Hunter Syndrome Market Forecast to 2034: ~1,200 Diagnosed Cases in 2023, FDA Priority Reviews, and Next-Gen Gene Therapies Reshaping the Treatment Landscape | DelveInsight

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The leading Hunter Syndrome Companies such as Homology Medicine, Denali Therapeutics, Regenxbio, Takeda, JCR Pharmaceuticals, GC Pharma, Regenxbio, Sangamo Therapeutics, ArmaGen, and others

In 2023, the US held the largest Hunter Syndrome market share among the 7MM, with around 500 prevalent cases. Current treatments include enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), and bone marrow transplantation (BMT). The only FDA-approved therapy is ELAPRASE, while IZCARGO and HUNTERASE ICV are approved in Japan. However, existing treatments do not address the cognitive symptoms, as ERTs cannot cross the blood–brain barrier. The pipeline shows promise, with REGENXBIO’s RGX-121 advancing as a potential first gene therapy for MPS II, backed by FDA alignment on accelerated approval using surrogate biomarkers. Additionally, Denali Therapeutics’ DNL310 demonstrated positive Phase I/II interim results at the 2024 WORLD Symposium, showing normalization of CSF heparan sulfate and reduction in neurofilament light chain (NfL).

 

DelveInsight’s report, “Hunter Syndrome Market Insight, Epidemiology, and Market Forecast – 2034,” provides a comprehensive analysis of Hunter Syndrome, covering its historical and projected epidemiology, along with market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. The report examines current treatment practices, emerging therapies, market share of available drugs, and market size forecasts from 2020 to 2034 across the seven major markets. It also highlights the treatment landscape, market drivers and barriers, unmet medical needs, and growth opportunities, offering insights into the future potential of the Hunter Syndrome market.

 

To Know in detail about the Hunter Syndrome market outlook, drug uptake, treatment scenario, and epidemiology trends, Click here: Hunter Syndrome Market Forecast

 

Some of the key facts of the Hunter Syndrome Market Report:

  • The US accounted for the largest Hunter Syndrome Market Size among the 7MM countries in 2023. The anticipated launch of targeted emerging therapies would bring about changes in treatment methodologies and facilitate market growth.
  • In February 2026, REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.
  • In January 2026, Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ISP-002, the company’s investigational therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
  • In July 2025, Denali Therapeutics Inc. (Nasdaq: DNLI) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for tividenofusp alfa for the treatment of Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026.
  • In May 2025, GC Biopharma, a South Korean pharmaceutical company, announced that the Phase 3 clinical trial results for 0048unterase (idursulfase beta), its investigational drug for Hunter Syndrome (MPS II), have been published in Genetics in Medicine, an SCIE-indexed journal.
  • The total number of Hunter Syndrome Diagnosed Prevalent Cases in the 7MM was nearly 1,200 cases in 2023 and is projected to increase during the forecasted period.
  • The total number of Hunter Syndrome Diagnosed Prevalent Cases in EU4 and the UK for hunter syndrome was estimated to be nearly 350 cases in 2023. Germany had the highest prevalent cases followed by the UK.
  • The leading Hunter Syndrome Companies such as Homology Medicine, Denali Therapeutics, Regenxbio, Takeda, JCR Pharmaceuticals, GC Pharma, Regenxbio, Sangamo Therapeutics, ArmaGen, and others
  • Promising Hunter Syndrome Therapies such as HMI-203, DNL310, RGX-121, SHP-609/ TAK-60, and others.

 

Hunter Syndrome Overview:

Hunter Syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This enzyme deficiency leads to the accumulation of glycosaminoglycans (GAGs) in cells, tissues, and organs, resulting in progressive damage. The disease primarily affects males and manifests with symptoms such as coarse facial features, enlarged liver and spleen, joint stiffness, respiratory issues, developmental delay, and in severe cases, neurocognitive decline. Current treatments include enzyme replacement therapy (ERT) and stem cell transplantation, which manage symptoms but do not cure the disease. Gene therapies are under investigation.

 

Discover Hunter Syndrome market drivers, barriers, company strategies, and emerging opportunities across the 7MM – Hunter Syndrome Treatment Market

 

Hunter Syndrome Epidemiology

In 2023, the 7MM recorded nearly 1,200 diagnosed prevalent cases of Hunter Syndrome, projected to rise in the coming years. The EU4 and UK accounted for about 350 cases, with Germany having the highest, followed by the UK. MPS II primarily affects males, with symptoms appearing around 2–4 years of age. About two-thirds of patients develop cognitive impairment in the neuronopathic form, with symptoms beginning at 1–3 years. Patients are classified into severe (65–70% of cases) and mild forms, though distribution varies slightly across countries.

