Mucopolysaccharidosis Market

Market Overview:

The 7 major mucopolysaccharidosis markets reached a value of USD 1.7 Billion in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 3.1 Billion by 2035, exhibiting a growth rate (CAGR) of 5.72% during 2025-2035.

The mucopolysaccharidosis market has been comprehensively analyzed in IMARC's new report titled "Mucopolysaccharidosis Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Mucopolysaccharidosis (MPS) refer to a group of rare, inherited metabolic disorders resulting from the absence or malfunctioning of specific enzymes needed to break down molecules called glycosaminoglycans (GAGs). These molecules accumulate in the body's cells, leading to progressive, multi-system damage. Symptoms vary based on the type of MPS but may include developmental delays, skeletal abnormalities, short stature, joint stiffness, coarse facial features, and problems with vision and hearing. Organ systems like the heart, central nervous system, and respiratory system can also be affected, leading to life-threatening complications. Because symptoms are often nonspecific and gradually progressive, diagnosis can be challenging. Typically, physicians may initially suspect MPS based on clinical presentation and may proceed with urine tests to check for elevated GAG levels. A definitive diagnosis is usually made by enzyme assays from blood samples or fibroblasts, and in some cases, genetic testing may be conducted to identify the specific mutation causing the disorder.

The increasing cases of genetic mutations affecting enzymes responsible for breaking down complex sugar molecules, leading to the accumulation of these substances in various tissues and organs, are primarily driving the mucopolysaccharidosis market. In addition to this, the inflating utilization of supportive care measures, including physical and occupational therapies, since they aim to enhance mobility, preserve joint function, and improve overall well-being, is also creating a positive outlook for the market. Moreover, the widespread adoption of hematopoietic stem cell transplantation (HSCT), especially in younger patients with certain types of MPS, is further propelling the market growth. This procedure involves the replacement of defective cells in the bone marrow with healthy ones, thereby correcting the enzyme deficiency. Additionally, the rising usage of efficacious enzyme replacement therapies (ERTs) like laronidase, idursulfase, elosulfase alfa, etc., which help to provide enzymatic activity to reduce the deficient enzymes in individuals suffering from the illness, is acting as another significant growth-inducing factor. Apart from this, the escalating popularity of innovative techniques, such as gene therapy, which focus on the introduction or correction of genetic material within a patient's cells to remedy or counteract the disorder, is expected to drive the mucopolysaccharidosis market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the mucopolysaccharidosis market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for mucopolysaccharidosis and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the mucopolysaccharidosis market in any manner.

Recent Developments:

• In February 2024, Denali Therapeutics Inc. released new data presentations that illustrate the broad potential of its BBB-crossing enzyme replacement medicines in development for the management of mucopolysaccharidosis (MPS). New research showed that tividenofusp alfa (DNL310) treatment for 104 weeks improved and stabilized clinical outcomes in MPS II. The single-arm, open-label Phase 1/2 study also showed a robust and sustained reduction in neurofilament light chain (NfL) and sustained normalization of CSF heparan sulfate.
• In February 2024, REGENXBIO Inc. reported topline results from the Phase I/II/III CAMPSIITE trial of RGX-121 for the treatment of patients up to 5 years old with MPS II, also known as Hunter syndrome, indicating that the pivotal phase of the trial met its primary endpoint with statistical significance.
• In October 2023, JCR Pharmaceuticals Co., Ltd. announced that it had dosed the first patient in Phase I/II clinical trial for mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) with JR-441, a blood-brain barrier (BBB) penetrating form of heparan N-sulfatase developed using JCR's proprietary J-Brain Cargo BBB-penetrating technology.
• In September 2023, JCR Pharmaceuticals Co., Ltd. reported major findings from the 52-week interim data of its global phase I/II research with JR-171 (lepunafusp alfa) in patients with MPS I. JR-171 is a BBB-penetrating recombinant α-L-iduronidase produced with JCR's patented J-Brain Cargo technology.

Key Highlights:

• The prevalence of all kinds of mucopolysaccharidosis is estimated to be one per 25,000 births.
• The incidence of MPS in the United States was determined to be 0.98 per 100,000 live births.
• The highest birth prevalence was observed for MPS II, which accounts for 55% of all MPS.
• In the United States, the birth rates for MPS IV, VI, and VII were 0.14, 0.04, and 0.027 per 100,000 live births, respectively.

Drugs:

ELAPRASE (idursulfase) is a medication that is prescribed to patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). It is a pure form of the I2S enzyme developed through recombinant DNA technology in a human cell line. The medicine has been found to improve walking capacity in individuals aged five and above.

DNL310 is an experimental iduronate-2-sulfatase (IDS) fusion protein designed to cure both the cognitive and physical symptoms of MPS II with a single weekly IV infusion. The medicine is designed to penetrate the BBB via receptor-mediated transcytosis into the brain, allowing for widespread transport of IDS into cells and tissues throughout the body, to treat disease symptoms.

RGX-121 is an investigational, one-time gene treatment that uses the NAV AAV9 vector to deliver IDS, which encodes the iduronate-2-sulfatase enzyme (I2S). The RGX-121-produced protein is structurally identical to normal I2S. RGX-121 is delivered directly to the central nervous system (CNS) via intracisternal or intracerebroventricular routes.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the mucopolysaccharidosis market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the mucopolysaccharidosis market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current mucopolysaccharidosis marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report

Key Questions Answered in this Report:

Market Insights

• How has the mucopolysaccharidosis market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the mucopolysaccharidosis market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the mucopolysaccharidosis market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2019-2035) of mucopolysaccharidosis across the seven major markets?
• What is the number of prevalent cases (2019-2035) of mucopolysaccharidosis by age across the seven major markets?
• What is the number of prevalent cases (2019-2035) of mucopolysaccharidosis by gender across the seven major markets?
• What is the number of prevalent cases (2019-2035) of mucopolysaccharidosis by type across the seven major markets?
• How many patients are diagnosed (2019-2035) with mucopolysaccharidosis across the seven major markets?
• What is the size of the mucopolysaccharidosis patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of mucopolysaccharidosis?
• What will be the growth rate of patients across the seven major markets?
   

Mucopolysaccharidosis: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for mucopolysaccharidosis drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the mucopolysaccharidosis market?
• What are the key regulatory events related to the mucopolysaccharidosis market?
• What is the structure of clinical trial landscape by status related to the mucopolysaccharidosis market?
• What is the structure of clinical trial landscape by phase related to the mucopolysaccharidosis market?
• What is the structure of clinical trial landscape by route of administration related to the mucopolysaccharidosis market?