戈謝氏症藥物市場、機會、成長動力、產業趨勢分析與預測，2024-2032

2023 年全球戈謝氏症藥物市場價值約16 億美元，預計2024 年至2032 年複合年成長率為2.9%。 ，導致葡萄糖腦苷脂在脾、肝、骨髓等器官中累積。這會導致貧血、疲勞、骨痛和器官腫大等症狀。戈謝病患病率的上升以及對有效治療的需求不斷增加正在推動市場成長。

主要市場促進因素包括人們對戈謝氏症的認知不斷提高、治療開發投資的增加以及政府對罕見疾病治療的支持增加。這些因素正在促進酵素替代療法（ERT）和底物替代療法（SRT）的進步。

整個戈謝氏症藥物產業根據疾病類型、治療類型、配銷通路和地區進行分類。

就疾病類型而言，由於 1 型疾病的盛行率較高，預計到 2032 年將達到 18 億美元。 1 型戈謝氏症主要以酵素替代療法治療，酵素替代療法提供合成形式的葡萄糖腦苷脂酶，防止有害葡萄糖腦苷脂的累積。針對 1 型的口服療法的推出預計將帶來成長機會。

根據治療類型，酵素替代療法 (ERT) 到 2023 年將佔 12 億美元，預計仍將佔據主導地位。 ERT 有助於減少重要器官中戈謝細胞的積累，減輕脾臟和肝臟腫大等症狀，並改善血紅蛋白水平和血小板計數等血液學參數。這有助於控制貧血、骨骼損傷和器官功能障礙等併發症，最終提高患者的生活品質。

市場按配銷通路進一步細分，到 2032 年，醫院藥房預計將達到 10 億美元。他們的專業知識和嚴格的品質控制方案有助於提高治療效果。

在美國，戈謝氏症藥物市場預計到2032 年將達到7.116 億美元。和批准戈謝氏症的新療法。

目錄

第 1 章：方法與範圍

第 2 章：執行摘要

第 3 章：產業洞察

• 產業生態系統分析
• 產業影響力
  • 成長動力
    • 戈謝氏症盛行率增加
    • 不斷增加開發戈謝氏症療法的投資
    • 提高及時診斷和治療的意識
    • 政府對罕見疾病治療的支持不斷增加
  • 產業陷阱與挑戰
    • 治療費用高
    • 存在嚴格的法規核准程序
• 成長潛力分析
• 監管環境
• 管道分析
• 2023 年定價分析
  • 按地區
  • 按關鍵人物
• 未來市場趨勢
• 波特的分析
• PESTEL分析

第 4 章：競爭格局

• 介紹
• 公司市佔率分析
• 公司矩陣分析
• 主要市場參與者的競爭分析
• 競爭定位矩陣
• 戰略儀表板

第 5 章：市場估計與預測：按疾病類型，2021 - 2032

• 主要趨勢
• 類型1
• 3型

第 6 章：市場估計與預測：按治療類型，2021 - 2032 年

• 主要趨勢
• 酵素替代療法
• 基質替代療法
• 其他治療類型

第 7 章：市場估計與預測：按配銷通路，2021 - 2032 年

• 主要趨勢
• 醫院藥房
• 零售藥房
• 網路藥房

第 8 章：市場估計與預測：按地區，2021 - 2032

• 主要趨勢
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 英國
  • 法國
  • 西班牙
  • 義大利
  • 荷蘭
  • 歐洲其他地區
• 亞太地區
  • 中國
  • 日本
  • 印度
  • 澳洲
  • 韓國
  • 亞太地區其他地區
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
  • 拉丁美洲其他地區
• 中東和非洲
  • 南非
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國
  • 中東和非洲其他地區

第 9 章：公司簡介

• Johnson and Johnson
• Pfizer Inc.
• Sanofi
• Takeda Pharmaceutical Company Limited
• Zywie LLC

The Global Gaucher Disease Drugs Market was valued at approximately USD 1.6 billion in 2023 and is anticipated to grow at a CAGR of 2.9% from 2024 to 2032. Gaucher disease is a rare, inherited metabolic condition caused by an enzyme glucocerebrosidase deficiency, leading to the accumulation of glucocerebroside in organs like the spleen, liver, and bone marrow. This results in symptoms such as anemia, fatigue, bone pain, and organ enlargement. The rising prevalence of Gaucher disease, along with increasing demand for effective treatments, is driving market growth.

