Increase in preference for gene therapy and substrate reduction therapy are majorly holding the responsibility of driving the metachromatic leukodystrophy treatment market. Metachromatic leukodystrophy ends up adversely affecting the nervous system to the extent of the body not being able to perform the daily chores. Timely detection and treatment can prevent the disease from reaching the last stage.
The companies operating in the metachromatic leukodystrophy treatment market are investing in devising advanced therapies for treating metachromatic leukodystrophy and seeking quick approvals regarding the same.
ARSA-Cel was recently approved for treating the disease. They are also working toward devising cost-effective medications in developing countries.
Metachromatic leukodystrophy (MLD) means a rare hereditary (genetic) disorder that results in building up of lipids in the cells, especially in brain, peripheral nerves, and spinal cord.
Such a build-up is an outcome of a deficiency of an enzyme that aids in breaking down the lipids called sulfatides. The nervous system and brain progressively lose function as this substance covering and protecting nerve cells (myelin) is ruptured.
The three forms of metachromatic leukodystrophy include juvenile form, late-infantile form, and adult form. The symptoms and signs may vary. Infantile form is amongst the most common forms and progresses rapidly as compared to the other forms.
The common cause of MLD is a mutation in ARSA gene, which causes the unnecessary build-up of lipids as mentioned above.
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Gene therapy is recommended in combination with hematopoietic stem cell transplantation (HSCT) for metachromatic leukodystrophy. The advantages are visible in early symptomatic or asymptomatic juvenile-onset disease. However, this course of treatment is not advisable for individuals suffering from late-infantile, symptomatic form.
In adult-onset and late juvenile forms, bone marrow transplantation has reported to slow the progression of disease.
In combination of gene therapy with the others as mentioned above, modified stem cells undergo transplantation back into the patient where they attach and multiply (engraft) within bone marrow.
These modified stem cells supply body with immune (myeloid) cells that manufacture ARSA enzyme, which, in turn, helps in breaking down harmful build-up of the sulfatides and may halt the progression of MLD.
Utilization of gene therapy at a larger scale to cure MLD is thus contributing toward metachromatic leukodystrophy treatment market growth.
Metachromatic leukodystrophy is a serious condition and results in fatality within 5-6 years during the early onset form. Proper diagnosis as well as treatment help in obtaining a better quality of life.
In metachromatic leukodystrophy, substrate reduction therapy (SRT) is applied for partially blocking the body from producing sulfatide (a fatty acid chemical accumulating inside lysosome of neuronal cells in absence of Arylsulfatase A, its regulating enzyme). SRT basically targets cerebroside sulfotransferase (CST) enzyme.
Substrate reduction therapy, in infantile-onset variants, slows the progression of MLD and increases life expectancy. Convenience involved in administering SRT is expanding the metachromatic leukodystrophy treatment market size.
| Attribute | Detail |
|---|---|
| Leading Region | North America |
As per the latest metachromatic leukodystrophy treatment market scenario, North America led the metachromatic leukodystrophy care landscape in 2023 and the status quo is expected to remain unchanged during the forecast period.
This is attributed to a marked increase in the number of cases of MLD in the U.S. followed by rise in healthcare spending. The government of Canada is also investing heavily on combating this ailment, which, even though looks mild at the starting, may cause serious consequences in future.
Asia Pacific is witnessing an influx of several awareness campaigns in this regard. Countries such as India and China are home to population that is entirely unmindful of the symptoms of metachromatic leukodystrophy. These countries are poised to be a promising market for MLD treatment in the near future.
The key players in the metachromatic leukodystrophy treatment are launching new products followed by speedy approvals by the regulatory authorities. For instance, in March 2024, the U.S. FDA approved the first and sole gene therapy meant to treat children suffering from MLD.
JCR Pharmaceuticals Co., Ltd., GSK plc, Recursion Pharmaceuticals Inc., and REGENXBIO Inc. are some of the key players covered in the metachromatic leukodystrophy treatment market report.
