Mucopolysaccharidosis I Market Set for Steady Growth Through 2034, Driven by Advancements in Enzyme Replacement Therapies and Gene-Based Innovations | DelveInsight

Mucopolysaccharidosis I (MPS I) is a rare, inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase, leading to the accumulation of glycosaminoglycans (GAGs) in various tissues. This progressive disease manifests across a spectrum of severity-from the severe Hurler syndrome to attenuated forms like Hurler-Scheie and Scheie syndromes-and affects multiple organs, including the heart, bones, eyes, and central nervous system.

DelveInsight's latest report, "Mucopolysaccharidosis I - Market Insight, Epidemiology, and Market Forecast - 2034," delivers a comprehensive analysis of the disease landscape across the 7MM (the US, EU4 [Germany, France, Italy, and Spain], the UK, and Japan). It includes deep dives into MPS I prevalence, diagnostic trends, current treatment algorithms, and evolving therapeutic opportunities. The report also segments patients by subtype, mutation patterns, and disease severity, providing a robust epidemiological forecast through 2034.

The current MPS I treatment landscape is dominated by enzyme replacement therapy (ERT), particularly with Aldurazyme (laronidase), and hematopoietic stem cell transplantation (HSCT) for selected severe cases. While these treatments have improved life expectancy and quality of life, significant unmet needs remain, especially in addressing neurological manifestations and achieving earlier diagnosis. Gene therapies, substrate reduction therapies, and next-generation ERTs are under development to bridge these gaps.

DelveInsight forecasts steady growth in the Mucopolysaccharidosis I market over the coming decade, fueled by increasing awareness, expanded newborn screening programs, earlier intervention, and the anticipated arrival of novel therapies. As the understanding of MPS I pathophysiology advances and biotech innovation accelerates, the market is expected to undergo a transformative shift toward more durable and CNS-penetrating treatments, unlocking new opportunities for patients, providers, and pharmaceutical stakeholders alike.

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Some of the key insights of the Mucopolysaccharidosis I Market Report:
• In 2024, the MPS I market in the 7MM was valued at ~USD 145 million, with steady growth expected by 2034.
• The U.S. market reached ~USD 70 million, accounting for 48% of the 7MM total.
• Growth is driven by emerging therapies such as OTL-203, Lepunafusp alfa (JR-171), and RGX-111.
• ALDURAZYME led the market, generating ~USD 145 million in 2024.
• The 7MM had ~660 diagnosed prevalent cases of MPS I in 2024.
• The U.S. recorded ~240 cases, expected to grow at a 0.6% CAGR by 2034.
• A multicenter, randomized Phase III trial comparing OTL‐203 versus allogeneic hematopoietic stem cell transplantation (HSCT) began in December 2023, with estimated completion in March 2031 (NCT06149403)
• Emerging therapies for mucopolysaccharidosis I include OTL-203, Lepunafusp alfa (JR-171), RGX-111, and others.
• Key companies involved in the treatment of mucopolysaccharidosis I include JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.

To know in detail about the mucopolysaccharidosis I market outlook, drug uptake, treatment scenario, and epidemiology trends, click here: https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Mucopolysaccharidosis I Overview
Mucopolysaccharidosis I (MPS I) is a rare, inherited lysosomal storage disorder caused by mutations in the IDUA gene, resulting in a deficiency of the enzyme alpha-L-iduronidase. This leads to the accumulation of glycosaminoglycans (GAGs) in cells, tissues, and organs, causing progressive multisystem damage. MPS I is clinically classified into three phenotypes-Hurler (severe), Hurler-Scheie (intermediate), and Scheie (mild)-based on the age of onset and severity of symptoms.

The disease can affect nearly every organ system, with manifestations ranging from skeletal deformities, cardiac and respiratory complications, hepatosplenomegaly, corneal clouding, and developmental delays to severe neurological decline, particularly in the Hurler phenotype. Without timely intervention, patients with severe MPS I often face a significantly shortened lifespan.

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Mucopolysaccharidosis I Epidemiology
The epidemiology section offers an overview of historical, current, and projected trends in the seven major countries (7MM) from 2020 to 2034. It helps identify the factors influencing these trends by examining various studies and perspectives from key opinion leaders. Additionally, the section provides an in-depth analysis of the diagnosed patient population and future trends.

Mucopolysaccharidosis I Epidemiology Segmentation:
The mucopolysaccharidosis I market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM, segmented into:
• Mucopolysaccharidosis I Diagnosed Prevalent Cases
• Mucopolysaccharidosis I Severity-Specific Diagnosed Prevalent Cases
• Mucopolysaccharidosis I Treated Cases

Mucopolysaccharidosis I Drugs Uptake and Pipeline Development Activities
The Drug Uptake section offers a detailed analysis of the adoption trends of newly launched and upcoming therapies for mucopolysaccharidosis I throughout the study period. It evaluates patient adoption rates, market penetration, and the commercial performance of each therapy, providing a clear understanding of the factors driving or hindering the market acceptance of these treatments.

