.7.3.3. Retail Pharmacies
   5.8. Market Attractiveness Analysis: Distribution Channel
6. Global Enzyme Replacement Therapy Market Outlook: Region
   6.1. Key Highlights
   6.2. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Region, 2019 - 2023
   6.3. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Region, 2024 - 2031
       6.3.1. North America
       6.3.2. Europe
       6.3.3. East Asia
       6.3.4. South Asia and Oceania
       6.3.5. Latin America
       6.3.6. Middle East & Africa
   6.4. Market Attractiveness Analysis: Region
7. North America Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   7.1. Key Highlights
   7.2. Pricing Analysis
   7.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       7.3.1. By Country
       7.3.2. By Therapeutic Conditions
       7.3.3. By Route of Administration
       7.3.4. By Distribution Channel
   7.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       7.4.1. U.S.
       7.4.2. Canada
   7.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       7.5.1. Enzyme Replacement Therapy for Fabry Disease
       7.5.2. Enzyme Replacement Therapy for Gaucher Disease
       7.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       7.5.4. Enzyme Replacement Therapy for Pompe Disease
       7.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       7.5.6. Others
   7.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       7.6.1. Oral Enzyme Replacement Therapy
       7.6.2. Injectable Enzyme Replacement Therapy
   7.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       7.7.1. Hospital Pharmacies
       7.7.2. Specialty Treatment Pharmacies
       7.7.3. Retail Pharmacies
   7.8. Market Attractiveness Analysis
8. Europe Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   8.1. Key Highlights
   8.2. Pricing Analysis
   8.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       8.3.1. By Country
       8.3.2. By Therapeutic Conditions
       8.3.3. By Route of Administration
       8.3.4. By Distribution Channel
   8.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       8.4.1. Germany
       8.4.2. France
       8.4.3. U.K.
       8.4.4. Italy
       8.4.5. Spain
       8.4.6. Russia
       8.4.7. Türkiye
       8.4.8. Rest of Europe
   8.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       8.5.1. Enzyme Replacement Therapy for Fabry Disease
       8.5.2. Enzyme Replacement Therapy for Gaucher Disease
       8.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       8.5.4. Enzyme Replacement Therapy for Pompe Disease
       8.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       8.5.6. Others
   8.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       8.6.1. Oral Enzyme Replacement Therapy
       8.6.2. Injectable Enzyme Replacement Therapy
   8.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       8.7.1. Hospital Pharmacies
       8.7.2. Specialty Treatment Pharmacies
       8.7.3. Retail Pharmacies
   8.8. Market Attractiveness Analysis
9. East Asia Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   9.1. Key Highlights
   9.2. Pricing Analysis
   9.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       9.3.1. By Country
       9.3.2. By Therapeutic Conditions
       9.3.3. By Route of Administration
       9.3.4. By Distribution Channel
   9.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       9.4.1. China
       9.4.2. Japan
       9.4.3. South Korea
   9.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       9.5.1. Enzyme Replacement Therapy for Fabry Disease
       9.5.2. Enzyme Replacement Therapy for Gaucher Disease
       9.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       9.5.4. Enzyme Replacement Therapy for Pompe Disease
       9.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       9.5.6. Others
   9.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       9.6.1. Oral Enzyme Replacement Therapy
       9.6.2. Injectable Enzyme Replacement Therapy
   9.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       9.7.1. Hospital Pharmacies
       9.7.2. Specialty Treatment Pharmacies
       9.7.3. Retail Pharmacies
   9.8. Market Attractiveness Analysis
10. South Asia & Oceania Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   10.1. Key Highlights
   10.2. Pricing Analysis
   10.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       10.3.1. By Country
       10.3.2. By Therapeutic Conditions
       10.3.3. By Route of Administration
       10.3.4. By Distribution Channel
   10.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       10.4.1. India
       10.4.2. Southeast Asia
       10.4.3. ANZ
       10.4.4. Rest of South Asia & Oceania
   10.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       10.5.1. Enzyme Replacement Therapy for Fabry Disease