These companies have been profiled based on parameters such as company overview, business strategies, financial overview, product portfolio, and business segments.
| Attribute | Detail |
|---|---|
| Market Size in 2023 | US$ 2.5 Bn |
| Market Forecast (Value) in 2034 | US$ 4.5 Bn |
| Growth Rate (CAGR) | 5.6% |
| Forecast Period | 2024-2034 |
| Historical Data Available for | 2020-2022 |
| Quantitative Units | US$ Bn for Value |
| Market Analysis | It includes segment analysis as well as regional level analysis. Moreover, qualitative analysis includes drivers, restraints, opportunities, key trends, Porter’s Five Forces analysis, value chain analysis, and key trend analysis. |
| Competition Landscape |
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| Format | Electronic (PDF) + Excel |
| Market Segmentation |
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| Regions Covered |
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| Countries Covered |
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| Companies Profiled |
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| Customization Scope | Available Upon Request |
| Pricing | Available Upon Request |
It was valued at US$ 2.5 Bn in 2023
It is projected to grow at a CAGR of 5.6% from 2024 to 2034
Preference for gene therapy and substrate reduction therapy
Hospitals segment accounted for the largest share in 2023
North America was the dominant region in 2023
JCR Pharmaceuticals Co., Ltd., GSK plc, Recursion Pharmaceuticals Inc., and REGENXBIO Inc.
