治療溶小體儲積症(LSD) 的全球市場：成長、傾向、預測（2020年∼2025年）

全球的溶小體儲積症 (LSD)治療市場在2020年到2025年之間，將以8.2%的年複合成長率成長。 溶小體儲積症是因為遺傳基因缺陷所致，基因缺陷導致溶小體酵素缺乏或功能失常。而溶小體儲積症包括法布瑞氏症、高雪氏症、龐貝氏症、戴-薩克司氏症、天冬氨□葡萄糖胺尿症、貝敦氏症、胱胺酸症等。其中高雪氏症的特徵是神經系統併發症，是LSD疾病最普遍的症狀。其他症狀包括發燒、疼痛、痲痺、刺痛、手腳灼熱感、疲勞感、皮膚發紅或發紫、呼吸困難、暈眩等。

目前有23種罕見藥物被批准用於治療溶小體儲積症。其中有4種疾病：法布瑞氏症、胱胺酸症、高雪氏症、以及龐貝氏症有多個被批准的治療方法。神經元蠟樣脂褐質儲積症（CLN2型）、 嬰兒型溶□體酸性脂肪□缺乏症（LAL-D）、以及黏多醣症第一型、第二型、第四型、第五型、第六型，有一種化合物已獲FDA批准用於治療。

根據國家高雪氏基金會，總人口中的5萬人到10萬人中會有1人罹患高雪氏症。第一型的高雪氏症是最普遍的，任何人都有可能罹患，發病機率為人口的4萬分之一。

溶小體儲積症(LSD)的發病率增加、人們對罕見疾病的認知提升、以及用於溶小體儲積症(LSD)的治療診斷和藥物開發的研發增加，都是推動溶小體儲積症(LSD)治療市場的主因。

此報告研究溶小體儲積症(LSD)治療市場，提供以下全面性資訊，包括：市場概況、市場規模的演變與預測（分為治療、相關疾病、地區）、市場趨勢、市場的成長因素及阻礙因素分析、市場機會、競爭格局、主要企業介紹等。

目次

第1章 介紹

• 研究的假設
• 研究範圍

第2章 研究手法

第3章 重點摘要

第4章 市場動力

• 市場概況
• 市場成長因素
  • 溶小體儲積症(LSD)發病率提高
  • 人們對於溶小體儲積症(LSD)的認知提升
  • 為了治療溶小體儲積症(LSD)的診斷及藥物開發的研發增加
• 阻礙市場因素
  • 治療費用昂貴
• 五力分析
  • 新參與者的威脅
  • 買家的議價能力
  • 供應商的議價能力
  • 替代產品的威脅
  • 競爭白熱化

第5章 市場區分

• 治療法
  • 酵素替代療法
  • 基質減量療法
• 相關疾病
  • 高雪氏症
  • 胱胺酸症
  • 龐貝氏症
  • 法布瑞氏症
  • 其他
• 地區
  • 北美
  • 歐洲
  • 亞太地區
  • 其他地區

第6章 競爭格局

• 企業介紹
  • Pfizer Inc
  • Takeda Pharmaceutical Company Limited (Shire Plc)
  • Sanofi (Genzyme Corporation)
  • BioMarin
  • Johnson & Johnson (Actelion Pharmaceuticals Ltd)
  • Amicus Therapeutics, Inc
  • Alexion Pharmaceuticals, Inc
  • Sigilon Therapeutics, Inc
  • Orphazyme A/S

第7章 市場機會及未來趨勢

The lysosomal storage disease treatment market is expected to register a CAGR of 8.2% during the forecast period. Lysosomal storage diseases are a group of rare inherited metabolic disorders that result from the lack or malfunctioning of lysosomal enzymes. Some of the lysosomal storage diseases are Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, aspartylglucosaminuria, Batten disease, cystinosis, and others. Among them Gaucher disease is the most common type of lysosomal disorder which is characterized by neurological complication. Some of the symptoms such as fever, pain, numbness, tingling, or burning in the hands and feet, tiredness, red or purple skin sores, trouble in breathing, dizziness and so on.

Currently, there are twenty-three orphan drugs approved for the treatment of 11 lysosomal storage diseases. Among them four diseases have multiple therapeutics approved, i.e. Fabry disease, cystinosis, Gaucher disease, and Pompe disease. Neuronal ceroid lipofuscinosis 2 (CLN2), Lysosomal acid lipase deficiency (LAL-D), and Mucopolysaccharidosis type I, II, IVA, VI, VII have one compound approved for treatment by the FDA.

According to the National Gaucher Foundation, Gaucher disease occurs in 1 in 50,000 to 100,000 people in the general population. Type 1 Gaucher disease is the most common form of the disorder. Gaucher disease can affect anyone, occurring in up to 1 in 40,000 live births in the general population.

Increase in incidence of lysosomal diseases, growing awareness among people regarding rare lysosomal storage disease and increasing research and development for diagnosis and drug development for treatment of lysosomal diseases are the key driving factors in lysosomal storage disease treatment market.

