리소좀 산성 리파아제 결핍증 치료 시장 규모, 점유율, 동향 분석 보고서 : 적응증별, 치료법별, 지역별, 부문별 예측(2025-2030년)

Lysosomal Acid Lipase Deficiency Treatment Market Size, Share & Trends Analysis Report By Indication (Wolman Disease, Cholesteryl Ester Storage Disease), By Treatment, By Region, And Segment Forecasts, 2025 - 2030

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한글목차

리소좀 산성 리파아제 결핍증 치료 시장 성장 및 동향:

Grand View Research의 최신 보고서에 따르면, 세계 리소좀 산성 리파제 결핍증 치료제 시장 규모는 2030년까지 4억 3,320만 달러에 달할 것으로 예상되며, 2025년부터 2030년까지 7.2%의 CAGR을 기록할 것으로 예측됩니다. 높은 미충족 수요, 유병률 증가, 효소 대체 요법의 보급이 시장을 이끄는 주요 요인 중 일부입니다.

LAL 결핍증(LAL-D)은 LAL 유전자의 돌연변이로 인해 환자가 콜레스테롤 에스테르와 트리글리세리드를 분해 할 수없는 희귀 한 유형의 리소좀 저장 장애입니다. LAL 결핍증은 진행성 질환으로 다기관 임상 증상, 심각한 의학적 합병증, 특히 영유아의 간부전으로 인한 조기 사망을 초래할 수 있는 진행성 질환입니다.

2015년까지 세계 LAL 결핍증 치료제 시장은 승인된 치료법이 없어 완만한 성장세를 보였습니다. 2015년 알렉시온 파마(Alexion Pharma)의 카누마(세베리파제 알파)가 승인되면서 효소대체요법(ERT)이 LAL-D 영역에 진입하면서 LAL-D 영역에 진입하게 되었습니다. 카누마는 리소좀의 콜레스테롤 에스테르와 트리아실글리세롤을 특이적으로 가수분해하는 효소로, LAL 결핍증 환자에게 정맥으로 투여됩니다. 현재 카누마는 주요 시장에서 월만병(WD)과 콜레스테롤 에스테르 저장증(CESD)에 대해 승인된 유일한 약물입니다.

치료법별로 보면 지지요법은 2017년 시장에서 주요 수익 기여자였습니다. 그러나 ERT 분야는 카누마의 지속적인 시장 진입에 힘입어 예측 기간 동안 가장 빠른 성장을 기록할 것으로 예상됩니다. 대부분의 환자가 진단을 받지 못했거나 오진으로 인해 질병의 예후는 여전히 좋지 않습니다. 따라서 질병의 생물학을 이해하고 내성을 극복하고 독성을 최소화하기 위한 효과적인 치료법을 개발하기 위한 광범위한 연구가 여전히 절실히 요구되고 있습니다.

리소좀 산성 리파아제 결핍증 치료 시장 보고서 하이라이트

• CESD는 백인과 히스패닉계 주민에게 많이 나타나며, 약 30만 명 중 1명이 앓고 있는 것으로 알려져 있습니다.
• 독일은 유럽 시장의 최전선에 위치하고 있으며, 높은 유병률과 ERT 계열 약물의 가용성이 시장을 주도하고 있습니다.
• Kanuma의 ERT 계열 승인은 LAL-D 결핍증 치료에 대한 근본적인 전환을 강조합니다.
• 미개척 LAL-D 환자군에서 유일한 장기 치료제로서 카누마의 선점 효과로 인해 ERT 분야는 예측 기간 동안 가장 빠른 성장을 보일 것으로 예상됩니다.
• 스타틴 계열은 동종 제네릭 의약품과의 경쟁 심화와 알렉시온의 카누마 도입 확대로 시장 점유율이 감소할 것으로 예상됩니다.
• LAL 결핍증 환자 수가 증가하고 치료법이 제한적이라는 점은 이 시장에서 새로운 치료법 개발에 투자하는 기업에게 큰 기회로 작용할 것입니다.
• 예후 불량으로 인한 질병 진단에 대한 인식 부족과 약품 수급 지연이 시장 성장을 저해할 수 있습니다.

