溶小体疾病治疗市场－全球产业规模、份额、趋势、机会和预测，按疾病类型、疗法、给药途径、最终用户、地区和竞争格局划分，2020-2030年预测

2024年全球溶小体疾病治疗市场规模为81亿美元，预计2030年将以7.10%的复合年增长率成长至122.2亿美元。全球溶小体疾病治疗市场涵盖溶小体贮积症（LSDs）的治疗介入。溶小体贮积症是一种罕见的遗传性疾病，其特征是酵素缺乏导致底物在细胞溶酶体内累积。市场成长的驱动因素包括公众意识的提高、诊断技术的进步（从而能够更早识别患者）以及酶替代疗法、底物减少疗法和基因疗法等生物技术的进步。孤儿药认定和相关激励措施的推动，促进了研发领域的拓展，进一步刺激了对此特殊治疗领域的投资。

主要市场驱动因素

主要市场挑战

主要市场趋势

目录

第一章：产品概述

第二章：研究方法

第三章：执行概要

第四章：客户之声

第五章：全球溶小体疾病治疗市场展望

• 市场规模及预测
  • 按价值
• 市占率及预测
  • 依疾病类型分类（黏多醣贮积症、庞贝氏症候群、法布瑞氏症、戈谢氏症、其他）
  • 依治疗方法（底物减少疗法、干细胞疗法、酵素替代疗法、其他）
  • 依给药途径（口服、注射、其他）
  • 按最终使用者（医院、专科诊所、家庭护理、其他）
  • 按地区
  • 按公司（2024 年）
• 市场地图

第六章：北美溶小体疾病治疗市场展望

• 市场规模及预测
• 市占率及预测
• 北美洲：国家分析
  • 美国
  • 加拿大
  • 墨西哥

第七章：欧洲溶小体疾病治疗市场展望

• 市场规模及预测
• 市占率及预测
• 欧洲：国家分析
  • 德国
  • 法国
  • 英国
  • 义大利
  • 西班牙

第八章：亚太地区溶小体疾病治疗市场展望

• 市场规模及预测
• 市占率及预测
• 亚太地区：国家分析
  • 中国
  • 印度
  • 日本
  • 韩国
  • 澳洲

第九章：中东和非洲溶小体疾病治疗市场展望

• 市场规模及预测
• 市占率及预测
• 中东和非洲：国家分析
  • 沙乌地阿拉伯
  • 阿联酋
  • 南非

第十章：南美洲溶小体疾病治疗市场展望

• 市场规模及预测
• 市占率及预测
• 南美洲：国家分析
  • 巴西
  • 哥伦比亚
  • 阿根廷

第十一章：市场动态

• 司机
• 挑战

第十二章：市场趋势与发展

• 併购（如有）
• 产品发布（如有）
• 最新进展

第十三章：全球溶小体疾病治疗市场：SWOT分析

第十四章：波特五力分析

• 产业竞争
• 新进入者的潜力
• 供应商议价能力
• 顾客的力量
• 替代产品的威胁

第十五章：竞争格局

• Eli Lilly and Company
• Takeda Pharmaceutical Company Limited
• Sanofi SA
• Novartis AG
• Johnson & Johnson Services, Inc.
• Amicus Therapeutics, Inc.
• Merck & Co., Inc.

第十六章：策略建议

第17章调查会社について・免责事项

The Global Lysosomal Disease Treatment Market, valued at USD 8.10 Billion in 2024, is projected to experience a CAGR of 7.10% to reach USD 12.22 Billion by 2030. The Global Lysosomal Disease Treatment Market encompasses therapeutic interventions for lysosomal storage disorders (LSDs), rare genetic conditions where enzyme deficiencies cause substrate accumulation within cellular lysosomes. Market growth drivers include increasing awareness, improved diagnostics leading to earlier patient identification, and biotechnology advancements in enzyme replacement, substrate reduction, and gene therapies. An expanding research and development landscape, bolstered by orphan drug designations and incentives, further stimulates investment in this specialized therapeutic area.

