The North America Lysosomal Disease Treatment Market would witness market growth of 5.2% CAGR during the forecast period (2022-2028).
Mannose-6-phosphate (M6P) receptors are used to direct the majority of lysosomal hydrolases to lysosomes. Similar to secreted proteins, lysosomal enzymes are glycoproteins that are synthesized in the ER and transported by the Golgi. When M6P residues are added, molecules are transported to the lysosome, but a portion of them (which varies depending on the particular enzyme in question) escape this pathway and are released from the cell. By targeting the internalized enzyme into the endocytic pathway with cell surface M6P receptors, this released enzyme is available for re-capture by the same cell or nearby cells.
Since sick cells can absorb exogenous enzyme, which is subsequently transferred to the lysosome where they can break down stored substrate and restore equilibrium, this aspect of lysosomal enzyme cell biology has significant therapeutic implications. It is also possible to utilize techniques like gene therapy and stem cell transplantation to create this exogenous enzyme outside of the body and inject it into the blood. With these later strategies, if a small number of cells are of donor origin or have been transduced with the correcting gene, they can deliver the corrective enzyme to the surrounding tissues through the secretion-recapture mechanism.
According to the World Health Organization, a rare disease affects no more than one person out of every 1000. To meet their unique needs and in light of their population, healthcare system, and resource availability, various nations have developed their definitions. A disease or ailment that affects fewer than 200,000 patients nationwide is considered to be rare in the US. A rare disease affects no more than 5 persons in 10,000 and can be fatal or severely disabling. Drug makers are incentivized by laws like the Orphan Drug Act (ODA) in the United States and Canada to stimulate the production of medications for rare disorders.
The US market dominated the North America Lysosomal Disease Treatment Market by Country in 2021, thereby, achieving a market value of $2,977.1 Million by 2028. The Canada market is experiencing a CAGR of 7.6% during (2022 - 2028). Additionally, The Mexico market is poised to grow at a CAGR of 6.6% during (2022 - 2028).
Based on Disease Type, the market is segmented into Gaucher's Diseases, Fabry Diseases, Mucopolysaccharidosis, Pompe’s Syndrome and Others. Based on Type of Therapy, the market is segmented into Enzyme Replacement Therapy, Substrate Reduction Therapy, Stem Cell Therapy and Others. Based on End User, the market is segmented into Hospitals, Clinics and Others. Based on countries, the market is segmented into U.S., Mexico, Canada, and Rest of North America.
Free Valuable Insights: The Global Lysosomal Disease Treatment Market is Predict to reach $11 Billion by 2028, at a CAGR of 6.3%
The market research report covers the analysis of key stake holders of the market. Key companies profiled in the report include Merck & Co., Inc., Johnson & Johnson (Janssen Global Services, LLC), Eli Lilly And Company, Pfizer, Inc., Takeda Pharmaceutical Company Limited, Sanofi S.A., Novartis AG, AstraZeneca PLC (Alexion Pharmaceuticals, Inc.), BioMarin Pharmaceutical Inc. and Sigilon Therapeutics, Inc.
Scope of the Study
Market Segments Covered in the Report:
By Disease Type
- Gaucher's Diseases
- Fabry Diseases
- Mucopolysaccharidosis
- Pompe’s Syndrome
- Others
By Type of Therapy
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- Stem Cell Therapy
- Others
By End User
By Country
- US
- Canada
- Mexico
- Rest of North America
Key Market Players
List of Companies Profiled in the Report:
- Merck & Co., Inc.
- Johnson & Johnson (Janssen Global Services, LLC)
- Eli Lilly And Company
- Pfizer, Inc.
- Takeda Pharmaceutical Company Limited
- Sanofi S.A.
- Novartis AG
- AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
- BioMarin Pharmaceutical Inc.
- Sigilon Therapeutics, Inc.
