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市场调查报告书
商品编码
1790497

美国溶小体储积症治疗市场规模、份额和趋势分析报告：按类型、疾病类型、国家和细分市场预测，2025 年至 2033 年

U.S. Lysosomal Storage Disease Treatment Market Size, Share & Trends Analysis Report By Type (Enzyme Replacement Therapy (ERT)), By Disease Type, By Country, And Segment Forecasts, 2025 - 2033
出版日期: | 出版商: Grand View Research | 英文 150 Pages | 商品交期: 2-10个工作天内

价格

美国溶小体储积症治疗市场摘要

据估计，2024 年美国溶小体储积症(LSD) 治疗市值为 14.1 亿美元，预计到 2033 年将达到 19.9 亿美元，2025 年至 2033 年的复合年增长率为 4.10%。这一增长是由罕见遗传患者的疾病上升、诊断能力的提高以及酵素替代疗法和基因疗法广泛普及所驱动。 2025 年 5 月，FDA 接受了 REGENXBIO 的基因治疗药物 RGX-121（用于治疗 MPS II）的生物製品许可申请 (BLA)。该药物显示脑脊髓液硫酸乙酰肝素降低 85%，显示其有潜力成为长期 ERT 的替代品。人们认识的提高促成了早期疗育并改善了治疗效果。在 FDA 孤儿药开发激励措施的支持下，治疗设计的发展和强大的产学合作继续推动创新。

一个关键驱动因素是基因疗法作为治疗 LSD 的创新方法的快速发展。与酵素替代疗法不同，基因疗法针对的是潜在的遗传原因，可能产生长期甚至永久性的影响。几种候选药物正在美国进行 MLD、 圣菲利柏氏症和高雪氏症的临床试验。 2024 年 3 月，FDA核准了Remmerdi (acidasgene autotensel)，这是美国首个针对早期 MLD 的基因疗法，结果显示，接受治疗的无症状性患者在 6 岁时的生存率为 100%，而接受治疗的无症状性在 6 年时的生存率为 58%。这些突破正在推动增加投资、加速采用并重塑 LSD 的长期管理。

另一个主要驱动因素是美国新生儿筛检计画的进展，这使得各种 LSD 能够更早诊断出来。各州正在逐步扩大 RUSP 建议的检测范围，以纳入庞贝氏症和 MPS i 等疾病。早期发现可以及时开始治疗，显着改善长期疗效。例如，佛罗里达州的新生儿筛检于 2024 年 7 月将使用串联式质谱质谱法的 MPS II 添加到其全州检测范围，从而实现更早的诊断和临床转诊。随着越来越多的州实施此类措施，预计确诊病例将会增加。这一趋势支持主动管理，并使早期治疗性介入与行业策略保持一致。

目录

第一章调查方法与范围

第二章执行摘要

第三章美国溶小体储积症（LSD）治疗市场变数、趋势与范围

  • 市场体系展望
  • 市场动态
    • 市场驱动因素分析
    • 市场限制因素分析
  • 商业环境分析
    • 产业分析-波特五力分析
    • PESTLE分析
    • 管道分析
    • 专利到期分析
    • 定价分析

第四章美国溶小体储积症（LSD）治疗市场：按类型分類的业务分析

  • 2024 年及 2033 年各类型市场占有率
  • 类型细分仪表板
  • 按类型分類的市场规模、预测和趋势分析（2021-2033 年）
  • 酵素替代疗法（ERT）
  • 基材还原疗法（SRT）
  • 其他类型

第五章美国溶小体储积症（LSD）治疗市场：依疾病类型分類的商业分析

  • 2024 年及 2033 年各疾病类型的市场占有率
  • 疾病类型细分仪表板
  • 市场规模、预测与趋势分析（按疾病类型，2021-2033 年）
  • 高雪氏症
  • 法布瑞氏症
  • 庞贝氏症
  • 黏多醣症（MPS）
  • 其他的

第六章 竞争态势

  • 参与者概览
  • 企业市场分析
  • 公司分类
  • 战略地图
  • 公司简介/上市公司
    • Pfizer Inc
    • Takeda Pharmaceutical Company Limited（Shire Plc）
    • Sanofi（Genzyme Corporation）
    • BioMarin
    • Johnson &Johnson（Actelion Pharmaceuticals Ltd）
    • Amicus Therapeutics, Inc
    • Alexion Pharmaceuticals, Inc
    • Sigilon Therapeutics, Inc
    • Orphazyme A/S
Product Code: GVR-4-68040-668-5

