ハンター症候群治療薬の世界市場 (2025年～2033年)

世界のハンター症候群治療薬市場は、2024年に7億4,706万米ドルに達し、2033年には14億1,779万米ドルに達すると予測され、予測期間（2025～2033年）のCAGRは7.5%で成長すると予測されています。

ムコ多糖症II型（MPS II）としても知られるハンター症候群は、まれな遺伝性疾患です。特定の複合糖を体内で分解することができず、様々な組織に異常蓄積することで発症します。この蓄積は臓器や組織に進行性の障害を引き起こし、様々な身体的・認知的障害をもたらします。

この症候群の症状は一般的に幼児期に現れ、発達の遅れ、難聴、関節のこわばり、呼吸器系の問題などがあります。時間が経つにつれて、この疾患は子供の成長や生活の質全体に深刻な影響を及ぼす可能性があります。X連鎖性劣性遺伝のため、女性は通常保因者となり、主に男性が罹患します。

市場力学：

促進要因と抑制要因

臨床試験中の革新的な新規治療法

現在進行中の革新的な治療法の臨床試験は、ハンター症候群治療薬市場の成長に大きな影響を与えそうです。新しい治療が臨床段階を経るにつれて、より効果的で的を絞った治療が可能になり、患者の転帰を大幅に改善できる可能性があります。これらの臨床試験は、遺伝子治療や次世代酵素補充療法（ERT）などの治療の安全性、有効性、長期的な利益を決定する上で極めて重要です。例えば、2024年9月、REGENXBIO社は、ムコ多糖症II型（ハンター症候群）の治療を目的としたRGX-121の第I/II/III相CAMPSIITE試験の良好な結果を発表しました。この結果は2024年のSSIEM年次シンポジウムで発表される予定です。これらの結果は、RGX-121がMPS IIに対する1回限りの遺伝子治療薬となる可能性を支持するものであり、神経認知機能の低下に対処する最初の治療法となり、米国における神経因性疾患患者の第一選択薬となる可能性があります。もし成功すれば、これらの治療法は疾患の進行を遅らせるだけでなく、より持続可能で長期的な解決策を提供し、治療の頻度と期間を減らして市場全体の成長をもたらす可能性があります。

さらに、臨床試験が成功すれば、特に現在の治療法が経済的に制約のある地域において、より競争力のある価格設定と利用しやすくなる可能性があります。臨床試験から得られる知見は、治療プロトコルをさらに形成し、ハンター症候群を管理する新たな道を開くことになり、最終的に市場成長を加速させます。治療のパイプラインが前進するにつれて、より効果的な治療が期待され、選択肢が拡大することで、ハンター症候群治療薬市場の継続的な進化が促進されるでしょう。

以下は、ハンター症候群治療薬の様々な臨床段階にある主要パイプライン医薬品の表現です。

高い治療費

治療費の高さは、ハンター症候群治療薬市場の成長にとって大きな課題となっています。イドゥルスルファーゼのような酵素補充療法（ERT）は効果的ですが、生涯にわたる投与が必要であるため、治療全体が患者や医療システムにとって経済的負担となっています。このような長期にわたる治療が必要であることは、高額な治療費と相まって、所得の低い患者にとっては障壁となります。特に医療予算が限られている低・中所得国では、経済的負担が治療へのアクセスを制限し、治療が遅れたり、最も効果的な治療法にアクセスできなかったりする可能性があり、市場成長にも影響を及ぼしかねません。

目次

第1章 分析手法と分析範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 臨床試験における革新的な新治療法
    • 治療開発のための研究開発活動の強化
  • 抑制要因
    • 高額な治療費
    • 限られた治療オプション
  • 機会
    • 新興市場における医薬品アクセスの拡大
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• 疫学
• 価格分析
• パイプライン分析
• 特許分析
• 規制分析

第6章 薬剤別

• イデュルスルファーゼ
• イズカルゴ
• ハンターラーゼICV
• その他

第7章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州地域
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第8章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第9章 企業プロファイル

• Takeda Pharmaceuticals U.S.A., Inc.
  • 企業概要
  • 製品ポートフォリオ
    • 製品説明
    • 製品の主要業績評価指標（KPI）
    • 製品販売の実績・予測
    • 製品販売量
  • 財務概要
    • 企業の収益
    • 収益シェア：地域別
    • 収益予測
  • 主な動向
    • 企業合併・買収 (M&A)
    • 主な製品開発活動
    • 規制当局の承認等
  • SWOT分析
• GC Biopharma
• CANbridge Life Sciences Ltd
• JCR Pharmaceuticals Co., Ltd.
• REGENXBIO Inc.
• Denali Therapeutics
• Avrobio, Inc.
• Inventiva
• 各企業に関する同様のデータも提供されます。

第10章 付録

The global hunter syndrome treatment market reached US$ 747.06 million in 2024 and is expected to reach US$ 1,417.79 million by 2033, growing at a CAGR of 7.5% during the forecast period 2025-2033.

