莫爾基奧症候群治療市場規模、佔有率和成長分析（按治療方法、疾病類型、通路和地區分類）—2026-2033年行業預測

預計到 2024 年，全球莫爾基奧症候群治療市場規模將達到 1.6462 億美元，到 2025 年將達到 1.7417 億美元，到 2033 年將達到 2.7344 億美元，預測期（2026-2033 年）的複合年成長率為 5.8%。

莫爾基奧症候群是一種罕見的遺傳性代謝失調疾病，會影響骨骼發育，屬於黏多醣症（MPS）範疇。該疾病在全球發病率的不斷上升是莫爾基奧症候群治療市場的主要驅動力。政府和企業部門日益成長的支持，以及對標靶治療需求的增加，進一步推動了市場成長。當前市場格局的特點是製造商之間為拓展治療選擇而進行的併購和聯盟活動日益增加。此外，醫療保健支出不斷成長、生活方式的改變、技術進步以及遺傳性疾病的普遍存在等因素也對市場產生了積極影響。然而，在對成本高度敏感的市場中，不完善的報銷政策、高昂的生產成本以及較低的治療認知度等挑戰可能會阻礙市場擴張。

全球莫爾基奧症候群治療市場促進因素

全球莫爾基奧症候群治療市場預計將迎來顯著成長，這主要得益於針對這種罕見疾病的新型治療方法研發的蓬勃發展。眾多產品正處於不同的研發階段，人們對其獲得監管部門核准的前景日益樂觀，尤其是在依洛硫酸酯酶α（VIMIZIM）近期取得成功之後。隨著各公司越來越重視罕見疾病治療，進入開發平臺的創新藥物可望滿足未被滿足的醫療需求，並改善患者照護。這一不斷變化的市場格局，以及莫爾基奧症候群患者臨床試驗和治療方案的進展，預示著強勁的市場發展趨勢。

限制全球莫爾基奧症候群治療市場的因素

全球莫爾基奧症候群治療市場面臨諸多限制因素，可能阻礙其成長。其中一個主要挑戰是治療費用高昂，例如維米珠單抗（VIMIZIM）等療法，每位患者每年的治療費用估計約為38萬美元。此外，新興市場對莫爾基奧症候群的認知和了解不足，也阻礙了治療方案的推廣應用。臨床和監管方面的障礙也增加了有效治療方法研發和普及的難度，這可能進一步限制市場的潛在擴張。這些因素共同構成了莫爾基奧症候群治療領域的挑戰。

全球莫爾基奧症候群治療市場趨勢

全球莫爾基奧症候群治療市場呈現顯著上升趨勢，這主要得益於主要行業參與者加大研發投入以及監管核准的不斷增加。領先的製藥公司正透過持續進行的III期臨床試驗和創新療法研究，將莫爾基奧症候群的治療方案納入其臨床研發管線。這種研發活動的增加反映了人們對該疾病日益成長的認知，進而引發大眾對有效治療方法的關注和需求。因此，在科學知識的進步、技術創新以及患者獲得治療方案途徑的改善等因素的推動下，預計該市場將實現強勁成長。

目錄

介紹

• 調查目標
• 調查範圍
• 定義

調查方法

• 資訊收集
• 二手資料和一手資料方法
• 市場規模預測
• 市場假設與限制

執行摘要

• 全球市場展望
• 供需趨勢分析
• 細分市場機會分析

市場動態與展望

• 市場規模
• 市場動態
  • 促進因素和機遇
  • 限制與挑戰
• 波特分析

關鍵市場考察

• 關鍵成功因素
• 競爭程度
• 關鍵投資機會
• 市場生態系統
• 市場吸引力指數（2025）
• PESTEL 分析
• 總體經濟指標
• 價值鏈分析
• 定價分析
• 技術分析
• 監管分析
• 專利分析
• 案例研究

全球莫爾基奧症候群治療市場規模（按治療方法和複合年成長率分類）（2026-2033 年）

• 酵素替代療法
• 其他

全球莫爾基奧症候群治療市場規模（按疾病類型和複合年成長率分類）（2026-2033 年）

• 莫爾基奧 A
• 莫爾基奧B型

全球莫爾基奧症候群治療市場規模（按分銷管道分類）及複合年成長率（2026-2033 年）

• 醫院藥房
• 零售藥房
• 網路藥房
• 其他

全球莫爾基奧症候群治療市場規模及複合年成長率（2026-2033）

• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 西班牙
  • 法國
  • 英國
  • 義大利
  • 其他歐洲地區
• 亞太地區
  • 中國
  • 印度
  • 日本
  • 韓國
  • 亞太其他地區
• 拉丁美洲
  • 巴西
  • 其他拉丁美洲地區
• 中東和非洲
  • 海灣合作理事會國家
  • 南非
  • 其他中東和非洲地區

