Mucopolysaccharidosis Type I Market Size, Share, Growth, Epidemiology and Forecast to 2036

Mucopolysaccharidosis Type I (MPS I) Market Insights, Epidemiology, and Market Forecast - 2036, provides a comprehensive assessment of the MPS I market across the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The report evaluates historical and forecasted epidemiology, patient burden, current treatment practices, emerging therapies, market dynamics, competitive intelligence, drug uptake, reimbursement, pricing, and unmet medical needs through 2036.
(Albany, New York) - 22nd Sep 2026 - DelveInsight's latest report, "Mucopolysaccharidosis Type I (MPS I) Market Insights, Epidemiology, and Market Forecast - 2036," provides a comprehensive assessment of the MPS I market across the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The report evaluates historical and forecasted epidemiology, patient burden, current treatment practices, emerging therapies, market dynamics, competitive intelligence, drug uptake, reimbursement, pricing, and unmet medical needs through 2036.

According to DelveInsight, the MPS I market across the 7MM was valued at approximately USD 155 million in 2025 and is projected to expand at a CAGR of 8.6% during 2026-2036. The report identifies increasing disease awareness, improved diagnosis, continued demand for enzyme replacement therapy, and the anticipated development of gene and cell therapies as important factors shaping the future MPS I treatment landscape.

Key Takeaways from the Mucopolysaccharidosis Type I Market

* The 7MM MPS I market was valued at approximately USD 155 million in 2025.
* DelveInsight reports an 8.6% CAGR during 2026-2036.
* The US represented the largest MPS I market in 2025, valued at approximately USD 75 million.
* The EU4 and UK together accounted for approximately USD 70 million in 2025.
* Japan represented approximately USD 10 million of the MPS I market in 2025.
* The 7MM had approximately 650 diagnosed prevalent MPS I cases in 2025.
* The US accounted for approximately 240 diagnosed prevalent cases in 2025.
* The EU4 and UK together contributed approximately 400 diagnosed prevalent cases, with the UK accounting for approximately 110 cases.
* Japan had the largest proportion of Scheie syndrome (MPS IS) cases, representing approximately 50% of all MPS I cases based on disease severity.
* The EU4 and UK accounted for approximately 240 Hurler syndrome (MPS IH) cases in 2025.
* Laronidase (ALDURAZYME) remains the established enzyme replacement therapy for MPS I.
* Key emerging therapies include OTL-203, lepunafusp alfa (JR-171), Iduronicrin genleukocel-T (ISP-001), and DNL622 (ETV:IDUA).
* OTL-203 is in Phase III clinical development, while JR-171 is in Phase I/II and ISP-001 is in Phase I.
* Key companies include BioMarin Pharmaceutical, Orchard Therapeutics, JCR Pharmaceuticals, Sanofi, IMMUSOFT, and others.

Keen to know more about the Mucopolysaccharidosis Type I market? Request a sample copy of the report for detailed market insights, epidemiology, treatment patterns, competitive intelligence, and forecast analysis.

[https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=kspr]

Key Factors Driving the Mucopolysaccharidosis Type I Market

Advancements in Enzyme Replacement Therapies

The continued use and development of enzyme replacement therapies remain important contributors to the MPS I market. Laronidase (ALDURAZYME) provides recombinant alpha-L-iduronidase to address the underlying enzyme deficiency and reduce glycosaminoglycan accumulation in affected tissues.

Although ERT provides important systemic benefits, its limitations in reaching the central nervous system are encouraging research into therapies capable of providing broader disease control.

Increasing Awareness and Early Diagnosis

MPS I can present with diverse and nonspecific symptoms, which can contribute to delayed or inaccurate diagnosis. Greater awareness among healthcare professionals and improvements in screening and molecular diagnostic approaches may facilitate earlier identification of affected patients.

Newborn screening and genetic testing are particularly important for identifying patients earlier and enabling timely intervention.

Rising Investment in Rare Disease Research

The high unmet medical need associated with MPS I is attracting pharmaceutical and biotechnology investment into orphan-drug development. Research is increasingly focused on therapies that can address both systemic and neurological manifestations of the disease.

Supportive Regulatory Incentives

Regulatory incentives for rare diseases, including Fast Track designation, Rare Pediatric Disease designation, and PRIME status, are supporting the development of novel MPS I therapies. OTL-203, for example, has received multiple regulatory designations supporting its clinical development.

Emergence of Gene and Cell Therapies

The MPS I pipeline is increasingly shifting toward potentially durable or one-time interventions. Gene therapy and engineered-cell approaches aim to introduce functional IDUA into the patient's cells and potentially provide more sustained enzyme production than conventional replacement therapy.

Competitive Landscape

The MPS I competitive landscape includes an established enzyme replacement therapy and several emerging gene, cell, and recombinant enzyme approaches.

Key companies include:

* BioMarin Pharmaceutical
* Orchard Therapeutics
* JCR Pharmaceuticals
* Sanofi
* IMMUSOFT
* Denali Therapeutics
* and others.