목차

제1장 조사 방법과 범위

제2장 주요 요약

제3장 리소좀 산성 리파아제 결핍증 치료 시장 변수, 동향, 범위

• 시장 계통 전망
• 시장 역학
  • 시장 성장 촉진요인 분석
  • 시장 성장 억제요인 분석
• 비즈니스 환경 분석
  • 업계 분석 - Porter's Five Forces 분석
  • PESTLE 분석

제4장 리소좀 산성 리파아제 결핍증 치료 시장 : 적응증 비즈니스 분석

• 적응증 시장 점유율, 2024년·2030년
• 적응증 부문 대시보드
• 시장 규모와 예측과 동향 분석, 적응증별, 2018-2030년
• 월만병(WD)
• 콜레스테롤 에스테르 축적증(CESD)

제5장 리소좀 산성 리파아제 결핍증 치료 시장 : 치료 비즈니스 분석

• 치료 시장 점유율, 2024년과 2030년
• 치료 부문 대시보드
• 시장 규모와 예측과 동향 분석, 치료별, 2018-2030년
• 효소보충요법(ERT)
• 지지요법
• 지질 개질제
• 기타

제6장 리소좀 산성 리파아제 결핍증 치료 시장 : 지역 추정·동향 분석

• 지역별 시장 점유율 분석, 2024년·2030년
• 지역 시장 대시보드
• 시장 규모, 예측 동향 분석, 2018-2030년 :
• 북미
  • 국가별, 2018-2030년
  • 미국
  • 캐나다
  • 멕시코
• 유럽
  • 국가별, 2018-2030년
  • 영국
  • 독일
  • 프랑스
  • 이탈리아
  • 스페인
  • 노르웨이
  • 덴마크
  • 스웨덴
• 아시아태평양
  • 국가별, 2018-2030년
  • 일본
  • 중국
  • 인도
  • 한국
  • 호주
  • 태국
• 라틴아메리카
  • 국가별, 2018-2030년
  • 브라질
  • 아르헨티나
• 중동 및 아프리카
  • 국가별, 2018-2030년
  • 남아프리카공화국
  • 사우디아라비아
  • 아랍에미리트
  • 쿠웨이트

제7장 경쟁 구도

• 참여 기업 개요
• 기업의 시장 포지션 분석
• 기업 분류
• 전략 매핑
• 기업 개요/리스트
  • Alexion Pharmaceuticals, Inc.(AstraZeneca)
  • Amgen Inc.
  • BioCrick BioTech
  • Lupin
  • Merck KGaA
  • Pfizer Inc.
  • Regeneron Pharmaceuticals Inc.
  • Teva Pharmaceutical Industries Ltd

ksm

영문 목차

영문목차

Lysosomal Acid Lipase Deficiency Treatment Market Growth & Trends:

The global lysosomal acid lipase deficiency treatment market size is expected to reach USD 433.2 million by 2030, registering a CAGR of 7.2% from 2025 to 2030, according to a new report by Grand View Research, Inc. High unmet needs, rising prevalence, and increasing uptake of enzyme replacement therapy are some of the primary factors driving the market.

LAL deficiency (LAL-D) is a rare type of lysosomal storage disorder in which patients are unable to breakdown cholesteryl esters and triglycerides due to a mutation in the LAL gene. As a result, there is an accumulation of lipids in the liver, spleen, and walls of blood vessels. LAL deficiency is a progressive disease with multi-systemic clinical manifestations, significant medical complications, and early mortality due to liver failure, especially among infants.