Chapter 1. Market Scope & Methodology
1.1 Market Definition
1.2 Objectives
1.3 Market Scope
1.4 Segmentation
1.4.1 North America Lysosomal Disease Treatment Market, by Disease Type
1.4.2 North America Lysosomal Disease Treatment Market, by Type of Therapy
1.4.3 North America Lysosomal Disease Treatment Market, by End User
1.4.4 North America Lysosomal Disease Treatment Market, by Country
1.5 Methodology for the research
Chapter 2. Market Overview
2.1 Introduction
2.1.1 Overview
2.1.1.1 Market composition & scenarios
2.2 Key Factors Impacting the Market
2.2.1 Market Drivers
2.2.2 Market Restraints
Chapter 3. North America Lysosomal Disease Treatment Market by Disease Type
3.1 North America Gaucher's Diseases Market by Country
3.2 North America Fabry Diseases Market by Country
3.3 North America Mucopolysaccharidosis Market by Country
3.4 North America Pompe’s Syndrome Market by Country
3.5 North America Others Market by Country
Chapter 4. North America Lysosomal Disease Treatment Market by Type of Therapy
4.1 North America Enzyme Replacement Therapy Market by Country
4.2 North America Substrate Reduction Therapy Market by Country
4.3 North America Stem Cell Therapy Market by Country
4.4 North America Others Market by Country
Chapter 5. North America Lysosomal Disease Treatment Market by End User
5.1 North America Hospitals Market by Country
5.2 North America Clinics Market by Country
5.3 North America Others Market by Country
Chapter 6. North America Lysosomal Disease Treatment Market by Country
6.1 US Lysosomal Disease Treatment Market
6.1.1 US Lysosomal Disease Treatment Market by Disease Type
6.1.2 US Lysosomal Disease Treatment Market by Type of Therapy
6.1.3 US Lysosomal Disease Treatment Market by End User
6.2 Canada Lysosomal Disease Treatment Market
6.2.1 Canada Lysosomal Disease Treatment Market by Disease Type
6.2.2 Canada Lysosomal Disease Treatment Market by Type of Therapy
6.2.3 Canada Lysosomal Disease Treatment Market by End User
6.3 Mexico Lysosomal Disease Treatment Market
6.3.1 Mexico Lysosomal Disease Treatment Market by Disease Type
6.3.2 Mexico Lysosomal Disease Treatment Market by Type of Therapy
6.3.3 Mexico Lysosomal Disease Treatment Market by End User
6.4 Rest of North America Lysosomal Disease Treatment Market
6.4.1 Rest of North America Lysosomal Disease Treatment Market by Disease Type
6.4.2 Rest of North America Lysosomal Disease Treatment Market by Type of Therapy
6.4.3 Rest of North America Lysosomal Disease Treatment Market by End User0.Chapter 7. Company Profiles
7.1 Merck & Co., Inc.
7.1.1 Company Overview
7.1.2 Financial Analysis
7.1.3 Segmental and Regional Analysis
7.1.4 Research & Development Expenses
7.2 Johnson & Johnson (Janssen Global Services, LLC)
7.2.1 Company Overview
7.2.2 Financial Analysis
7.2.3 Segmental &Regional Analysis
7.2.4 Research & Development Expenses
7.3 Eli Lilly And Company
7.3.1 Company Overview
7.3.2 Financial Analysis
7.3.3 Regional Analysis
7.3.4 Research & Development Expenses
7.3.5 Recent strategies and developments:
7.3.5.1 Acquisition and Mergers:
7.4 Pfizer, Inc.
7.4.1 Company Overview
7.4.2 Financial Analysis
7.4.3 Regional & Segmental Analysis
7.4.4 Research & Development Expense
7.4.5 Recent strategies and developments:
7.4.5.1 Partnerships, Collaborations, and Agreements:
7.5 Takeda Pharmaceutical Company Limited
7.5.1 Company Overview
7.5.2 Financial Analysis
7.5.3 Regional Analysis
7.5.4 Research & Development Expense
7.6 Sanofi S.A.
7.6.1 Company Overview
7.6.2 Financial Analysis
7.6.3 Segmental and Regional Analysis