The Therapeutics Assessment further highlights the mucopolysaccharidosis I drugs, demonstrating the most rapid uptake. It examines the underlying drivers contributing to their swift adoption and compares the market share of these therapies to identify those gaining significant traction.

Additionally, the report provides an in-depth overview of the current therapeutic pipeline for mucopolysaccharidosis I, covering investigational drugs at various stages of development. It profiles the key pharmaceutical and biotech companies actively involved in advancing targeted treatments and presents the latest updates on partnerships, mergers and acquisitions, licensing deals, and other strategic developments shaping the future of mucopolysaccharidosis I therapeutics.

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Mucopolysaccharidosis I Market Outlook
The current Mucopolysaccharidosis I treatment landscape is largely defined by enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), each tailored to disease severity. While ALDURAZYME (laronidase) remains the only approved Mucopolysaccharidosis I drug, it primarily addresses somatic symptoms and lacks CNS penetration. HSCT, on the other hand, provides better neurological protection but is limited to severe cases due to procedural risks.

Given these therapeutic limitations, there is a pressing need for more effective Mucopolysaccharidosis I therapies that target both systemic and neurological manifestations. Innovative treatment strategies are being explored, including gene therapy and next-generation ERT, aiming to overcome current barriers such as immune responses and inadequate CNS delivery.

Several promising Mucopolysaccharidosis I drugs are advancing through the pipeline, including OTL-203, Lepunafusp alfa, RGX-111, and Iduronicrin genleukocel-T. These investigational agents hold the potential to transform the treatment paradigm, improve patient outcomes, and expand options beyond the current standard of care.

With rising awareness, early diagnosis through newborn screening, and ongoing clinical development, the Mucopolysaccharidosis I treatment market is poised for meaningful growth and innovation over the next decade.

Mucopolysaccharidosis I Market Drivers
• The ongoing development of innovative MPS I therapies such as gene therapy (e.g., OTL-203, RGX-111) and next-generation enzyme replacement therapies is expected to significantly improve treatment outcomes and expand options for patients.
• Increased implementation of newborn screening programs and rising disease awareness are facilitating earlier diagnosis, allowing timely intervention and improving the overall prognosis of MPS I patients.

Mucopolysaccharidosis I Market Barriers
• Current approved MPS I therapies, such as ALDURAZYME, do not effectively address central nervous system involvement due to poor blood-brain barrier penetration, leaving a critical treatment gap for patients with neurological symptoms.
• The high cost of lifelong enzyme replacement therapies and complex procedures like HSCT can limit access, particularly in low- and middle-income countries, affecting overall treatment uptake and market expansion.

Scope of the Mucopolysaccharidosis I Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan].
• Key Mucopolysaccharidosis I Companies: JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.
• Key Mucopolysaccharidosis I Therapies: OTL-203, Lepunafusp alfa (JR-171), RGX-111, and others.
• Mucopolysaccharidosis I Therapeutic Assessment: Mucopolysaccharidosis I currently marketed, and Mucopolysaccharidosis I emerging therapies.
• Mucopolysaccharidosis I Market Dynamics: Mucopolysaccharidosis I market drivers and Mucopolysaccharidosis I market barriers.
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies.
• Mucopolysaccharidosis I Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis I Market Access and Reimbursement.

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Table of Contents
1. Mucopolysaccharidosis I Market Report Introduction
2. Executive Summary for Mucopolysaccharidosis I
3. SWOT analysis of Mucopolysaccharidosis I
4. Mucopolysaccharidosis I Patient Share (%) Overview at a Glance
5. Mucopolysaccharidosis I Market Overview at a Glance
6. Mucopolysaccharidosis I Disease Background and Overview
7. Mucopolysaccharidosis I Epidemiology and Patient Population
8. Country-Specific Patient Population of Mucopolysaccharidosis I
9. Mucopolysaccharidosis I Current Treatment and Medical Practices
10. Mucopolysaccharidosis I Unmet Needs
11. Mucopolysaccharidosis I Emerging Therapies
12. Mucopolysaccharidosis I Market Outlook
13. Country-Wise Mucopolysaccharidosis I Market Analysis (2020-2034)
14. Mucopolysaccharidosis I Market Access and Reimbursement of Therapies
15. Mucopolysaccharidosis I Market Drivers
16. Mucopolysaccharidosis I Market Barriers
17. Mucopolysaccharidosis I Appendix
18. Mucopolysaccharidosis I Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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