溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗市场:现状分析与未来预测 (2024年～2032年)

溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗市场预计在预测期内 (2024-2032) 以 11.4% 的复合年增长率大幅增长。推动 LAL-D 治疗市场的关键成长因素包括基因疗法的持续进步（其在治疗该疾病方面具有关键作用）以及可用于治疗该疾病症状的酵素替代疗法。公众意识的提高、诊断能力的提高、国家和国际对罕见疾病药物开发的支持以及 LAL-D 发病率的上升也促进了市场的成长。

根据适应症，市场分为两个部分：沃尔曼病和胆固醇酯沉积病。沃尔曼病（WD）因其发病早、病情严重且需要立即治疗而占据当前市场主导地位。由于该疾病相对罕见，且若不及时治疗可能会产生严重后果，因此引起了医疗和製药行业利益相关者的关注。然而，市场成长最快的部分是胆固醇酯沉积病（CESD）。 CESD 也是一种罕见疾病，但其发病从儿童到成年是逐渐的，因此更加多样化。因此患者生命週期长、数量高。此外，人们认识的提高、鑑别能力的提高以及 sebelipase alfa 等用于 WD 和 CESD 的治疗方法的批准都是 CESD 领域预期增长的原因。此外，治疗药物的增加和 CESD 患者存活率的上升也是推动预期市场成长的因素。

根据治疗方法，市场分为酵素替代疗法 (ERT)、支持性护理、脂质调节药物等。其中，ERT 在目前的 LAL-D 治疗市场中占据主导地位，这主要是因为直接酵素替代疗法对沃尔曼病和胆固醇酯累积病 (CESD) 均具有明确的疗效。由于可以使用 sebelipase alfa 来取代缺失的酶，ERT 仍然是管理 LAL-D 和改善患者预后的基石。然而，由于使用调脂药物和 ERT 同时治疗 LAL-D 中的脂质累积问题，预计未来几年调脂药物领域将以最高的复合年增长率成长。这些药物可能对控制 CESD 患者的胆固醇和血脂水平特别有用，并且越来越多地被视为此目的联合疗法的组成部分。脂质调节药物引入治疗管理方案可望解放市场领域，同时提高意识并改变治疗方案。

根据分销管道，市场分为医院药房、零售药房、网路药房和其他。在这些细分市场中，医院药房占据最大的市场占有率，因为这些机构为医疗机构提供有价值的服务。医院药局提供并调配Kanuma等必要药品，这些药品对于LAL-D患者的治疗至关重要。然而，由于消费者对便利性和移动性的偏好，线上药局市场的价值预计未来几年将大幅成长。

为了更了解溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗市场的市场渗透率，我们根据北美（美国、加拿大和北美其他地区）、欧洲（德国、法国、英国、西班牙、义大利和欧洲其他地区）、亚太地区（中国、日本、印度和亚太地区其他地区）和世界其他地区的全球影响力对其进行了分析。由于 LAL-D 的普及率不断提高，预计预测期内（2024-2032 年）亚太地区将以显着的复合年增长率成长。溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗市场正专注于协作开发活动，以改善产品开发并带来新药。

例如，2024 年 10 月，阿斯特捷利康与石药集团有限公司 (CSPC) 签订了独家许可协议，以推进新型早期小脂蛋白 (a) (Lp(a)) 干扰剂的开发，这种干扰剂可能为患有血脂异常（一种严重的 LAL-D）的患者带来其他益处。

市场的主要参与者包括 Alexion Pharmaceuticals (AstraZeneca)、Amicus Therapeutics, Inc.、Pfizer Inc.、Lupin Pharmaceutical, Inc.、Sun Pharmaceutical Industries Limited、Zydus Group、Teva Pharmaceuticals USA, Inc.、Glenmark Pharmaceuticals Inc.、Therincmot

目录

第1章 市场概要

• 市场定义
• 主要目的
• 相关利益者
• 限制事项

第2章 分析方法或前提条件

• 分析流程
• 分析方法
• 受访者简介

第3章 摘要整理

• 产业摘要
• 各市场区隔预测
  • 市场成长的强度
• 地区展望

第4章 市场动态

• 促进因素
• 机会
• 阻碍因素
• 趋势
• PESTEL分析
• 需求面分析
• 供给面分析
  • 企业合併·收购 (M&A)
  • 投资情境

第5章 全球溶小体酸性脂肪酶缺乏症 (LAL-D) 治疗的市场收益 (2022～2032年)

第6章 市场分析:各适应症

• 沃尔曼病
• 胆固醇酯沉积病

第7章 市场分析:各治疗法

• 酵素补充疗法 (ERT)
• 支持疗法
• 脂质改质剂
• 其他

第8章 市场分析:各流通管道

• 医院药局
• 零售药局
• 线上
• 其他

第9章 市场分析:各地区

• 北美
  • 美国
  • 加拿大
  • 其他北美地区
• 欧洲
  • 德国
  • 英国
  • 法国
  • 义大利
  • 西班牙
  • 其他欧洲地区
• 亚太地区
  • 中国
  • 日本
  • 印度
  • 其他亚太地区
• 全球其他地区

