07-05-2025
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Mucopolysaccharidosis Type I Market to Witness Upsurge in Growth at a CAGR of 8% During the Forecast Period (2025-2034)
The future of MPS I treatment is poised for significant advancements, driven by ongoing research and innovative therapies. Emerging therapies, such as stem cell gene therapy, IDUA Gene therapy, and Recombinant DNA, including OTL-203, Lepunafusp alfa (JR-171), RGX-111, and Iduronicrin genleukocel-T (ISP-001), offer novel approaches. These therapies have the potential to reshape the treatment landscape by improving patient outcomes and reducing the limitations of existing treatments.
LAS VEGAS, May 7, 2025 /PRNewswire/ — DelveInsight's Mucopolysaccharidosis Type I Market Insights report includes a comprehensive understanding of current treatment practices, Mucopolysaccharidosis type I emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Mucopolysaccharidosis Type I Market Report
According to DelveInsight's analysis, the market size for Mucopolysaccharidosis type I was found to be USD 145 million in the 7MM in 2024.
in the 7MM in 2024. In 2024, the MPS I market size in the US was highest, accounting for 48% of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies.
of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies. According to DelveInsight's estimates, in 2024, there were approximately 660 diagnosed prevalent cases of MPS I in the 7MM. Of these, the United States accounted for 36% of the cases.
diagnosed prevalent cases of MPS I in the 7MM. Of these, accounted for of the cases. Leading Mucopolysaccharidosis type I companies developing emerging therapies, such as Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years.
and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years. The promising Mucopolysaccharidosis type I therapies in the pipeline include OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others.
and others. In March 2025 , REGENXBIO Inc. finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in the United States and Asia .
finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in and . In February 2025 , at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study.
at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study. In
January 2025 , Immusoft announced positive results from the first engineered B Cell (ISP-001) in a human clinical trial, to be presented at the WORLDSymposium.
Discover which therapies are expected to grab the major MPS I market share @ Mucopolysaccharidosis Type I Market Report
Mucopolysaccharidosis Type I Overview
MPS I is a rare inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), which is essential for the breakdown of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate. When these substances accumulate, they lead to progressive damage in cells and tissues, impacting various organ systems, including the skeletal, cardiovascular, respiratory, and nervous systems. The condition presents as a spectrum, with the most severe form being Hurler syndrome and the mildest form known as Scheie syndrome.
Diagnosis of MPS I involves testing for elevated GAG levels, enzyme activity assays, and genetic analysis, all of which support early intervention, prognosis determination, and genetic counseling. Early detection is further aided by newborn screening programs. Ongoing disease management relies on comprehensive assessments such as pulmonary function tests, sleep studies, hearing evaluations, eye exams, imaging of the bones, and cognitive testing. Molecular diagnostics and early screening are critical to improving patient outcomes and guiding long-term care.
Mucopolysaccharidosis Type I Epidemiology Segmentation
The Mucopolysaccharidosis type I epidemiology section provides insights into the historical and current Mucopolysaccharidosis type I patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Mucopolysaccharidosis type I market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Diagnosed Prevalence of MPS I
Severity-specific Diagnosed Prevalent Cases of MPS I
Treated Cases of MPS I
Hurler Syndrome Treatment Market
The treatment landscape for MPS I (Hurler syndrome) is progressing gradually, with growing insights into both the disease's biology and its management. Traditional therapies like ERT and HSCT remain central, but advances in precision medicine and new pharmacological options are beginning to show promise. Despite these developments, a universal, comprehensive treatment approach remains elusive due to the disease's complexity and variability among patients, requiring a tailored, multidisciplinary strategy.
Supportive care plays a critical role and includes various surgical procedures such as adenotonsillectomy, hernia repair, ventriculoperitoneal shunting, heart valve replacement, carpal tunnel release, and spinal decompression. Therapies like physical, occupational, and speech therapy, along with respiratory aids like CPAP, hearing devices, and medications for managing pain and gastrointestinal symptoms, are also essential. Vision issues may require corneal transplants, although surgical interventions in Hurler syndrome can be risky due to anesthetic complications.
