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시장보고서
상품코드
2076246
리소좀 축적 질환 치료제 시장 : 치료법별, 적응증별, 최종사용자별, 지역별Lysosomal Storage Diseases Therapeutics Market, By Treatment, By Indication, By End User, and By Region |
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리소좀 축적 질환 치료제 시장은 2026년에 126억 달러로 추정되며, 2033년에 224억 4,000만 달러에 달할 것으로 전망됩니다. 2026년부터 2033년까지 CAGR 8.6%로 성장할 것으로 예측됩니다.
| 보고서 범위 | 보고서 상세 | ||
|---|---|---|---|
| 기준 연도: | 2025년 | 2026년 시장 규모: | 126억 달러 |
| 과거 데이터 기간: | 2020-2024년 | 예측 기간: | 2026-2033년 |
| 2026-2033년까지 예측 기간의 CAGR: | 8.60% | 2033년 시장 규모 예측: | 224억 4,000만 달러 |
리소좀 축적증(LSD)은 단일 유전자의 결함으로 인해 발생하는 질환입니다. LSD의 약 70%는 효소 결함에 기인하며, 나머지는 효소 활성화 인자 또는 관련 단백질의 결함에 기인합니다. 특정 염색체 위치에 있는 유전자가 특정 효소를 암호화하고 있으며, 효소를 암호화하는 과정에서 이상이 발생하면 비활성 효소가 생성됩니다. 마찬가지로, 활성화 인자의 결함은 활성화 인자 유전자의 돌연변이에 기인합니다. 이러한 이상은 효소가 축적되는 장기에 질환을 유발하여 임상적 징후와 증상을 결정짓습니다. 영유아와 소아는 성인에 비해 더 심각한 증상을 겪는 경향이 있습니다. 같은 질환이라 하더라도, 소아와 성인의 경우 임상적 특징이 다릅니다. 예를 들어, 발달 단계에 있는 소아의 뇌는 장애의 영향을 받기 쉬워 기능 부전의 증상이나 징후가 나타나지만, 성인의 경우 이러한 증상이 경미하거나 아예 나타나지 않는 경우가 있습니다. 일반적으로 효소 검사가 첫 번째 진단 검사가 되지만, 유전자 변이에 대한 유전자 분석을 실시하면 진단의 정확도가 높아집니다. 이러한 질환에 대해서는 많은 최신 치료법이 존재합니다. 장기 손상이 진행되기 전 초기 단계에서 이러한 치료법을 적용하면, 손상을 예방하거나 지연시키고, 삶의 질을 향상시키며, 수명을 연장할 수 있을 것으로 기대됩니다.
시장 역학
리소좀 축적증에 대한 인식이 높아지고, 정부가 실시하는 계몽 프로그램, 신제품 출시 등이 전 세계 리소좀 축적 질환 치료제 시장의 성장에 기여하고 있습니다. 예를 들어, 2023년 5월 11일, 미국에 본사를 둔 제약회사 Chiesi USA, Inc.는 자사의 제품 'Elfabrio'가 미국 식품의약국(FDA)의 승인을 획득했다고 발표했습니다. 이 제품은 효소 대체 요법(ERT)에 페그니갈시다제 알파를 사용하여, 파브리병으로 진단받은 성인의 치료에 사용됩니다.
본 조사의 주요 특징:
- 본 조사에서는 다양한 부문의 잠재적 수익 기회를 파악하고, 이 시장에서 매력적인 투자 제안 매트릭스에 대해 설명하고 있습니다.
- 또한, 본 조사에서는 시장 촉진요인, 억제요인, 기회, 신제품 출시 및 승인, 시장 동향, 지역별 전망, 주요 기업들이 채택하는 경쟁 전략에 관한 중요한 인사이트도 제공하고 있습니다.
- 본 조사에서는 다음의 매개변수(기업 개요, 제품 포트폴리오, 주요 하이라이트, 재무 실적, 전략)를 바탕으로 전 세계 리소좀 축적 질환 치료제 시장의 주요 기업 개요을 작성하고 있습니다.