7.6.4 Research & Development Expense
7.6.5 Recent strategies and developments:
7.6.5.1 Approvals and Trials:
7.7 Novartis AG
7.7.1 Company Overview
7.7.2 Financial Analysis
7.7.3 Segmental and Regional Analysis
7.7.4 Research & Development Expense
7.8 AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
7.8.1 Company Overview
7.8.2 Financial Analysis
7.8.3 Regional Analysis
7.8.4 Research & Development Expenses
7.9 BioMarin Pharmaceutical Inc.
7.9.1 Company Overview
7.9.2 Financial Analysis
7.9.3 Regional Analysis
7.9.4 Research & Development Expenses
7.9.5 Recent strategies and developments:
7.9.5.1 Approvals and Trials:
7.10. Sigilon Therapeutics, Inc.
7.10.1 Company Overview
7.10.2 Financial Analysis
7.10.3 Research & Development Expenses
TABLE 1 North America Lysosomal Disease Treatment Market, 2018 - 2021, USD Million
TABLE 2 North America Lysosomal Disease Treatment Market, 2022 - 2028, USD Million
TABLE 3 North America Lysosomal Disease Treatment Market by Disease Type, 2018 - 2021, USD Million
TABLE 4 North America Lysosomal Disease Treatment Market by Disease Type, 2022 - 2028, USD Million
TABLE 5 North America Gaucher's Diseases Market by Country, 2018 - 2021, USD Million
TABLE 6 North America Gaucher's Diseases Market by Country, 2022 - 2028, USD Million
TABLE 7 North America Fabry Diseases Market by Country, 2018 - 2021, USD Million
TABLE 8 North America Fabry Diseases Market by Country, 2022 - 2028, USD Million
TABLE 9 North America Mucopolysaccharidosis Market by Country, 2018 - 2021, USD Million
TABLE 10 North America Mucopolysaccharidosis Market by Country, 2022 - 2028, USD Million
TABLE 11 North America Pompe’s Syndrome Market by Country, 2018 - 2021, USD Million
TABLE 12 North America Pompe’s Syndrome Market by Country, 2022 - 2028, USD Million
TABLE 13 North America Others Market by Country, 2018 - 2021, USD Million
TABLE 14 North America Others Market by Country, 2022 - 2028, USD Million
TABLE 15 North America Lysosomal Disease Treatment Market by Type of Therapy, 2018 - 2021, USD Million
TABLE 16 North America Lysosomal Disease Treatment Market by Type of Therapy, 2022 - 2028, USD Million
TABLE 17 North America Enzyme Replacement Therapy Market by Country, 2018 - 2021, USD Million
TABLE 18 North America Enzyme Replacement Therapy Market by Country, 2022 - 2028, USD Million
TABLE 19 North America Substrate Reduction Therapy Market by Country, 2018 - 2021, USD Million
TABLE 20 North America Substrate Reduction Therapy Market by Country, 2022 - 2028, USD Million
TABLE 21 North America Stem Cell Therapy Market by Country, 2018 - 2021, USD Million
TABLE 22 North America Stem Cell Therapy Market by Country, 2022 - 2028, USD Million
TABLE 23 North America Others Market by Country, 2018 - 2021, USD Million
TABLE 24 North America Others Market by Country, 2022 - 2028, USD Million
TABLE 25 North America Lysosomal Disease Treatment Market by End User, 2018 - 2021, USD Million
TABLE 26 North America Lysosomal Disease Treatment Market by End User, 2022 - 2028, USD Million
TABLE 27 North America Hospitals Market by Country, 2018 - 2021, USD Million
TABLE 28 North America Hospitals Market by Country, 2022 - 2028, USD Million
TABLE 29 North America Clinics Market by Country, 2018 - 2021, USD Million
TABLE 30 North America Clinics Market by Country, 2022 - 2028, USD Million
TABLE 31 North America Others Market by Country, 2018 - 2021, USD Million
TABLE 32 North America Others Market by Country, 2022 - 2028, USD Million
TABLE 33 North America Lysosomal Disease Treatment Market by Country, 2018 - 2021, USD Million