ALDURAZYME (laronidase), a recombinant version of the human IDUA enzyme produced using Chinese hamster ovary cells, was approved in the U.S., EU4, the UK (in 2003), and Japan (in 2006). It is indicated for patients with Hurler and Hurler-Scheie types of MPS I, and for symptomatic Scheie patients with moderate to severe manifestations. While ALDURAZYME carries a boxed warning due to potential safety concerns, it remains the only approved ERT for MPS I, with no biosimilars available despite the expiration of its patent.
To know more about ALDURAZYME mechanism of action and laronidase cost, visit @ ALDURAZYME Patent Expiration
Mucopolysaccharidosis Type I Pipeline Therapies and Key Companies
OTL-203: Orchard Therapeutics/Kyowa Kirin
Lepunafusp alfa (JR-171): JCR Pharmaceuticals
RGX-111: REGENXBIO/Nippon Shinyaku
Iduronicrin genleukocel-T (ISP-001): IMMUSOFT
Discover more about MPS I drugs in development @ Mucopolysaccharidosis I Clinical Trials Market
Mucopolysaccharidosis Type I Market Dynamics
The MPS I market dynamics are expected to change in the coming years. Current treatments for MPS I, including ERT and hematopoietic stem cell transplantation, have demonstrated effectiveness in managing symptoms and improving the quality of life for patients with severe forms of the disease. Advances in genetic testing and the expansion of newborn screening programs have enabled earlier diagnosis, allowing for timely intervention and significantly improved patient outcomes. Increased awareness of MPS I, coupled with enhanced genetic counseling, has further contributed to earlier detection and better disease management.
Additionally, the growing interest in rare diseases has attracted increased funding and support for research, accelerating the development of new and potentially more effective treatments for MPS I. These combined efforts are shaping a more promising future for patients and families affected by this rare lysosomal storage disorder.
Furthermore, potential therapies are being investigated for the treatment of MPS I, and it is safe to predict that the treatment space will significantly impact the mucopolysaccharidosis therapeutics market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the MPS I market in the 7MM.
However, several factors may impede the growth of the MPS I market. ERTs for MPS I, such as intravenous iduronidase, are lifelong treatments typically administered every week. While ERT has shown effectiveness in alleviating many somatic symptoms, it is unable to cross the blood-brain barrier, rendering it ineffective in addressing the neurological manifestations associated with severe forms of MPS I. This represents a significant limitation in the current therapeutic approach.
As an autosomal recessive disorder, MPS I requires both parents to be carriers, which complicates family planning and increases the likelihood of recurrence in future pregnancies. Despite the benefits of available treatments like ALDURAZYME, the financial burden associated with long-term therapy remains a challenge for many patients and families. Additionally, serious adverse reactions—including life-threatening anaphylaxis—have been reported in some patients during or up to three hours after ALDURAZYME infusion, underscoring the need for careful monitoring and consideration of risk-benefit balance in treatment planning.
Moreover, MPS I treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the MPS I market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the MPS I market growth.
Mucopolysaccharidosis Type I Market Report Metrics
Details
Study Period
2020–2034
Coverage
7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Mucopolysaccharidosis Type I Market CAGR
8 %
Mucopolysaccharidosis Type I Market Size in 2024
USD 145 Million
Key Mucopolysaccharidosis Type I Companies
Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, and others
Key Pipeline Mucopolysaccharidosis Type I Therapies
OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others
Scope of the Mucopolysaccharidosis Type I Market Report
Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and emerging therapies