- 본 보고서를 통해 얻은 인사이트를 바탕으로, 기업의 마케팅 담당자 및 경영진은 향후 제품 출시, 제품 업그레이드, 시장 확대, 마케팅 전략에 관해 정보에 입각한 의사결정을 내릴 수 있게 됩니다.
- '세계 리소좀 축적 질환 치료제 시장 보고서'는 투자자, 공급업체, 제품 제조업체, 유통업체, 신규 진입 기업, 금융 애널리스트 등 이 산업의 다양한 이해관계자를 대상으로 합니다.
- 이해관계자 여러분은 전 세계 리소좀 축적 질환 치료제 시장 분석에 활용되는 다양한 전략 매트릭스를 활용함으로써 의사결정을 원활하게 진행할 수 있습니다.
목차
제1장 조사 목적과 가정
제2장 시장 전망
제3장 시장 역학·규제·동향 분석
제4장 세계의 리소좀 축적 질환 치료제 시장 : 치료법별, 2021-2033년
제5장 세계의 리소좀 축적 질환 치료제 시장 : 적응증별, 2021-2033년
제6장 세계의 리소좀 축적 질환 치료제 시장 : 최종사용자별, 2021-2033년
제7장 세계의 리소좀 축적 질환 치료제 시장 : 지역별, 2021-2033년
제8장 경쟁 구도
제9장 섹션
KSM 26.07.07Lysosomal Storage Diseases Therapeutics Market is estimated to be valued at USD 12.60 Bn in 2026 and is expected to reach USD 22.44 Bn by 2033, growing at a compound annual growth rate (CAGR) of 8.6% from 2026 to 2033.
| Report Coverage | Report Details | ||
|---|---|---|---|
| Base Year: | 2025 | Market Size in 2026: | USD 12.60 Bn |
| Historical Data for: | 2020 To 2024 | Forecast Period: | 2026 To 2033 |
| Forecast Period 2026 to 2033 CAGR: | 8.60% | 2033 Value Projection: | USD 22.44 Bn |
Lysosomal storage diseases (LSDs) are diseases caused by defects in single-genes. Enzyme defects cause nearly seventy percent of the LSDs, and the rest are defects in enzyme activator or associated proteins. A gene on a particular chromosome locus transcribes a particular enzyme, improper enzyme-coding results in inactive enzymes. Similarly, defective activators result from mutations in activator genes. These conditions cause disease of the organs in which they accumulate and decide the clinical signs and symptoms. Infants and children suffer more severely compared to adults. The clinical features are unique in many children and adults for the same disease. For example, the child's developing brain is more susceptible to insults and manifests symptoms and signs of dysfunction, while this may be milder or absent in adults. Enzyme testing is usually the initial diagnostic test, but genetic analysis of the gene mutations adds precision. There are many modern therapeutic techniques for these conditions. When applied early before organ damage sets in, these therapies have the potential to prevent or delay damage, improve quality of life, and increase lifespan.
Market Dynamics
Increasing significance of lysosomal storage diseases, arrangement of awareness programs by governments and the launch of new products are attributed to the growth of the global lysosomal storage diseases therapeutics market. For instance, on May 11, 2023, Chiesi USA, Inc., a U.S. based pharmaceutical company announced that its product Elfabrio was approved by U.S. Food and Drug Administration. It uses the enzyme pegunigalsidase alfa for enzyme replacement therapy (ERT) to treat adults with confirmed Fabry disease.
Key features of the study:
- It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
- This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
- It profiles key players in the global lysosomal storage diseases therapeutics market based on the following parameters-Company highlights, products portfolio, key highlights, financial performance, and strategies
- Key companies covered as a part of this study include Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.
- Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
- Global lysosomal storage diseases therapeutics market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
- Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global lysosomal storage diseases therapeutics market.
