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1456953

高雪氏症治療市場 – 2024 年至 2029 年預測

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個工作天內

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簡介目錄

高雪氏症治療市場預計在預測期內將以 2.24% 的複合年成長率成長，從 2022 年的 1,069,222,000 美元增至 2029 年的 1,248,479,000 美元。

高雪氏症的特徵是Glucocerebroside在細胞和一些器官中積聚，導致肝臟和脾臟腫大。一種遺傳性疾病，具有疲勞、瘀傷、貧血和血小板計數低等症狀，由Glucocerebrosidase脂酶的遺傳缺陷引起。高雪氏症是一種罕見的脂肪細胞疾病，由Glucocerebrosidase，該酶會分解Glucocerebroside等脂肪化合物，導致戈謝細胞在脾、肝和骨髓中積聚。這種疾病分為三種類型。它們是I型、II型和III型。 I 型是最常見的疾病類型，由於盛行率不斷增加，預計將主導高雪氏症市場。短期治療目標包括貧血、出血傾向、內臟器官問題和一般健康狀況。長期治療旨在解決呼吸系統問題。有效的治療取決於患者和家人對病情和可用治療方法的教育。酵素替代療法（ERT）由於其採用率高於其他治療方法，預計將主導全球高雪氏症市場。 ERT 是Glucocerebrosidase的合成或重組形式，靜脈注射，以取代缺失的酵素並減少Glucocerebroside脂累積。由於核准用於 ERT 的藥物增加，預計該細分市場將擴大。

由於高雪氏症在全球的盛行率不斷上升以及製藥公司越來越注重開發創新治療方法，預計高雪氏症治療市場將顯著成長。此外，人們越來越擔心高雪氏症在世界各地的患病率不斷上升、政府支持和資金的增加以及世界多個國家的計劃。人均醫療保健支出的增加是預計推動成長的一些因素全球高雪氏症市場。

市場促進因素：

  • 溶小體儲積症盛行率的增加預示著市場的成長

溶小體儲積症日益流行預計將推動市場成長。在高雪氏症患者中，巨噬細胞中富含的溶小體變大並充滿未消化的Glucocerebroside苷脂。溶小體貯積症是一種遺傳性代謝疾病，其特徵是由於酵素缺乏而導致有毒化學物質的異常累積。酵素替代療法（ERT）已在改善高雪氏症I 型、II 型和 III 型高雪氏症病患者的貧血、血小板計數低、肝臟和脾臟腫大以及骨骼異常方面取得了成功。例如，根據美國國立衛生研究院的數據，高雪氏症是德系猶太人後裔中常見的遺傳性疾病，每 450 人中就有 1 人受到影響。根據美國國家戈謝基金會的數據，每 10 萬人中就有 1 人受到影響，2,500 名美國人受到影響。

  • 由於政府的努力和支持，醫療支出大幅增加。

津貼、研究基金和法規結構等政府支持正在促進高雪氏症治療市場的成長。支持政策為研發投資創造了良好的環境。改善患者獲得高雪氏症治療的機會，例如減少財務障礙和擴大醫療保險覆蓋範圍，將對市場成長產生正面影響。患者權益組織和支持團體可以提高認知、早期診斷並改善獲得治療的機會。適當的保險範圍和報銷政策對於確保患者獲得高雪氏症治療也至關重要。根據醫療保險和醫療補助服務中心2023年12月美國醫療支出資料，2022年國民醫療支出（NHE）成長4.1%，達到4.5兆美元，佔GDP的17.3%。 2022年醫療保險支出將成長5.9%，達到9,443億美元，佔NHE總額的21%。 2022年醫療補助支出將成長9.6%，達到8,057億美元，佔NHE總額的18%。 2022 年私人健康保險支出也成長 5.9%，達到 12,898 億美元。

