한글목차

세계 헐러 증후군 치료 시장은 2024년에 3억 7,765만 달러에 달했습니다. 2033년에는 5억 2,934만 달러에 달할 것으로 예측되며, 예측 기간(2025-2033년) 동안 3.9%의 연평균 복합 성장률(CAGR)을 보일 것으로 예상됩니다.

헐러 증후군은 뮤코다당증(MPS i)으로도 알려져 있으며, 11개의 뮤코다당증(MPS) 질환 중 하나입니다. 이전에는 가거일 증후군이라고 불렸습니다. 유전적 결함으로 인한 리소좀 이상이 원인입니다. 글리코사미노글리칸(GAG 또는 점액 다당류)의 파괴 과정에 영향을 미치는 효소인 αL이두로니다제(αL-iduronidase)가 존재하지 않습니다.

시장 역학:

성장 촉진요인과 억제요인

효소대체요법(ERT) 적용 확대

효소대체요법(ERT)은 부족한 효소를 보충하여 질병의 진행을 늦추고 환자의 생존율과 삶의 질을 향상시키는 효소대체요법(ERT)의 채택이 증가함에 따라 세계 헐러 증후군 치료 시장이 급성장하고 있습니다. 효소 제제의 발전, 정부 지원, 희귀질환 의약품에 대한 혜택, 헬스케어에 대한 투자로 인해 ERT의 접근성이 향상되고 있습니다. 제약회사들이 효소 치료의 최적화에 집중함에 따라 시장이 확대되고 있습니다.

효소대체요법(ERT)은 체내 글리코사미노글리칸을 분해하여 헐러 증후군의 증상을 완화시킬 수 있습니다. 그러나 ERT는 혈뇌장벽을 통과하여 발달 중인 뇌에 도달할 수 없어 신경인지 기능 저하와 지적 장애를 유발합니다. 이는 ERT가 뇌 기능에 대처하지 못해 환자에게 신경인지 기능 저하와 지적 장애의 위험이 남아있기 때문입니다.

개발도상국에서의 치료 옵션의 제한된 가용성

저개발국이나 개발도상국에서는 의료 혜택이 부족한 경우가 많습니다. 이는 일반적으로 이들 지역에서는 헐러 증후군에 대한 시장이 제한적이라는 것을 의미합니다. 예를 들어, 효소대체요법(ERT)과 조혈모세포이식(HSCT)은 이 질환에 대한 가장 효과적인 치료법이지만, 이들 국가에서는 쉽게 이용할 수 없습니다. 그러나 대부분의 가난한 국가에서는 앞서 강조한 수요 패턴으로 인해 필요한 의료 인프라나 치료를 시행하고 신속하게 진단을 내릴 수 있는 전문가를 단계적으로 확보하지 못할 수 있습니다.

목차

제1장 분석 방법이및 분석 범위

제2장 정의와 개요

제3장 주요 요약

제4장 시장 역학

• 영향요인
  • 성장 촉진요인
  • 성장 억제요인
  • 기회
  • 영향 분석

제5장 전략적 인사이트와 업계 전망

• 시장 리더와 선구자
  • 새로운 선구자와 주요 기업
  • 최대 판매를 자랑하는 브랜드를 확립한 리더
  • 확립된 제품을 보유한 시장 리더
• CXO 시점
• 최신 개발과 Break through
• 사례 연구/진행 중인 연구
• 규제와 상환 상황
  • 북미
  • 유럽
  • 아시아태평양
  • 라틴아메리카
  • 중동 및 아프리카
• Porter의 Five Forces 분석
• 공급망 분석
• 특허 분석
• SWOT 분석
• 미충족 요구와 갭
• 시장 진입 및 확장을 위한 추천 전략
• 시나리오 분석 : 베스트 케이스/베이스 케이스/워스트 케이스 예측
• 가격 분석과 가격 시장 역학
• KOL(Key Opinion Leader)

제6장 치료 유형별

• 효소보충요법(ERT)
• 조혈모세포 이식(HSCT)
• 골수이식(BMT)
• 기타

제7장 연령층별

• 성인
• 소아

제8장 최종사용자별

• 병원
• 전문 클리닉
• 기타

제9장 지역별

• 북미
  • 미국
  • 캐나다
  • 멕시코
• 유럽
  • 독일
  • 영국
  • 프랑스
  • 스페인
  • 이탈리아
  • 기타 유럽
• 남미
  • 브라질
  • 아르헨티나
  • 기타 남미
• 아시아태평양
  • 중국
  • 인도
  • 일본
  • 한국
  • 기타 아시아태평양
• 중동 및 아프리카

제10장 경쟁 구도

• 경쟁 시나리오
• 시장 현황 및 점유율 분석
• 인수합병(M&A) 분석

제11장 기업 개요

• BioMarin
  • 기업 개요
  • 제품 포트폴리오
    • 제품 설명
    • 제품 핵심성과지표(KPI)
    • 제품 판매 실적과 예측
    • 제품 판매량
  • 재무 개요
    • 기업 매출
    • 매출 점유율 : 지역별
    • 매출 예측
  • 주요 발전
    • 인수합병(M&A)
    • 주요 제품 개발 활동
    • 규제기관 승인 등
  • SWOT 분석
• Sanofi
• Orchard Therapeutics
  • 파이프라인 제품 설명
  • 제품 핵심성과지표(KPI)
  • 주요 활동
  • 시장 참여 타임라인
  • 제품 보급률
  • 판매 예측과 예측
• JCR Pharmaceuticals
• Immusoft
• Regenxbio
• Paradigm Biopharmaceuticals Ltd
• 각각 기업에 대해서도 같은 데이터가 제공됩니다.

제12장 부록

영문목차

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