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ハーラー症候群治療の世界市場 (2025年～2033年)

Global Hurler Syndrome Treatment Market - 2025-2033

出版日
ページ情報
英文 220 Pages
納期
即日から翌営業日
カスタマイズ可能
適宜更新あり
価格
価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=146.82円
ハーラー症候群治療の世界市場 (2025年～2033年)
出版日: 2025年03月11日
発行: DataM Intelligence
ページ情報: 英文 220 Pages
納期: 即日から翌営業日
GIIご利用のメリット
  • 全表示
  • 概要
  • 目次
概要

世界のハーラー症候群治療市場は、2024年に3億7,765万米ドルに達し、2033年には5億2,934万米ドルに達すると予測され、予測期間（2025～2033年）にはCAGR 3.9%で成長する見込みです。

ハーラー症候群はムコ多糖症（MPS i）としても知られ、11のムコ多糖症（MPS）疾患のひとつです。以前はガーゴイル症と呼ばれていました。遺伝的欠陥によるリソソーム異常が原因です。グリコサミノグリカン（GAGまたはムコ多糖類）の破壊過程に影響する酵素であるαLイドゥロニダーゼが存在しません。

市場力学：

促進要因と抑制要因

酵素補充療法（ERT）の採用拡大

世界のハーラー症候群治療市場は、酵素補充療法（ERT）の採用増加により活況を呈しています。ERTは不足した酵素を補うことで疾患の進行を遅らせ、患者の生存率とQOLを向上させます。酵素製剤の進歩、政府の支援、希少疾病用医薬品の優遇措置、ヘルスケアへの投資により、ERTはより利用しやすくなっています。製薬会社が酵素療法の最適化に注力するにつれ、市場は拡大しています。

酵素補充療法（ERT）は、体内のグリコサミノグリカンを分解することにより、ハーラー症候群の症状を緩和することができます。しかし、ERTは血液脳関門を通過して発達中の脳に到達することができず、神経認知機能の低下や知的障害を引き起こします。これは、ERTが脳機能に対処できず、患者に神経認知機能の低下や知的障害のリスクが残るためです。

開発途上地域における治療選択肢の限られた利用可能性

低開発国や発展途上諸国では、医療が行き届かないことが多いです。このことは通常、これらの地域ではハーラー症候群の市場が限られていることを意味します。例えば、酵素補充療法（ERT）や造血幹細胞移植（HSCT）は、この疾患に対する最も効果的な治療法ですが、これらの国では容易に利用できません。しかし、ほとんどの貧しい国々では、必要な医療インフラや、治療を実施したり診断を迅速に下すことのできる専門家が、先ほど強調したような需要パターンの結果、段階的に確保できない可能性があります。

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
      • 酵素補充療法（ERT）の採用拡大
    • 抑制要因
      • 発展途上地域での治療選択肢の制約
    • 機会
    • 影響分析

第5章 戦略的洞察と業界展望

  • 市場のリーダーと先駆者
    • 新たな先駆者と主要企業
    • 最大の売上を誇るブランドを確立したリーダー
    • 確立された製品を持つ市場リーダー
  • CXOの視点
  • 最新の開発とブレークスルー
  • ケーススタディ/進行中の研究
  • 規制と償還の情勢
    • 北米
    • 欧州
    • アジア太平洋
    • ラテンアメリカ
    • 中東・アフリカ
  • ポーターのファイブフォース分析
  • サプライチェーン分析
  • 特許分析
  • SWOT分析
  • アンメットニーズとギャップ
  • 市場参入と拡大のための推奨戦略
  • シナリオ分析：ベストケース・ベースケース・ワーストケースの予測
  • 価格分析と価格市場力学
  • キーオピニオンリーダー (KOL)