第10章 价值链分析

• 限制分析
• 市场参与企业一览

第11章 竞争情形

• 竞争仪表板
• 企业的市场定位分析
• 波特的五力分析

第12章 企业简介

• Alexion Pharmaceuticals (AstraZeneca)
  • 企业概要
  • 主要的财务指标
  • SWOT分析
  • 产品系列
  • 近几年趋势
• Amicus Therapeutics, Inc.
• Pfizer Inc.
• Lupin Pharmaceutical, Inc.
• Sun Pharmaceutical Industries Limited
• Zydus Group
• Teva Pharmaceuticals USA, Inc.
• Glenmark Pharmaceuticals Inc
• Thermo Fisher Scientific inc.
• Merck & Co., Inc.

第13章 缩写与前提条件

第14章 附录

Lysosomal Acid Lipase Deficiency (LAL-D) is a genetic disorder that results from mutations involving the LIPA gene, which codes for the LAL enzyme. The diagnosis is made using genetic tests, enzyme activity tests, and Imaging. LAL-D is a very rare autosomal recessive disease resulting from the deficiency of the LAL enzyme. LAL is essential for the breakdown of lipids that bring into the cell's lysosomes, such as cholesterol esters and triglycerides. In the absence of or low levels of LAL, these lipids accumulate in many organs to cause serious organ injury and dysfunction such as in the liver, spleen, and intestines. LAL-D manifests in two forms: The first is Wolman disease which is a severe, infantile form of the disease and the second one is Cholesteryl Ester Storage Disease (CESD), which is a mild or late-onset form of the disease. Wolman disease is usually manifest by the first few months of life, clinical signs include hepatomegaly, gastric dilation, growth failure, and jaundice. It is a very aggressive disease and if not treated, the disease is fatal. CESD, however, may be asymptomatic until childhood or adulthood, and the patients present with liver disorders, fatty deposition, and heart disease. The main management includes enzyme replacement therapy ERT involving sebelipase alfa in a bid to manage the deficient enzyme.

The Lysosomal Acid Lipase Deficiency (LAL-D) Treatment Market is expected to grow with a significant CAGR of 11.4% during the forecast period (2024-2032). Some of the main growth factors that drive the LAL-D treatment market include the continuous progression in gene therapy that holds a vital function in the treatment of the disease and the availability of enzyme replacement therapy that treats the symptoms of the disorder. Also, awareness among people, enhanced diagnostic capabilities, state and international support in the development of orphan drugs, and a higher incidence of LAL-D contribute to the growth of the market.

Depending on the Indication the market is divided into two segments namely the Wolman Disease and Cholesteryl Ester Storage Disease. Wolman Disease (WD) dominates the current market due to the increased severity and early onset of the symptoms that call for immediate treatment. This is a relatively rare disease that has severe consequences, if not treated quickly, hence it has attracted attention from stakeholders in the medical field and the pharmaceutical industries. However, the fastest-growing segment of the market is Cholesteryl Ester Storage Disease (CESD). Though CESD is also rare, its subject population is more diverse in nature due to the gradual onset of the disease which can be diagnosed in childhood, or in adulthood. This leads to increased volume and longevity of the patient's life cycles. Furthermore, raising consciousness, enhancement in identification, and authorization to use treatments such as sebelipase alfa for both WD and CESD are the reasons behind the anticipated growth of the CESD segment. The increasing number of therapeutic products and higher survival rates of patients with CESD also provide evidence for the expected market growth.

Based on the Treatment, the market is segmented into Enzyme Replacement Therapy (ERT), Supportive Care, Lipid Modifying Agents, and Others. Out of these, ERT is most dominant in the current LAL-D treatment market largely due to the established efficacy of direct enzyme replacement in both Wolman Disease and Cholesteryl Ester Storage Disease (CESD). As sebelipase alfa which replaces the deficient enzyme is now available, ERT continues to be the cornerstone with which LAL-D can be managed and the patient's prognosis enhanced. Nonetheless, it would be observed that the Lipid Modifying Agents segment is expected to grow at the highest CAGR in the coming years due to the simultaneous use of Lipid Modifying Agents along with ERT to address lipid accumulation in LAL-D. These drugs are useful in managing cholesterol and lipid profiles, especially for CESD patients, and are increasingly features as components of combination regimes for this purpose. It is expected that the introduction of lipid-modifying agents into therapeutic management plans will unleash the market segment as awareness rises and treatment protocols change.

According to the distribution channel, the market is categorized into hospital pharmacies, retail pharmacies, online, and others segments. Out of these segments, the hospital pharmacy has taken the largest market share because these institutions offer invaluable services to healthcare organizations. They offer and dispense the needed medicine such as Kanuma, which is important in treating patients with LAL-D. However, it is predicted that the value of the online pharmacy segment will rise significantly in the future due to customer orientation toward convenience and mobility.

