骨骼發育不良藥物市場 - 按發育不良類型、治療、配銷通路- 全球預測

由於骨骼發育不良的盛行率和診斷率不斷上升，預計 2024 年至 2032 年間，骨骼發育不良藥物市場規模的複合年成長率將達到 4.2%。據報道，非致命性骨骼發育不良的發生率約為 26,000 分之一至 28,000 分之一，影響全球約 25 萬人。

基因研究的進步和診斷技術的改進使得這些罕見疾病的識別更加準確和早期，從而導致對標靶治療的更高需求。醫療保健專業人員和公眾的認知和理解不斷增強，也有助於提高診斷率，進一步推動對專業治療的需求。製藥公司也正在投資臨床試驗並開發針對這些疾病的潛在遺傳原因的新型候選藥物。政府的激勵措施，例如孤兒藥指定和撥款，正在進一步支持新療法的開發和商業化。

本行業分為不典型增生類型、治療方法、配銷通路和地區。

基於治療，預計到 2032 年，人類單株抗體領域的骨骼發育不良市場將出現大幅複合年成長率，因為它有可能為這些複雜的遺傳性疾病提供有針對性的、有效的治療。單株抗體具有高度特異性，使其能夠結合併中和參與骨骼發育不良發病機制的特定蛋白質或細胞。與傳統治療相比，這種精確度可以改善治療效果並減少副作用。

就配銷通路而言，醫院藥房部門的骨骼發育不良市場將在 2024 年至 2032 年間呈指數級成長，因為它們在罕見疾病專門治療的實施和管理中發揮關鍵作用。醫院藥房有能力處理複雜且昂貴的藥物，為患者提供量身定做的藥物治療方案，同時確保正確的劑量和監測。

預計亞太地區骨骼發育不良產業將在 2024 年至 2032 年期間顯著成長。這一成長歸因於人們意識的提高、醫療基礎設施的改善以及對罕見疾病研究的投資不斷增加。中國、印度和日本等國家醫療保健設施的擴張和專業醫療服務的提供正在改善先進療法的取得。通常由政府措施和私部門資金支持的研發活動投資也將有利於區域市場的成長。

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
    • 骨骼發育不良盛行率增加
    • 提高認知和篩檢
    • 醫療保健支出增加
    • 老年人口增加
  • 產業陷阱與挑戰
    • 治療費用高
    • 監管和報銷挑戰
• 成長潛力分析
• 技術景觀
• 監管環境
• 波特的分析
• PESTEL分析

第 4 章：競爭格局

• 介紹
• 公司市佔率分析
• 競爭定位矩陣
• 戰略展望矩陣

第 5 章：市場估計與預測：按發育異常類型，2021 - 2032 年

• 主要趨勢
• 莫爾基奧A症候群
• X連鎖低血磷症
• 低磷酸酯酶症
• 軟骨發育不全
• 進行性骨化性纖維發育不良
• 多發性骨軟骨瘤
• 其他發育不良類型

第 6 章：市場估計與預測：按處理方式，2021 - 2032 年

• 主要趨勢
• 酵素替代療法
• 人類單株抗體
• 其他治療

第 7 章：市場估計與預測：按配銷通路分類，2021 - 2032 年

• 主要趨勢
• 醫院藥房
• 零售藥局
• 網路藥局

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
  • 日本
  • 中國
  • 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
  • 沙烏地阿拉伯
  • 南非
  • 阿拉伯聯合大公國
  • 中東和非洲其他地區

第 9 章：公司簡介

• AbbVie Inc.
• Alexion Pharmaceuticals, Inc.
• Amgen Inc.
• BioMarin Pharmaceutical Inc.
• Horizon Therapeutics plc
• Johnson & Johnson
• Mallinckrodt Pharmaceuticals
• Novartis AG
• Pfizer Inc.
• Regeneron Pharmaceuticals, Inc.
• Sanofi
• Sarepta Therapeutics, Inc.
• Takeda Pharmaceutical Company Limited
• Ultragenyx Pharmaceutical Inc.
• Vertex Pharmaceuticals Incorporated

Skeletal dysplasia drugs market size is projected to witness 4.2% CAGR between 2024 and 2032 driven by the increasing prevalence and diagnosis of skeletal dysplasia. As per reports, non-lethal skeletal dysplasia occurs in approximately 1 in 26,000 to 1 in 28,000 live births, impacting around 250,000 people worldwide.

