Mucopolysaccharidosis Type I Market to Witness Upsurge in Growth at a CAGR of 8% During the Forecast Period (2025-2034) | DelveInsight

Key Takeaways from the Mucopolysaccharidosis Type I Market Report

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:

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

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 tocross 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.

Scope of the Mucopolysaccharidosis Type I Market Report

Download the report to understand which factors are driving MPS I market trends @ Mucopolysaccharidosis Type I Market Trends

Table of Contents

Related Reports

Mucopolysaccharidosis Type I Pipeline

Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key MPS I companies, including Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, among others.

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Mucopolysaccharidosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS companies, including Takeda Pharmaceutical, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Sanofi, among others.

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Mucopolysaccharidosis Type III Market

Mucopolysaccharidosis Type III Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS III companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Lysogene, Inventiva, among others.

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