The marketed treatment landscape is currently centered on Laronidase (ALDURAZYME), while the emerging pipeline includes:

* OTL-203 - Orchard Therapeutics/Kyowa Kirin
* Lepunafusp alfa (JR-171) - JCR Pharmaceuticals
* Iduronicrin genleukocel-T (ISP-001) - IMMUSOFT
* DNL622 (ETV:IDUA) - Denali Therapeutics

Recent Developments in the Mucopolysaccharidosis Type I Market

* July 2025: Orchard Therapeutics reported that the final patient had been treated in the registrational trial of OTL-203 for MPS I Hurler syndrome.
* September 2024: JCR Pharmaceuticals presented data at the SSIEM Annual Symposium regarding investigational lysosomal storage disorder treatments, including neurobehavioral and somatic improvements observed in MPS I patients treated with JR-171.
* September 2022: IMMUSOFT announced that the US FDA had cleared its Investigational New Drug (IND) application for ISP-001, an engineered B-cell therapy being developed for MPS I.
* September 2021: The US FDA granted Fast Track Designation to JCR Pharmaceuticals for JR-171, targeting CNS symptoms associated with MPS I.

What is Mucopolysaccharidosis Type I?

Mucopolysaccharidosis Type I is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase (IDUA). This enzyme is responsible for breaking down glycosaminoglycans (GAGs), particularly dermatan sulfate and heparan sulfate. When IDUA activity is deficient, these substances accumulate within cells and tissues, causing progressive dysfunction across multiple organ systems.

MPS I can affect the skeletal, cardiovascular, respiratory, and nervous systems, with clinical severity ranging from severe forms associated with Hurler syndrome to attenuated forms such as Hurler-Scheie and Scheie syndromes.

Diagnosis involves GAG analysis, enzyme activity testing, and genetic testing. Additional assessments can include pulmonary function testing, polysomnography, audiometry, ophthalmological examinations, skeletal imaging, and cognitive evaluations. Newborn screening and molecular testing can support earlier diagnosis and management.

Current Treatment Landscape

Treatment selection depends largely on disease severity, age, clinical manifestations, and neurological involvement.

Laronidase (ALDURAZYME)

Laronidase (ALDURAZYME) is a recombinant form of human alpha-L-iduronidase produced using recombinant DNA technology. It is administered by intravenous infusion and replaces the deficient enzyme to facilitate degradation of accumulated GAGs.

ALDURAZYME is approved in the US, Europe, and Japan for MPS I and primarily addresses systemic manifestations. However, its limited penetration across the blood-brain barrier restricts its ability to address CNS manifestations.

The therapy carries a boxed warning concerning serious hypersensitivity reactions, including anaphylaxis, as well as acute respiratory complications associated with administration.

Hematopoietic Stem Cell Transplantation

Hematopoietic stem cell transplantation (HSCT) is an established treatment approach for severe MPS I, particularly in young children. Donor-derived cells can produce IDUA and may provide benefits for neurological disease by enabling enzyme production within the CNS.

However, HSCT does not fully prevent all disease manifestations, with persistent challenges involving skeletal abnormalities, joint contractures, and corneal clouding.

Emerging Therapies in MPS I

OTL-203

OTL-203, developed by Orchard Therapeutics and Kyowa Kirin, is a one-time gene therapy using a patient's own hematopoietic stem and progenitor cells. The cells are genetically modified ex vivo using a lentiviral vector carrying functional IDUA complementary DNA.

OTL-203 is currently in Phase III clinical development in North America and Europe. The report identifies a planned approval timeframe of 2029/2030. The program has received US FDA Fast Track and Rare Pediatric Disease designations as well as PRIME status from the European Medicines Agency.

Lepunafusp Alfa (JR-171)

Lepunafusp alfa (JR-171) is an investigational recombinant fusion protein developed by JCR Pharmaceuticals. It is designed to cross the blood-brain barrier using JCR's J-Brain Cargo platform and deliver the deficient IDUA enzyme to the CNS.

JR-171 is in Phase I/II clinical development, with a provisional US launch year of 2031 in the report.

Iduronicrin Genleukocel-T (ISP-001)

ISP-001, developed by IMMUSOFT, is an engineered B-cell therapy designed to provide sustained production of functional IDUA. It is currently in Phase I clinical development.

DNL622 (ETV:IDUA)

DNL622 (ETV:IDUA) from Denali Therapeutics is a preclinical approach designed to deliver iduronidase replacement therapy with the objective of addressing CNS disease.

Mucopolysaccharidosis Type I Epidemiology

DelveInsight estimates that the 7MM had approximately 650 diagnosed prevalent MPS I cases in 2025. The epidemiological analysis evaluates diagnosed prevalent cases, disease-severity-specific populations, and treated cases across the US, EU4, UK, and Japan.

Key epidemiological findings include:

* The 7MM diagnosed prevalent population was approximately 650 cases in 2025.
* The US accounted for nearly 240 cases, representing the largest individual market.
* The EU4 and UK together accounted for approximately 400 cases.
* The UK represented approximately 110 cases in 2025.
* Japan had the largest proportion of Scheie syndrome (MPS IS) cases, accounting for approximately 50% of Japanese MPS I cases.
* The EU4 and UK accounted for approximately 240 Hurler syndrome (MPS IH) cases in 2025.

