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市场调查报告书
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1456953

高雪氏症治疗市场 – 2024 年至 2029 年预测

Gaucher Disease Treatment Market - Forecasts from 2024 to 2029
出版日期: | 出版商: Knowledge Sourcing Intelligence | 英文 125 Pages | 商品交期: 最快1-2个工作天内

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简介目录

高雪氏症治疗市场预计在预测期内将以 2.24% 的复合年增长率成长，从 2022 年的 1,069,222,000 美元增至 2029 年的 1,248,479,000 美元。

高雪氏症的特征是Glucocerebroside在细胞和一些器官中积聚，导致肝臟和脾臟肿大。一种遗传性疾病，具有疲劳、瘀伤、贫血和血小板计数低等症状，由Glucocerebrosidase脂酶的遗传缺陷引起。高雪氏症是一种罕见的脂肪细胞疾病，由Glucocerebrosidase，该酶会分解Glucocerebroside等脂肪化合物，导致戈谢细胞在脾、肝和骨髓中积聚。这种疾病分为三种类型。它们是I型、II型和III型。 I 型是最常见的疾病类型，由于盛行率不断增加，预计将主导高雪氏症市场。短期治疗目标包括贫血、出血倾向、内臟器官问题和一般健康状况。长期治疗旨在解决呼吸系统问题。有效的治疗取决于患者和家人对病情和可用治疗方法的教育。酵素替代疗法（ERT）由于其采用率高于其他治疗方法，预计将主导全球高雪氏症市场。 ERT 是Glucocerebrosidase的合成或重组形式，静脉注射，以取代缺失的酵素并减少Glucocerebroside脂累积。由于核准用于 ERT 的药物增加，预计该细分市场将扩大。

由于高雪氏症在全球的盛行率不断上升以及製药公司越来越注重开发创新治疗方法，预计高雪氏症治疗市场将显着成长。此外，人们越来越担心高雪氏症在世界各地的患病率不断上升、政府支持和资金的增加以及世界多个国家的倡议。人均医疗保健支出的增加是预计推动增长的一些因素全球高雪氏症市场。

市场驱动因素：

  • 溶小体储积症盛行率的增加预示着市场的成长

溶小体储积症日益流行预计将推动市场成长。在高雪氏症患者中，巨噬细胞中富含的溶小体变大并充满未消化的Glucocerebroside苷脂。溶小体贮积症是一种遗传性代谢疾病，其特征是由于酵素缺乏而导致有毒化学物质的异常累积。酵素替代疗法（ERT）已在改善高雪氏症I 型、II 型和 III 型高雪氏症病患者的贫血、血小板计数低、肝臟和脾臟肿大以及骨骼异常方面取得了成功。例如，根据美国国立卫生研究院的数据，高雪氏症是德系犹太人后裔中常见的遗传性疾病，每 450 人中就有 1 人受到影响。根据美国国家戈谢基金会的数据，每 10 万人中就有 1 人受到影响，2,500 名美国人受到影响。

  • 由于政府的努力和支持，医疗支出大幅增加。

津贴、研究基金和法规结构等政府支持正在促进高雪氏症治疗市场的成长。支持政策为研发投资创造了良好的环境。改善患者获得高雪氏症治疗的机会，例如减少财务障碍和扩大医疗保险覆盖范围，将对市场成长产生正面影响。患者权益组织和支持团体可以提高认识、早期诊断并改善获得治疗的机会。适当的保险范围和报销政策对于确保患者获得高雪氏症治疗也至关重要。根据医疗保险和医疗补助服务中心2023年12月美国医疗支出资料，2022年国民医疗支出（NHE）成长4.1%，达到4.5兆美元，占GDP的17.3%。 2022年医疗保险支出将成长5.9%，达到9,443亿美元，占NHE总额的21%。 2022年医疗补助支出将成长9.6%，达到8,057亿美元，占NHE总额的18%。 2022 年私人健康保险支出也成长 5.9%，达到 12,898 亿美元。