TABLE 34 North America Lysosomal Disease Treatment Market by Country, 2022 - 2028, USD Million
TABLE 35 US Lysosomal Disease Treatment Market, 2018 - 2021, USD Million
TABLE 36 US Lysosomal Disease Treatment Market, 2022 - 2028, USD Million
TABLE 37 US Lysosomal Disease Treatment Market by Disease Type, 2018 - 2021, USD Million
TABLE 38 US Lysosomal Disease Treatment Market by Disease Type, 2022 - 2028, USD Million
TABLE 39 US Lysosomal Disease Treatment Market by Type of Therapy, 2018 - 2021, USD Million
TABLE 40 US Lysosomal Disease Treatment Market by Type of Therapy, 2022 - 2028, USD Million
TABLE 41 US Lysosomal Disease Treatment Market by End User, 2018 - 2021, USD Million
TABLE 42 US Lysosomal Disease Treatment Market by End User, 2022 - 2028, USD Million
TABLE 43 Canada Lysosomal Disease Treatment Market, 2018 - 2021, USD Million
TABLE 44 Canada Lysosomal Disease Treatment Market, 2022 - 2028, USD Million
TABLE 45 Canada Lysosomal Disease Treatment Market by Disease Type, 2018 - 2021, USD Million
TABLE 46 Canada Lysosomal Disease Treatment Market by Disease Type, 2022 - 2028, USD Million
TABLE 47 Canada Lysosomal Disease Treatment Market by Type of Therapy, 2018 - 2021, USD Million
TABLE 48 Canada Lysosomal Disease Treatment Market by Type of Therapy, 2022 - 2028, USD Million
TABLE 49 Canada Lysosomal Disease Treatment Market by End User, 2018 - 2021, USD Million
TABLE 50 Canada Lysosomal Disease Treatment Market by End User, 2022 - 2028, USD Million
TABLE 51 Mexico Lysosomal Disease Treatment Market, 2018 - 2021, USD Million
TABLE 52 Mexico Lysosomal Disease Treatment Market, 2022 - 2028, USD Million
TABLE 53 Mexico Lysosomal Disease Treatment Market by Disease Type, 2018 - 2021, USD Million
TABLE 54 Mexico Lysosomal Disease Treatment Market by Disease Type, 2022 - 2028, USD Million
TABLE 55 Mexico Lysosomal Disease Treatment Market by Type of Therapy, 2018 - 2021, USD Million
TABLE 56 Mexico Lysosomal Disease Treatment Market by Type of Therapy, 2022 - 2028, USD Million
TABLE 57 Mexico Lysosomal Disease Treatment Market by End User, 2018 - 2021, USD Million
TABLE 58 Mexico Lysosomal Disease Treatment Market by End User, 2022 - 2028, USD Million
TABLE 59 Rest of North America Lysosomal Disease Treatment Market, 2018 - 2021, USD Million
TABLE 60 Rest of North America Lysosomal Disease Treatment Market, 2022 - 2028, USD Million
TABLE 61 Rest of North America Lysosomal Disease Treatment Market by Disease Type, 2018 - 2021, USD Million
TABLE 62 Rest of North America Lysosomal Disease Treatment Market by Disease Type, 2022 - 2028, USD Million
TABLE 63 Rest of North America Lysosomal Disease Treatment Market by Type of Therapy, 2018 - 2021, USD Million
TABLE 64 Rest of North America Lysosomal Disease Treatment Market by Type of Therapy, 2022 - 2028, USD Million
TABLE 65 Rest of North America Lysosomal Disease Treatment Market by End User, 2018 - 2021, USD Million
TABLE 66 Rest of North America Lysosomal Disease Treatment Market by End User, 2022 - 2028, USD Million
TABLE 67 KEY INFORMATION - MERCK & CO., INC.
TABLE 68 Key information –Johnson & Johnson
TABLE 69 Key Information – Eli Lilly And Company
TABLE 70 Key Information – Pfizer, Inc.
TABLE 71 Key Information – Takeda Pharmaceutical Company Limited
TABLE 72 Key Information – Sanofi S.A.
TABLE 73 Key Information – Novartis AG
TABLE 74 KEY INFORMATION – ASTRAZENECA PLC
TABLE 75 Key Information – BioMarin Pharmaceutical Inc.
TABLE 76 Key Information – Sigilon Therapeutics, Inc.
List of Figures
FIG 1 Methodology for the research