PUBLISHER: DataM Intelligence | PRODUCT CODE: 1082815
PUBLISHER: DataM Intelligence | PRODUCT CODE: 1082815
Global Metachromatic leukodystrophy Treatment Market - 2022-2029
Market Overview
The global metachromatic leukodystrophy treatment market size was valued US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).
Metachromatic leukodystrophy is a rare hereditary (genetic) condition characterized by the accumulation of fatty substances (lipids) in cells, mainly in the brain, spinal cord, and peripheral nerves. A lack of an enzyme that helps break down lipids called sulfatides causes this accumulation. Because the substance that covers and protects nerve cells (myelin) is destroyed, the brain and nervous system gradually lose function. Metachromatic leukodystrophy is currently incurable. Early detection and treatment may help manage some signs and symptoms and postpone the progression of the illness, depending on the form and age of start.
Market Dynamics
Innovation in the products for treating demyelinating conditions is expected to drive market growth.
Joanne Kurtzberg an American professor of pediatrics and pathology at Duke University has developed a product called Duke O Cell or DUOC-01, which is a cell generated from umbilical cord blood that has been demonstrated to help people with demyelinating diseases. It's been used as a bridge therapy to help youngsters with inherited metabolic problems engraft faster. Moreover, Atidarsagene autotemcel (arsa-cel) is a gene therapy medicinal product based on autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding the ARSA cDNA driving supranormal ARSA expression in HSPCs and their progeny, with preliminary evidence of safety and efficacy in patients with early-onset MLD. Thus, from the above statements, the market is expected to drive in the forecast period.
Restraint:
The high cost of therapies for the treatment of Metachromatic leukodystrophy and there is no single FDA-approved pharmacological product on the market that can completely cure metachromatic leukodystrophy are the factors the market is expected to hamper in the forecast period.
COVID-19 Impact Analysis
The COVID-19 pandemic has moderately impacted healthcare systems and the market. Standard operating procedures for Personal Protective Equipment (PPE) for home care nurses were unclear during the pandemic. The doctors contacted all patients by email or telephone, as well as sent letters to those who did not have access to email or the internet. With little evidence of the impact of SARS-CoV-2 infections on rare diseases early in the pandemic, we decided that the danger of developing SARS-CoV-2 outweighed the risk of treatment interruption. Moreover, disruptions in therapy, whether planned or inevitable, resulted in a deterioration of clinical parameters and quality of life, as well as potentially negative outcomes such as cardiac events and/or renal function decline.
Additionally, the severity of the impact of the COVID-19 pandemic on business, including on preclinical and clinical development, its supply chain, and commercial programs got affected. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.
Segment Analysis
Gene Therapy segment is expected to hold the largest market share in metachromatic leukodystrophy treatment market
The gene therapy segment is expected to dominate in 2021. The segment's growth is beneficial because gene therapy showed benefits in pediatric patients with early-onset metachromatic leukodystrophy (MLD). There is currently no cure for MLD. In contrast, the European Medicines Agency (EMA) has recommended that the gene therapy Libmeldy, which consists of cryopreserved autologous CD34+ cells encoding the arylsulfatase-A gene, be given marketing authorization in the European Union to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is for children who have been diagnosed as carriers of the faulty gene but have not yet shown symptoms and have the 'late infantile' or 'early juvenile' variants of MLD. It is used in children who have been diagnosed with the early juvenile form of the disease and have begun to show symptoms while still being able to walk independently and before cognitive impairment. Thus, from the above statements, the market segment is expected to hold the largest market share in the forecast period.