市场限制因素：

  • 高处理成本可能抑制市场成长

标准治疗方法酶替代疗法 (ERT) 的高成本可能会阻碍高雪氏症治疗的市场成长，这对某些患者的治疗构成障碍，并对医疗保健系统和保险公司构成挑战。

北美地区预计将占据主要市场占有率

由于多种因素，预计北美将主导高雪氏症治疗市场。高雪氏症在美国的盛行率特别高，这有助于扩大高雪氏症治疗的市场占有率。该地区先进的医疗基础设施、专业治疗中心、研究机构和具有罕见疾病专业知识的医疗保健专业人员为诊断和治疗提供了便利。北美积极参与医学研发，加上高昂的医疗成本，支持了罕见疾病先进治疗方法的开发、生产和普及。该地区还提供广泛的治疗选择，包括酵素替代疗法、基材减少疗法和潜在新兴的基因疗法。患者倡导和支持团体可以提高意识并改善获得治疗的机会。适当的保险范围和报销政策对于确保患者获得高雪氏症治疗至关重要。法律规范，特别是美国食品药物管理局，在药物核准和市场准入方面发挥关键作用。

主要市场区隔

按治疗类型

  • 酵素替代疗法（ERT）
  • 基材减少疗法（SRT）
  • 手术过程

依疾病类型

  • Ⅰ型
  • II型
  • Ⅲ型

按最终用户

  • 医院
  • 专科门诊
  • 临床研究所

按地区

  • 北美洲
  • 美国
  • 加拿大
  • 墨西哥
  • 南美洲
  • 巴西
  • 阿根廷
  • 其他的
  • 欧洲
  • 英国
  • 德国
  • 法国
  • 其他的
  • 中东/非洲 沙乌地阿拉伯/非洲
  • 沙乌地阿拉伯
  • 阿拉伯聯合大公国
  • 南非
  • 其他的
  • 亚太地区
  • 中国
  • 日本
  • 印度
  • 韩国
  • 泰国
  • 印尼
  • 台湾
  • 其他的

目录

第一章 简介

  • 市场概况
  • 市场定义
  • 调查范围
  • 市场区隔
  • 货币
  • 先决条件
  • 基准年和预测年时间表
  • 相关利益者的主要利益

第二章调查方法

  • 研究设计
  • 调查过程

第三章执行摘要

  • 主要发现
  • CXO观点

第四章市场动态

  • 市场驱动因素
  • 市场限制因素
  • 波特五力分析
  • 产业价值链分析
  • 分析师观点

第五章高雪氏症治疗市场：依治疗类型

  • 介绍
  • 酵素替代疗法（ERT）
    • 市场机会和趋势
    • 成长前景
    • 地域盈利
  • 基材去除疗法（SRT）
    • 市场机会趋势
    • 成长前景
    • 地理收益
  • 手术过程
    • 市场机会和趋势
    • 成长前景
    • 地域盈利

第六章高雪氏症治疗市场：依疾病类型

  • 介绍
  • Ⅰ型
    • 市场机会和趋势
    • 成长前景
    • 地域盈利
  • II型
    • 市场机会和趋势
    • 成长前景
    • 地域盈利
  • Ⅲ型
    • 市场机会和趋势
    • 成长前景
    • 地域盈利

第七章高雪氏症治疗市场：依最终使用者分类

  • 介绍
  • 医院
    • 市场机会和趋势
    • 成长前景
    • 地域盈利
  • 专科门诊
    • 市场机会和趋势
    • 成长前景
    • 地域盈利
  • 临床研究所
    • 市场机会和趋势
    • 成长前景
    • 地域盈利

第八章高雪氏症治疗市场：按地区

  • 介绍
  • 北美洲
    • 按治疗类型
    • 依疾病类型
    • 按最终用户
    • 按国家/地区
  • 南美洲
    • 按治疗类型
    • 依疾病类型
    • 按最终用户
    • 按国家/地区
  • 欧洲
    • 按治疗类型
    • 依疾病类型
    • 按最终用户
    • 按国家/地区
  • 中东/非洲
    • 按治疗类型
    • 依疾病类型
    • 按最终用户
    • 按国家/地区
  • 亚太地区
    • 按治疗类型
    • 依疾病类型
    • 按最终用户
    • 按国家/地区