Hunter syndrome, also known as Mucopolysaccharidosis Type II (MPS II), is a rare, inherited genetic disorder. It occurs when the body is unable to break down certain complex sugars, leading to their abnormal accumulation in various tissues. This buildup causes progressive damage to organs and tissues, resulting in a range of physical and cognitive impairments.

The symptoms of the syndrome typically appear in early childhood, and they can include developmental delays, hearing loss, joint stiffness, and respiratory issues. Over time, the condition can severely impact a child's growth and overall quality of life. Since it is inherited in an X-linked recessive manner, it primarily affects males, while females are usually carriers.

Market Dynamics: Drivers & Restraints

Innovative Novel Therapies in Clinical Trials

The ongoing clinical trials for innovative therapies are poised to have a major impact on the growth of the Hunter syndrome treatment market. As new treatments progress through clinical stages, they bring the potential for more effective, targeted therapies that could significantly improve patient outcomes. These clinical trials are crucial in determining the safety, efficacy, and long-term benefits of treatments such as gene therapies and next-generation enzyme replacement therapies (ERTs). For instance, in September 2024, REGENXBIO announced positive results from the Phase I/II/III CAMPSIITE trial of RGX-121 for treating Mucopolysaccharidosis Type II (Hunter syndrome). The results will be presented at the 2024 SSIEM Annual Symposium. These support RGX-121 as a potential one-time gene therapy for MPS II, with the possibility of becoming the first treatment to address the neurocognitive decline and serve as a first-line option for patients with neuronopathic disease in the U.S. If successful, these therapies could not only slow disease progression but also provide more sustainable, long-term solutions, reducing the frequency and duration of treatment resulting in the overall market growth.

Additionally, successful clinical trial outcomes may result in more competitive pricing and greater accessibility, especially in regions where current therapies are financially restrictive. The insights gained from clinical trials will further shape treatment protocols and open up new avenues for managing Hunter syndrome, ultimately accelerating market growth. As the pipeline of therapies moves forward, the promise of more effective treatments and the expansion of options will fuel the continued evolution of the Hunter syndrome treatment market.

Below is the representation of key pipeline drugs that are in various clinical phases of Hunter syndrome treatment.

High Treatment Costs

The high cost of treatment poses a significant challenge to the growth of the Hunter syndrome treatment market. Enzyme replacement therapies (ERTs) like idursulfase, while effective, require lifelong administration, which makes the overall treatment financially burdensome for patients and healthcare systems. This long-term treatment requirement, coupled with high treatment costs, creates a barrier for patients with lower incomes. The financial strain limits access to care, particularly in low- and middle-income countries with limited healthcare budgets, potentially leading to delayed treatments or the inability to access the most effective therapies, which can even affect the market growth.

Segment Analysis

The global Hunter syndrome treatment market is segmented based on drug and region.

Idursulfase in the drug segment is dominating the market.

The idursulfase is expected to continue its dominance in the Hunter syndrome treatment market, largely due to its proven effectiveness as an enzyme replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). As a recombinant version of the enzyme iduronate-2-sulfatase, which is deficient in MPS II patients, idursulfase has shown significant clinical benefits.

Additionally, the treatment's long-term safety and ability to slow disease progression have made it the standard of care for MPS II. These established benefits ensure that idursulfase continues to dominate the market, with patients and healthcare providers relying on it for the effective management of Hunter syndrome.

Geographical Analysis

North America dominated the Hunter Syndrome Treatment market.

The North America region has the highest market share in the global hunter syndrome treatment market. A significant contributor to this dominance is the presence of major biopharmaceutical companies in the region, many of which are focused on developing cutting-edge treatments for Hunter syndrome.

The key companies have a robust pipeline of promising therapies in various stages of development, offering hope for more effective treatment options in the future. For instance, in February 2025, Denali Therapeutics announced the primary analysis of its Phase 1/2 study on tividenofusp alfa (DNL310) in 47 Hunter syndrome (MPS II) participants, covering a 24-week treatment period and long-term follow-up. With the recent Breakthrough Therapy designation, Denali plans to submit a biologics license application (BLA) in early 2025 for accelerated approval, aiming to deliver the treatment to the Hunter syndrome community by late 2025 or early 2026.

Thus, the above factors are expected to hold North America in the dominant position and will continue to play a crucial role in the ongoing progress of the market.

Competitive Landscape

The global market players in the Hunter Syndrome treatment market are Takeda Pharmaceuticals U.S.A., Inc., GC Biopharma, CANbridge Life Sciences Ltd, and JCR Pharmaceuticals Co., Ltd., among others.