第6章 治療の種類別

  • 酵素補充療法（ERT）
  • 造血幹細胞移植（HSCT）
  • 骨髄移植（BMT）
  • その他

第7章 年齢層別

  • 成人
  • 小児

第8章 エンドユーザー別

  • 病院
  • 専門クリニック
  • その他

第9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10章 競合情勢

  • 競合シナリオ
  • 市況・シェア分析
  • M&A分析

第11章 企業プロファイル

  • BioMarin
    • 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
    • 主な発展
      • 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
  • Sanofi
  • Orchard Therapeutics
    • パイプライン製品の説明
    • 製品の主要業績評価指標（KPI）
    • 主な活動
    • 市場参入のタイムライン
    • 製品普及率
    • 売上予測と予測
  • JCR Pharmaceuticals
  • Immusoft
  • Regenxbio
  • Paradigm Biopharmaceuticals Ltd
  • 各々の企業に対しても同様のデータが提供されます。

第12章 付録

目次
Product Code: PH9238

The Global Hurler Syndrome Treatment Market reached US$ 377.65 million in 2024 and is expected to reach US$ 529.34 million by 2033, growing at a CAGR of 3.9% during the forecast period 2025-2033.

Hurler syndrome is also known as mucopolysaccharidosis (MPS I), one of the eleven mucopolysaccharidoses (MPS) disorders. It was formerly referred to as gargoylism. It is caused by an inherited lysosomal problem due to a genetic defect. There is no alpha L iduronidase, an enzyme that affects the destruction process of glycosaminoglycans (GAGs or mucopolysaccharides).

Market Dynamics: Drivers & Restraints

Growing adoption of Enzyme Replacement Therapy (ERT)

The global Hurler syndrome treatment market is thriving due to the increasing adoption of enzyme replacement therapy (ERT). ERT slows disease progression by supplementing deficient enzymes, improving patient survival and quality of life. Advancements in enzyme formulations, government support, orphan drug incentives, and healthcare investments are making ERT more accessible. As pharmaceutical companies focus on optimizing enzyme therapies, the market expands.

Enzyme replacement therapy (ERT) can alleviate Hurler syndrome symptoms by breaking down glycosaminoglycans in the body. However, ERT cannot cross the blood-brain barrier to reach the developing brain, causing neurocognitive decline and intellectual disability. This is because ERT cannot address brain function, leaving patients at risk for neurocognitive decline and intellectual disability.

Limited Availability of Treatment Options in Developing Regions

Availability of medical care is often sparse in underdeveloped or developing countries. This usually implies that the market of Hurler syndrome in these areas is also limited since there are minimum treatment options to save lives. For example, enzyme replacement therapy (ERT) and haematopoietic or bone marrow transplantation (HSCT), are the most effective treatments for the condition may not be readily available in these countries. However, in most of the poor countries, the required health care infrastructure and professionals capable of carrying out treatment or making the diagnosis promptly may not be able to be staged as a result of the demand pattern just highlighted.

Segment Analysis

The global hurler syndrome treatment market is segmented based on treatment type, Age Group, end user, and region.

Treatment Type:

Enzyme Replacement Therapy (ERT) in the treatment type segment is expected to dominate the market with the highest share.

As of now, only one enzyme replacement therapy, i.e., laronidase, is approved for the treatment of Hurler syndrome. Sold under the brand name Aldurazyme, manufactured by Sanofi, this therapy has been available in the market since 2003 across 75 countries. As compared to other treatment options, which are mostly experimental, enzyme replacement therapy turned out to be the most promising approach for the treatment of Hurler syndrome. By addressing the underlying enzymatic deficiency associated with the condition, Aldurazyme has become a long-term care option for the patient population. The drug has generated sales of 321 million in 2024, majorly generating sales from recurring prescriptions. Moreover, several enzyme replacement therapies are in the clinical pipeline, which are anticipated to make market entry in the forecast period.

Geographical Analysis

North America is expected to hold a significant position in the Hurler Syndrome Treatment market share

North America holds a substantial position in the hurler syndrome treatment market due to advanced healthcare infrastructure and the presence of approved therapies. The United States sees a very dominant role, owing to the increase in government initiatives and the wide range of orphan drug designations encouraging innovative product launches.