Key Market Drivers

The robust pipeline and continuous development of novel therapeutic modalities significantly propel the global lysosomal disease treatment market. Innovations in enzyme replacement therapies, gene therapies, and substrate reduction therapies offer improved efficacy and address previously untreatable manifestations of these complex disorders. For example, the U. S. Food and Drug Administration approved Pombiliti(TM) and Opfolda(TM), a two-component therapy for adults with late-onset Pompe disease, representing a significant advancement in treatment options. According to Amicus Therapeutics, in September 2023, this approval marked the first and only two-component therapy for eligible adults living with the condition. Such advancements expand the treatable patient population and enhance therapeutic outcomes, fostering market growth through increased product adoption and sustained patient engagement.

Key Market Challenges

The high cost associated with specialized treatments for lysosomal diseases presents a significant impediment to market growth. These substantial expenses strain healthcare budgets globally, leading to restrictive reimbursement policies and protracted negotiation processes with payers. Such financial barriers directly limit patient access to approved therapies, even when innovative interventions are available. This directly decelerates market penetration and revenue generation for lysosomal disease treatments.

Key Market Trends

Personalized medicine integration in therapy development signifies a shift towards tailoring treatments to individual patient profiles, utilizing genetic and molecular biomarker information to optimize therapeutic outcomes. This approach moves beyond conventional generalized treatments, focusing on patient-specific needs for enhanced efficacy and reduced adverse effects, particularly crucial in complex rare diseases like lysosomal storage disorders. As per the Personalized Medicine Coalition, a nonprofit based in the US, the FDA sanctioned 16 innovative personalized treatments for rare disease sufferers in 2023, representing a substantial increase from six approvals in 2022.

Key Market Players

• Eli Lilly and Company
• Takeda Pharmaceutical Company Limited
• Sanofi SA
• Novartis AG
• Johnson & Johnson Services, Inc.
• Amicus Therapeutics, Inc.
• Merck & Co., Inc.

Report Scope:

In this report, the Global Lysosomal Disease Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Lysosomal Disease Treatment Market, By Disease Type:

• Mucopolysaccharidosis
• Pompes Syndrome
• Fabry Diseases
• Gaucher's Disease
• Other

Lysosomal Disease Treatment Market, By Therapy:

• Substrate Reduction Therapy
• Stem Cell Therapy
• Enzyme Replacement Therapy
• Other

Lysosomal Disease Treatment Market, By Route Of Administration:

• Oral
• Parenteral
• Other

Lysosomal Disease Treatment Market, By End User:

• Hospitals
• Specialty Clinics
• Homecare
• Other

Lysosomal Disease Treatment Market, By Region:

• North America
  • United States
  • Canada
  • Mexico
• Europe
  • France
  • United Kingdom
  • Italy
  • Germany
  • Spain
• Asia Pacific
  • China
  • India
  • Japan
  • Australia
  • South Korea
• South America
  • Brazil
  • Argentina
  • Colombia
• Middle East & Africa
  • South Africa
  • Saudi Arabia
  • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies presents in the Global Lysosomal Disease Treatment Market.

Available Customizations:

Global Lysosomal Disease Treatment Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

• Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

• 1.1. Market Definition
• 1.2. Scope of the Market
  • 1.2.1. Markets Covered
  • 1.2.2. Years Considered for Study
  • 1.2.3. Key Market Segmentations

2. Research Methodology

• 2.1. Objective of the Study
• 2.2. Baseline Methodology
• 2.3. Key Industry Partners
• 2.4. Major Association and Secondary Sources
• 2.5. Forecasting Methodology
• 2.6. Data Triangulation & Validation
• 2.7. Assumptions and Limitations

3. Executive Summary

• 3.1. Overview of the Market
• 3.2. Overview of Key Market Segmentations
• 3.3. Overview of Key Market Players
• 3.4. Overview of Key Regions/Countries
• 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global Lysosomal Disease Treatment Market Outlook

