- Enzyme Replacement Therapy
- Supportive Care
- Gene Therapy
Mucopolysaccharidosis Type VII (MPS VII) Treatment Market size was valued at USD 372.1million in 2023 and is expected to grow at 5.3% CAGR from 2024 to 2030. Mucopolysaccharidosis Type VII (MPS VII), also known as Sly syndrome, is an ultra-rare lysosomal storage disorder caused by a deficiency of the enzyme beta-glucuronidase. This deficiency leads to the accumulation of glycosaminoglycans in various tissues, resulting in progressive cellular damage and multi-organ dysfunction. The global mucopolysaccharidosis type VII treatment market has been experiencing growth due to several key drivers. Increased awareness among healthcare professionals, advancements in diagnostic techniques, and the development of enzyme replacement therapy (ERT) have significantly contributed to market expansion. The approval of Mepsevii (vestronidase alfa-vjbk) by the FDA in November 2017 and by the EMA in August 2018 marked a milestone in mucopolysaccharidosis type VII treatment, offering the first specific therapy for this condition. However, the extreme rarity of mucopolysaccharidosis type VII, with an estimated incidence of less than 1 in 250,000 births, results in a very limited patient population.
This rarity can discourage investment in research and development. Additionally, the high cost of treatment and challenges in early diagnosis due to the nonspecific nature of initial symptoms pose obstacles to market growth. Despite these challenges, the mucopolysaccharidosis type VII treatment market presents several opportunities. The potential for developing gene therapies and other advanced treatment modalities offers promising avenues for market expansion. Increasing collaborations between pharmaceutical companies, research institutions, and patient advocacy groups are likely to drive innovation and expand treatment options. Recent trends in the mucopolysaccharidosis type VII treatment market include a growing focus on personalized medicine and the exploration of combination therapies. Advancements in newborn screening techniques are also improving early diagnosis and treatment initiation for mucopolysaccharidosis type VII patients. A recent development in the field occurred in October 2023, when Ultragenyx Pharmaceutical Inc. announced positive long-term data from the Phase 3 study of Mepsevii in mucopolysaccharidosis type VII patients. The study demonstrated sustained improvements in motor function and reduction in urinary glycosaminoglycans over 144 weeks of treatment, further supporting the efficacy of enzyme replacement therapy in mucopolysaccharidosis type VII.
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The global mucopolysaccharidosis type VII treatment market is characterized by intense research activities aimed at developing more effective and convenient treatment options. Pharmaceutical and biotechnology companies are investing in the development of gene therapies and exploring innovative drug delivery methods to improve treatment outcomes. The increasing focus on personalized medicine and advancements in genetic testing techniques are expected to shape the future of mucopolysaccharidosis type VII treatment. Moreover, patient advocacy groups and rare disease organizations play a crucial role in raising awareness about mucopolysaccharidosis type VII, potentially leading to earlier diagnosis and treatment initiation. This, coupled with improving healthcare infrastructure in developing countries, is likely to expand the patient pool and drive market growth.
The Mucopolysaccharidosis Type VII (MPS VII) Treatment Market was valued at USD 372.1million in 2023 and is expected to grow at 5.3% CAGR from 2024 to 2030.
North America is expected to hold a major market share in the mucopolysaccharidosis type VII treatment market.
Enzyme Replacement Therapy currently holds the highest market share in the mucopolysaccharidosis type VII treatment market.
The Asia-Pacific region is estimated to grow at the highest CAGR over the forecast period.
Increasing awareness, improved diagnostic techniques, development of novel therapies, and expanding research activities are the major growth drivers in the mucopolysaccharidosis type VII treatment market.