競爭資訊

• 前五大公司對比
• 主要企業的市場定位（2025 年）
• 主要市場參與者所採取的策略
• 近期市場趨勢
• 公司市佔率分析（2025 年）
• 主要企業公司簡介
  • 公司詳情
  • 產品系列分析
  • 依業務板塊進行公司股票分析
  • 2023-2025年營收年比比較

主要企業簡介

• BioMarin Pharmaceuticals Inc.（US）
• Genzyme Corporation（US）
• Shire plc（Ireland）
• JCR Pharmaceuticals Co., Ltd.（Japan）
• Sanofi（France）
• Sangamo Therapeutics（US）
• ArmaGen, Inc.（US）
• GREEN CROSS CORP（South Korea）
• Concert Pharmaceuticals Inc.（US）
• Alexion Pharmaceuticals Inc.（US）
• Novo Nordisk A/S（Denmark）
• Eli Lilly and Company（US）
• Boehringer Ingelheim International GmbH（Germany）
• AstraZeneca（UK）
• Amgen Inc.（US）
• Merck KGaA（Germany）
• Actelion Pharmaceuticals Ltd.（Switzerland）
• Biocon（India）
• Ultragenyx Pharmaceutical Inc.（US）
• Enzyvant Therapeutics Inc.（US）

結論與建議

Global Morquio Syndrome Treatment Market size was valued at USD 164.62 Million in 2024 and is poised to grow from USD 174.17 Million in 2025 to USD 273.44 Million by 2033, growing at a CAGR of 5.8% during the forecast period (2026-2033).

Morquio syndrome, an uncommon inherited metabolic disorder affecting skeletal development, falls under the mucopolysaccharidosis (MPS) category. The growing incidence of this condition globally is a significant driver of the Morquio syndrome treatment market. Increased support from government and corporate sectors, along with heightened demand for targeted therapies, is further fueling market growth. The landscape is characterized by rising mergers and partnerships among manufacturers aimed at enhancing treatment options. Additionally, factors such as growing healthcare expenditures, evolving lifestyles, technological advancements, and the prevalence of genetic disorders are positively influencing the market. However, challenges such as inadequate reimbursement policies in cost-sensitive markets, high production costs, and limited treatment awareness may hinder market expansion.

Top-down and bottom-up approaches were used to estimate and validate the size of the Global Morquio Syndrome Treatment market and to estimate the size of various other dependent submarkets. The research methodology used to estimate the market size includes the following details: The key players in the market were identified through secondary research, and their market shares in the respective regions were determined through primary and secondary research. This entire procedure includes the study of the annual and financial reports of the top market players and extensive interviews for key insights from industry leaders such as CEOs, VPs, directors, and marketing executives. All percentage shares split, and breakdowns were determined using secondary sources and verified through Primary sources. All possible parameters that affect the markets covered in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data.

Global Morquio Syndrome Treatment Market Segments Analysis

Global Morquio Syndrome Treatment Market is segmented by Treatment, Disease Type, Distribution Channel and region. Based on Treatment, the market is segmented into Enzyme Replacement Therapy and Others. Based on Disease Type, the market is segmented into Morquio A and Morquio B. Based on Distribution Channel, the market is segmented into Hospital Pharmacies, Retail Pharmacies, Online Pharmacies and Others. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Global Morquio Syndrome Treatment Market

The Global Morquio Syndrome Treatment market is poised for significant growth, driven by an upsurge in research and development of new therapies targeting this rare disease. With numerous products advancing through various stages of development, there is heightened optimism surrounding their potential regulatory approval, particularly following the recent success of elosulfase alfa (VIMIZIM). As companies increasingly prioritize rare disease treatments, the influx of innovative medications in the pipeline promises to fulfill unmet medical needs and enhance patient care. This evolving landscape suggests a robust market trajectory, fueled by advancements in clinical trials and therapeutic options for those affected by Morquio syndrome.