• 5.1. Market Size & Forecast
  • 5.1.1. By Value
• 5.2. Market Share & Forecast
  • 5.2.1. By Disease Type (Mucopolysaccharidosis, Pompes Syndrome, Fabry Diseases, Gaucher's Disease, Other)
  • 5.2.2. By Therapy (Substrate Reduction Therapy, Stem Cell Therapy, Enzyme Replacement Therapy, Other)
  • 5.2.3. By Route Of Administration (Oral, Parenteral, Other)
  • 5.2.4. By End User (Hospitals, Specialty Clinics, Homecare, Other)
  • 5.2.5. By Region
  • 5.2.6. By Company (2024)
• 5.3. Market Map

6. North America Lysosomal Disease Treatment Market Outlook

• 6.1. Market Size & Forecast
  • 6.1.1. By Value
• 6.2. Market Share & Forecast
  • 6.2.1. By Disease Type
  • 6.2.2. By Therapy
  • 6.2.3. By Route Of Administration
  • 6.2.4. By End User
  • 6.2.5. By Country
• 6.3. North America: Country Analysis
  • 6.3.1. United States Lysosomal Disease Treatment Market Outlook
    • 6.3.1.1. Market Size & Forecast
      • 6.3.1.1.1. By Value
    • 6.3.1.2. Market Share & Forecast
      • 6.3.1.2.1. By Disease Type
      • 6.3.1.2.2. By Therapy
      • 6.3.1.2.3. By Route Of Administration
      • 6.3.1.2.4. By End User
  • 6.3.2. Canada Lysosomal Disease Treatment Market Outlook
    • 6.3.2.1. Market Size & Forecast
      • 6.3.2.1.1. By Value
    • 6.3.2.2. Market Share & Forecast
      • 6.3.2.2.1. By Disease Type
      • 6.3.2.2.2. By Therapy
      • 6.3.2.2.3. By Route Of Administration
      • 6.3.2.2.4. By End User
  • 6.3.3. Mexico Lysosomal Disease Treatment Market Outlook
    • 6.3.3.1. Market Size & Forecast
      • 6.3.3.1.1. By Value
    • 6.3.3.2. Market Share & Forecast
      • 6.3.3.2.1. By Disease Type
      • 6.3.3.2.2. By Therapy
      • 6.3.3.2.3. By Route Of Administration
      • 6.3.3.2.4. By End User

7. Europe Lysosomal Disease Treatment Market Outlook

• 7.1. Market Size & Forecast
  • 7.1.1. By Value
• 7.2. Market Share & Forecast
  • 7.2.1. By Disease Type
  • 7.2.2. By Therapy
  • 7.2.3. By Route Of Administration
  • 7.2.4. By End User
  • 7.2.5. By Country
• 7.3. Europe: Country Analysis
  • 7.3.1. Germany Lysosomal Disease Treatment Market Outlook
    • 7.3.1.1. Market Size & Forecast
      • 7.3.1.1.1. By Value
    • 7.3.1.2. Market Share & Forecast
      • 7.3.1.2.1. By Disease Type
      • 7.3.1.2.2. By Therapy
      • 7.3.1.2.3. By Route Of Administration
      • 7.3.1.2.4. By End User
  • 7.3.2. France Lysosomal Disease Treatment Market Outlook
    • 7.3.2.1. Market Size & Forecast
      • 7.3.2.1.1. By Value
    • 7.3.2.2. Market Share & Forecast
      • 7.3.2.2.1. By Disease Type
      • 7.3.2.2.2. By Therapy
      • 7.3.2.2.3. By Route Of Administration
      • 7.3.2.2.4. By End User
  • 7.3.3. United Kingdom Lysosomal Disease Treatment Market Outlook
    • 7.3.3.1. Market Size & Forecast
      • 7.3.3.1.1. By Value
    • 7.3.3.2. Market Share & Forecast
      • 7.3.3.2.1. By Disease Type
      • 7.3.3.2.2. By Therapy
      • 7.3.3.2.3. By Route Of Administration
      • 7.3.3.2.4. By End User
  • 7.3.4. Italy Lysosomal Disease Treatment Market Outlook
    • 7.3.4.1. Market Size & Forecast
      • 7.3.4.1.1. By Value
    • 7.3.4.2. Market Share & Forecast
      • 7.3.4.2.1. By Disease Type
      • 7.3.4.2.2. By Therapy
      • 7.3.4.2.3. By Route Of Administration
      • 7.3.4.2.4. By End User
  • 7.3.5. Spain Lysosomal Disease Treatment Market Outlook
    • 7.3.5.1. Market Size & Forecast
      • 7.3.5.1.1. By Value
    • 7.3.5.2. Market Share & Forecast
      • 7.3.5.2.1. By Disease Type
      • 7.3.5.2.2. By Therapy
      • 7.3.5.2.3. By Route Of Administration
      • 7.3.5.2.4. By End User