1. Preface
1.1. Market Definition and Scope
1.2. Market Segmentation
1.3. Key Research Objectives
1.4. Research Highlights
2. Assumptions and Research Methodology
3. Executive Summary: Global Metachromatic Leukodystrophy Treatment Market
4. Market Overview
4.1. Introduction
4.1.1. Product Definition
4.1.2. Industry Evolution/Developments
4.2. Overview
4.3. Market Dynamics
4.3.1. Drivers
4.3.2. Restraints
4.3.3. Opportunities
4.4. Global Metachromatic Leukodystrophy Treatment Market Analysis and Forecast, 2020-2034
5. Key Insights
5.1. Pipeline Analysis
5.2. Key Product/Brand Analysis
5.3. Key Mergers & Acquisitions
5.4. COVID-19 Pandemic Impact on Industry
6. Global Metachromatic Leukodystrophy Treatment Market Analysis and Forecast, by Drug Type
6.1. Introduction & Definition
6.2. Key Findings/Developments
6.3. Market Value Forecast, by Drug Type, 2020-2034
6.3.1. Atidarsagene Autotemcel
6.3.2. DUOC-01
6.3.3. OTL-200
6.3.4. Others (AGT-183, etc.)
6.4. Market Attractiveness Analysis, by Drug Type
7. Global Metachromatic Leukodystrophy Treatment Market Analysis and Forecast, by End-user
7.1. Introduction & Definition
7.2. Key Findings/Developments
7.3. Market Value Forecast, by End-user, 2020-2034
7.3.1. Hospitals
7.3.2. Specialty Clinics
7.3.3. Others (Research Institutes, etc.)
7.4. Market Attractiveness Analysis, by End-user
8. Global Metachromatic Leukodystrophy Treatment Market Analysis and Forecast, by Region
8.1. Key Findings
8.2. Market Value Forecast, by Region, 2020-2034
8.2.1. North America
8.2.2. Europe
8.2.3. Asia Pacific
8.2.4. Latin America
8.2.5. Middle East & Africa
8.3. Market Attractiveness Analysis, by Region
9. North America Metachromatic Leukodystrophy Treatment Market Analysis and Forecast
9.1. Introduction
9.2. Key Findings
9.3. Market Value Forecast, by Drug Type, 2020-2034
9.3.1. Atidarsagene Autotemcel
9.3.2. DUOC-01
9.3.3. OTL-200
9.3.4. Others (AGT-183, etc.)
9.4. Market Value Forecast, by End-user, 2020-2034
9.4.1. Hospitals
9.4.2. Specialty Clinics
9.4.3. Others (Research Institutes, etc.)
9.5. Market Value Forecast, by Country, 2020-2034
9.5.1. U.S.
9.5.2. Canada
9.6. Market Attractiveness Analysis
9.6.1. By Drug Type
9.6.2. By End-user
9.6.3. By Country
10. Europe Metachromatic Leukodystrophy Treatment Market Analysis and Forecast
10.1. Introduction
10.2. Key Findings
10.3. Market Value Forecast, by Drug Type, 2020-2034
10.3.1. Atidarsagene Autotemcel
10.3.2. DUOC-01
10.3.3. OTL-200
10.3.4. Others (AGT-183, etc.)
10.4. Market Value Forecast, by End-user, 2020-2034
10.4.1. Hospitals
10.4.2. Specialty Clinics
10.4.3. Others (Research Institutes, etc.)
10.5. Market Value Forecast, by Country/Sub-region, 2020-2034
10.5.1. Germany
10.5.2. U.K.
10.5.3. France
10.5.4. Italy
10.5.5. Spain
10.5.6. Rest of Europe
10.6. Market Attractiveness Analysis
10.6.1. By Drug Type
10.6.2. By End-user
10.6.3. By Country/Sub-region
11. Asia Pacific Metachromatic Leukodystrophy Treatment Market Analysis and Forecast
11.1. Introduction
11.2. Key Findings
11.3. Market Value Forecast, by Drug Type, 2020-2034
11.3.1. Atidarsagene Autotemcel
11.3.2. DUOC-01
11.3.3. OTL-200
11.3.4. Others (AGT-183, etc.)
11.4. Market Value Forecast, by End-user, 2020-2034
11.4.1. Hospitals
11.4.2. Specialty Clinics
11.4.3. Others (Research Institutes, etc.)
11.5. Market Value Forecast, by Country/Sub-region, 2020-2034
11.5.1. China
11.5.2. Japan
11.5.3. India
11.5.4. Australia & New Zealand
11.5.5. Rest of Asia Pacific
11.6. Market Attractiveness Analysis
11.6.1. By Drug Type
11.6.2. By End-user
11.6.3. By Country/Sub-region
12. Latin America Metachromatic Leukodystrophy Treatment Market Analysis and Forecast
12.1. Introduction
12.2. Key Findings
12.3. Market Value Forecast, by Drug Type, 2020-2034
12.3.1. Atidarsagene Autotemcel
12.3.2. DUOC-01
12.3.3. OTL-200
12.3.4. Others (AGT-183, etc.)
12.4. Market Value Forecast, by End-user, 2020-2034
12.4.1. Hospitals
12.4.2. Specialty Clinics
12.4.3. Others (Research Institutes, etc.)
12.5. Market Value Forecast, by Country/Sub-region, 2020-2034
12.5.1. Brazil
12.5.2. Mexico
12.5.3. Rest of Latin America
12.6. Market Attractiveness Analysis
12.6.1. By Drug Type
12.6.2. By End-user
12.6.3. By Country/Sub-region
13. Middle East & Africa Metachromatic Leukodystrophy Treatment Market Analysis and Forecast
13.1. Introduction
13.2. Key Findings
13.3. Market Value Forecast, by Drug Type, 2020-2034
13.3.1. Atidarsagene Autotemcel
13.3.2. DUOC-01
13.3.3. OTL-200
13.3.4. Others (AGT-183, etc.)
13.4. Market Value Forecast, by End-user, 2020-2034
13.4.1. Hospitals
13.4.2. Specialty Clinics
13.4.3. Others (Research Institutes, etc.)
13.5. Market Value Forecast, by Country/Sub-region, 2020-2034
13.5.1. GCC Countries
13.5.2. South Africa
13.5.3. Rest of Middle East & Africa
13.6. Market Attractiveness Analysis
13.6.1. By Drug Type
13.6.2. By End-user
13.6.3. By Country/Sub-region
14. Competition Landscape
14.1. Market Player - Competitive Matrix (by Tier and Size of Companies)
14.2. Market Share Analysis, by Company (2023)
14.3. Company Profiles
14.3.1. JCR Pharmaceuticals Co., Ltd.
14.3.1.1. Company Overview
14.3.1.2. Product Portfolio
14.3.1.3. SWOT Analysis
14.3.1.4. Financial Overview
14.3.1.5. Strategic Overview
14.3.2. GSK plc
14.3.2.1. Company Overview
14.3.2.2. Product Portfolio
14.3.2.3. SWOT Analysis
14.3.2.4. Financial Overview
14.3.2.5. Strategic Overview
14.3.3. Recursion Pharmaceuticals Inc.
14.3.3.1. Company Overview
14.3.3.2. Product Portfolio
14.3.3.3. SWOT Analysis
14.3.3.4. Financial Overview
14.3.3.5. Strategic Overview
14.3.4. REGENXBIO Inc.
14.3.4.1. Company Overview
14.3.4.2. Product Portfolio
14.3.4.3. SWOT Analysis
14.3.4.4. Financial Overview
14.3.4.5. Strategic Overview
List of Tables
Table 01: Global Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 02: Global Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