Restraints in the Global Morquio Syndrome Treatment Market

The Global Morquio Syndrome Treatment market faces several restraints that may hinder its growth. A significant challenge is the high cost associated with treatment options, with estimates suggesting that therapies like VIMIZIM could amount to approximately $380,000 per patient each year. Additionally, a lack of awareness and understanding of Morquio syndrome in emerging markets poses a barrier to treatment adoption. Furthermore, clinical and regulatory hurdles add complexity to the development and accessibility of effective therapies, which could further limit the market's potential expansion. Collectively, these factors contribute to a challenging environment for the Morquio syndrome treatment sector.

Market Trends of the Global Morquio Syndrome Treatment Market

The Global Morquio Syndrome Treatment market is experiencing a notable upward trend, driven by intensified research and development efforts from key industry players and growing regulatory approvals. Major pharmaceutical companies are increasingly including Morquio syndrome in their clinical pipelines, with ongoing Phase 3 trials and innovative therapeutic approaches under investigation. This surge in activity reflects a broader awareness of the condition, leading to heightened public interest and demand for effective treatments. As a result, the market is poised for robust growth, propelled by advancements in scientific understanding, technological innovation, and improved access to treatment options for affected individuals.

Table of Contents

Introduction

• Objectives of the Study
• Scope of the Report
• Definitions

Research Methodology

• Information Procurement
• Secondary & Primary Data Methods
• Market Size Estimation
• Market Assumptions & Limitations

Executive Summary

• Global Market Outlook
• Supply & Demand Trend Analysis
• Segmental Opportunity Analysis

Market Dynamics & Outlook

• Market Overview
• Market Size
• Market Dynamics
  • Drivers & Opportunities
  • Restraints & Challenges
• Porters Analysis
  • Competitive rivalry
  • Threat of substitute
  • Bargaining power of buyers
  • Threat of new entrants
  • Bargaining power of suppliers

Key Market Insights

• Key Success Factors
• Degree of Competition
• Top Investment Pockets
• Market Ecosystem
• Market Attractiveness Index, 2025
• PESTEL Analysis
• Macro-Economic Indicators
• Value Chain Analysis
• Pricing Analysis
• Technology Analysis
• Regulatory Analysis
• Patent Analysis
• Case Studies

Global Morquio Syndrome Treatment Market Size by Treatment & CAGR (2026-2033)

• Market Overview
• Enzyme Replacement Therapy
• Others

Global Morquio Syndrome Treatment Market Size by Disease Type & CAGR (2026-2033)

• Market Overview
• Morquio A
• Morquio B

Global Morquio Syndrome Treatment Market Size by Distribution Channel & CAGR (2026-2033)

• Market Overview
• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies
• Others

Global Morquio Syndrome Treatment Market Size & CAGR (2026-2033)

• North America (Treatment, Disease Type, Distribution Channel)
  • US
  • Canada
• Europe (Treatment, Disease Type, Distribution Channel)
  • Germany
  • Spain
  • France
  • UK
  • Italy
  • Rest of Europe
• Asia Pacific (Treatment, Disease Type, Distribution Channel)
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Latin America (Treatment, Disease Type, Distribution Channel)
  • Brazil
  • Rest of Latin America
• Middle East & Africa (Treatment, Disease Type, Distribution Channel)
  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

Competitive Intelligence

• Top 5 Player Comparison
• Market Positioning of Key Players, 2025
• Strategies Adopted by Key Market Players
• Recent Developments in the Market
• Company Market Share Analysis, 2025
• Company Profiles of All Key Players
  • Company Details
  • Product Portfolio Analysis
  • Company's Segmental Share Analysis
  • Revenue Y-O-Y Comparison (2023-2025)

Key Company Profiles

• BioMarin Pharmaceuticals Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Genzyme Corporation (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Shire plc (Ireland)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• JCR Pharmaceuticals Co., Ltd. (Japan)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sanofi (France)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sangamo Therapeutics (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• ArmaGen, Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• GREEN CROSS CORP (South Korea)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Concert Pharmaceuticals Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Alexion Pharmaceuticals Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Novo Nordisk A/S (Denmark)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Eli Lilly and Company (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Boehringer Ingelheim International GmbH (Germany)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AstraZeneca (UK)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amgen Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Merck KGaA (Germany)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Actelion Pharmaceuticals Ltd. (Switzerland)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Biocon (India)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Ultragenyx Pharmaceutical Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Enzyvant Therapeutics Inc. (US)
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments

Conclusion & Recommendations