8. Asia Pacific Lysosomal Disease Treatment Market Outlook

• 8.1. Market Size & Forecast
  • 8.1.1. By Value
• 8.2. Market Share & Forecast
  • 8.2.1. By Disease Type
  • 8.2.2. By Therapy
  • 8.2.3. By Route Of Administration
  • 8.2.4. By End User
  • 8.2.5. By Country
• 8.3. Asia Pacific: Country Analysis
  • 8.3.1. China Lysosomal Disease Treatment Market Outlook
    • 8.3.1.1. Market Size & Forecast
      • 8.3.1.1.1. By Value
    • 8.3.1.2. Market Share & Forecast
      • 8.3.1.2.1. By Disease Type
      • 8.3.1.2.2. By Therapy
      • 8.3.1.2.3. By Route Of Administration
      • 8.3.1.2.4. By End User
  • 8.3.2. India Lysosomal Disease Treatment Market Outlook
    • 8.3.2.1. Market Size & Forecast
      • 8.3.2.1.1. By Value
    • 8.3.2.2. Market Share & Forecast
      • 8.3.2.2.1. By Disease Type
      • 8.3.2.2.2. By Therapy
      • 8.3.2.2.3. By Route Of Administration
      • 8.3.2.2.4. By End User
  • 8.3.3. Japan Lysosomal Disease Treatment Market Outlook
    • 8.3.3.1. Market Size & Forecast
      • 8.3.3.1.1. By Value
    • 8.3.3.2. Market Share & Forecast
      • 8.3.3.2.1. By Disease Type
      • 8.3.3.2.2. By Therapy
      • 8.3.3.2.3. By Route Of Administration
      • 8.3.3.2.4. By End User
  • 8.3.4. South Korea Lysosomal Disease Treatment Market Outlook
    • 8.3.4.1. Market Size & Forecast
      • 8.3.4.1.1. By Value
    • 8.3.4.2. Market Share & Forecast
      • 8.3.4.2.1. By Disease Type
      • 8.3.4.2.2. By Therapy
      • 8.3.4.2.3. By Route Of Administration
      • 8.3.4.2.4. By End User
  • 8.3.5. Australia Lysosomal Disease Treatment Market Outlook
    • 8.3.5.1. Market Size & Forecast
      • 8.3.5.1.1. By Value
    • 8.3.5.2. Market Share & Forecast
      • 8.3.5.2.1. By Disease Type
      • 8.3.5.2.2. By Therapy
      • 8.3.5.2.3. By Route Of Administration
      • 8.3.5.2.4. By End User

