Mucopolysaccharidoses Market is expected to reach US$ 4,389.85 million by 2033 | Top key players - Sanofi, Takeda Pharmaceuticals, GC Biopharma corporate.

Market Size and Growth:

The Global Mucopolysaccharidoses Market size reached US$ 2,313.08 million in 2024 and is expected to reach US$ 4,389.85 million by 2033, growing at a CAGR of 7.5% during the forecast period 2025-2033.

The Mucopolysaccharidoses Market report, published by DataM Intelligence, provides in-depth insights and analysis on key market trends, growth opportunities, and emerging challenges. Committed to delivering actionable intelligence, DataM Intelligence empowers businesses to make informed decisions and stay ahead of the competition. Through a combination of qualitative and quantitative research methods, it offers comprehensive reports that help clients navigate complex market landscapes, drive strategic growth, and seize new opportunities in an ever-evolving global market.

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The Mucopolysaccharidoses Market encompasses therapies, diagnostics, and supportive care for rare inherited lysosomal storage disorders caused by enzyme deficiencies leading to glycosaminoglycan accumulation. It includes enzyme replacement therapies, gene therapies, hematopoietic stem cell transplantation, and diagnostic tools, driven by rising awareness, early screening programs, and ongoing advancements in rare disease treatment.

Recent Key Developments of United States:

✅ November 2025: Prime Therapeutics submitted an FDA application for Clemidsogene lanparvovec (RGX-121), a one-time gene therapy for MPS II (Hunter syndrome) in boys aged 4 months to 5 years, as a potential alternative to weekly enzyme replacement.

✅ October 2025: Spruce Biosciences received FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB), based on long-term data showing CSF HS-NRE normalization.

✅ September 2025: Japan's Ministry of Health granted Orphan Drug Designation to JR-446 for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B), with implications for US market access through collaborations like MEDIPAL Holdings and JCR Pharmaceuticals.

Recent Key Developments of Europe:

✅ November 2025: UCL scientists manufactured a world-first ex vivo gene therapy for a boy with Hunter syndrome (MPS II), transplanted at Royal Manchester Children's Hospital, showing exponential progress in speech, agility, and cognition nine months post-treatment in February 2025.

✅ September 2025: JCR Pharmaceuticals presented long-term clinical data on Pabinafusp Alfa for MPS II at ICIEM 2025 in Kyoto, advancing brain-penetrant therapies relevant to European research pipelines.

List of the Key Players in the Mucopolysaccharidoses Market:

Sanofi
Takeda Pharmaceuticals
GC Biopharma corporate.
CANbridge Life Sciences Ltd.
JCR Pharmaceuticals Co., Ltd.
BioMarin Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc.

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This Report Covers:

✔ Go-to-market Strategy.

✔ Neutral perspective on the market performance.

✔Development trends, competitive landscape analysis, supply side analysis, demand side analysis, year-on-year growth, competitive benchmarking, vendor identification, and other significant analysis, as well as development status.

✔Customized regional/country reports as per request and country level analysis.

✔ Potential & niche segments and regions exhibiting promising growth covered.

✔ Analysis of Market Size (historical and forecast), Total Addressable Market (TAM), Serviceable Available Market (SAM), Serviceable Obtainable Market (SOM), Market Growth, Technological Trends, Market Share, Market Dynamics, Competitive Landscape and Major Players (Innovators, Start-ups, Laggard, and Pioneer).

Segments Covered in the Mucopolysaccharidoses Market:

By Type: MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS IV (Morquio Syndrome), MPS VI (Maroteaux-Lamy Syndrome), MPS VII (Sly Syndrome), Others.

By Drug: Laronidase, Idursulfase, Pabinafusp Alfa, Elosulfase Alfa, Galsulfase, Vestronidase Alfa-vjbk, Others.

Regional Analysis:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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Chapter Outline

⏩ Market Overview: It contains five chapters, as well as information about the research scope, major manufacturers covered, market segments, Mucopolysaccharidoses market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Global Mucopolysaccharidoses Market is evaluated here in terms of value, turnover, revenues, and market share by organization, as well as market rate, competitive landscape, and recent developments, transaction, growth, sale, and market shares of top companies.

⏩ Companies Profiles: The Global Mucopolysaccharidoses market's leading players are studied based on sales, main products, gross profit margin, revenue, price, and growth production.

⏩ Market Outlook by Region: The report goes through gross margin, sales, income, supply, market share, CAGR, and market size by region in this segment. North America, Europe, Asia Pacific, Middle East & Africa, and South America are among the regions and countries studied in depth in this study.

⏩ Market Segments: It contains the deep research study which interprets how different end-user/application/type segments contribute to the Mucopolysaccharidoses Market.

⏩ Market Forecast: Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

⏩ Research Findings: This section of the report showcases the findings and analysis of the report.

⏩ Conclusion: This portion of the report is the last section of the report where the conclusion of the research study is provided.

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