U.S. Lysosomal Storage Disease Treatment Market Summary

The U.S. lysosomal storage disease treatment market size was estimated at USD 1.41 billion in 2024 and is projected to reach USD 1.99 billion by 2033, growing at a CAGR of 4.10% from 2025 to 2033. The rising prevalence of rare genetic disorders, better diagnostic capabilities, and broader access to enzyme replacement and gene-based therapies drive this growth. In May 2025, the FDA accepted REGENXBIO's BLA for RGX-121, a gene therapy for MPS II, showing 85% CSF heparan sulfate reduction and potential to replace long-term ERT. Increased awareness has led to earlier intervention and improved outcomes. Advancements in therapy design and strong industry-academic collaborations continue to drive innovation, supported by FDA incentives for orphan drug development.

One of the key drivers is the rapid evolution of gene therapy as a transformative approach for treating LSDs. Unlike enzyme replacement therapies, gene therapies target the root genetic cause, offering potential long-term or permanent benefits. In the U.S., several candidates for conditions such as MLD, Sanfilippo syndrome, and Gaucher disease are advancing through clinical stages. In March 2024, the FDA approved Lenmeldy (atidarsagene autotemcel), the first gene therapy in the U.S. for early-stage MLD, showing 100% survival at age 6 in treated presymptomatic patients versus 58% in untreated cases. Such milestones are increasing investment, accelerating adoption, and reshaping long-term LSD management.

Another major driver is the advancement of newborn screening programs across the U.S., which has led to earlier diagnosis of various LSDs. States are progressively expanding their recommended panels under the RUSP to include conditions such as Pompe disease and MPS I. Early detection enables the timely initiation of treatment, significantly improving long-term disease outcomes. For instance, in July 2024, Florida Newborn Screening added MPS II to its statewide panel using tandem mass spectrometry, enabling early diagnosis and clinical referral. As more states implement such measures, diagnosed cases are expected to rise. This trend supports proactive management and aligns industry strategies with early therapeutic intervention.

U.S. Lysosomal Storage Disease (LSDs) Treatment Market Report Segmentation

This report forecasts revenue growth at regional levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Grand View Research has segmented the U.S. lysosomal storage disease treatment market report based on type and disease type:

  • Type Outlook (Revenue, USD Billion, 2021 - 2033)
  • Enzyme Replacement Therapy (ERT)
  • Imiglucerase (Cerezyme)
  • Alglucosidase alfa (Myozyme/Lumizyme)
  • Idursulfase (Elaprase)
  • Velaglucerase alfa
  • Others
  • Substrate Reduction Therapy (SRT)
  • Other Types
  • Disease Type Outlook (Revenue, USD Billion, 2021 - 2033)
  • Gaucher Disease
  • Fabry Disease
  • Pompe Disease
  • Mucopolysaccharidoses (MPS)
  • Others

Table of Contents

Chapter 1. Methodology and Scope

  • 1.1. Market Segmentation & Scope
  • 1.2. Segment Definitions
    • 1.2.1. Type
    • 1.2.2. Disease Type
  • 1.3. Estimates and Forecast Timeline
  • 1.4. Research Methodology
  • 1.5. Information Procurement
    • 1.5.1. Purchased Database
    • 1.5.2. GVR's Internal Database
    • 1.5.3. Secondary Sources
    • 1.5.4. Primary Research
  • 1.6. Information Analysis
    • 1.6.1. Data Analysis Models
  • 1.7. Market Formulation & Data Visualization
  • 1.8. Model Details
    • 1.8.1. Commodity Flow Analysis
  • 1.9. List of Secondary Sources
  • 1.10. Objectives

Chapter 2. Executive Summary

  • 2.1. Market Snapshot
  • 2.2. Segment Snapshot
  • 2.3. Competitive Landscape Snapshot

Chapter 3. U.S. Lysosomal Storage Disease (LSDs) Treatment Market Variables, Trends, & Scope

