Hunter Syndrome Pipeline Analysis Report 2025

Hunter syndrome, a rare genetic disorder, primarily affects children assigned male at birth (AMAB) and occurs in approximately 1 in 100,000 to 170,000 AMAB children. It imposes a significant burden due to progressive symptoms impacting multiple organs. There is a high unmet clinical need for advanced therapies, as current treatment options, such as enzyme replacement therapy, offer only symptomatic relief. The Hunter syndrome pipeline analysis by the publisher provides a detailed overview of emerging treatment options, including enzyme replacement therapies (ERT) and substrate reduction therapies currently under clinical development for managing this rare genetic disorder. Furthermore, the growing focus on gene therapies, novel drug formulations, and targeted treatments is likely to support the Hunter syndrome pipeline growth in the coming years.

Report Coverage

The Hunter Syndrome Pipeline Analysis Report by the publisher gives comprehensive insights into Hunter syndrome therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Hunter syndrome. The Hunter syndrome report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Hunter syndrome pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with Hunter syndrome treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Hunter syndrome.

Hunter Syndrome Pipeline Outlook

Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase. This leads to the buildup of glycosaminoglycans in cells, affecting multiple organs. It is an X-linked disorder, primarily affecting children assigned male at birth (AMAB), and results in progressive developmental delays, organ dysfunction, and reduced life expectancy.

Hunter syndrome treatments focus on managing symptoms and slowing disease progression. Enzyme replacement therapy (ERT) with Idursulfase helps reduce symptoms but does not cure the disease. Other emerging approaches, such as gene therapy and substrate reduction therapy, are being explored to improve outcomes and address the high unmet clinical need. In September 2021, Takeda collaborated with JCR Pharmaceuticals to commercialize JR-141, a next-generation investigational therapy for Hunter syndrome designed to cross the blood-brain barrier and target neuronopathic symptoms.

Hunter Syndrome Epidemiology

Hunter syndrome is a rare genetic disorder, primarily affecting children assigned male at birth (AMAB), with an incidence of 1 in 100,000 to 170,000 AMAB children. Mucopolysaccharidosis Type II is the most prevalent form of mucopolysaccharidosis, with a reported prevalence of 0.13 to 2.16 per 100,000 live births in Europe and 0.84 per 100,000 in Japan. Advancements in gene therapies and targeted treatments are poised to drive the growth of the Hunter syndrome pipeline, addressing the high unmet clinical need.

Hunter Syndrome Pipeline Therapeutic Assessment

This section of the report covers the analysis of Hunter syndrome drug candidates based on several segmentations, including:

By Phase

The pipeline report covers 50+ drug analyses based on phase:

• Late-Stage Products (Phase 3 and Phase 4)
• Mid-Stage Products (Phase 2)
• Early-Stage Products (Phase I)
• Preclinical and Discovery Stage Products

By Drug Class

The Hunter syndrome pipeline analysis report covers 50+ drug analyses based on drug classes:

• Small Molecules
• Gene Therapies
• Enzyme Replacement Therapies
• Hematopoietic Stem Cell Therapies

By Route of Administration

The pipeline assessment report covers 50+ drug analyses based on the route of administration.

• Oral
• Parenteral
• Others

Hunter Syndrome - Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase II covers a significant share of the total Hunter syndrome clinical trials, at 42.86%, reflecting strong ongoing research and development in Hunter syndrome therapies. Phase III accounts for 28.57%, highlighting promising advancements nearing regulatory review. Phase I follows with 21.43%, while phase IV contributes 7.14%, indicating continued post-approval studies. These developments positively drive innovation and future growth in the Hunter syndrome treatment.

Hunter Syndrome Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the Hunter syndrome pipeline analysis include small molecules, gene therapies, enzyme replacement therapies, and hematopoietic stem cell therapies. The Hunter syndrome report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Hunter syndrome. Gene-based drug therapies for rare diseases are also advancing, offering promising alternatives to traditional treatments. For instance, a world-first gene therapy for Hunter syndrome is under investigation at the Royal Manchester Children’s Hospital. Moreover, this therapy is designed to cross the blood-brain barrier, potentially reducing enzyme infusion reliance and addressing cognitive decline.

Hunter Syndrome Clinical Trials Therapeutic Assessment - Competitive Dynamics

The report for the Hunter syndrome pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Hunter syndrome therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Hunter syndrome clinical trials:

• Shire plc
• GC Biopharma Corp.
• Denali Therapeutics Inc.
• JCR Pharmaceuticals Co., Ltd.
• Regenxbio Inc.
• Generium
• Takeda Pharmaceuticals
• Sanofi
• Inventiva

Hunter Syndrome Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Hunter syndrome. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Hunter syndrome drug candidates.

Drug: Elaprase

Elaprase is currently under Phase IV study, conducted by Shire plc, to evaluate long-term height and weight changes in Hunter syndrome patients treated with the drug before the age of six. The study, utilizing patient registry data, involves around 21 participants and is expected to be completed by July 25, 2025. Elaprase is a formulation of idursulfase, a recombinant enzyme that hydrolyses glycosaminoglycan sulfate residues in lysosomes, which reduce substrate accumulation continuously.

Drug: JR-141

JR-141 is currently under a Phase III extension study, sponsored by JCR Pharmaceuticals Co., Ltd., to evaluate its long-term safety and efficacy in treating mucopolysaccharidosis Type II (Hunter syndrome). The study aims to assess the investigational drug’s sustained benefits. It is expected to conclude by February 28, 2028, and plans to enroll approximately 80 participants across multiple sites. JR-141 fuse the I2S enzyme with an anti-transferrin receptor antibody, which crosses the blood-brain barrier through receptor-mediated transcytosis and promotes lysosomal enzyme uptake.

Drug: Tividenofusp Alfa

Tividenofusp Alfa (DNL310) is currently under a Phase I/II clinical study, sponsored by Denali Therapeutics Inc., to evaluate its safety and efficacy in treating Hunter syndrome. The study aims to assess the safety, pharmacokinetics, and pharmacodynamics of this CNS-penetrant enzyme replacement therapy. With an estimated 47 participants enrolled, the trial is expected to be completed by July 2027. DNL310 delivers iduronate-2-sulfatase to lysosomes using a transport vehicle that crosses the blood-brain barrier via receptor-mediated transcytosis.

Key Questions Answered in the Hunter Syndrome - Pipeline Insight Report

• Which companies/institutions are leading the Hunter syndrome drug development?
• What is the efficacy and safety profile of Hunter syndrome pipeline drugs?
• Which company is leading the Hunter syndrome pipeline development activities?
• What is the current Hunter syndrome commercial assessment?
• What are the opportunities and challenges present in the Hunter syndrome pipeline landscape?
• What is the efficacy and safety profile of Hunter syndrome pipeline drugs?
• Which company is conducting major trials for Hunter syndrome drugs?
• Which companies/institutions are involved in Hunter syndrome collaborations aimed at providing enhanced therapeutic alternatives for patients?
• What are the geographies covered for clinical trials in Hunter syndrome?

Reasons To Buy This Report

The Hunter Syndrome Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Hunter syndrome. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Hunter syndrome collaborations, regulatory environments, and potential growth opportunities.

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