Geographical Analysis
North America region holds the largest market share in the global metachromatic leukodystrophy treatment market
In 2021, North America accounted for the highest revenue share. The increasing prevalence of metachromatic leukodystrophy, increased number of diagnosed patients, ongoing clinical trials, rise in government funding for research & development for the treatment, and launch of contact registry by the organizations in the region are some of the factors the market is expected to boost in the forecast period. For instance, metachromatic leukodystrophy ranges from 1/40,000 births in the United States of America. Moreover, The National Organization for Rare Disorders, Inc. has initiated a large-scale study to investigate Metachromatic Leukodystrophy (MLD), a brain disease that causes irreparable white matter destruction. The Metachromatic Leukodystrophy Community United Research and Engagement Survey (MLDCures) is a new project that provides a platform for Metachromatic Leukodystrophy patients worldwide to contribute information about the disease (MLD). Its goal is to create an international resource that scientists can use in future research. Additionally, Cure MLD is collaborating on the study with the National Organization for Rare Disorders (NORD), an independent organization that developed the IAMRARE platform to aid in the identification and treatment of all 7,000 rare diseases. Thus, from the above statements, the North American region is expected to hold the largest market share in the forecast period.
Competitive Landscape
Major key players in the metachromatic leukodystrophy treatment market are ArmaGen, Inc., GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, Orchard Therapeutics plc, Magenta Therapeutics, Recursion Pharmaceuticals Inc. and RegenxBio Inc.
ArmaGen, Inc.:
Overview:
ArmaGen is a privately held, clinical-stage biotechnology company, with headquarters and research operations located in Calabasas, CA focused on developing revolutionary therapies for severe neurological disorders. The company is advancing innovative therapies for the treatment of currently unaddressed neurological complications of lysosomal storage disorders (LSDs), as well as neurodegenerative diseases such as Alzheimer's and Parkinson's. Moreover, ArmaGen has eight compounds in development. The company's lead product candidates are AGT-182 for Hunter syndrome, which is in Phase 1 clinical trials and AGT-181 for Hurler syndrome, which is in Phase 2 trials. The company is advancing its pipeline through licensing and collaboration agreements, in-house development efforts, and future partnering opportunities.
Product Portfolio:
AGT-183: AGT-183 is an investigational enzyme replacement therapy (ERT) for the treatment of metachromatic leukodystrophy (MLD), a lysosomal storage disease that arises from a deficiency of the enzyme arylsulfatase (ASA).
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Target Audience
Service Providers/ Buyers
Industry Investors/Investment Bankers
Education & Research Institutes
Research Professionals
Emerging Companies
Manufacturers
Table of Contents
1. Methodology and Scope
- 1.1. Research Methodology
- 1.2. Research Objective and Scope of the Report
2. Market Definition and Overview
3. Executive Summary
- 3.1. Market Snippet by Type
- 3.2. Market Snippet by Diagnosis
- 3.3. Market Snippet by Treatment Type
- 3.4. Market Snippet by End user
- 3.5. Market Snippet by Region
4. Market Dynamics
- 4.1. Market Impacting Factors
- 4.1.1. Drivers
- 4.1.1.1. Innovation in the products for treating demyelinating conditions is expected to drive market growth.
- 4.1.2. Restraints:
- 4.1.2.1. The high cost of therapies for the treatment of Metachromatic leukodystrophy is expected to hamper the market growth in the forecast period.