  • 3.1. Market Lineage Outlook
  • 3.2. Market Dynamics
    • 3.2.1. Market Driver Analysis
    • 3.2.2. Market Restraint Analysis
  • 3.3. Business Environment Analysis
    • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
      • 3.3.1.1. Supplier Power
      • 3.3.1.2. Buyer Power
      • 3.3.1.3. Substitution Threat
      • 3.3.1.4. Threat of New Entrants
      • 3.3.1.5. Competitive Rivalry
    • 3.3.2. PESTLE Analysis
    • 3.3.3. Pipeline Analysis
    • 3.3.4. Patent Expiry Analysis
    • 3.3.5. Pricing Analysis

Chapter 4. U.S. Lysosomal Storage Disease (LSDs) Treatment Market: Type Business Analysis

  • 4.1. Type Market Share, 2024 & 2033
  • 4.2. Type Segment Dashboard
  • 4.3. Market Size & Forecasts and Trend Analysis, by Type, 2021 to 2033 (USD Billion)
  • 4.4. Enzyme Replacement Therapy (ERT)
    • 4.4.1. Enzyme Replacement Therapy (ERT) Market, 2021 - 2033 (USD Billion)
    • 4.4.2. Imiglucerase (Cerezyme)
      • 4.4.2.1. Imiglucerase (Cerezyme) Market, 2021 - 2033 (USD Billion)
    • 4.4.3. Alglucosidase alfa (Myozyme/Lumizyme)
      • 4.4.3.1. Alglucosidase alfa (Myozyme/Lumizyme) Market, 2021 - 2033 (USD Billion)
    • 4.4.4. Idursulfase (Elaprase)
      • 4.4.4.1. Idursulfase (Elaprase) Market, 2021 - 2033 (USD Billion)
    • 4.4.5. Velaglucerase alfa
      • 4.4.5.1. Velaglucerase alfa Market, 2021 - 2033 (USD Billion)
    • 4.4.6. Others
      • 4.4.6.1. Others Market, 2021 - 2033 (USD Billion)
  • 4.5. Substrate Reduction Therapy (SRT)
    • 4.5.1. Substrate Reduction Therapy (SRT) Market, 2021 - 2033 (USD Billion)
  • 4.6. Other Types
    • 4.6.1. Other Types Market, 2021 - 2033 (USD Billion)

Chapter 5. U.S. Lysosomal Storage Disease (LSDs) Treatment Market: Disease Type Business Analysis

  • 5.1. Disease Type Market Share, 2024 & 2033
  • 5.2. Disease Type Segment Dashboard
  • 5.3. Market Size & Forecasts and Trend Analysis, by Disease Type, 2021 to 2033 (USD Billion)
  • 5.4. Gaucher Disease
    • 5.4.1. Gaucher Disease Market, 2021 - 2033 (USD Billion)
  • 5.5. Fabry Disease
    • 5.5.1. Fabry Disease Market, 2021 - 2033 (USD Billion)
  • 5.6. Pompe Disease
    • 5.6.1. Pompe Disease Market, 2021 - 2033 (USD Billion)
  • 5.7. Mucopolysaccharidoses (MPS)
    • 5.7.1. Mucopolysaccharidoses (MPS) Market, 2021 - 2033 (USD Billion)
  • 5.8. Others
    • 5.8.1. Others Market, 2021 - 2033 (USD Billion)

