The North America Lysosomal Disease Treatment Market is expected to 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.
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.
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.
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
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- Stem Cell Therapy
- Others
- Hospitals
- Clinics
- Others
- 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.
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Table of Contents
Chapter 1. Market Scope & Methodology
Chapter 2. Market Overview
Chapter 3. North America Lysosomal Disease Treatment Market by Disease Type
Chapter 4. North America Lysosomal Disease Treatment Market by Type of Therapy
Chapter 5. North America Lysosomal Disease Treatment Market by End User
Chapter 6. North America Lysosomal Disease Treatment Market by Country
Chapter 7. Company Profiles
Companies Mentioned
- 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.
Methodology
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