市場限制因素：

  • 高處理成本可能抑制市場成長

標準治療方法酶替代療法 (ERT) 的高成本可能會阻礙高雪氏症治療的市場成長，這對某些患者的治療構成障礙，並對醫療保健系統和保險公司構成挑戰。

北美地區預計將佔據主要市場佔有率

由於多種因素，預計北美將主導高雪氏症治療市場。高雪氏症在美國的盛行率特別高，這有助於擴大高雪氏症治療的市場佔有率。該地區先進的醫療基礎設施、專業治療中心、研究機構和具有罕見疾病專業知識的醫療保健專業人員為診斷和治療提供了便利。北美積極參與醫學研發，加上高昂的醫療成本，支持了罕見疾病先進治療方法的開發、生產和普及。該地區還提供廣泛的治療選擇，包括酵素替代療法、基材減少療法和潛在新興的基因療法。患者倡導和支持團體可以提高意識並改善獲得治療的機會。適當的保險範圍和報銷政策對於確保患者獲得高雪氏症治療至關重要。法律規範，特別是美國食品藥物管理局，在藥物核准和市場准入方面發揮關鍵作用。

主要市場區隔

按治療類型

  • 酵素替代療法（ERT）
  • 基材減少療法（SRT）
  • 手術過程

依疾病類型

  • Ⅰ型
  • II型
  • Ⅲ型

按最終用戶

  • 醫院
  • 專科門診
  • 臨床研究所

按地區

  • 北美洲
  • 美國
  • 加拿大
  • 墨西哥
  • 南美洲
  • 巴西
  • 阿根廷
  • 其他
  • 歐洲
  • 英國
  • 德國
  • 法國
  • 其他
  • 中東/非洲 沙烏地阿拉伯/非洲
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國
  • 南非
  • 其他
  • 亞太地區
  • 中國
  • 日本
  • 印度
  • 韓國
  • 泰國
  • 印尼
  • 台灣
  • 其他

目錄

第1章 簡介

  • 市場概況
  • 市場定義
  • 調查範圍
  • 市場區隔
  • 貨幣
  • 先決條件
  • 基準年和預測年時間表
  • 相關利益者的主要利益

第2章調查方法

  • 研究設計
  • 調查過程

第3章執行摘要

  • 主要發現
  • CXO觀點

第4章市場動態

  • 市場促進因素
  • 市場限制因素
  • 波特五力分析
  • 產業價值鏈分析
  • 分析師觀點

第5章高雪氏症治療市場：依治療類型

  • 介紹
  • 酵素替代療法（ERT）
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利
  • 基材去除療法（SRT）
    • 市場機會趨勢
    • 成長前景
    • 地理收益
  • 手術過程
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利

第6章高雪氏症治療市場：依疾病類型

  • 介紹
  • Ⅰ型
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利
  • II型
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利
  • Ⅲ型
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利

第7章高雪氏症治療市場：依最終使用者分類

  • 介紹
  • 醫院
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利
  • 專科門診
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利
  • 臨床研究所
    • 市場機會和趨勢
    • 成長前景
    • 地域盈利

第8章高雪氏症治療市場：按地區

  • 介紹
  • 北美洲
    • 按治療類型
    • 依疾病類型
    • 按最終用戶
    • 按國家/地區
  • 南美洲
    • 按治療類型
    • 依疾病類型
    • 按最終用戶
    • 按國家/地區
  • 歐洲
    • 按治療類型
    • 依疾病類型
    • 按最終用戶
    • 按國家/地區
  • 中東/非洲
    • 按治療類型
    • 依疾病類型
    • 按最終用戶
    • 按國家/地區
  • 亞太地區
    • 按治療類型
    • 依疾病類型
    • 按最終用戶
    • 按國家/地區

第9章競爭環境及分析

  • 主要企業及策略分析
  • 市場佔有率分析
  • 合併、收購、協議和合作
  • 競爭對手儀表板

第10章 公司簡介

  • AVROBIO Inc.
  • Gain Therapeutics, Inc.
  • Pfizer Inc.
  • Prevail Therapeutics
  • Sanofi
  • Evotech
  • CANbridge Life Sciences Ltd.
  • Lysogene
  • Orphazyme
  • Freeline
簡介目錄
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