Table 03: Global Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Region, 2020-2034
Table 04: North America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Country, 2020-2034
Table 05: North America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 06: North America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
Table 07: Europe Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Country/Sub-region, 2020-2034
Table 08: Europe Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 09: Europe Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
Table 10: Asia Pacific Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Country/Sub-region, 2020-2034
Table 11: Asia Pacific Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 12: Asia Pacific Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
Table 13: Latin America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Country/Sub-region, 2020-2034
Table 14: Latin America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 15: Latin America Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
Table 16: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Country/Sub-region, 2020-2034
Table 17: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by Drug Type, 2020-2034
Table 18: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast, by End-user, 2020-2034
List of Figures
Figure 01: Global Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) and Distribution (%), by Region, 2023 and 2034
Figure 02: Global Metachromatic Leukodystrophy Treatment Market Revenue (US$ Bn), by Drug Type, 2023
Figure 03: Global Metachromatic Leukodystrophy Treatment Market Value Share, by Drug Type, 2023
Figure 04: Global Metachromatic Leukodystrophy Treatment Market Revenue (US$ Bn), by End-user, 2023
Figure 05: Global Metachromatic Leukodystrophy Treatment Market Value Share, by End-user, 2023
Figure 06: Global Metachromatic Leukodystrophy Treatment Market Value Share, by Region, 2023
Figure 07: Global Metachromatic Leukodystrophy Treatment Market Value (US$ Bn) Forecast, 2020-2034
Figure 08: Global Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 09: Global Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 10: Global Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 11: Global Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
Figure 12: Global Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Region, 2023 and 2034
Figure 13: Global Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Region, 2020-2034
Figure 14: North America Metachromatic Leukodystrophy Treatment Market Value (US$ Bn) Forecast and Y-o-Y Growth (%), 2020-2034
Figure 15: North America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Country, 2020-2034
Figure 16: North America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Country, 2023 and 2034
Figure 17: North America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 18: North America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 19: North America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 20: North America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
Figure 21: Europe Metachromatic Leukodystrophy Treatment Market Value (US$ Bn) Forecast and Y-o-Y Growth (%), 2020-2034
Figure 22: Europe Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Country/Sub-region, 2020-2034
Figure 23: Europe Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Country/Sub-region, 2023 and 2034
Figure 24: Europe Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 25: Europe Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 26: Europe Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 27: Europe Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
Figure 28: Asia Pacific Metachromatic Leukodystrophy Treatment Market Value (US$ Bn) Forecast and Y-o-Y Growth (%), 2020-2034
Figure 29: Asia Pacific Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Country/Sub-region, 2020-2034
Figure 30: Asia Pacific Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Country/Sub-region, 2023 and 2034
Figure 31: Asia Pacific Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 32: Asia Pacific Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 33: Asia Pacific Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 34: Asia Pacific Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
Figure 35: Latin America Metachromatic Leukodystrophy Treatment Market Value (US$ Bn) Forecast and Y-o-Y Growth (%), 2020-2034
Figure 36: Latin America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Country/Sub-region, 2020-2034
Figure 37: Latin America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Country/Sub-region, 2023 and 2034
Figure 38: Latin America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 39: Latin America Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 40: Latin America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 41: Latin America Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
Figure 42: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Size (US$ Bn) Forecast and Y-o-Y Growth (%), 2020-2034
Figure 43: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Country/Sub-region, 2020-2034
Figure 44: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Country/Sub-region, 2023 and 2034
Figure 45: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by Drug Type, 2023 and 2034
Figure 46: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Value Share Analysis, by End-user, 2023 and 2034
Figure 47: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by Drug Type, 2020-2034
Figure 48: Middle East & Africa Metachromatic Leukodystrophy Treatment Market Attractiveness Analysis, by End-user, 2020-2034
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Metachromatic Leukodystrophy Treatment Market