Segmented by:

  • Total Diagnosed Prevalence of Hunter Syndrome
  • Severity-based Diagnosed Prevalence of Hunter Syndrome

 

Download the report to understand which factors are driving Hunter Syndrome epidemiology trends @ Hunter Syndrome Patient Population

 

Hunter Syndrome Drugs Uptake and Pipeline Development Activities

  • Tividenofusp alfa (DNL310): Denali Therapeutics

Tividenofusp alfa (DNL310) is a fusion protein composed of IDS fused to Denali’s proprietary Enzyme Transport Vehicle (ETV), which is engineered to cross the BBB via receptor-mediated transcytosis into the brain and to enable broad delivery of IDS into cells and tissues throughout the body with the goal of addressing the behavioral, cognitive, and physical manifestations of MPS II. In March 2021, the US Food and Drug Administration granted Fast Track Designation to DNL310 for the treatment of patients with MPS II. In May 2022, the European Medicines Agency granted DNL310 Priority Medicines designation.

 

  • RGX-121: Regenxbio

RGX121 is an investigational adeno-associated Virus (AAV) therapeutic for the treatment of MPS II. RGX121 is designed to use the NAV AAV9 vector to deliver the human IDS gene to cells in the Central Nervous System (CNS). RGX-121 is the only product in late-stage development with the potential to address neurocognitive development in patients diagnosed under the age of 2 years. • In August 2025, REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) extended its review timeline of the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The Prescription Drug User Fee Act (PDUFA) goal date has been extended from November 9, 2025 to February 8, 2026.

 

Get a comprehensive view of drug uptake trends, clinical progress, regulatory designations, and market opportunities shaping the Hunter Syndrome market @ Hunter Syndrome Medication and FDA Approvals

 

Hunter Syndrome Market Drivers

  • Unmet medical need: Current therapies (ERT, HSCT, BMT) only manage symptoms and do not address cognitive decline, creating strong demand for innovative treatments.
  • Advancements in gene therapy: Promising candidates like REGENXBIO’s RGX-121 and Denali Therapeutics’ DNL310 are advancing toward approval with encouraging trial results.
  • Regulatory incentives: FDA Orphan Drug Designation, Rare Pediatric Disease Designation, and accelerated approval pathways support innovation.
  • Rising awareness and diagnosis: Growing recognition of lysosomal storage disorders is improving early detection and patient identification.
  • Pipeline expansion: Multiple therapies in late-stage development provide opportunities for market growth.
  • Improved supportive care: Initiatives like Curant Rare’s IWILFIN Cares® program enhance patient access and quality of life.

 

Hunter Syndrome Market Barriers

  • Ultra-rare prevalence: With only ~1,200 diagnosed cases across the 7MM in 2023, the small patient pool limits commercial potential.
  • High treatment costs: ERTs and emerging gene therapies are extremely expensive, restricting accessibility.
  • Limited efficacy of current therapies: ERTs do not cross the blood–brain barrier, leaving cognitive symptoms unaddressed.
  • Heterogeneity of disease progression: Variability between mild and severe cases complicates treatment strategies and trial design.
  • Clinical trial challenges: Recruiting small pediatric populations delays development timelines.
  • Regulatory complexities: Ensuring safety and long-term efficacy in gene therapies adds additional hurdles.

 

To learn more about Hunter Syndrome companies working in the treatment market, visit @ Hunter Syndrome Clinical Trials and Therapeutic Assessment

 

Scope of the Hunter Syndrome Market Report

  • Study Period: 2020–2034
  • Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
  • Key Hunter Syndrome Companies: United Therapeutics, EUSA Pharma, Y-mAbs Therapeutics, Clarity Pharmaceuticals, and many others.
  • Key Hunter Syndrome Therapies: UNITUXIN (dinutuximab), QARZIBA (dinutuximab beta), Omburtamab, SARTATE, and others.
  • Hunter Syndrome Therapeutic Assessment: Hunter Syndrome current marketed and Hunter Syndrome emerging therapies
  • Hunter Syndrome Market Dynamics: Hunter Syndrome market drivers and Hunter Syndrome market barriers
  • Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
  • Hunter Syndrome Unmet Needs, KOL’s views, Analyst’s views, Hunter Syndrome Market Access and Reimbursement

 

Table of Contents

1. Hunter Syndrome Market Report Introduction

2. Executive Summary for Hunter Syndrome

3. SWOT analysis of Hunter Syndrome

4. Hunter Syndrome Patient Share (%) Overview at a Glance

5. Hunter Syndrome Market Overview at a Glance

6. Hunter Syndrome Disease Background and Overview

7. Hunter Syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Hunter Syndrome

9. Hunter Syndrome Current Treatment and Medical Practices

10. Hunter Syndrome Unmet Needs

11. Hunter Syndrome Emerging Therapies

12. Hunter Syndrome Market Outlook

13. Country-Wise Hunter Syndrome Market Analysis (2020–2034)

14. Hunter Syndrome Market Access and Reimbursement of Therapies

15. Hunter Syndrome Market Drivers

16. Hunter Syndrome Market Barriers

17. Hunter Syndrome Appendix

18. Hunter Syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

 

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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