리소좀 산성 리파아제 결핍증 치료 시장, 기회, 성장 촉진요인, 산업 동향 분석 및 예측(2024-2032년)

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