第九章竞争环境及分析

  • 主要企业及策略分析
  • 市场占有率分析
  • 合併、收购、协议和合作
  • 竞争对手仪表板

第十章 公司简介

  • AVROBIO Inc.
  • Gain Therapeutics, Inc.
  • Pfizer Inc.
  • Prevail Therapeutics
  • Sanofi
  • Evotech
  • CANbridge Life Sciences Ltd.
  • Lysogene
  • Orphazyme
  • Freeline
简介目录
Product Code: KSI061612288

The Gaucher disease treatment market is projected to grow at a CAGR of 2.24% during the forecast period, reaching US$1248.479 million by 2029, from US$1069.222 million in 2022.

Gaucher disease is characterized by the build-up of glucocerebroside in cells and some organs, resulting in enlargement of the liver as well as the spleen. It is a genetic disease that shows symptoms such as fatigue, bruising, anemia, and a low blood platelet count and is caused by a hereditary deficit of the enzyme glucocerebrosidase. Gaucher disease is a rare liposomal disorder caused by a deficiency in the enzyme glucocerebrosidase, which breaks down fatty compounds like glucocerebroside, causing Gaucher cells to accumulate in the spleen, liver, and bone marrow. The disease has three types: Type I, Type II, and Type III. Type I is the most prevalent form and is expected to dominate the Gaucher disorder market due to its increased incidence. Short-term treatment objectives include anemia, bleeding propensity, visceral issues, and general health. Long-term therapy aims to address respiratory issues. Effective treatment depends on patient and family education on the condition and available treatments. Enzyme replacement therapy (ERT) is expected to dominate the global Gaucher Disease market due to its higher adoption rate than other therapies. ERT involves regular oral or intravenous administration of a synthetic or recombinant version of glucocerebrosidase, aiming to replace the lacking enzyme and reduce glucocerebroside buildup. The segment is expected to expand due to more approved drugs in ERT.

The Gaucher disease treatment market is expected to grow significantly due to the increasing global prevalence of Gaucher and the growing focus of pharmaceutical companies on developing innovative treatments for the disease. Furthermore, rising concern for the growing incidences of Gaucher disease amongst people worldwide, combined with increasing government support and funding and rising per capita healthcare expenditure by several nations worldwide, are some of the factors expected to drive the growth of the global Gaucher disease market.

MARKET DRIVERS:

  • Increasing incidence of lysosomal diseases is anticipated in the market growth

The increasing prevalence of lysosomal disease is expected to drive market growth. Lysosomes, abundant in macrophages, in patients with Gaucher disease become enlarged and filled with undigested glucocerebroside, which can eventually be visualized as "crumpled tissue paper" by electron microscopy. Lysosomal storage disorders are inherited metabolic disorders characterized by abnormal accumulation of harmful chemicals due to enzyme shortages. Enzyme replacement therapy (ERT) has shown success in improving anemia, low platelet counts, liver and spleen enlargement, and skeletal abnormalities in individuals with Gaucher disease type I, and also in those with Gaucher disease types II and III. For instance, according to National Institution of Health, Gaucher disease is a genetic disorder more prevalent among Ashkenazi Jewish descendants, with an incidence of 1 in 450 persons. In the general public, it affects 1 in 100,000 people, with 2500 Americans suffering from the condition, according to the National Gaucher Foundation.

  • Government initiatives and support have led to a significant increase in healthcare expenditure.

