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市場調查報告書
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1790497

美國溶小體儲積症治療市場規模、佔有率和趨勢分析報告：按類型、疾病類型、國家和細分市場預測，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個工作天內

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美國溶小體儲積症治療市場摘要

據估計，2024 年美國溶小體儲積症(LSD) 治療市值為 14.1 億美元，預計到 2033 年將達到 19.9 億美元，2025 年至 2033 年的複合年成長率為 4.10%。這一成長是由罕見遺傳患者的疾病上升、診斷能力的提高以及酵素替代療法和基因療法廣泛普及所驅動。 2025 年 5 月，FDA 接受了 REGENXBIO 的基因治療藥物 RGX-121（用於治療 MPS II）的生物製品許可申請 (BLA)。該藥物顯示腦脊髓液硫酸乙醯肝素降低 85%，顯示其有潛力成為長期 ERT 的替代品。人們認知的提高促成了早期療育並改善了治療效果。在 FDA 孤兒藥開發激勵措施的支持下，治療設計的發展和強大的產學合作繼續推動創新。

一個關鍵促進因素是基因療法作為治療 LSD 的創新方法的快速發展。與酵素替代療法不同，基因療法針對的是潛在的遺傳原因，可能產生長期甚至永久性的影響。幾種候選藥物正在美國進行 MLD、 聖菲利柏氏症和高雪氏症的臨床試驗。 2024 年 3 月，FDA核准了Remmerdi (acidasgene autotensel)，這是美國首個針對早期 MLD 的基因療法，結果顯示，接受治療的無症狀性患者在 6 歲時的生存率為 100%，而接受治療的無症狀性在 6 年時的生存率為 58%。這些突破正在推動增加投資、加速採用並重塑 LSD 的長期管理。

另一個主要促進因素是美國新生兒篩檢計畫的進展，這使得各種 LSD 能夠更早診斷出來。各州正在逐步擴大 RUSP 建議的檢測範圍，以納入龐貝氏症和 MPS i 等疾病。早期發現可以及時開始治療，顯著改善長期療效。例如，佛羅裡達州的新生兒篩檢於 2024 年 7 月將使用串聯式質譜質譜法的 MPS II 添加到其全州檢測範圍，從而實現更早的診斷和臨床轉診。隨著越來越多的州實施此類措施，預計確診病例將會增加。這一趨勢支持主動管理，並使早期治療性介入與行業策略保持一致。

目錄

第1章調查方法與範圍

第2章執行摘要

第3章美國溶小體儲積症（LSD）治療市場變數、趨勢與範圍

  • 市場體系展望
  • 市場動態
    • 市場促進因素分析
    • 市場限制因素分析
  • 商業環境分析
    • 產業分析-波特五力分析
    • PESTLE分析
    • 管道分析
    • 專利到期分析
    • 定價分析

第4章美國溶小體儲積症（LSD）治療市場：按類型分類的業務分析

  • 2024 年及 2033 年各類型市場佔有率
  • 類型細分儀表板
  • 按類型分類的市場規模、預測和趨勢分析（2021-2033 年）
  • 酵素替代療法（ERT）
  • 基材還原療法（SRT）
  • 其他類型

第5章美國溶小體儲積症（LSD）治療市場：依疾病類型分類的商業分析

  • 2024 年及 2033 年各疾病類型的市場佔有率
  • 疾病類型細分儀表板
  • 市場規模、預測與趨勢分析（按疾病類型，2021-2033 年）
  • 高雪氏症
  • 法布瑞氏症
  • 龐貝氏症
  • 黏多醣症（MPS）
  • 其他

第6章 競爭態勢

  • 參與者概覽
  • 企業市場分析
  • 公司分類
  • 戰略地圖
  • 公司簡介/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