1.Executive Summary |
2.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market Introduction |
2.1.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market - Taxonomy |
2.2.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market - Definitions |
2.2.1.Treatment type |
2.2.2.Treatment type |
2.2.3.Region |
3.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market Dynamics |
3.1. Drivers |
3.2. Restraints |
3.3. Opportunities/Unmet Needs of the Market |
3.4. Trends |
3.5. Product Landscape |
3.6. New Product Launches |
3.7. Impact of COVID 19 on Market |
4.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market Analysis, 2019 - 2023 and Forecast 2024 - 2030 |
4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) |
4.3. Market Opportunity Analysis |
5.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market By Treatment type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
5.1. Enzyme Replacement Therapy |
5.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
5.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
5.1.3. Market Opportunity Analysis |
5.2. Supportive Care |
5.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
5.2.3. Market Opportunity Analysis |
5.3. Gene Therapy |
5.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
5.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
5.3.3. Market Opportunity Analysis |
6.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market By Treatment type, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
6.1. Hospital Pharmacies |
6.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
6.1.3. Market Opportunity Analysis |
6.2. Specialty Pharmacies |
6.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
6.2.3. Market Opportunity Analysis |
6.3. Others |
6.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
6.3.3. Market Opportunity Analysis |
7.Global Mucopolysaccharidosis Type VII (MPS VII) Treatment Market By Region, 2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
7.1. North America |
7.1.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
7.1.3. Market Opportunity Analysis |
7.2. Europe |
7.2.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
7.2.3. Market Opportunity Analysis |
7.3. Asia Pacific (APAC) |
7.3.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
7.3.3. Market Opportunity Analysis |
7.4. Middle East and Africa (MEA) |
7.4.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
7.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
7.4.3. Market Opportunity Analysis |
7.5. Latin America |
7.5.1. Market Analysis, 2019 - 2023 and Forecast, 2024 - 2030, (Sales Value USD Million) |
7.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%) |
7.5.3. Market Opportunity Analysis |
8.North America Mucopolysaccharidosis Type VII (MPS VII) Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
8.1. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
8.1.1.Enzyme Replacement Therapy |
8.1.2.Supportive Care |
8.1.3.Gene Therapy |
8.2. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
8.2.1.Hospital Pharmacies |
8.2.2.Specialty Pharmacies |
8.2.3.Others |
8.3. Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
8.3.1.United States of America (USA) |
8.3.2.Canada |
9.Europe Mucopolysaccharidosis Type VII (MPS VII) Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
9.1. Treatment type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
9.1.1.Enzyme Replacement Therapy |
9.1.2.Supportive Care |
9.1.3.Gene Therapy |
9.2. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
9.2.1.Hospital Pharmacies |
9.2.2.Specialty Pharmacies |
9.2.3.Others |
9.3. Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
9.3.1.Germany |
9.3.2.France |
9.3.3.Italy |
9.3.4.United Kingdom (UK) |
9.3.5.Spain |
10.Asia Pacific (APAC) Mucopolysaccharidosis Type VII (MPS VII) Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
10.1. Treatment type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
10.1.1.Enzyme Replacement Therapy |
10.1.2.Supportive Care |
10.1.3.Gene Therapy |
10.2. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
10.2.1.Hospital Pharmacies |
10.2.2.Specialty Pharmacies |
10.2.3.Others |
10.3. Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
10.3.1.China |
10.3.2.India |
10.3.3.Australia and New Zealand (ANZ) |
10.3.4.Japan |
10.3.5.Rest of APAC |
11.Middle East and Africa (MEA) Mucopolysaccharidosis Type VII (MPS VII) Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
11.1. Treatment type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
11.1.1.Enzyme Replacement Therapy |
11.1.2.Supportive Care |
11.1.3.Gene Therapy |
11.2. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
11.2.1.Hospital Pharmacies |
11.2.2.Specialty Pharmacies |
11.2.3.Others |
11.3. Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
11.3.1.GCC Countries |
11.3.2.South Africa |
11.3.3.Rest of MEA |
12.Latin America Mucopolysaccharidosis Type VII (MPS VII) Treatment Market ,2019 - 2023 and Forecast 2024 - 2030 (Sales Value USD Million) |
12.1. Treatment type Analysis and Forecast by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
12.1.1.Enzyme Replacement Therapy |
12.1.2.Supportive Care |
12.1.3.Gene Therapy |
12.2. Treatment type Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
12.2.1.Hospital Pharmacies |
12.2.2.Specialty Pharmacies |
12.2.3.Others |
12.3. Country Analysis 2019 - 2023 and Forecast 2024 - 2030 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%) |
12.3.1.Brazil |
12.3.2.Mexico |
12.3.3.Rest of LA |
13. Competition Landscape |
13.1. Market Player Profiles (Introduction, Brand/Product Sales, Financial Analysis, Product Offerings, Key Developments, Collaborations, M & A, Strategies, and SWOT Analysis) |
13.2.1.Ultragenyx Pharmaceutical Inc. |
13.2.2.BioMarin Pharmaceutical Inc. |
13.2.3.RegenxBio Inc. |
13.2.4.Sanofi Genzyme |
13.2.5.Takeda Pharmaceutical Company Limited |
13.2.6.Horizon Therapeutics plc |
13.2.7.ArmaGen Inc. |
13.2.8.Lysogene |
13.2.9.Denali Therapeutics Inc. |
14. Research Methodology |
15. Appendix and Abbreviations |