Epidemiology Segmentation

DelveInsight segments MPS I epidemiology into:

* Diagnosed Prevalent Cases of MPS I
* Severity-specific Diagnosed Prevalent Cases of MPS I
* Treated Cases of MPS I

Unmet Needs in Mucopolysaccharidosis Type I

Despite the availability of ERT and HSCT, significant unmet needs remain:

* Delayed and inaccurate diagnosis
* Limited CNS penetration of current ERT
* Persistent bone and joint complications
* Limited access to specialized care
* Need for more durable treatment approaches
* Research and development gaps
* Need for standardized treatment guidelines

The inability of conventional systemic ERT to adequately address neurological manifestations remains a particularly important challenge, supporting research into blood-brain barrier-penetrating therapies and gene-based approaches.

Mucopolysaccharidosis Type I Market Outlook

The MPS I market is expected to evolve as next-generation therapies progress through clinical development. The combination of improved diagnosis, increasing awareness, established ERT, and the development of gene and cell therapies is expected to influence the treatment paradigm over the forecast period.

The development of OTL-203, JR-171, ISP-001, and other innovative approaches reflects a shift toward treatments designed to provide more comprehensive disease control, including potential benefits for CNS manifestations.

Scope of the Mucopolysaccharidosis Type I Market Report

* Study Period: 2022-2036
* Historical Period: 2022-2025
* Forecast Period: 2026-2036
* Base Year: 2025
* Geographies: US, Germany, France, Italy, Spain, UK, and Japan
* 7MM Market Size, 2025: ~USD 155 million
* Market CAGR: 8.6% during 2026-2036
* US Market Size, 2025: ~USD 75 million
* EU4 + UK Market Size, 2025: ~USD 70 million
* Japan Market Size, 2025: ~USD 10 million
* 7MM Diagnosed Prevalent Cases, 2025: ~650
* Epidemiology Segmentation: Diagnosed prevalent cases, severity-specific diagnosed prevalent cases, and treated cases
* Market Segmentation: Region/geographies, drugs/therapies, and class of therapies
* Key Companies: BioMarin Pharmaceutical, Orchard Therapeutics, JCR Pharmaceuticals, Sanofi, IMMUSOFT, Denali Therapeutics, and others
* Key Marketed Therapy: Laronidase (ALDURAZYME)
* Key Emerging Therapies: OTL-203, lepunafusp alfa (JR-171), Iduronicrin genleukocel-T (ISP-001), DNL622 (ETV:IDUA), and others
* Analysis: Addressable patient population, market drivers and barriers, cost assumptions and pricing analogues, KOL views, SWOT analysis, reimbursement, attribute analysis, and unmet needs
* Key Strengths: Epidemiology-based bottom-up forecasting, AI-enabled market research, 11-year forecast, regional market outlook, patient burden trends, treatment addressable market, competitive landscape, company insights, price trends and analogue assessment, therapy adoption/uptake, and peak patient share analysis.

To Know More About the Mucopolysaccharidosis Type I Market Report

Mucopolysaccharidosis Type I Market Insights, Epidemiology, and Market Forecast - 2036 [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=kspr]

Table of Contents

* Key Insights
* Report Introduction
* MPS I Market Overview at a Glance
* Executive Summary
* Key Events
* Disease Background and Overview
* Causes and Risk Factors
* Clinical Types
* Signs and Symptoms
* Pathogenesis
* Diagnosis and Diagnostic Algorithm
* Treatment Landscape
* Treatment Guidelines
* Epidemiology and Patient Burden
* Patient Journey
* Marketed Therapies
* Emerging Therapies and Pipeline Analysis
* Drug Uptake and Patient Share
* Market Size and Forecast
* Competitive Landscape
* Market Drivers and Barriers
* Unmet Medical Needs
* Market Access and Reimbursement
* Pricing and Analogue Assessment
* KOL and SME Insights
* SWOT and Attribute Analysis
* Regional Market Analysis
* Company and Therapy Profiles
* Market Outlook
* Conclusions and Strategic Insights

About DelveInsight

DelveInsight is a leading market research and consulting firm specializing in the life sciences and healthcare industries. Founded in 2014, the company provides comprehensive market intelligence, epidemiology, and insights across pharmaceuticals, biotechnology, medical devices, and emerging therapies. DelveInsight helps healthcare organizations make informed strategic decisions through data-driven research and industry expertise.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kirti Sharma
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=mucopolysaccharidosis-type-i-market-size-share-growth-epidemiology-and-forecast-to-2036]
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/consulting/due-diligence-services

Legal Disclaimer: Information contained on this page is provided by an independent third-party content provider. ABNewswire makes no warranties or responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you are affiliated with this article or have any complaints or copyright issues related to this article and would like it to be removed, please contact retract@swscontact.com



This release was published on openPR.