United States Mucopolysaccharidosis Treatment Market 2025: Industry Developments, Future Growth, Share & Industry Insights | REGENXBIO Inc, Sangamo Therapeutics, Abeona Therapeutics, and Orchard Therapeutics

Mucopolysaccharidosis Treatment Market size reached US$ 2.78 Billion in 2023 with a rise of US$ 3.02 Billion in 2024 and is expected to reach US$ 6.76 Billion by 2033, growing at a CAGR of 9.4% during the forecast period 2025-2033.

DataM Intelligence has published a new research report on "Mucopolysaccharidosis Treatment Market Size 2025". The report explores comprehensive and insightful Information about various key factors like Regional Growth, Segmentation, CAGR, Business Revenue Status of Top Key Players and Drivers. The purpose of this report is to provide a telescopic view of the current market size by value and volume, opportunities, and development status.

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Industry Latest News 2025:

✅ 14 Jan 2025 - Nippon Shinyaku and REGENXBIO entered into an exclusive license agreement for gene‐therapies RGX‐121 (for MPS II) and RGX‐111 (for MPS I): Nippon Shinyaku takes development/commercialisation rights in U.S. & Asia; REGENXBIO retains rest‐of‐world.

✅ 07 Jul 2025 - Orchard Therapeutics announced last patient treated in the registrational trial of OTL‐203 for MPS I (Hurler syndrome).

✅ 05 Sept 2025 - REGENXBIO reported positive 12‐month pivotal data from its phase I/II/III trial of RGX‐121 for MPS II: sustained biomarker reductions and neurodevelopment stability.

✅ 18 Aug 2025 - REGENXBIO announced that the U.S. Food and Drug Administration (FDA) extended its review of the BLA for RGX‐121 (MPS II) from Nov 9 2025 to Feb 8 2026.

✅ 08 Sept 2025 - JCR Pharmaceuticals presented long‐term (up to 5 years) clinical data on Pabinafusp alfa (JR‐141) in MPS II (Hunter syndrome), showing sustained neurocognitive & somatic benefit.

✅ 29 Sept 2025 - JCR Pharmaceuticals and MEDIPAL announced that Japan's Ministry of Health, Labour and Welfare granted Orphan Drug Designation to investigational therapy JR‐446 for MPS IIIB.

✅ 07 Oct 2025 - Immusoft announced that a patient in its lead programme for MPS I (ISP‐001 engineered B‐cell therapy) has been safely re‐dosed 18 months later, showing positive functional outcomes.

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Major Key Players: BioMarin Pharmaceutical Inc., Sanofi, Takeda Pharmaceuticals Inc., Clinigen Limited, and Ultragenyx Pharmaceutical Inc., REGENXBIO Inc., Sangamo Therapeutics, Abeona Therapeutics, and Orchard Therapeutics.

Market Growth Drivers:

✅ Advancements in Enzyme Replacement Therapies (ERT): The development of innovative ERTs has improved patient outcomes, driving adoption in regions with advanced healthcare facilities.

✅ Growing Rare Disease Awareness: Increased awareness among healthcare professionals and patients has led to early diagnosis and better disease management, boosting demand for treatments.

✅ Government Initiatives and Reimbursement Policies: Supportive policies and insurance coverage for rare disease treatments in North America and Europe enhance market accessibility.

✅ Emergence of Gene Therapy and Precision Medicine: Ongoing research in gene therapy is opening new avenues for long-term treatment solutions, attracting investment and clinical trials globally.

✅ Rising Investment in Pediatric Healthcare: As MPS primarily affects children, heightened focus on pediatric healthcare infrastructure contributes to market expansion.

Segments Covered in the Mucopolysaccharidosis Treatment Market

By Disease Type (MPS Type 1 ((Hurler syndrome), MPS Type 2 (Hunter syndrome), MPS Type 3 (Sanfilippo syndrome), MPS Type 4 (Morquio syndrome), MPS Type 6 (Maroteaux-Lamy syndrome), MPS Type 7 (Sly syndrome), MPS Type 9 (Natowicz syndrome)

By Treatment Type (Enzyme Replacement Therapy, Haematopoietic Stem Cell Transplantation (HSCT), Gene Therapy, Others)

By End User (Hospitals, Infusion Centers, Ambulatory Surgical Centers)

By Region (North America, Europe, South America, Asia Pacific, Middle East, and Africa)

Regional Analysis for Mucopolysaccharidosis Treatment 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)

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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, Mucopolysaccharidosis Treatment Market segments, study objectives, and years considered.

⏩ Market Landscape: The competition in the Global Mucopolysaccharidosis Treatment 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 Mucopolysaccharidosis Treatment 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 Mucopolysaccharidosis Treatment 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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Frequently asked questions:

➠ What is the global sales value, production value, consumption value, import and export of Mucopolysaccharidosis Treatment Market?

➠ Who are the global key manufacturers of the Mucopolysaccharidosis Treatment Market Industry? How is their operating situation (capacity, production, sales, price, cost, gross, and revenue)?

➠ What are the Mucopolysaccharidosis Treatment Market opportunities and threats faced by the vendors in the global Mucopolysaccharidosis Treatment Market Industry?

➠ Which application/end-user or product type may seek incremental growth prospects? What is the market share of each type and application?

➠ What focused approach and constraints are holding the Mucopolysaccharidosis Treatment Market?

➠ What are the different sales, marketing, and distribution channels in the global industry?

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