For a better understanding of the market penetration of Lysosomal Acid Lipase Deficiency (LAL-D) Treatments market is analyzed based on its worldwide presence in countries such as North America (U.S., Canada, and the Rest of North America), Europe (Germany, France, U.K., Spain, Italy, Rest of Europe), Asia-Pacific (China, Japan, India, Rest of Asia-Pacific), Rest of World. Asia-Pacific is expected to grow with a significant CAGR in the forecast period (2024-2032) owing to the growing incidence of LAL-D. The emphasis is made on the development of collaboration activities to improve the product development in the Lysosomal Acid Lipase Deficiency (LAL-D) Treatment market for bringing new drug.

For instance, In October 2024, AstraZeneca entered into an exclusive license agreement with CSPC Pharmaceutical Group Ltd (CSPC) to advance the development of an early stage, novel small molecule Lipoprotein (a) (Lp(a)) disruptor that has the potential to offer additional benefits for patients with dyslipidaemia, which is a critical symptom of LAL-D.

Some of the major players operating in the market include Alexion Pharmaceuticals (AstraZeneca), Amicus Therapeutics, Inc., Pfizer Inc., Lupin Pharmaceutical, Inc., Sun Pharmaceutical Industries Limited, Zydus Group, Teva Pharmaceuticals USA, Inc., Glenmark Pharmaceuticals Inc., Thermo Fisher Scientific inc., Merck & Co., Inc.

TABLE OF CONTENTS

1.MARKET INTRODUCTION

• 1.1. Market Definitions
• 1.2. Main Objective
• 1.3. Stakeholders
• 1.4. Limitation

2.RESEARCH METHODOLOGY OR ASSUMPTION

• 2.1. Research Process of the Lysosomal Acid Lipase Deficiency (LAL-D) Treatment Market
• 2.2. Research Methodology of the Lysosomal Acid Lipase Deficiency (LAL-D) Treatment Market
• 2.3. Respondent Profile

3.EXECUTIVE SUMMARY

• 3.1. Industry Synopsis
• 3.2. Segmental Outlook
  • 3.2.1. Market Growth Intensity
• 3.3. Regional Outlook

4.MARKET DYNAMICS

• 4.1. Drivers
• 4.2. Opportunity
• 4.3. Restraints
• 4.4. Trends
• 4.5. PESTEL Analysis
• 4.6. Demand Side Analysis
• 4.7. Supply Side Analysis
  • 4.7.1. Merger & Acquisition
  • 4.7.2. Investment Scenario

5.GLOBAL LYSOSOMAL ACID LIPASE DEFICIENCY (LAL-D) TREATMENT MARKET REVENUE (USD MN), 2022-2032F

6.MARKET INSIGHTS BY INDICATION

• 6.1. Wolman Disease
• 6.2. Cholesteryl Ester Storage Disease

7.MARKET INSIGHTS BY TREATMENT

• 7.1. Enzyme Replacement Therapy (ERT)
• 7.2. Supportive Care
• 7.3. Lipid Modifying Agents
• 7.4. Others

8.MARKET INSIGHTS BY DISTRIBUTION CHANNEL

• 8.1. Hospital Pharmacies
• 8.2. Retail Pharmacies
• 8.3. Online
• 8.4. Others

9.MARKET INSIGHTS BY REGION

• 9.1. North America
  • 9.1.1. U.S.
  • 9.1.2. Canada
  • 9.1.3. Rest of North America
• 9.2. Europe
  • 9.2.1. Germany
  • 9.2.2. U.K.
  • 9.2.3. France
  • 9.2.4. Italy
  • 9.2.5. Spain
  • 9.2.6. Rest of Europe
• 9.3. Asia-Pacific
  • 9.3.1. China
  • 9.3.2. Japan
  • 9.3.3. India
  • 9.3.4. Rest of Asia-Pacific
• 9.4. Rest of World

10.VALUE CHAIN ANALYSIS

• 10.1. Marginal Analysis
• 10.2. List of Market Participants

11.COMPETITIVE LANDSCAPE

• 11.1. Competition Dashboard
• 11.2. Competitor Market Positioning Analysis
• 11.3. Porter Five Forces Analysis

12.COMPANY PROFILES

• 12.1. Alexion Pharmaceuticals (AstraZeneca)
  • 12.1.1. Company Overview
  • 12.1.2. Key Financials
  • 12.1.3. SWOT Analysis
  • 12.1.4. Product Portfolio
  • 12.1.5. Recent Developments
• 12.2. Amicus Therapeutics, Inc.
• 12.3. Pfizer Inc.
• 12.4. Lupin Pharmaceutical, Inc.
• 12.5. Sun Pharmaceutical Industries Limited
• 12.6. Zydus Group
• 12.7. Teva Pharmaceuticals USA, Inc.
• 12.8. Glenmark Pharmaceuticals Inc
• 12.9. Thermo Fisher Scientific inc.
• 12.10. Merck & Co., Inc.

13.ACRONYMS & ASSUMPTION

14.ANNEXURE