溶小體疾病治療市場－全球產業規模、佔有率、趨勢、機會和預測，按疾病類型、療法、給藥途徑、最終用戶、地區和競爭格局分類，2020-2030年預測

2024年全球溶小體疾病治療市場規模為81億美元，預計2030年將以7.10%的複合年成長率成長至122.2億美元。全球溶小體疾病治療市場涵蓋溶小體貯積症（LSDs）的治療介入。溶小體貯積症是一種罕見的遺傳性疾病，其特徵是酵素缺乏導致底物在細胞溶酶體內累積。市場成長的促進因素包括公眾意識的提高、診斷技術的進步（從而能夠更早識別患者）以及酶替代療法、底物減少療法和基因療法等生物技術的進步。孤兒藥認定和相關激勵措施的推動，促進了研發領域的拓展，進一步刺激了對此特殊治療領域的投資。

主要市場促進因素

主要市場挑戰

主要市場趨勢

目錄

第1章：產品概述

第2章：研究方法

第3章：執行概要

第4章：客戶之聲

第5章：全球溶小體疾病治療市場展望

• 市場規模及預測
  • 按價值
• 市佔率及預測
  • 依疾病類型分類（黏多醣貯積症、龐貝氏症候群、法布瑞氏症、戈謝氏症、其他）
  • 依治療方法（底物減少療法、幹細胞療法、酵素替代療法、其他）
  • 依給藥途徑（口服、注射、其他）
  • 按最終使用者（醫院、專科診所、家庭護理、其他）
  • 按地區
  • 按公司（2024 年）
• 市場地圖

第6章：北美溶小體疾病治療市場展望

• 市場規模及預測
• 市佔率及預測
• 北美洲：國家分析
  • 美國
  • 加拿大
  • 墨西哥

第7章：歐洲溶小體疾病治療市場展望

• 市場規模及預測
• 市佔率及預測
• 歐洲：國家分析
  • 德國
  • 法國
  • 英國
  • 義大利
  • 西班牙

第8章：亞太地區溶小體疾病治療市場展望

• 市場規模及預測
• 市佔率及預測
• 亞太地區：國家分析
  • 中國
  • 印度
  • 日本
  • 韓國
  • 澳洲

第9章：中東和非洲溶小體疾病治療市場展望

• 市場規模及預測
• 市佔率及預測
• 中東和非洲：國家分析
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國
  • 南非

第10章：南美洲溶小體疾病治療市場展望

• 市場規模及預測
• 市佔率及預測
• 南美洲：國家分析
  • 巴西
  • 哥倫比亞
  • 阿根廷

第11章：市場動態

• 促進要素
• 挑戰

第12章：市場趨勢與發展

• 併購（如有）
• 產品發布（如有）
• 最新進展

第13章：全球溶小體疾病治療市場：SWOT分析

第14章：波特五力分析

• 產業競爭
• 新進入者的潛力
• 供應商議價能力
• 顧客的力量
• 替代產品的威脅

第15章：競爭格局

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

第16章：策略建議

第17章調查會社について,免責事項

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