Detailed Segmentation:
- Global Lysosomal Storage Diseases Therapeutics Market, By Treatment (Revenue, USD Bn, 2021 - 2033)
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Substrate Reduction Therapy
- Others
- Global Lysosomal Storage Diseases Therapeutics Market, By Indication (Revenue, USD Bn, 2021 - 2033)
- Gaucher's Disease
- Fabry Disease
- Pompe's Syndrome
- Mucopolysaccharidosis
- Others
- Global Lysosomal Storage Diseases Therapeutics Market, By End User (Revenue, USD Bn, 2021 - 2033)
- Hospitals
- Clinics
- Global Lysosomal Storage Diseases Therapeutics Market, By Geography (Revenue, USD Bn, 2021 - 2033)
- North America
- U.S.
- Canada
- Europe
- U.K.
- Germany
- Italy
- France
- Spain
- Russia
- Rest of Europe
- Asia Pacific
- China
- India
- Japan
- ASEAN
- Australia
- South Korea
- Rest of Asia Pacific
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Middle East:
- GCC
- Israel
- Rest of Middle East
- Africa
- Central Africa
- South Africa
- North Africa
- Company Profiles
- Shire plc
- Pfizer, Inc.
- Sanofi SA
- BioMarin Pharmaceutical Inc
- Actelion Ltd.
- Raptor Pharmaceutical Corp.
- Protalix Biotherapeutics Inc.
- Amicus Therapeutics, Inc.
Table of Contents
1. Research Objectives and Assumptions
- Research Objectives
- Assumptions
- Abbreviations
2. Market Purview
- Report Description
- Market Definition and Scope
- Executive Summary
- Market Snapshot, By Treatment
- Market Snapshot, By Indication
- Market Snapshot, By End user
- Market Snapshot, By Region
- Coherent Opportunity Map (COM)
3. Market Dynamics, Regulations, and Trends Analysis
- Market Dynamics
- Drivers
- Restraints
- Market Opportunities
- Impact Analysis
- Recent Product Launches
- Epidemiology
- Merger, Acquisition, and Collaborations
- Regulatory Scenario
- Key Developments
- PEST Analysis
4. Global Lysosomal Storage Diseases Therapeutics Market, By Treatment, 2021-2033, (US$ Billion)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2022 -2033
- Segment Trends
- Enzyme Replacement Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Stem Cell Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Substrate Reduction Therapy
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
5. Global Lysosomal Storage Diseases Therapeutics Market, By Indication, 2021-2033, (US$ Billion)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2022 -2033
- Segment Trends
- Gaucher's Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Fabry Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Pompe's Disease
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Mucopolysaccharidosis
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
6. Global Lysosomal Storage Diseases Therapeutics Market, By End user, 2021-2033, (US$ Billion)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2022 -2033
- Segment Trends
- Hospitals
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
- Clinics
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2021 - 2033, (US$ Bn)
7. Global Lysosomal Storage Diseases Therapeutics Market, By Region, 2021-2033, (US$ Billion)
- Introduction
- Market Share Analysis, By Region, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, For Regions, 2022 -2033
- Regional Trends
- North America
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 - 2033, (US$ Billion)
- U.S.
- Canada
- Latin America
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021- 2033, (US$ Billion)
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Europe
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021- 2033, (US$ Billion)
- U.K.
- Germany
- Italy
- France
- Spain
- Russia
- Rest of Europe
- Asia Pacific
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication , 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021- 2033, (US$ Billion)
- China
- India
- Japan
- ASEAN
- Australia
- South Korea
- Rest of Asia Pacific
- Middle East
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021- 2033, (US$ Billion)
- GCC
- Israel
- Rest of Middle East
- Africa
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Indication, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By End User, 2021 - 2033, (US$ Billion)
- Market Size and Forecast, and Y-o-Y Growth, By Country, 2021- 2033, (US$ Billion)
- North Africa
- Central Africa
- South Africa
8. Competitive Landscape
- Shire plc
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Pfizer, Inc.
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Sanofi SA
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- BioMarin Pharmaceutical Inc
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Actelion Ltd.
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Raptor Pharmaceutical Corp
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Protalix Biotherapeutics Inc.
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
- Amicus Therapeutics, Inc.
- Company Highlights
- Product Portfolio
- Key Highlights
- Financial Performance
- Market Strategies
9. Section
- Research Methodology
- About Us
