Mucopolysaccharidosis I (MPS I) Market is projected to reach USD 2.89 billion by 2034

The global Mucopolysaccharidosis I (MPS I) Market was valued at USD 1.42 billion in 2024 and is projected to reach USD 2.89 billion by 2034, growing at a CAGR of 7.3% during the forecast period (2025-2034). Increased newborn screening, rising availability of enzyme replacement therapies (ERT), growing adoption of hematopoietic stem cell transplantation (HSCT), and strong momentum in gene therapy development are the primary drivers of market expansion.

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MPS I is a rare lysosomal storage disorder caused by mutations in the IDUA gene, resulting in deficiency of the α-L-iduronidase enzyme and accumulation of glycosaminoglycans (GAGs) in multiple tissues. The disease presents across a clinical spectrum-ranging from the severe Hurler syndrome to the attenuated Hurler-Scheie and Scheie phenotypes. Without treatment, MPS I leads to progressive organ dysfunction, skeletal abnormalities, cognitive impairment, cardiopulmonary issues, and reduced lifespan.

Therapeutic progress over the past two decades has significantly improved outcomes, especially when treatment is initiated early.

Key Market Highlights
• 2024 Market Size: USD 1.42 billion
• 2034 Market Forecast: USD 2.89 billion
• CAGR (2025-2034): 7.3%
• Most Common Treatments: Enzyme replacement therapy & HSCT
• High-Mortality Phenotype: Severe MPS I (Hurler)

Market Growth Drivers
1. Rising Adoption of Enzyme Replacement Therapy (ERT)
Approved ERTs improve:
• Respiratory function
• Mobility
• Organ enlargement
• Quality of life
Growing treatment penetration in developing regions continues to expand the market.
2. Increasing Use of Hematopoietic Stem Cell Transplantation
HSCT remains the standard of care for severe MPS I when performed early, preserving cognitive function and prolonging survival.
3. Growth in Newborn Screening Programs
Several countries have added MPS I to newborn screening panels, enabling early diagnosis and timely intervention.
4. Strong Pipeline Activity in Gene Therapy
AAV and lentiviral gene therapy candidates aim to provide long-term or potentially curative benefit.
5. Improved Multidisciplinary Management
Patients require lifelong care involving cardiology, orthopedics, neurology, pulmonology, and physiotherapy, increasing overall healthcare demand.

Market Restraints
• Very high cost of enzyme replacement therapy
• Variability in treatment response
• Limited availability of HSCT expertise
• Challenges in CNS penetration for systemic therapies
• Underdiagnosis in low-income regions

Market Opportunities
1. Gene Therapy & Genome Editing
Advancements in viral vectors and genome correction strategies offer strong commercial potential.
2. Intrathecal & CNS-Targeted Therapies
Novel delivery routes are under development to address neurological manifestations.
3. Expansion of Screening in Emerging Markets
Countries in Asia, the Middle East, and Latin America are exploring newborn and high-risk screening initiatives.
4. Digital Patient Monitoring Tools
Wearables and remote monitoring tools enhance long-term disease tracking.
5. Improved Access to ERT via Global Health Partnerships
Subsidy programs and rare-disease foundations can accelerate therapy uptake.

Segmentation Overview
By Treatment Type
• Enzyme replacement therapy (ERT)
• Hematopoietic stem cell transplantation (HSCT)
• Gene therapy (pipeline)
• Supportive care (cardiac, respiratory, orthopedic)

By Disease Phenotype
• Severe MPS I (Hurler)
• Attenuated MPS I (Hurler-Scheie)
• Mild MPS I (Scheie)

By Diagnostic Method
• Enzyme activity testing
• Genetic testing (IDUA sequencing)
• Urinary GAG analysis
• Newborn screening

By End User
• Hospitals & specialty clinics
• Stem cell transplant centers
• Genetic testing laboratories
• Research institutions

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Regional Insights
North America - Largest Market
Strong newborn screening adoption, established ERT availability, advanced HSCT centers, and active gene therapy trials drive regional dominance.
Europe - Strong Rare Disease Infrastructure
Widespread healthcare access, cross-border treatment programs, and early diagnosis pathways support consistent growth.
Asia Pacific - Fastest-Growing Region
Improved newborn screening access, rising healthcare investment, and expanding genetic testing capabilities fuel strong demand.
Latin America & Middle East/Africa - Emerging Markets
Diagnosis and treatment access are improving through collaborations with global nonprofits and pharmaceutical access programs.

Competitive Landscape
Key companies involved in MPS I treatment, diagnostics, and therapy development include:
• Sanofi (Aldurazyme - ERT)
• BioMarin
• Orchard Therapeutics (gene therapy research)
• Takeda
• Pfizer (advanced gene therapy capabilities)
• Regenxbio
• Rocket Pharmaceuticals
• Thermo Fisher Scientific (genetic testing technologies)
• Labcorp & Quest Diagnostics
These players are actively supporting advancements in ERT, HSCT, diagnostics, and curative genetic medicine.

Recent Market Developments
• Ongoing Phase I/II trials for AAV-based gene therapies
• Improvements in HSCT outcomes through reduced toxicity regimens
• Expansion of newborn screening in the U.S., Europe, and Asia
• New biomarker development for monitoring disease progression
• Increased global research collaborations on lysosomal disorders

Future Outlook (2025-2034)
The MPS I Market is expected to evolve significantly with:
• Broader use of gene therapy as clinical results mature
• Expansion of newborn screening enabling earlier and more effective intervention
• Growth of intrathecal and CNS-targeted therapies
• Greater global access to enzyme replacement therapy
• Digital tools aiding in long-term disease management
With strong therapeutic advances and rising diagnostic penetration, the market is projected to grow from USD 1.42 billion in 2024 to USD 2.89 billion by 2034, signaling sustained momentum in the rare metabolic disease segment.

This report is also available in the following languages : Japanese (ムコ多糖症I市場), Korean (점액다당증 I 시장), Chinese (粘多糖贮积症 I 市场), French (Mucopolysaccharidose I Marché), German (Mukopolysaccharidose I Markt), and Italian (Mercato della mucopolisaccaridosi I), etc.

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