Mucopolysaccharidosis Type I current marketed and emerging therapies Mucopolysaccharidosis Type I Market Dynamics: Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook
Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis Type I Market Access and Reimbursement
Download the report to understand which factors are driving MPS I market trends @ Mucopolysaccharidosis Type I Market Trends
Table of Contents
1
Key Insights
2
Report Introduction
3
MPS I Market Overview at a Glance
3.1
Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2020
3.2
Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2034
4
Executive Summary
5
Key Events
6
Disease Background and Overview
6.1
Introduction
6.2
Causes and Risk Factors
6.3
Clinical Types
6.4
Symptoms
6.5
Pathogenesis
6.6
Diagnosis
6.6.1
Laboratory Diagnosis
6.6.2
Biomarkers
6.6.3
Diagnostic Algorithm
6.6.4
Diagnostic Guidelines
6.7
Treatment
6.7.1
Treatment Algorithm
6.7.2
Treatment Guidelines
7
Epidemiology and Market Methodology
8
Epidemiology and Patient Population
8.1
Key Findings
8.2
Assumptions and Rationale: 7MM
8.2.1
Diagnosed Prevalent Cases of MPS I
8.2.2
Severity-specific Diagnosed Prevalent Cases of MPS I
8.2.3
Treated Cases of MPS I
8.3
Total Diagnosed Prevalent Cases of MPS I in the 7MM
8.4
The United States
8.4.1
Diagnosed Prevalent Cases of MPS I in the US
8.4.2
Severity-specific Diagnosed Prevalent Cases of MPS I in the US
8.4.3
Treated Cases of MPS I in the US
8.5
EU4 and the UK
8.5.1
Diagnosed Prevalent Cases of MPS I in EU4 and the UK
8.5.2
Severity-specific Diagnosed Prevalent Cases of MPS I in EU4 and the UK
8.5.3
Treated Cases of MPS I in EU4 and the UK
8.6
Japan
8.6.1
Diagnosed Prevalent Cases of MPS I in Japan
8.6.2
Severity-specific Diagnosed Prevalent Cases of MPS I in Japan
8.6.3
Treated Cases of MPS I in Japan
9
Patient Journey
10
Marketed Therapies
10.1
ALDURAZYME (laronidase): BioMarin Pharmaceutical/Sanofi
10.1.1
Product Description
10.1.2
Regulatory Milestones
10.1.3
Other Developmental Activities
10.1.4
Clinical Trials Information
10.1.5
Safety and Efficacy
To be continued in the report…
11
Emerging Drug Profiles
11.1
Key Cross Competition of Emerging Drugs
11.2
OTL-203: Orchard Therapeutics/Kyowa Kirin
11.2.1
Drug Description
11.2.2
Other Developmental Activities
11.2.3
Clinical Trials Information
11.2.4
Safety and Efficacy
11.2.5
Analysts' Views
11.3
Lepunafusp alfa (JR-171): JCR Pharmaceuticals
11.3.1
Drug Description
11.3.2
Other Developmental Activities
11.3.3
Clinical Trials Information
11.3.4
Safety and Efficacy
11.3.5
Analysts' Views
11.4
RGX-111: REGENXBIO/Nippon Shinyaku
11.4.1
Drug Description
11.4.2
Other Developmental Activities
11.4.3
Clinical Trials Information
11.4.4
Safety and Efficacy
11.4.5
Analysts' Views
11.5
Iduronicrin genleukocel-T (ISP-001): IMMUSOFT
11.5.1
Drug Description
11.5.2
Other Developmental Activities
11.5.3
Clinical Trials Information
11.5.4
Safety and Efficacy
11.5.5
Analysts' Views
To be continued in the report…
12
MPS I: Market Analysis
12.1
Key Findings
12.2
Market Outlook
12.3
Attribute Analysis
12.4
Key Market Forecast Assumptions
12.4.1
Cost Assumptions and Rebates
12.4.2
Pricing Trends
12.4.3
Analogue Assessment
12.4.4
Launch Year and Therapy Uptake
12.5
Total Market Size of MPS I in the 7MM
12.6
Market Size of MPS I by Therapies in the 7MM
12.7
Market Size of MPS I in the United States
12.7.1
Total Market Size of MPS I
12.7.2
Market Size of MPS I by Therapies in the United States
12.8
Market Size of MPS I in EU4 and the UK
12.8.1
Total Market Size of MPS I
12.8.2
Market Size of MPS I by Therapies in EU4 and the UK
12.9
Market Size of MPS I in Japan
12.9.1
Total Market Size of MPS I
12.9.2
Market Size of MPS I by Therapies in Japan
13
Key Opinion Leaders' Views
14
Unmet Needs
15
SWOT Analysis
16
Market Access and Reimbursement
16.1
The United States
16.1.1
CMS
16.2
In EU4 and the UK
16.2.1
Germany
16.2.2
France
16.2.3
Italy
16.2.4
Spain
16.2.5
The United Kingdom
16.3
Japan
16.3.1
MHLW
17
Appendix
17.1
Acronyms and Abbreviations
17.2
Bibliography
17.3
Report Methodology
18
DelveInsight Capabilities
19
Disclaimer
20
About DelveInsight
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