Government support, including grants, research funding, and regulatory frameworks, has contributed to the growth of the Gaucher disease treatment market. Supportive policies create a favorable environment for investment in research and development. Improving patient access to Gaucher disease treatments, such as reducing financial barriers and expanding healthcare coverage, positively impacts market growth. Patient advocacy organizations and support groups can increase awareness, early diagnosis, and improve access to treatments. Adequate insurance coverage and reimbursement policies are also crucial for ensuring patients have access to Gaucher disease treatments. According to the Centers for Medicare and Medicaid Services data of December 2023 about healthcare expenditure in United States, National Health Expenditure (NHE) grew 4.1% to $4.5 trillion in 2022, accounting for 17.3% of GDP. Medicare spending increased 5.9% to $944.3 billion in 2022, representing 21% of total NHE. Medicaid spending increased 9.6% to $805.7 billion in 2022, accounting for 18% of total NHE. Private health insurance spending also increased 5.9% to $1,289.8 billion in 2022.

MARKET RESTRAINT:

  • High cost of treatment could restrain the growth of the market

The market growth of Gaucher's disease treatment may be hindered by the high cost of enzyme replacement therapy (ERT), which is a standard treatment, which may pose barriers to access for some patients and pose challenges for healthcare systems and insurers.

North America region is anticipated to hold a significant market share

North America is expected to dominate the Gaucher Disease Treatment Market due to several factors. The country's high prevalence of Gaucher Disease, particularly in the United States, contributes to a larger market share for Gaucher disease treatments. The region's advanced healthcare infrastructure, specialized treatment centers, research institutions, and healthcare professionals with expertise in rare diseases facilitate diagnosis and treatment. North America's active involvement in medical research and development, coupled with high healthcare expenditure, supports the development, production, and adoption of advanced therapies for rare diseases. The region also offers a broader range of treatment options, including enzyme replacement therapy, substrate reduction therapy, and potentially emerging gene therapies. Patient advocacy and support groups can increase awareness and improve access to treatments. Adequate insurance coverage and reimbursement policies are crucial for ensuring patients have access to Gaucher disease treatments. The regulatory framework, particularly the U.S. Food and Drug Administration, plays a significant role in drug approvals and market access.

Key Developments

  • October 2023- AceLink Therapeutics has initiated a Phase 2 trial in China for AL01211, a novel oral glucosylceramide synthase inhibitor, to treat Fabry disease. The open-label study evaluates the safety, pharmacokinetics, pharmacodynamics, and treatment effects of AL01211 in males with classic Fabry disease who have not been previously treated. The trial aims to enroll 18 patients across six sites in China. AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase inhibitor being developed to treat glycosphingolipid storage diseases, including Fabry Disease and Type 1 Gaucher Disease.
  • February 2023- Gain Therapeutics, a biotechnology company, presented pre-clinical data from its Gaucher disease program at the 19th Annual WORLDSymposium. The data showed that GT-02329 restores B-glucocerebrosidase (GCase) activity, depletes toxic lipid substrate accumulation, reduces neuroinflammation, and improves neuromuscular function in an animal model of neuronopathic Gaucher disease. The study supports the potential of GCase-targeting small molecule therapy for neuronopathic Gaucher disease.

Key Market Segments

By Treatment Type

  • Enzyme Replacement Therapy (ERT)
  • Substrate Reduction Therapy (SRT)
  • Surgical Procedure

By Disease Type

  • Type I
  • Type II
  • Type III

By End-Users

  • Hospitals
  • Ambulatory Specialty Clinics
  • Clinical Research Institutes

By Geography

  • North America
  • USA
  • Canada
  • Mexico
  • South America
  • Brazil
  • Argentina
  • Others
  • Europe
  • United Kingdom
  • Germany
  • France
  • Others
  • Middle East and Africa
  • Saudi Arabia
  • UAE
  • South Africa
  • Others
  • Asia Pacific
  • China
  • Japan
  • India
  • South Korea
  • Thailand
  • Indonesia
  • Taiwan
  • Others

TABLE OF CONTENTS

1. INTRODUCTION

  • 1.1. Market Overview
  • 1.2. Market Definition
  • 1.3. Scope of the Study
  • 1.4. Market Segmentation
  • 1.5. Currency
  • 1.6. Assumptions
  • 1.7. Base, and Forecast Years Timeline
  • 1.8. Key Benefits to the Stakeholder