For instance, in November 2023, Orchard Therapeutics received Fast Track designation from the FDA for OTL-203, an investigational hematopoietic stem cell gene therapy for the treatment of the Hurler subtype of mucopolysaccharidosis. This designation aims to expedite the development of medicines that address serious medical needs, potentially allowing for quicker submission and review timelines for therapeutic candidates. Once the product is launched, the population in the U.S. may get early market access to this therapy and benefit from it.

Moreover, the only approved drug for hurler syndrome, i.e., Aldurazyme (laronidase) by Sanofi, has been widely available in the U.S. since 2003, and the company generates major sales from the country.

Competitive Landscape

The major global players in the Hurler Syndrome Treatment market include Sanofi and BioMarin, among others.

Various other emerging players include, Orchard Therapeutics, JCR Pharmaceuticals, Immusoft, Armagen, Regenxbio, Paradigm Biopharmaceuticals Ltd, and others.

Key Developments

  • In February 2024, Orchard Therapeutics, acquired by Kyowa Kirin, has randomized the first patient in a registrational trial at M Health Fairview Masonic Children's Hospital to evaluate the efficacy and safety of OTL-203, an investigational hematopoietic stem cell gene therapy, in patients with Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares OTL-203 treatment to standard care with allogeneic hematopoietic stem cell transplant (HSCT) and is expected to enroll 40 MPS-IH patients.
  • In December 2023, Orchard Therapeutics, acquired by Kyowa Kirin, has randomized the first patient in a registrational trial at M Health Fairview Masonic Children's Hospital to evaluate the efficacy and safety of OTL-203, an investigational hematopoietic stem cell gene therapy, in patients with Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares OTL-203 treatment to standard care with allogeneic hematopoietic stem cell transplant (HSCT) and is expected to enroll 40 MPS-IH patients.

Why Purchase the Report?

  • Pipeline & Innovations: Reviews ongoing clinical trials, product pipelines, and forecasts upcoming advancements in medical devices and pharmaceuticals.
  • Product 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 product 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 Hurler Syndrome Treatment market report delivers a detailed analysis with 61 key tables, more than 59 visually impactful figures, and 220 pages of expert insights, providing a complete view of the market landscape.

Target Audience 2024

  • Manufacturers: Pharmaceutical, Medical Device, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
  • Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
  • Treatment Type & Innovation: AI/Robotics Providers, 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 Treatment Type
  • 3.2. Snippet by Age Group
  • 3.3. Snippet by End User
  • 3.4. Snippet by Region

4. Dynamics

  • 4.1. Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Growing Adoption of Enzyme Replacement Therapy (ERT)
    • 4.1.2. Restraints
      • 4.1.2.1. Limited Availability of Treatment Options in Developing Regions
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Strategic Insights and Industry Outlook

  • 5.1. Market Leaders and Pioneers
    • 5.1.1. Emerging Pioneers and Prominent Players
    • 5.1.2. Established leaders with largest selling Brand
    • 5.1.3. Market leaders with established Product
  • 5.2. CXO Perspectives
  • 5.3. Latest Developments and Breakthroughs
  • 5.4. Case Studies/Ongoing Research
  • 5.5. Regulatory and Reimbursement Landscape
    • 5.5.1. North America
    • 5.5.2. Europe
    • 5.5.3. Asia Pacific
    • 5.5.4. Latin America
    • 5.5.5. Middle East & Africa
  • 5.6. Porter's Five Forces Analysis
  • 5.7. Supply Chain Analysis
  • 5.8. Patent Analysis
  • 5.9. SWOT Analysis
  • 5.10. Unmet Needs and Gaps
  • 5.11. Recommended Strategies for Market Entry and Expansion
  • 5.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
  • 5.13. Pricing Analysis and Price Dynamics
  • 5.14. Key Opinion Leaders