       10.5.2. Enzyme Replacement Therapy for Gaucher Disease
       10.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       10.5.4. Enzyme Replacement Therapy for Pompe Disease
       10.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       10.5.6. Others
   10.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       10.6.1. Oral Enzyme Replacement Therapy
       10.6.2. Injectable Enzyme Replacement Therapy
   10.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       10.7.1. Hospital Pharmacies
       10.7.2. Specialty Treatment Pharmacies
       10.7.3. Retail Pharmacies
   10.8. Market Attractiveness Analysis
11. Latin America Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   11.1. Key Highlights
   11.2. Pricing Analysis
   11.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       11.3.1. By Country
       11.3.2. By Therapeutic Conditions
       11.3.3. By Route of Administration
       11.3.4. By Distribution Channel
   11.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       11.4.1. Brazil
       11.4.2. Mexico
       11.4.3. Rest of Latin America
   11.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       11.5.1. Enzyme Replacement Therapy for Fabry Disease
       11.5.2. Enzyme Replacement Therapy for Gaucher Disease
       11.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       11.5.4. Enzyme Replacement Therapy for Pompe Disease
       11.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       11.5.6. Others
   11.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       11.6.1. Oral Enzyme Replacement Therapy
       11.6.2. Injectable Enzyme Replacement Therapy
   11.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       11.7.1. Hospital Pharmacies
       11.7.2. Specialty Treatment Pharmacies
       11.7.3. Retail Pharmacies
   11.8. Market Attractiveness Analysis
12. Middle East & Africa Enzyme Replacement Therapy Market Outlook: Historical (2019 - 2023) and Forecast (2024 - 2031)
   12.1. Key Highlights
   12.2. Pricing Analysis
   12.3. Historical Market Size (US$ Bn) and Volume (Units) Analysis, By Market, 2019 - 2023
       12.3.1. By Country
       12.3.2. By Therapeutic Conditions
       12.3.3. By Route of Administration
       12.3.4. By Distribution Channel
   12.4. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Country, 2024 - 2031
       12.4.1. GCC Countries
       12.4.2. Egypt
       12.4.3. South Africa
       12.4.4. Northern Africa
       12.4.5. Rest of Middle East & Africa
   12.5. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Therapeutic Conditions, 2024 - 2031
       12.5.1. Enzyme Replacement Therapy for Fabry Disease
       12.5.2. Enzyme Replacement Therapy for Gaucher Disease
       12.5.3. Enzyme Replacement Therapy for Mucopolysaccharidosis (MPS)
       12.5.4. Enzyme Replacement Therapy for Pompe Disease
       12.5.5. Enzyme Replacement Therapy for Lysosomal Acid Lipase Deficiency
       12.5.6. Others
   12.6. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Route of Administration, 2024 - 2031
       12.6.1. Oral Enzyme Replacement Therapy
       12.6.2. Injectable Enzyme Replacement Therapy
   12.7. Current Market Size (US$ Bn) and Volume (Units) Analysis and Forecast, By Distribution Channel, 2024 - 2031
       12.7.1. Hospital Pharmacies
       12.7.2. Specialty Treatment Pharmacies
       12.7.3. Retail Pharmacies
   12.8. Market Attractiveness Analysis
13. Competition Landscape
   13.1. Market Share Analysis, 2023
   13.2. Market Structure
       13.2.1. Competition Intensity Mapping By Market
       13.2.2. Competition Dashboard
   13.3. Company Profiles (Details - Overview, Financials, Strategy, Recent Developments)
       13.3.1. Sanofi Genzyme
           13.3.1.1. Overview
           13.3.1.2. Segments and Therapeutic Conditions
           13.3.1.3. Key Financials
           13.3.1.4. Market Developments
           13.3.1.5. Market Strategy
       13.3.2. Shire (now part of Takeda Pharmaceuticals)
       13.3.3. BioMarin Pharmaceutical Inc.
       13.3.4. Amicus Therapeutics
       13.3.5. Alexion Pharmaceuticals
       13.3.6. Ultragenyx Pharmaceutical Inc.
       13.3.7. Sarepta Therapeutics
       13.3.8. Biomarin
       13.3.9. Genzyme (now part of Sanofi)
       13.3.10. Takeda Pharmaceuticals
       13.3.11. Pfizer Inc.
       13.3.12. AbbVie Inc.
       13.3.13. Vertex Pharmaceuticals
       13.3.14. Sangamo Therapeutics
       13.3.15. Regeneron Pharmaceuticals Inc.
       13.3.16. Spark Therapeutics (now part of Roche)
       13.3.17. Orchard Therapeutics
       13.3.18. Novartis AG
       13.3.19. CSL Behring
       13.3.20. Audentes Therapeutics
14. Appendix
   14.1. Research Methodology
   14.2. Research Assumptions
   14.3. Acronyms and Abbreviations