2. RESEARCH METHODOLOGY

  • 2.1. Research Design
  • 2.2. Research Processes

3. EXECUTIVE SUMMARY

  • 3.1. Key Findings
  • 3.2. CXO Perspective

4. MARKET DYNAMICS

  • 4.1. Market Drivers
  • 4.2. Market Restraints
  • 4.3. Porter's Five Forces Analysis
    • 4.3.1. Bargaining Power of Suppliers
    • 4.3.2. Bargaining Power of Buyers
    • 4.3.3. Threat of New Entrants
    • 4.3.4. Threat of Substitutes
    • 4.3.5. Competitive Rivalry in the Industry
  • 4.4. Industry Value Chain Analysis
  • 4.5. Analyst View

5. GAUCHER DISEASE TREATMENT MARKET, BY TREATMENT TYPE

  • 5.1. Introduction
  • 5.2. Enzyme Replacement Therapy (ERT)
    • 5.2.1. Market Opportunities and Trends
    • 5.2.2. Growth Prospects
    • 5.2.3. Geographic Lucrativeness
  • 5.3. Substrate Reduction Therapy (SRT)
    • 5.3.1. Market Opportunities and Trends
    • 5.3.2. Growth Prospects
    • 5.3.3. Geographic Lucrativeness
  • 5.4. Surgical Procedures
    • 5.4.1. Market Opportunities and Trends
    • 5.4.2. Growth Prospects
    • 5.4.3. Geographic Lucrativeness

6. GAUCHER DISEASE TREATMENT MARKET, BY DISEASE TYPE

  • 6.1. Introduction
  • 6.2. Type I
    • 6.2.1. Market Opportunities and Trends
    • 6.2.2. Growth Prospects
    • 6.2.3. Geographic Lucrativeness
  • 6.3. Type II
    • 6.3.1. Market Opportunities and Trends
    • 6.3.2. Growth Prospects
    • 6.3.3. Geographic Lucrativeness
  • 6.4. Type III
    • 6.4.1. Market Opportunities and Trends
    • 6.4.2. Growth Prospects
    • 6.4.3. Geographic Lucrativeness

7. GAUCHER DISEASE TREATMENT MARKET, BY END-USERS

  • 7.1. Introduction
  • 7.2. Hospitals
    • 7.2.1. Market Opportunities and Trends
    • 7.2.2. Growth Prospects
    • 7.2.3. Geographic Lucrativeness
  • 7.3. Ambulatory Speciality Clinics
    • 7.3.1. Market Opportunities and Trends
    • 7.3.2. Growth Prospects
    • 7.3.3. Geographic Lucrativeness
  • 7.4. Clinical Research Institutes
    • 7.4.1. Market Opportunities and Trends
    • 7.4.2. Growth Prospects
    • 7.4.3. Geographic Lucrativeness