Key market drivers include rising awareness about Gaucher disease, growing investments in treatment development, and increasing government support for rare disease therapies. These factors are promoting advancements in enzyme replacement therapy (ERT) and substrate replacement therapy (SRT).

The overall Gaucher Disease Drugs Industry is categorized based on Disease Type, Therapy Type, Distribution Channel, and Region.

With respect to disease type, the type 1 segment is projected to reach USD 1.8 billion by 2032 due to its higher prevalence. Type 1 Gaucher disease is primarily treated with enzyme replacement therapies, which supply a synthetic version of glucocerebrosidase, preventing the accumulation of harmful glucocerebroside. The introduction of oral therapies targeting Type 1 is expected to present growth opportunities.

Based on therapy type, enzyme replacement therapy (ERT) accounted for USD 1.2 billion in 2023 and is anticipated to remain a dominant segment. ERT helps reduce the accumulation of Gaucher cells in vital organs, alleviating symptoms such as enlarged spleen and liver and improving hematological parameters like hemoglobin levels and platelet counts. This helps manage complications such as anemia, bone damage, and organ dysfunction, ultimately improving the quality of life for patients.

The market is further segmented by distribution channel, with hospital pharmacies expected to reach USD 1 billion by 2032. Hospital pharmacies play a key role in offering the latest therapies, providing specialized knowledge, and ensuring high-quality medication management. Their expertise and strict quality control protocols contribute to enhanced treatment outcomes.

In the U.S., the Gaucher disease drugs market is projected to reach USD 711.6 million by 2032. The country's strong pharmaceutical R&D capabilities and a supportive regulatory environment, such as the FDA's Orphan Drug Act, contribute significantly to market growth, facilitating the development and approval of new treatments for Gaucher disease.

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 prevalence of Gaucher disease
    • 3.2.1.2 Growing investments for developing Gaucher disease therapies
    • 3.2.1.3 Increasing awareness towards timely diagnosis and treatment
    • 3.2.1.4 Rising government support for rare disease therapies
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 High cost of therapies
    • 3.2.2.2 Presence of stringent regulatory approval procedures
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Pipeline analysis
• 3.6 Pricing analysis, 2023
  • 3.6.1 By region
  • 3.6.2 By key player
• 3.7 Future market trends
• 3.8 Porter's analysis
• 3.9 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 Disease Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Type 1
• 5.3 Type 3

Chapter 6 Market Estimates and Forecast, By Therapy Type, 2021 - 2032 ($ Mn)

• 6.1 Key trends
• 6.2 Enzyme replacement therapy
• 6.3 Substrate replacement therapy
• 6.4 Other therapy types

Chapter 7 Market Estimates and Forecast, By Distribution Channel, 2021 - 2032 ($ Mn)

• 7.1 Key trends
• 7.2 Hospital pharmacy
• 7.3 Retail pharmacy
• 7.4 Online pharmacy

Chapter 8 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Mn)

• 8.1 Key trends
• 8.2 North America
  • 8.2.1 U.S.
  • 8.2.2 Canada
• 8.3 Europe
  • 8.3.1 Germany
  • 8.3.2 UK
  • 8.3.3 France
  • 8.3.4 Spain
  • 8.3.5 Italy
  • 8.3.6 Netherlands
  • 8.3.7 Rest of Europe
• 8.4 Asia Pacific
  • 8.4.1 China
  • 8.4.2 Japan
  • 8.4.3 India
  • 8.4.4 Australia
  • 8.4.5 South Korea
  • 8.4.6 Rest of Asia Pacific
• 8.5 Latin America
  • 8.5.1 Brazil
  • 8.5.2 Mexico
  • 8.5.3 Argentina
  • 8.5.4 Rest of Latin America
• 8.6 Middle East and Africa
  • 8.6.1 South Africa
  • 8.6.2 Saudi Arabia
  • 8.6.3 UAE
  • 8.6.4 Rest of Middle East and Africa

Chapter 9 Company Profiles

• 9.1 Johnson and Johnson
• 9.2 Pfizer Inc.
• 9.3 Sanofi
• 9.4 Takeda Pharmaceutical Company Limited
• 9.5 Zywie LLC