- 4.1.3. Opportunity
- 4.1.4. Impact Analysis
- 4.1.1. Drivers
5. Industry Analysis
- 5.1. Supply Chain Analysis
- 5.2. Pricing Analysis
- 5.3. Unmet Needs
6. COVID-19 Analysis
- 6.1. Analysis of Covid-19 on the Market
- 6.1.1. Before COVID-19 Market Scenario
- 6.1.2. Present COVID-19 Market Scenario
- 6.1.3. After COVID-19 or Future Scenario
- 6.2. Pricing Dynamics Amid Covid-19
- 6.3. Demand-Supply Spectrum
- 6.4. Government Initiatives Related to the Market During Pandemic
- 6.5. Manufacturers Strategic Initiatives
- 6.6. Conclusion
7. By Type
- 7.1. Introduction
- 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 7.1.2. Market Attractiveness Index, By Type Segment
- 7.2. Late-Infantile MLD*
- 7.2.1. Introduction
- 7.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 7.3. Juvenile MLD
- 7.4. Adult MLD
8. By Diagnosis
- 8.1. Introduction
- 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 8.1.2. Market Attractiveness Index, By Diagnosis Segment
- 8.2. Blood tests*
- 8.2.1. Introduction
- 8.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 8.3. Genetic tests
- 8.4. Nerve conduction study
- 8.5. Magnetic resonance imaging (MRI)
- 8.6. Psychological and cognitive tests
- 8.7. Others
9. By Treatment Type
- 9.1. Introduction
- 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 9.1.2. Market Attractiveness Index, By Treatment Type Segment
- 9.2. Pharmacotherapy*
- 9.2.1. Introduction
- 9.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 9.3. Gene Therapy
- 9.3.1. AGT-183
- 9.3.2. GSK-2696274
- 9.3.3. Others
- 9.4. Enzyme replacement or enhancement therapy
- 9.5. Substrate reduction therapy
- 9.6. Others
10. By End user
- 10.1. Introduction
- 10.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 10.1.2. Market Attractiveness Index, By End user Segment
- 10.2. Hospital*
- 10.2.1. Introduction
- 10.2.2. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029
- 10.3. Clinics
- 10.4. Diagnostic Centers
- 10.5. Others
11. By Region
- 11.1. Introduction
- 11.1.1. Market Size Analysis, US$ Million, 2020-2029 and Y-o-Y Growth Analysis (%), 2021-2029, By Region
- 11.1.2. Market Attractiveness Index, By Region
- 11.2. North America
- 11.2.1. Introduction
- 11.2.2. Key Region-Specific Dynamics
- 11.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 11.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 11.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 11.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 11.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 11.2.7.1. U.S.
- 11.2.7.2. Canada
- 11.2.7.3. Mexico
- 11.3. Europe
- 11.3.1. Introduction
- 11.3.2. Key Region-Specific Dynamics
- 11.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 11.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 11.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 11.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 11.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 11.3.7.1. Germany
- 11.3.7.2. U.K.
- 11.3.7.3. France
- 11.3.7.4. Italy
- 11.3.7.5. Spain
- 11.3.7.6. Rest of Europe
- 11.4. South America
- 11.4.1. Introduction
- 11.4.2. Key Region-Specific Dynamics
- 11.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 11.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 11.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 11.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 11.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 11.4.7.1. Brazil
- 11.4.7.2. Argentina
- 11.4.7.3. Rest of South America
- 11.5. Asia Pacific
- 11.5.1. Introduction
- 11.5.2. Key Region-Specific Dynamics
- 11.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 11.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 11.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 11.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
- 11.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
- 11.5.7.1. China
- 11.5.7.2. India
- 11.5.7.3. Japan
- 11.5.7.4. Australia
- 11.5.7.5. Rest of Asia Pacific
- 11.6. Middle East and Africa
- 11.6.1. Introduction
- 11.6.2. Key Region-Specific Dynamics
- 11.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
- 11.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Diagnosis
- 11.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
- 11.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End user
12. Competitive Landscape
- 12.1. Key Developments and Strategies
- 12.2. Company Share Analysis
- 12.3. Product Benchmarking
- 12.4. List of Key Companies to Watch
13. Company Profiles
- 13.1. ArmaGen, Inc.*
- 13.1.1. Company Overview
- 13.1.2. Product Portfolio and Description
- 13.1.3. Key Highlights
- 13.1.4. Financial Overview
- 13.2. GlaxoSmithKline plc
- 13.3. Takeda Pharmaceutical Company Limited
- 13.4. Orchard Therapeutics plc
- 13.5. Magenta Therapeutics
- 13.6. Recursion Pharmaceuticals Inc.
- 13.7. RegenxBio Inc.
LIST NOT EXHAUSTIVE
14. Global Metachromatic leukodystrophy Treatment Market - DataM
- 14.1. Appendix
- 14.2. About Us and Applications
- 14.3. Contact Us