Key Developments

• In January 2025, Denali Therapeutics announced that the U.S. FDA has granted a Breakthrough Therapy Designation for tividenofusp alfa (DNL310) to treat Hunter syndrome (MPS II). This adds to the existing fast-track, Orphan Drug, and Rare Pediatric Disease designations already granted for the therapy.
• In April 2023, JCR Pharmaceuticals and Sumitomo Pharma entered into a co-promotion agreement for IZCARGO I.V. Infusion 10 mg in Japan. IZCARGO is a recombinant treatment for mucopolysaccharidosis type II, currently marketed by JCR.
• In December 2022, JCR Pharmaceuticals announced that the U.S. FDA has granted Rare Pediatric Disease Designation (RPDD) to JR-141 (pabinafusp alfa) for the treatment of Mucopolysaccharidosis type II (Hunter syndrome).

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials and product pipelines and forecasts upcoming pharmaceutical advancements.
• Type Performance & Market Positioning: Analyze product performance, market positioning, and growth potential to optimize strategies.
• Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: This covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyze competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient Type delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance product safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global hunter syndrome treatment market report would provide approximately 45 tables, 46 figures, and 180 pages.

Target Audience 2024

• Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Drug
• 3.2. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Innovative Novel Therapies in Clinical Trials
    • 4.1.1.2. Increasing R&D Activities for the Development of Treatment
  • 4.1.2. Restraints
    • 4.1.2.1. High Treatment Costs
    • 4.1.2.2. Limited Treatment Options
  • 4.1.3. Opportunities
    • 4.1.3.1. Expanding Drug Access in Emerging Markets
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Forces Analysis
• 5.2. Epidemiology
• 5.3. Pricing Analysis
• 5.4. Pipeline Analysis
• 5.5. Patent Analysis
• 5.6. Regulatory Analysis

6. By Drug

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 6.1.2. Market Attractiveness Index, By Drug
• 6.2. idursulfase*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. IZCARGO
• 6.4. Hunterase ICV
• 6.5. Others

7. By Region

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 7.1.2. Market Attractiveness Index, By Region
• 7.2. North America
  • 7.2.1. Introduction
  • 7.2.2. Key Region-Specific Dynamics
  • 7.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 7.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 7.2.4.1. U.S.
    • 7.2.4.2. Canada
    • 7.2.4.3. Mexico
• 7.3. Europe
  • 7.3.1. Introduction
  • 7.3.2. Key Region-Specific Dynamics
  • 7.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 7.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 7.3.4.1. Germany
    • 7.3.4.2. U.K.
    • 7.3.4.3. France
    • 7.3.4.4. Spain
    • 7.3.4.5. Italy
    • 7.3.4.6. Rest of Europe
• 7.4. South America
  • 7.4.1. Introduction
  • 7.4.2. Key Region-Specific Dynamics
  • 7.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 7.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 7.4.4.1. Brazil
    • 7.4.4.2. Argentina
    • 7.4.4.3. Rest of South America
• 7.5. Asia-Pacific
  • 7.5.1. Introduction
  • 7.5.2. Key Region-Specific Dynamics
  • 7.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 7.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 7.5.4.1. China
    • 7.5.4.2. India
    • 7.5.4.3. Japan
    • 7.5.4.4. South Korea
    • 7.5.4.5. Rest of Asia-Pacific
• 7.6. Middle East and Africa
  • 7.6.1. Introduction
  • 7.6.2. Key Region-Specific Dynamics
  • 7.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug

8. Competitive Landscape

• 8.1. Competitive Scenario
• 8.2. Market Positioning/Share Analysis
• 8.3. Mergers and Acquisitions Analysis

9. Company Profiles

Key Market Players

• 9.1. Takeda Pharmaceuticals U.S.A., Inc.*
  • 9.1.1. Company Overview
  • 9.1.2. Product Portfolio
    • 9.1.2.1. Product Description
    • 9.1.2.2. Product Key Performance Indicators (KPIs)
    • 9.1.2.3. Historic and Forecasted Product Sales
    • 9.1.2.4. Product Sales Volume
  • 9.1.3. Financial Overview
    • 9.1.3.1. Company Revenue's
    • 9.1.3.2. Geographical Revenue Shares
    • 9.1.3.3. Revenue Forecasts
  • 9.1.4. Key Developments
    • 9.1.4.1. Mergers & Acquisitions
    • 9.1.4.2. Key Product Development Activities
    • 9.1.4.3. Regulatory Approvals, etc.
  • 9.1.5. SWOT Analysis
• 9.2. GC Biopharma
• 9.3. CANbridge Life Sciences Ltd
• 9.4. JCR Pharmaceuticals Co., Ltd.

Emerging Market Players

• 9.5. REGENXBIO Inc.
• 9.6. Denali Therapeutics
• 9.7. Avrobio, Inc.
• 9.8. Inventiva
• Similar data will be provided for each market player.

LIST NOT EXHAUSTIVE

10. Appendix

• 10.1. About Us and Services
• 10.2. Contact Us