Until 2015, the global LAL deficiency treatment market witnessed modest growth due to a lack of approved treatments for the disease. Medical care was limited to supportive care, with surgery and lipid-modifying drugs such as statins remained the mainstay for treatment. In 2015, approval of Alexion Pharma's Kanuma (sebelipase alfa) marked the entry of enzyme replacement therapy (ERT) in the LAL-D space. Kanuma is a hydrolytic lysosomal cholesterol ester and triacylglycerol specific enzyme, administered intravenously in patients with LAL deficiency. Currently, Kanuma is the only drug approved for both Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD) in the key markets.

Based on treatment, supportive care was the leading revenue contributor in the market in 2017. However, the ERT segment is expected to register the fastest growth during the forecast period, supported by continued uptake of Kanuma. Disease prognosis remains poor, as a majority of patients are either undiagnosed or misdiagnosed. Thus, there remains a strong need for extensive research to understand disease biology and develop effective therapeutic options with the goal to overcome resistance and minimize toxicity.

Lysosomal Acid Lipase Deficiency Treatment Market Report Highlights:

• CESD manifests later in life and is more prevalent among Caucasians and Hispanic populations, affecting approximately 1 in 300,000 individuals.
• Germany is at the forefront of the Europe market and is driven by high disease prevalence and availability of drugs in the ERT class.
• Approval of Kanuma in ERT class highlight a fundamental shift in treatment approach to LAL-D deficiency.
• The ERT segment is estimated to be the fastest-growing during the forecast period, owing to Kanuma's first-mover advantage as the only long-term treatment in untapped LAL-D patient population pool.
• Statins are anticipated to lose market share due to rising competition from generics within the class and increasing uptake of Alexion's Kanuma.
• Rising eligible patient population and limited therapeutic intervention for LAL deficiency offer a strong commercial opportunity for players to invest in developing novel treatments in this market.
• Lack of awareness in disease diagnosis and delayed access to medications due to poor prognosis may restrain the growth of the market.

Table of Contents

Chapter 1. Methodology and Scope

• 1.1. Market Segmentation & Scope
• 1.2. Segment Definitions
  • 1.2.1. Indication
  • 1.2.2. Treatment
• 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. Lysosomal Acid Lipase Deficiency 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

Chapter 4. Lysosomal Acid Lipase Deficiency Treatment Market: Indication Business Analysis

• 4.1. Indication Market Share, 2024 & 2030
• 4.2. Indication Segment Dashboard
• 4.3. Market Size & Forecasts and Trend Analysis, by Indication, 2018 to 2030 (USD Million)
• 4.4. Wolman Disease (WD)
  • 4.4.1. Wolman Disease (WD) Market, 2018 - 2030 (USD Million)
• 4.5. Cholesteryl Ester Storage Disease (CESD)
  • 4.5.1. Cholesteryl Ester Storage Disease (CESD) Market, 2018 - 2030 (USD Million)

Chapter 5. Lysosomal Acid Lipase Deficiency Treatment Market: Treatment Business Analysis

• 5.1. Treatment Market Share, 2024 & 2030
• 5.2. Treatment Segment Dashboard
• 5.3. Market Size & Forecasts and Trend Analysis, by Treatment, 2018 to 2030 (USD Million)
• 5.4. Enzyme Replacement Therapy (ERT)
  • 5.4.1. Enzyme Replacement Therapy (ERT) Market, 2018 - 2030 (USD Million)
• 5.5. Supportive Care
  • 5.5.1. Supportive Care Market, 2018 - 2030 (USD Million)
• 5.6. Lipid Modifying Agents
  • 5.6.1. Lipid Modifying Agents Market, 2018 - 2030 (USD Million)
• 5.7. Others
  • 5.7.1. Others Market, 2018 - 2030 (USD Million)

Chapter 6. Lysosomal Acid Lipase Deficiency Treatment Market: Regional Estimates & Trend Analysis

