骨骼发育不良药物市场 - 按发育不良类型、治疗、配销通路- 全球预测

由于骨骼发育不良的盛行率和诊断率不断上升，预计 2024 年至 2032 年间，骨骼发育不良药物市场规模的复合年增长率将达到 4.2%。据报道，非致命性骨骼发育不良的发生率约为 26,000 分之一至 28,000 分之一，影响全球约 25 万人。

基因研究的进步和诊断技术的改进使得这些罕见疾病的识别更加准确和早期，从而导致对标靶治疗的更高需求。医疗保健专业人员和公众的认识和理解不断增强，也有助于提高诊断率，进一步推动对专业治疗的需求。製药公司也正在投资临床试验并开发针对这些疾病的潜在遗传原因的新型候选药物。政府的激励措施，例如孤儿药指定和拨款，正在进一步支持新疗法的开发和商业化。

本行业分为不典型增生类型、治疗方法、配销通路和地区。

基于治疗，预计到 2032 年，人类单株抗体领域的骨骼发育不良市场将出现大幅复合年增长率，因为它有可能为这些复杂的遗传性疾病提供有针对性的、有效的治疗。单株抗体具有高度特异性，使其能够结合併中和参与骨骼发育不良发病机制的特定蛋白质或细胞。与传统治疗相比，这种精确度可以改善治疗效果并减少副作用。

就配销通路而言，医院药房部门的骨骼发育不良市场将在 2024 年至 2032 年间呈指数级增长，因为它们在罕见疾病专门治疗的实施和管理中发挥关键作用。医院药房有能力处理复杂且昂贵的药物，为患者提供量身定制的药物治疗方案，同时确保正确的剂量和监测。

预计亚太地区骨骼发育不良产业将在 2024 年至 2032 年期间显着成长。这一增长归因于人们意识的提高、医疗基础设施的改善以及对罕见疾病研究的投资不断增加。中国、印度和日本等国家医疗保健设施的扩张和专业医疗服务的提供正在改善先进疗法的取得。通常由政府措施和私部门资金支持的研发活动投资也将有利于区域市场的成长。

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • 骨骼发育不良盛行率增加
    • 提高认识和筛检
    • 医疗保健支出增加
    • 老年人口增加
  • 产业陷阱与挑战
    • 治疗费用高
    • 监管和报销挑战
• 成长潜力分析
• 技术景观
• 监管环境
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司市占率分析
• 竞争定位矩阵
• 战略展望矩阵

第 5 章：市场估计与预测：按发育异常类型，2021 - 2032 年

• 主要趋势
• 莫尔基奥A症候群
• X连锁低血磷症
• 低磷酸酯酶症
• 软骨发育不全
• 进行性骨化性纤维发育不良
• 多发性骨软骨瘤
• 其他发育不良类型

第 6 章：市场估计与预测：按处理方式，2021 - 2032 年

• 主要趋势
• 酵素替代疗法
• 人类单株抗体
• 其他治疗

第 7 章：市场估计与预测：按配销通路划分，2021 - 2032 年

• 主要趋势
• 医院药房
• 零售药局
• 网路药局

第 8 章：市场估计与预测：按地区，2021 - 2032

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 荷兰
  • 欧洲其他地区
• 亚太地区
  • 日本
  • 中国
  • 印度
  • 澳洲
  • 韩国
  • 亚太地区其他地区
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
  • 拉丁美洲其他地区
• 中东和非洲
  • 沙乌地阿拉伯
  • 南非
  • 阿联酋
  • 中东和非洲其他地区

第 9 章：公司简介

• 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