United States Mucopolysaccharidoses Market 2025 | Growth Drivers, Market Forecast, Competitive Landscape & Business Opportunities

Market Size and Growth

The Global Mucopolysaccharidoses market 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.

Key Development:

United States: Recent Industry Developments

✅ In August 2025, Denali Therapeutics announced the initiation of a Phase 2 trial for its investigational enzyme replacement therapy targeting MPS II. The trial focuses on enhanced blood-brain barrier penetration and improved cognitive outcomes. It reflects a significant step in advancing next-generation therapies for rare lysosomal disorders.

✅ In July 2025, Regenxbio expanded its clinical manufacturing facility in Maryland to support gene therapy production for MPS I and II candidates. The expansion strengthens supply chain reliability for advanced rare disease trials. The investment highlights growing U.S. focus on scalable gene therapy infrastructure.

✅ In June 2025, Orchard Therapeutics received FDA Fast Track designation for its ex vivo gene therapy candidate for MPS IIIA. The designation enables expedited review and closer engagement with regulators. It underlines increasing U.S. momentum toward transformative cell and gene therapies for MPS.

Japan: Recent Industry Developments

✅ In August 2025, JCR Pharmaceuticals launched clinical development of its next-generation enzyme replacement therapy for MPS II. The therapy leverages J-Brain Cargo® technology to improve CNS delivery. It marks a major milestone in Japan's innovation pipeline for rare diseases.

✅ In July 2025, Takeda announced a strategic collaboration with Japanese academic institutions to accelerate gene therapy research for MPS disorders. The partnership focuses on translational programs integrating local expertise. It reflects Japan's push to strengthen domestic rare disease R&D capabilities.

✅ In May 2025, Astellas Pharma invested in early-stage biotech firms specializing in lysosomal storage diseases, including MPS. The funding aims to expand preclinical pipelines and establish global licensing opportunities. It signals Japan's growing commitment to diversify rare disease treatment strategies.

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Mucopolysaccharidoses (MPS) Market refers to the global industry focused on developing therapies, diagnostics, and supportive care for rare genetic lysosomal storage disorders caused by enzyme deficiencies leading to glycosaminoglycan (GAG) buildup. The market is driven by advancements in enzyme replacement therapies (ERT), gene therapy research, rising awareness of rare diseases, and increasing healthcare investments to improve patient outcomes.

Recent Mergers and Acquisitions:

✅ In April 2025, Spruce Biosciences acquired Tralesinidase Alfa (TA-ERT) for Sanfilippo syndrome type B (MPS IIIB). The acquisition aligns with its shift toward rare neurodegenerative enzyme replacement therapies, strengthening its position in lysosomal storage diseases.

✅ In April 2025, Spruce Biosciences also announced a broader corporate strategy focused on expanding its rare disease pipeline. The acquisition of TA-ERT is central to this roadmap, enabling advancement of late-stage clinical assets in MPS.

✅ In January 2025, Nippon Shinyaku entered into an exclusive licensing and commercialization agreement with REGENXBIO for RGX-121 and RGX-111, gene therapy candidates for MPS II and MPS I. While not a full acquisition, the deal represents a significant rights transfer in the global MPS treatment landscape.

Key Players:

=> Sanofi, Takeda Pharmaceuticals, GC Biopharma Corporate., JCR Pharmaceuticals Co., Ltd., BioMarin Pharmaceutical Inc., and Ultragenyx Pharmaceutical Inc., among others.

Growth Forecast Projected:

The Global Mucopolysaccharidoses Market is anticipated to rise at a considerable rate during the forecast period, between 2025 and 2033. In 2024, the market is growing at a steady rate, and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

Research Process:

Both primary and secondary data sources have been used in the global Mucopolysaccharidoses Market research report. During the research process, a wide range of industry-affecting factors are examined, including governmental regulations, market conditions, competitive levels, historical data, market situation, technological advancements, upcoming developments, in related businesses, as well as market volatility, prospects, potential barriers, and challenges.

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Key Segments:

➥ 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 for Mucopolysaccharidoses Market:

⇥ 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)

Benefits of the Report:

Chapter 1: Sets the stage by outlining the report's coverage, summarizing key market segments by region, product type, and application. Presents a snapshot of market sizes, growth potential across segments, and anticipated industry evolution both short and long term.

Chapter 2: Highlights pivotal market insights and uncovers the most significant emerging trends driving change within the industry.

Chapter 3: Offers an in-depth look at the competitive landscape among Mucopolysaccharidoses producers, including revenue shares, strategic moves, and recent mergers and acquisitions.

Chapter 4: Presents comprehensive profiles of the market's key players, delving into details such as revenue, profit margins, product portfolios, and company milestones.

Chapters 5 & 6: Analyze Mucopolysaccharidoses revenue at both regional and country levels, providing quantitative breakdowns of market sizes, growth opportunities, and development prospects worldwide.

Chapter 7: Focuses on different market segments by type, examining their individual sizes and potential, guiding readers toward high-impact, untapped market areas.

Chapter 8: Explores segmentation by application, evaluating industry growth potential in various downstream markets and pinpointing promising sectors for expansion.

Chapter 9: Provides a thorough review of the industry's supply chain mapping out both upstream and downstream activities.

Chapter 10: Concludes with a summary of the report's key findings and highlights the most critical takeaways for industry stakeholders.

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FAQ

What is the current size of the Mucopolysaccharidoses Market?

A: In 2024, the Mucopolysaccharidoses Market was valued at US$ 2,313.08 million, reflecting its strong industry presence.

Q2: How large is the Mucopolysaccharidoses Market expected to be by 2033?

A: By 2033, industry forecasts suggest the Mucopolysaccharidoses Market will grow to around US$ 4,389.85 million , demonstrating significant expansion.

Q3: What is the growth rate of the Mucopolysaccharidoses Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 7.5% during the forecast period from 2025 to 2033.

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