• 6.1. Regional Market Share Analysis, 2024 & 2030
• 6.2. Regional Market Dashboard
• 6.3. Market Size, & Forecasts Trend Analysis, 2018 to 2030:
• 6.4. North America
  • 6.4.1. North America Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
  • 6.4.2. U.S.
    • 6.4.2.1. Key Country Dynamics
    • 6.4.2.2. Regulatory Framework
    • 6.4.2.3. Competitive Insights
    • 6.4.2.4. U.S. Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.4.3. Canada
    • 6.4.3.1. Key Country Dynamics
    • 6.4.3.2. Regulatory Framework
    • 6.4.3.3. Competitive Insights
    • 6.4.3.4. Canada Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.4.4. Mexico
    • 6.4.4.1. Key Country Dynamics
    • 6.4.4.2. Regulatory Framework
    • 6.4.4.3. Competitive Insights
    • 6.4.4.4. Mexico Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
• 6.5. Europe
  • 6.5.1. Europe Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
  • 6.5.2. UK
    • 6.5.2.1. Key Country Dynamics
    • 6.5.2.2. Regulatory Framework
    • 6.5.2.3. Competitive Insights
    • 6.5.2.4. UK Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.3. Germany
    • 6.5.3.1. Key Country Dynamics
    • 6.5.3.2. Regulatory Framework
    • 6.5.3.3. Competitive Insights
    • 6.5.3.4. Germany Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.4. France
    • 6.5.4.1. Key Country Dynamics
    • 6.5.4.2. Regulatory Framework
    • 6.5.4.3. Competitive Insights
    • 6.5.4.4. France Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.5. Italy
    • 6.5.5.1. Key Country Dynamics
    • 6.5.5.2. Regulatory Framework
    • 6.5.5.3. Competitive Insights
    • 6.5.5.4. Italy Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.6. Spain
    • 6.5.6.1. Key Country Dynamics
    • 6.5.6.2. Regulatory Framework
    • 6.5.6.3. Competitive Insights
    • 6.5.6.4. Spain Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.7. Norway
    • 6.5.7.1. Key Country Dynamics
    • 6.5.7.2. Regulatory Framework
    • 6.5.7.3. Competitive Insights
    • 6.5.7.4. Norway Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.8. Denmark
    • 6.5.8.1. Key Country Dynamics
    • 6.5.8.2. Regulatory Framework
    • 6.5.8.3. Competitive Insights
    • 6.5.8.4. Denmark Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5.9. Sweden
    • 6.5.9.1. Key Country Dynamics
    • 6.5.9.2. Regulatory Framework
    • 6.5.9.3. Competitive Insights
    • 6.5.9.4. Sweden Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
• 6.6. Asia Pacific
  • 6.6.1. Asia Pacific Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
  • 6.6.2. Japan
    • 6.6.2.1. Key Country Dynamics
    • 6.6.2.2. Regulatory Framework
    • 6.6.2.3. Competitive Insights
    • 6.6.2.4. Japan Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6.3. China
    • 6.6.3.1. Key Country Dynamics
    • 6.6.3.2. Regulatory Framework
    • 6.6.3.3. Competitive Insights
    • 6.6.3.4. China Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6.4. India
    • 6.6.4.1. Key Country Dynamics
    • 6.6.4.2. Regulatory Framework
    • 6.6.4.3. Competitive Insights
    • 6.6.4.4. India Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6.5. South Korea
    • 6.6.5.1. Key Country Dynamics
    • 6.6.5.2. Regulatory Framework
    • 6.6.5.3. Competitive Insights
    • 6.6.5.4. South Korea Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6.6. Australia
    • 6.6.6.1. Key Country Dynamics
    • 6.6.6.2. Regulatory Framework
    • 6.6.6.3. Competitive Insights
    • 6.6.6.4. Australia Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6.7. Thailand
    • 6.6.7.1. Key Country Dynamics
    • 6.6.7.2. Regulatory Framework
    • 6.6.7.3. Competitive Insights
    • 6.6.7.4. Thailand Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
• 6.7. Latin America
  • 6.7.1. Latin America Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
  • 6.7.2. Brazil
    • 6.7.2.1. Key Country Dynamics
    • 6.7.2.2. Regulatory Framework
    • 6.7.2.3. Competitive Insights
    • 6.7.2.4. Brazil Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.7.3. Argentina
    • 6.7.3.1. Key Country Dynamics
    • 6.7.3.2. Regulatory Framework
    • 6.7.3.3. Competitive Insights
    • 6.7.3.4. Argentina Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
• 6.8. MEA
  • 6.8.1. MEA Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
  • 6.8.2. South Africa
    • 6.8.2.1. Key Country Dynamics
    • 6.8.2.2. Regulatory Framework
    • 6.8.2.3. Competitive Insights
    • 6.8.2.4. South Africa Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.8.3. Saudi Arabia
    • 6.8.3.1. Key Country Dynamics
    • 6.8.3.2. Regulatory Framework
    • 6.8.3.3. Competitive Insights
    • 6.8.3.4. Saudi Arabia Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.8.4. UAE
    • 6.8.4.1. Key Country Dynamics
    • 6.8.4.2. Regulatory Framework
    • 6.8.4.3. Competitive Insights
    • 6.8.4.4. UAE Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.8.5. Kuwait
    • 6.8.5.1. Key Country Dynamics
    • 6.8.5.2. Regulatory Framework
    • 6.8.5.3. Competitive Insights
    • 6.8.5.4. Kuwait Lysosomal Acid Lipase Deficiency Treatment Market Estimates And Forecasts, 2018 - 2030 (USD Million)