Advancements in genetic research and improved diagnostic techniques have resulted in more accurate and early identification of these rare conditions, leading to the higher demand for targeted therapies. The growing awareness and understanding among healthcare professionals and the public are also contributing to increased diagnosis rates, further driving the need for specialized treatments. Pharmaceutical companies are also investing in clinical trials and developing novel drug candidates that target the underlying genetic causes of these conditions. Government incentives, such as orphan drug designations and grants, are further supporting the development and commercialization of new therapies.

The industry is divided into dysplasia type, treatment, distribution channel, and region.

Based on treatment, the skeletal dysplasia market from the human monoclonal antibody segment is projected to observe substantial CAGR through 2032 due to its potential to provide targeted, effective treatments for these complex genetic disorders. Monoclonal antibodies offer a high degree of specificity, allowing them to bind to and neutralize specific proteins or cells involved in the pathogenesis of skeletal dysplasia. This precision can lead to improved therapeutic outcomes and reduced side effects compared to traditional treatments.

With respect to distribution channel, the skeletal dysplasia market from the hospital pharmacies segment will expand exponentially between 2024 and 2032 owing to their critical role in the administration and management of specialized treatments for rare conditions. Hospital pharmacies are equipped to handle complex as well as costly medications to provide tailored drug regimens for patients while ensuring proper dosing and monitoring.

Asia Pacific skeletal dysplasia industry is anticipated to grow at a notable pace over 2024-2032. The growth is attributed to the increasing awareness, improving healthcare infrastructure, and the rising investments in rare disease research. The expansion of healthcare facilities and the availability of specialized medical services in countries like China, India, and Japan are improving access to advanced therapies. Investments in R&D activities, often supported by government initiatives and private sector funding will also favor the regional market growth.

Table of Contents

Chapter 1 Methodology & Scope

• 1.1 Market scope & definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates & 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 skeletal dysplasia
    • 3.2.1.2 Increasing awareness and screening
    • 3.2.1.3 Rising healthcare expenditure
    • 3.2.1.4 Increasing geriatric population
  • 3.2.2 Industry pitfalls & challenges
    • 3.2.2.1 High cost of therapy
    • 3.2.2.2 Regulatory and reimbursement challenges
• 3.3 Growth potential analysis
• 3.4 Technological landscape
• 3.5 Regulatory landscape
• 3.6 Porter's analysis
• 3.7 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company market share analysis
• 4.3 Competitive positioning matrix
• 4.4 Strategy outlook matrix

Chapter 5 Market Estimates and Forecast, By Dysplasia Type, 2021 - 2032 ($ Mn)

• 5.1 Key trends
• 5.2 Morquio A syndrome
• 5.3 X-linked hypophosphatemia
• 5.4 Hypophosphatasia
• 5.5 Achondroplasia
• 5.6 Fibrodysplasia ossificans progressive
• 5.7 Multiple osteochondromas
• 5.8 Other dysplasia types

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

• 6.1 Key trends
• 6.2 Enzyme replacement therapy
• 6.3 Human monoclonal antibody
• 6.4 Other treatments

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

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

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 Japan
  • 8.4.2 China
  • 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 Saudi Arabia
  • 8.6.2 South Africa
  • 8.6.3 UAE
  • 8.6.4 Rest of Middle East and Africa

Chapter 9 Company Profiles

• 9.1 AbbVie Inc.
• 9.2 Alexion Pharmaceuticals, Inc.
• 9.3 Amgen Inc.
• 9.4 BioMarin Pharmaceutical Inc.
• 9.5 Horizon Therapeutics plc
• 9.6 Johnson & Johnson
• 9.7 Mallinckrodt Pharmaceuticals
• 9.8 Novartis AG
• 9.9 Pfizer Inc.
• 9.10 Regeneron Pharmaceuticals, Inc.
• 9.11 Sanofi
• 9.12 Sarepta Therapeutics, Inc.
• 9.13 Takeda Pharmaceutical Company Limited
• 9.14 Ultragenyx Pharmaceutical Inc.
• 9.15 Vertex Pharmaceuticals Incorporated