8. GAUCHER DISEASE TREATMENT MARKET, BY GEOGRAPHY

  • 8.1. Introduction
  • 8.2. North America
    • 8.2.1. By Treatment Type
    • 8.2.2. By Disease Type
    • 8.2.3. By End-Users
    • 8.2.4. By Country
      • 8.2.4.1. United States
        • 8.2.4.1.1. Market Opportunities and Trends
        • 8.2.4.1.2. Growth Prospects
      • 8.2.4.2. Canada
        • 8.2.4.2.1. Market Opportunities and Trends
        • 8.2.4.2.2. Growth Prospects
      • 8.2.4.3. Mexico
        • 8.2.4.3.1. Market Opportunities and Trends
        • 8.2.4.3.2. Growth Prospects
  • 8.3. South America
    • 8.3.1. By Treatment Type
    • 8.3.2. By Disease Type
    • 8.3.3. By End-Users
    • 8.3.4. By Country
      • 8.3.4.1. Brazil
        • 8.3.4.1.1. Market Opportunities and Trends
        • 8.3.4.1.2. Growth Prospects
      • 8.3.4.2. Argentina
        • 8.3.4.2.1. Market Opportunities and Trends
        • 8.3.4.2.2. Growth Prospects
      • 8.3.4.3. Others
        • 8.3.4.3.1. Market Opportunities and Trends
        • 8.3.4.3.2. Growth Prospects
  • 8.4. Europe
    • 8.4.1. By Treatment Type
    • 8.4.2. By Disease Type
    • 8.4.3. By End-Users
    • 8.4.4. By Country
      • 8.4.4.1. United Kingdom
        • 8.4.4.1.1. Market Opportunities and Trends
        • 8.4.4.1.2. Growth Prospects
      • 8.4.4.2. Germany
        • 8.4.4.2.1. Market Opportunities and Trends
        • 8.4.4.2.2. Growth Prospects
      • 8.4.4.3. France
        • 8.4.4.3.1. Market Opportunities and Trends
        • 8.4.4.3.2. Growth Prospects
      • 8.4.4.4. Italy
        • 8.4.4.4.1. Market Opportunities and Trends
        • 8.4.4.4.2. Growth Prospects
      • 8.4.4.5. Spain
        • 8.4.4.5.1. Market Opportunities and Trends
        • 8.4.4.5.2. Growth Prospects
      • 8.4.4.6. Others
        • 8.4.4.6.1. Market Opportunities and Trends
        • 8.4.4.6.2. Growth Prospects
  • 8.5. Middle East and Africa
    • 8.5.1. By Treatment Type
    • 8.5.2. By Disease Type
    • 8.5.3. By End-Users
    • 8.5.4. By Country
      • 8.5.4.1. Saudi Arabia
        • 8.5.4.1.1. Market Opportunities and Trends
        • 8.5.4.1.2. Growth Prospects
      • 8.5.4.2. UAE
        • 8.5.4.2.1. Market Opportunities and Trends
        • 8.5.4.2.2. Growth Prospects
      • 8.5.4.3. Others
        • 8.5.4.3.1. Market Opportunities and Trends
        • 8.5.4.3.2. Growth Prospects
  • 8.6. Asia Pacific
    • 8.6.1. By Treatment Type
    • 8.6.2. By Disease Type
    • 8.6.3. By End-Users
    • 8.6.4. By Country
      • 8.6.4.1. Japan
        • 8.6.4.1.1. Market Opportunities and Trends
        • 8.6.4.1.2. Growth Prospects
      • 8.6.4.2. China
        • 8.6.4.2.1. Market Opportunities and Trends
        • 8.6.4.2.2. Growth Prospects
      • 8.6.4.3. India
        • 8.6.4.3.1. Market Opportunities and Trends
        • 8.6.4.3.2. Growth Prospects
      • 8.6.4.4. South Korea
        • 8.6.4.4.1. Market Opportunities and Trends
        • 8.6.4.4.2. Growth Prospects
      • 8.6.4.5. Taiwan
        • 8.6.4.5.1. Market Opportunities and Trends
        • 8.6.4.5.2. Growth Prospects
      • 8.6.4.6. Thailand
        • 8.6.4.6.1. Market Opportunities and Trends
        • 8.6.4.6.2. Growth Prospects
      • 8.6.4.7. Indonesia
        • 8.6.4.7.1. Market Opportunities and Trends
        • 8.6.4.7.2. Growth Prospects
      • 8.6.4.8. Others
        • 8.6.4.8.1. Market Opportunities and Trends
        • 8.6.4.8.2. Growth Prospects

9. COMPETITIVE ENVIRONMENT AND ANALYSIS

  • 9.1. Major Players and Strategy Analysis
  • 9.2. Market Share Analysis
  • 9.3. Mergers, Acquisitions, Agreements, and Collaborations
  • 9.4. Competitive Dashboard

10. COMPANY PROFILES

  • 10.1. AVROBIO Inc.
  • 10.2. Gain Therapeutics, Inc.
  • 10.3. Pfizer Inc.
  • 10.4. Prevail Therapeutics
  • 10.5. Sanofi
  • 10.6. Evotech
  • 10.7. CANbridge Life Sciences Ltd.
  • 10.8. Lysogene
  • 10.9. Orphazyme
  • 10.10. Freeline