溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗市场、机会、成长动力、产业趋势分析与预测，2024-2032 年

2023 年，全球溶小体酸性脂肪酶缺乏症(LAL-D) 治疗市场价值为2.974 亿美元，预计2024 年至2032 年复合年增长率为7.2%。认识和诊断的提高推动的以及基因检测和酶替代疗法（ERT）方面的进步。 《遗传代谢疾病杂誌》2022 年的一项研究报告称，接受 ERT 的患者在第一年内症状改善了 50%，强调了其疗效并创造了市场机会。

整个溶小体酸性脂肪酶缺乏症（LAL-D）治疗产业根据适应症、治疗类型和地区进行分类。

LAL-D 治疗市场依适应症分为沃尔曼病 (WD) 和胆固醇酯贮积症 (CESD)。 WD 在全球市场占据主导地位，预计在预测期内复合年增长率为 7.3%。 WD 的严重性和早发性推动了对有效治疗的需求，包括 ERT 和标靶治疗。此外，人们对早期诊断的认识不断提高，基因检测的进步也进一步推动了对 WD 治疗的需求。此外，正在进行的 WD 新疗法研究预计将有助于未来几年的市场成长。

按治疗类型划分，市场包括调脂药物、支持性治疗、酵素替代疗法 (ERT) 和其他治疗方法。 2023 年，ERT 占据最高市场份额，达到 57.5%。 ERT 在治疗严重肝臟和代谢併发症方面的有效性使其成为首选治疗方法。 Sebelipase alfa 是唯一被批准用于 LAL-D 的 ERT，可改善肝功能、减少代谢症状并预防疾病进展，从而增强其主导地位。此外，在新兴市场扩大 ERT 治疗范围以及提高患者对治疗方案的认识预计将促进 ERT 领域的成长。更有效的 ERT 治疗方法的开发预计也将提高患者的治疗效果和治疗依从性。

2023年，美国LAL-D治疗市场价值为1.001亿美元，预计到预测期结束时将达到1.788亿美元。美国因其先进的医疗基础设施和专注于罕见疾病研究而领先北美市场。政府措施和 FDA 批准 ERT 作为第一线治疗药物极大地促进了市场成长。领先製药公司和研究机构的存在进一步加速了有效治疗方法的开发和可用性。

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • 对个人化医疗的需求不断增加
    • 基因检测的进展
    • 提高早期诊断和治疗意识
    • 罕见疾病研究投资不断增加
  • 产业陷阱与挑战
    • 治疗费用高
    • 严格的监管要求
• 成长潜力分析
• 监管环境
• 管道分析
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司市占率分析
• 公司矩阵分析
• 主要市场参与者的竞争分析
• 竞争定位矩阵
• 战略仪表板

第 5 章：市场估计与预测：按指标划分，2021 - 2032 年

• 主要趋势
• 沃尔曼病 (WD)
• 胆固醇酯贮积症（CESD）

第 6 章：市场估计与预测：按治疗类型，2021 - 2032 年

• 主要趋势
• 酵素替代疗法（ERT）
• 支持性护理
• 脂质调节剂
• 其他治疗类型

第 7 章：市场估计与预测：按地区，2021 - 2032

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 欧洲其他地区
• 世界其他地区

第 8 章：公司简介

• Alexion Pharmaceuticals
• Amgen Inc.
• AstraZeneca
• BioCrick BioTech
• Lupin
• Merck and Co., Inc.
• Pfizer Inc.
• Regeneron Pharmaceuticals, Inc.
• Teva Pharmaceutical Industries Ltd.
• Thermo Fisher Scientific Inc.
• Zydus Group

The Global Lysosomal Acid Lipase Deficiency (LAL-D) Treatment Market was valued at USD 297.4 million in 2023 and is projected to grow at a CAGR of 7.2% from 2024 to 2032. This growth is driven by increased awareness and diagnosis of rare genetic disorders, as well as advancements in genetic testing and enzyme replacement therapy (ERT). A 2022 study in the Journal of Inherited Metabolic Disease reported a 50% improvement in symptoms for patients receiving ERT within the first year, highlighting its efficacy and creating market opportunities.

The overall lysosomal acid lipase deficiency (LAL-D) treatment industry is classified based on the indication, treatment type, and region.

The LAL-D treatment market is segmented by indication into Wolman disease (WD) and cholesteryl ester storage disease (CESD). WD dominated the global market and is expected to grow at a 7.3% CAGR over the forecast period. WD's severity and early onset drive demand for effective treatments, including ERTs and targeted therapies. Moreover, increasing awareness about early diagnosis and advancements in genetic testing are further fueling the demand for WD treatments. Additionally, ongoing research into novel therapies for WD is anticipated to contribute to market growth in the coming years.

By treatment type, the market includes lipid-modifying agents, supportive care, enzyme replacement therapy (ERT), and other treatments. In 2023, ERT held the highest market share at 57.5%. ERT's effectiveness in managing severe hepatic and metabolic complications makes it the preferred treatment. Sebelipase alfa, the only approved ERT for LAL-D, improves liver function, reduces metabolic symptoms, and prevents disease progression, reinforcing its dominance. Furthermore, expanding access to ERT in emerging markets and increasing patient awareness about treatment options are expected to boost the ERT segment's growth. The development of more efficient ERT delivery methods is also projected to enhance patient outcomes and treatment adherence.

In 2023, the U.S. LAL-D treatment market was valued at USD 100.1 million and is anticipated to reach USD 178.8 million by the end of the forecast period. The U.S. leads the North American market due to its advanced healthcare infrastructure and focus on rare disease research. Government initiatives and FDA approvals for ERTs as first-line treatments have significantly contributed to market growth. The presence of leading pharmaceutical companies and research institutions further accelerates the development and availability of effective treatments.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360° synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Increasing demand for personalized medicine
    • 3.2.1.2 Advancements in genetic testing
    • 3.2.1.3 Growing awareness for early diagnosis and treatment
    • 3.2.1.4 Rising investments in rare disease research
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 High cost of the treatment
    • 3.2.2.2 Stringent regulatory requirement
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Pipeline analysis
• 3.6 Porter's analysis
• 3.7 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company market share analysis
• 4.3 Company matrix analysis
• 4.4 Competitive analysis of major market players
• 4.5 Competitive positioning matrix
• 4.6 Strategy dashboard

Chapter 5 Market Estimates and Forecast, By Indication, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Wolman disease (WD)
• 5.3 Cholesteryl ester storage disease (CESD)

Chapter 6 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Enzyme replacement therapy (ERT)
• 6.3 Supportive care
• 6.4 Lipid modifying agents
• 6.5 Other treatment types

Chapter 7 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 North America
  • 7.2.1 U.S.
  • 7.2.2 Canada
• 7.3 Europe
  • 7.3.1 Germany
  • 7.3.2 UK
  • 7.3.3 France
  • 7.3.4 Spain
  • 7.3.5 Italy
  • 7.3.6 Rest of Europe
• 7.4 Rest of the World

Chapter 8 Company Profiles

• 8.1 Alexion Pharmaceuticals
• 8.2 Amgen Inc.
• 8.3 AstraZeneca
• 8.4 BioCrick BioTech
• 8.5 Lupin
• 8.6 Merck and Co., Inc.
• 8.7 Pfizer Inc.
• 8.8 Regeneron Pharmaceuticals, Inc.
• 8.9 Teva Pharmaceutical Industries Ltd.
• 8.10 Thermo Fisher Scientific Inc.
• 8.11 Zydus Group