Chapter 6. Competitive Landscape

  • 6.1. Participant Overview
  • 6.2. Company Market Position Analysis
  • 6.3. Company Categorization
  • 6.4. Strategy Mapping
  • 6.5. Company Profiles/Listing
    • 6.5.1. Pfizer Inc
      • 6.5.1.1. Overview
      • 6.5.1.2. Financial Performance
      • 6.5.1.3. Type Benchmarking
      • 6.5.1.4. Strategic Initiatives
    • 6.5.2. Takeda Pharmaceutical Company Limited (Shire Plc)
      • 6.5.2.1. Overview
      • 6.5.2.2. Financial Performance
      • 6.5.2.3. Type Benchmarking
      • 6.5.2.4. Strategic Initiatives
    • 6.5.3. Sanofi (Genzyme Corporation)
      • 6.5.3.1. Overview
      • 6.5.3.2. Financial Performance
      • 6.5.3.3. Type Benchmarking
      • 6.5.3.4. Strategic Initiatives
    • 6.5.4. BioMarin
      • 6.5.4.1. Overview
      • 6.5.4.2. Financial Performance
      • 6.5.4.3. Type Benchmarking
      • 6.5.4.4. Strategic Initiatives
    • 6.5.5. Johnson & Johnson (Actelion Pharmaceuticals Ltd)
      • 6.5.5.1. Overview
      • 6.5.5.2. Financial Performance
      • 6.5.5.3. Type Benchmarking
      • 6.5.5.4. Strategic Initiatives
    • 6.5.6. Amicus Therapeutics, Inc
      • 6.5.6.1. Overview
      • 6.5.6.2. Financial Performance
      • 6.5.6.3. Type Benchmarking
      • 6.5.6.4. Strategic Initiatives
    • 6.5.7. Alexion Pharmaceuticals, Inc
      • 6.5.7.1. Overview
      • 6.5.7.2. Financial Performance
      • 6.5.7.3. Type Benchmarking
      • 6.5.7.4. Strategic Initiatives
    • 6.5.8. Sigilon Therapeutics, Inc
      • 6.5.8.1. Overview
      • 6.5.8.2. Financial Performance
      • 6.5.8.3. Type Benchmarking
      • 6.5.8.4. Strategic Initiatives
    • 6.5.9. Orphazyme A/S
      • 6.5.9.1. Overview
      • 6.5.9.2. Financial Performance
      • 6.5.9.3. Type Benchmarking
      • 6.5.9.4. Strategic Initiatives

List of Tables

  • Table 1 List of secondary sources
  • Table 2 List of abbreviations
  • Table 3 U.S. lysosomal storage disease (LSDs) treatment market, by type, 2021 - 2033 (USD Billion)
  • Table 4 U.S. lysosomal storage disease (LSDs) treatment market, by disease type, 2021 - 2033 (USD Billion)

List of Figures

  • Fig. 1 U.S. lysosomal storage disease (LSDs) treatment market segmentation
  • Fig. 2 Market research process
  • Fig. 3 Data triangulation techniques
  • Fig. 4 Primary research pattern
  • Fig. 5 Market research approaches
  • Fig. 6 Value-chain-based sizing & forecasting
  • Fig. 7 Market formulation & validation
  • Fig. 8 Market snapshot
  • Fig. 9 Competitive landscape
  • Fig. 10 U.S. lysosomal storage disease (LSDs) treatment market dynamics
  • Fig. 11 U.S. lysosomal storage disease (LSDs) treatment market: Porter's five forces analysis
  • Fig. 12 U.S. lysosomal storage disease (LSDs) treatment market: PESTLE analysis
  • Fig. 13 Type market, 2021 - 2033 (USD Billion)
  • Fig. 14 Enzyme replacement therapy market, 2021 - 2033 (USD Billion)
  • Fig. 15 Imiglucerase (Cerezyme) market, 2021 - 2033 (USD Billion)
  • Fig. 16 Alglucosidase alfa (Myozyme/Lumizyme) market, 2021 - 2033 (USD Billion)
  • Fig. 17 Idursulfase (Elaprase) market, 2021 - 2033 (USD Billion)
  • Fig. 18 Velaglucerase alfa market, 2021 - 2033 (USD Billion)
  • Fig. 19 Others market, 2021 - 2033 (USD Billion)
  • Fig. 20 Substrate reduction therapy market, 2021 - 2033 (USD Billion)
  • Fig. 21 Other types market, 2021 - 2033 (USD Billion)
  • Fig. 22 Disease type market, 2021 - 2033 (USD Billion)
  • Fig. 23 Gaucher disease market, 2021 - 2033 (USD Billion)
  • Fig. 24 Fabry disease market, 2021 - 2033 (USD Billion)
  • Fig. 25 Pompe disease market, 2021 - 2033 (USD Billion)
  • Fig. 26 Mucopolysaccharidoses market, 2021 - 2033 (USD Billion)
  • Fig. 27 Others market, 2021 - 2033 (USD Billion)
  • Fig. 28 Company categorization
  • Fig. 29 Company market position analysis
  • Fig. 30 Strategic framework