溶小體酸性脂肪酶缺乏症 (LAL-D) 治療市場:現狀分析與未來預測 (2024年～2032年)

溶小體酸性脂肪酶缺乏症 (LAL-D) 治療市場預計在預測期內 (2024-2032) 以 11.4% 的複合年增長率大幅增長。推動 LAL-D 治療市場的關鍵成長因素包括基因療法的持續進步（其在治療該疾病方面具有關鍵作用）以及可用於治療該疾病症狀的酵素替代療法。公眾意識的提高、診斷能力的提高、國家和國際對罕見疾病藥物開發的支持以及 LAL-D 發病率的上升也促進了市場的成長。

根據適應症，市場分為兩個部分：沃爾曼病和膽固醇酯沉積病。沃爾曼病（WD）因其發病早、病情嚴重且需要立即治療而佔據當前市場主導地位。由於該疾病相對罕見，且若不及時治療可能會產生嚴重後果，因此引起了醫療和製藥行業利益相關者的關注。然而，市場成長最快的部分是膽固醇酯沉積病（CESD）。 CESD 也是一種罕見疾病，但其發病從兒童到成年是逐漸的，因此更加多樣化。因此患者生命週期長、數量高。此外，人們認識的提高、鑑別能力的提高以及 sebelipase alfa 等用於 WD 和 CESD 的治療方法的批准都是 CESD 領域預期增長的原因。此外，治療藥物的增加和 CESD 患者存活率的上升也是推動預期市場成長的因素。

根據治療方法，市場分為酵素替代療法 (ERT)、支持性護理、脂質調節藥物等。其中，ERT 在目前的 LAL-D 治療市場中佔據主導地位，這主要是因為直接酵素替代療法對沃爾曼病和膽固醇酯累積病 (CESD) 均具有明確的療效。由於可以使用 sebelipase alfa 來取代缺失的酶，ERT 仍然是管理 LAL-D 和改善患者預後的基石。然而，由於使用調脂藥物和 ERT 同時治療 LAL-D 中的脂質累積問題，預計未來幾年調脂藥物領域將以最高的複合年增長率成長。這些藥物可能對控制 CESD 患者的膽固醇和血脂水平特別有用，並且越來越多地被視為此目的聯合療法的組成部分。脂質調節藥物引入治療管理方案可望解放市場領域，同時提高意識並改變治療方案。

根據分銷管道，市場分為醫院藥房、零售藥房、網路藥房和其他。在這些細分市場中，醫院藥房佔據最大的市場佔有率，因為這些機構為醫療機構提供有價值的服務。醫院藥局提供並調配Kanuma等必要藥品，這些藥品對於LAL-D患者的治療至關重要。然而，由於消費者對便利性和移動性的偏好，線上藥局市場的價值預計未來幾年將大幅成長。

為了更了解溶小體酸性脂肪酶缺乏症 (LAL-D) 治療市場的市場滲透率，我們根據北美（美國、加拿大和北美其他地區）、歐洲（德國、法國、英國、西班牙、義大利和歐洲其他地區）、亞太地區（中國、日本、印度和亞太地區其他地區）和世界其他地區的全球影響力對其進行了分析。由於 LAL-D 的普及率不斷提高，預計預測期內（2024-2032 年）亞太地區將以顯著的複合年增長率成長。溶小體酸性脂肪酶缺乏症 (LAL-D) 治療市場正專注於協作開發活動，以改善產品開發並帶來新藥。

例如，2024 年 10 月，阿斯特捷利康與石藥集團有限公司 (CSPC) 簽訂了獨家許可協議，以推進新型早期小脂蛋白 (a) (Lp(a)) 幹擾劑的開發，這種幹擾劑可能為患有血脂異常（一種嚴重的 LAL-D）的患者帶來其他益處。

市場的主要參與者包括 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

目錄

第1章 市場概要

• 市場定義
• 主要目的
• 相關利益者
• 限制事項

第2章 分析方法或前提條件

• 分析流程
• 分析方法
• 受訪者簡介

第3章 摘要整理

• 產業摘要
• 各市場區隔預測
  • 市場成長的強度
• 地區展望

第4章 市場動態

• 促進因素
• 機會
• 阻礙因素
• 趨勢
• PESTEL分析
• 需求面分析
• 供給面分析
  • 企業合併·收購 (M&A)
  • 投資情境

第5章 全球溶小體酸性脂肪酶缺乏症 (LAL-D) 治療的市場收益 (2022～2032年)

第6章 市場分析:各適應症

• 沃爾曼病
• 膽固醇酯沉積病

第7章 市場分析:各治療法

• 酵素補充療法 (ERT)
• 支持療法
• 脂質改質劑
• 其他

第8章 市場分析:各流通管道

• 醫院藥局
• 零售藥局
• 線上
• 其他

第9章 市場分析:各地區

• 北美
  • 美國
  • 加拿大
  • 其他北美地區
• 歐洲
  • 德國
  • 英國
  • 法國
  • 義大利
  • 西班牙
  • 其他歐洲地區
• 亞太地區
  • 中國
  • 日本
  • 印度
  • 其他亞太地區
• 全球其他地區

第10章 價值鏈分析

• 限制分析
• 市場參與企業一覽

第11章 競爭情形

• 競爭儀表板
• 企業的市場定位分析
• 波特的五力分析

第12章 企業簡介

• Alexion Pharmaceuticals (AstraZeneca)
  • 企業概要
  • 主要的財務指標
  • SWOT分析
  • 產品系列
  • 近幾年趨勢
• 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 縮寫與前提條件

第14章 附錄

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