Key Market Trends

Enzyme Replacement Therapy Segment is Expected to Hold a Major Market Share in the Lysosomal Storage Disease Treatment Market

• Enzyme replacement therapy is a treatment which replaces an enzyme that is absent or deficient in the body. Some of the available enzyme replacement therapy for lysosomal storage diseases are Velaglucerase Alfa, Taliglucerase Alfa, Agalsidase beta, Laronidase, Imiglucerase and among others.
• National Institute of Neurological Disorders and Stroke (NINDS), estimates that 5,000 to 10,000 people have pompe disease worldwide. In addition, incidence of infantile onset pompe disease varies between ethnic groups and regions. In some countries, such as in African Americans, the incidence is as high as one in 14,000 people.
• Enzyme replacement therapy segment holds a significant market share in the lysosomal storage disease treatment market and is anticipated to show similar trend over the forecast period due to improving organ function and effective in reducing toxic storage material in patients suffering from lysosomal disorders.
• Increase in incidence of lysosomal diseases and increasing research and development for diagnosis and drug development for treatment of lysosomal diseases are the key driving factors in the enzyme replacement therapy segment.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

North America is expected to hold a major market share in the global lysosomal storage disease treatment market due to increasing incidence of lysosomal storage disorders in this region. According to the National Organization for Rare Disorders (NORD), approximately 6,000 individuals are affected with Gaucher disease in the United States. Moreover, Type 1 Gaucher disease is the most common form of the disease in western countries, making up roughly 95 percent of patients. Furthermore, increasing research and development for diagnosis and drug development for treatment of lysosomal diseases and presence of well-established healthcare infrastructure is also fueling the growth of the overall regional market to a large extent.

Competitive Landscape

The lysosomal storage disease treatment market is consolidated competitive and consists of a few major players. In terms of market share, few of the major players are currently dominating the market. Some of the leading market players include Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Therapeutics, Inc, Alexion Pharmaceuticals, Inc, Sigilon Therapeutics, Inc and Orphazyme A/S.

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Table of Contents

1 INTRODUCTION

• 1.1 Study Assumptions
• 1.2 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS

• 4.1 Market Overview
• 4.2 Market Drivers
  • 4.2.1 Increasing Incidences of Lysosomal Diseases
  • 4.2.2 Growing Awareness Among People Worldwide Regarding Rare Lysosomal storage Disease
  • 4.2.3 Increasing Research and Development for Diagnosis and Drug Development for Treatment of Lysosomal Diseases
• 4.3 Market Restraints
  • 4.3.1 High Cost of Treatment
• 4.4 Porter's Five Force Analysis
  • 4.4.1 Threat of New Entrants
  • 4.4.2 Bargaining Power of Buyers/Consumers
  • 4.4.3 Bargaining Power of Suppliers
  • 4.4.4 Threat of Substitute Products
  • 4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION

• 5.1 By Therapy Type
  • 5.1.1 Enzyme Replacement Therapy
    • 5.1.1.1 Velaglucerase Alfa
    • 5.1.1.2 Taliglucerase Alfa
    • 5.1.1.3 Agalsidase beta
    • 5.1.1.4 Laronidase
    • 5.1.1.5 Imiglucerase
    • 5.1.1.6 Other Enzyme Replacement Therapies
  • 5.1.2 Substrate Reduction Therapy
    • 5.1.2.1 Eliglustat
    • 5.1.2.2 Miglustat
    • 5.1.2.3 Other Substrate Reduction Therapies
• 5.2 By Application
  • 5.2.1 Gaucher disease
  • 5.2.2 Cystinosis
  • 5.2.3 Pompe Disease
  • 5.2.4 Fabry Disease
  • 5.2.5 Other Applications
• 5.3 Geography
  • 5.3.1 North America
    • 5.3.1.1 United States
    • 5.3.1.2 Canada
    • 5.3.1.3 Mexico
  • 5.3.2 Europe
    • 5.3.2.1 Germany
    • 5.3.2.2 United Kingdom
    • 5.3.2.3 France
    • 5.3.2.4 Italy
    • 5.3.2.5 Spain
    • 5.3.2.6 Rest of Europe
  • 5.3.3 Asia-Pacific
    • 5.3.3.1 China
    • 5.3.3.2 Japan
    • 5.3.3.3 India
    • 5.3.3.4 Australia
    • 5.3.3.5 South Korea
    • 5.3.3.6 Rest of Asia-Pacific
  • 5.3.4 Rest of the World

6 COMPETITIVE LANDSCAPE

• 6.1 Company Profiles
  • 6.1.1 Pfizer Inc
  • 6.1.2 Takeda Pharmaceutical Company Limited (Shire Plc)
  • 6.1.3 Sanofi (Genzyme Corporation)
  • 6.1.4 BioMarin
  • 6.1.5 Johnson & Johnson (Actelion Pharmaceuticals Ltd)
  • 6.1.6 Amicus Therapeutics, Inc
  • 6.1.7 Alexion Pharmaceuticals, Inc
  • 6.1.8 Sigilon Therapeutics, Inc
  • 6.1.9 Orphazyme A/S

7 MARKET OPPORTUNITIES AND FUTURE TRENDS