6. By Treatment Type

  • 6.1. Introduction
    • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 6.1.2. Market Attractiveness Index, By Treatment Type
  • 6.2. Enzyme Replacement Therapy (ERT)*
    • 6.2.1. Introduction
    • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 6.3. Hematopoietic Stem Cell Transplant (HSCT)
  • 6.4. Bone Marrow Transplantation (BMT)
  • 6.5. Others

7. By Age Group

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 7.1.2. Market Attractiveness Index, By Age Group
  • 7.2. Adult*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. Pediatric

8. By End User

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
    • 8.1.2. Market Attractiveness Index, By End User
  • 8.2. Hospitals*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Specialty Clinics
  • 8.4. Others

9. By Region

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
    • 9.1.2. Market Attractiveness Index, By Region
  • 9.2. North America
    • 9.2.1. Introduction
    • 9.2.2. Key Region-Specific Dynamics
    • 9.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
    • 9.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.2.6.1. U.S.
      • 9.2.6.2. Canada
      • 9.2.6.3. Mexico
  • 9.3. Europe
    • 9.3.1. Introduction
    • 9.3.2. Key Region-Specific Dynamics
    • 9.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
    • 9.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.3.6.1. Germany
      • 9.3.6.2. U.K.
      • 9.3.6.3. France
      • 9.3.6.4. Spain
      • 9.3.6.5. Italy
      • 9.3.6.6. Rest of Europe
  • 9.4. South America
    • 9.4.1. Introduction
    • 9.4.2. Key Region-Specific Dynamics
    • 9.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
    • 9.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.4.6.1. Brazil
      • 9.4.6.2. Argentina
      • 9.4.6.3. Rest of South America
  • 9.5. Asia-Pacific
    • 9.5.1. Introduction
    • 9.5.2. Key Region-Specific Dynamics
    • 9.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
    • 9.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.5.6.1. China
      • 9.5.6.2. India
      • 9.5.6.3. Japan
      • 9.5.6.4. South Korea
      • 9.5.6.5. Rest of Asia-Pacific
  • 9.6. Middle East and Africa
    • 9.6.1. Introduction
    • 9.6.2. Key Region-Specific Dynamics
    • 9.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User

10. Competitive Landscape

  • 10.1. Competitive Scenario
  • 10.2. Market Positioning/Share Analysis
  • 10.3. Mergers and Acquisitions Analysis

11. Company Profiles

Key Market Players

  • 11.1. BioMarin*
    • 11.1.1. Company Overview
    • 11.1.2. Product Portfolio
      • 11.1.2.1. Product Description
      • 11.1.2.2. Product Key Performance Indicators (KPIs)
      • 11.1.2.3. Historic and Forecasted Product Sales
      • 11.1.2.4. Product Sales Volume
    • 11.1.3. Financial Overview
      • 11.1.3.1. Company Revenue's
      • 11.1.3.2. Geographical Revenue Shares
      • 11.1.3.3. Revenue Forecasts
    • 11.1.4. Key Developments
      • 11.1.4.1. Mergers & Acquisitions
      • 11.1.4.2. Key Product Development Activities
      • 11.1.4.3. Regulatory Approvals, etc.
    • 11.1.5. SWOT Analysis
  • 11.2. Sanofi
  • Similar data will be provided for each market player.

Emerging Market Players

  • 11.3. Orchard Therapeutics*
    • 11.3.1. Pipeline Products Description
    • 11.3.2. Product Key Performance Indicators (KPIs)
    • 11.3.3. Key Activities
    • 11.3.4. Market Entry Timelines
    • 11.3.5. Product Penetration Rate
    • 11.3.6. Sales Estimation and Projections
  • 11.4. JCR Pharmaceuticals
  • 11.5. Immusoft
  • 11.6. Regenxbio
  • 11.7. Paradigm Biopharmaceuticals Ltd
  • Similar data will be provided for each market player.

LIST NOT EXHAUSTIVE

12. Appendix

  • 12.1. About Us and Services
  • 12.2. Contact Us