9. Middle East & Africa Lysosomal Disease Treatment Market Outlook

• 9.1. Market Size & Forecast
  • 9.1.1. By Value
• 9.2. Market Share & Forecast
  • 9.2.1. By Disease Type
  • 9.2.2. By Therapy
  • 9.2.3. By Route Of Administration
  • 9.2.4. By End User
  • 9.2.5. By Country
• 9.3. Middle East & Africa: Country Analysis
  • 9.3.1. Saudi Arabia Lysosomal Disease Treatment Market Outlook
    • 9.3.1.1. Market Size & Forecast
      • 9.3.1.1.1. By Value
    • 9.3.1.2. Market Share & Forecast
      • 9.3.1.2.1. By Disease Type
      • 9.3.1.2.2. By Therapy
      • 9.3.1.2.3. By Route Of Administration
      • 9.3.1.2.4. By End User
  • 9.3.2. UAE Lysosomal Disease Treatment Market Outlook
    • 9.3.2.1. Market Size & Forecast
      • 9.3.2.1.1. By Value
    • 9.3.2.2. Market Share & Forecast
      • 9.3.2.2.1. By Disease Type
      • 9.3.2.2.2. By Therapy
      • 9.3.2.2.3. By Route Of Administration
      • 9.3.2.2.4. By End User
  • 9.3.3. South Africa Lysosomal Disease Treatment Market Outlook
    • 9.3.3.1. Market Size & Forecast
      • 9.3.3.1.1. By Value
    • 9.3.3.2. Market Share & Forecast
      • 9.3.3.2.1. By Disease Type
      • 9.3.3.2.2. By Therapy
      • 9.3.3.2.3. By Route Of Administration
      • 9.3.3.2.4. By End User

10. South America Lysosomal Disease Treatment Market Outlook

• 10.1. Market Size & Forecast
  • 10.1.1. By Value
• 10.2. Market Share & Forecast
  • 10.2.1. By Disease Type
  • 10.2.2. By Therapy
  • 10.2.3. By Route Of Administration
  • 10.2.4. By End User
  • 10.2.5. By Country
• 10.3. South America: Country Analysis
  • 10.3.1. Brazil Lysosomal Disease Treatment Market Outlook
    • 10.3.1.1. Market Size & Forecast
      • 10.3.1.1.1. By Value
    • 10.3.1.2. Market Share & Forecast
      • 10.3.1.2.1. By Disease Type
      • 10.3.1.2.2. By Therapy
      • 10.3.1.2.3. By Route Of Administration
      • 10.3.1.2.4. By End User
  • 10.3.2. Colombia Lysosomal Disease Treatment Market Outlook
    • 10.3.2.1. Market Size & Forecast
      • 10.3.2.1.1. By Value
    • 10.3.2.2. Market Share & Forecast
      • 10.3.2.2.1. By Disease Type
      • 10.3.2.2.2. By Therapy
      • 10.3.2.2.3. By Route Of Administration
      • 10.3.2.2.4. By End User
  • 10.3.3. Argentina Lysosomal Disease Treatment Market Outlook
    • 10.3.3.1. Market Size & Forecast
      • 10.3.3.1.1. By Value
    • 10.3.3.2. Market Share & Forecast
      • 10.3.3.2.1. By Disease Type
      • 10.3.3.2.2. By Therapy
      • 10.3.3.2.3. By Route Of Administration
      • 10.3.3.2.4. By End User

11. Market Dynamics

• 11.1. Drivers
• 11.2. Challenges

12. Market Trends & Developments

• 12.1. Merger & Acquisition (If Any)
• 12.2. Product Launches (If Any)
• 12.3. Recent Developments

13. Global Lysosomal Disease Treatment Market: SWOT Analysis

14. Porter's Five Forces Analysis

• 14.1. Competition in the Industry
• 14.2. Potential of New Entrants
• 14.3. Power of Suppliers
• 14.4. Power of Customers
• 14.5. Threat of Substitute Products

15. Competitive Landscape

• 15.1. Eli Lilly and Company
  • 15.1.1. Business Overview
  • 15.1.2. Products & Services
  • 15.1.3. Recent Developments
  • 15.1.4. Key Personnel
  • 15.1.5. SWOT Analysis
• 15.2. Takeda Pharmaceutical Company Limited
• 15.3. Sanofi SA
• 15.4. Novartis AG
• 15.5. Johnson & Johnson Services, Inc.
• 15.6. Amicus Therapeutics, Inc.
• 15.7. Merck & Co., Inc.

16. Strategic Recommendations

17. About Us & Disclaimer