Chapter 7. Competitive Landscape

• 7.1. Participant Overview
• 7.2. Company Market Position Analysis
• 7.3. Company Categorization
• 7.4. Strategy Mapping
• 7.5. Company Profiles/Listing
  • 7.5.1. Alexion Pharmaceuticals, Inc. (AstraZeneca)
    • 7.5.1.1. Overview
    • 7.5.1.2. Financial Performance
    • 7.5.1.3. Product Benchmarking
    • 7.5.1.4. Strategic Initiatives
  • 7.5.2. Amgen Inc.
    • 7.5.2.1. Overview
    • 7.5.2.2. Financial Performance
    • 7.5.2.3. Product Benchmarking
    • 7.5.2.4. Strategic Initiatives
  • 7.5.3. BioCrick BioTech
    • 7.5.3.1. Overview
    • 7.5.3.2. Financial Performance
    • 7.5.3.3. Product Benchmarking
    • 7.5.3.4. Strategic Initiatives
  • 7.5.4. Lupin
    • 7.5.4.1. Overview
    • 7.5.4.2. Financial Performance
    • 7.5.4.3. Product Benchmarking
    • 7.5.4.4. Strategic Initiatives
  • 7.5.5. Merck KGaA
    • 7.5.5.1. Overview
    • 7.5.5.2. Financial Performance
    • 7.5.5.3. Product Benchmarking
    • 7.5.5.4. Strategic Initiatives
  • 7.5.6. Pfizer Inc.
    • 7.5.6.1. Overview
    • 7.5.6.2. Financial Performance
    • 7.5.6.3. Product Benchmarking
    • 7.5.6.4. Strategic Initiatives
  • 7.5.7. Regeneron Pharmaceuticals Inc.
    • 7.5.7.1. Overview
    • 7.5.7.2. Financial Performance
    • 7.5.7.3. Product Benchmarking
    • 7.5.7.4. Strategic Initiatives
  • 7.5.8. Teva Pharmaceutical Industries Ltd
    • 7.5.8.1. Overview
    • 7.5.8.2. Financial Performance
    • 7.5.8.3. Product Benchmarking
    • 7.5.8.4. Strategic Initiatives

관련자료