全球粘多糖贮积症市场 – 行业趋势和 2031 年预测

粘多糖贮积症市场分析及规模

在粘多糖贮积症 (MPS) 市场中，研发 (R&D) 在促进对这些罕见遗传疾病的了解和治疗方案方面发挥着至关重要的作用。制药公司和研究机构专注于研发创新疗法，包括酶替代疗法(ERT)、基因疗法和小分子疗法，旨在解决 MPS 患者潜在的生化缺陷。研发工作还扩展到改进诊断工具，以便尽早准确检测 MPS 亚型，以及探索新的生物标志物和疾病途径。

2023 年全球粘多糖贮积症市场规模价值 22.7 亿美元，预计到 2031 年将达到 44.9 亿美元，2024 年至 2031 年预测期内的复合年增长率为 8.90%。

除了对市场价值、增长率、细分、地理覆盖范围和主要参与者等市场情景的洞察之外，Data Bridge Market Research 策划的市场报告还包括深度专家分析、患者流行病学、管道分析、定价分析和监管框架。

报告范围和市场细分       

Market Definition

Mucopolysaccharidosis (MPS) refers to a group of inherited metabolic disorders caused by the absence or malfunctioning of enzymes needed to break down complex sugars called glycosaminoglycans (GAGs). This results in the accumulation of GAGs in cells throughout the body, leading to progressive damage to organs and tissues. MPS can manifest in various forms with a range of symptoms affecting physical appearance, organ function, and cognitive abilities, depending on the specific type and severity of the disorder.

Mucopolysaccharidosis Market Dynamics

Drivers                                                                                          

• Growing Awareness among Healthcare Providers

Awareness among healthcare providers leads to improved detection, diagnosis, and management of these rare genetic disorders. Increased understanding of the diverse clinical presentations of MPS among healthcare professionals enhances their ability to identify symptoms early, reducing diagnostic delays and ensuring timely intervention. This awareness also promotes multidisciplinary collaboration among specialists, fostering comprehensive care pathways tailored to the specific needs of MPS patients. Moreover, heightened awareness encourages healthcare providers to stay updated on the latest advancements in MPS therapies and guidelines, facilitating optimal treatment decisions and improving patient outcomes.

• Rising Advancements in Therapeutics

Rising advancements in therapeutics expands treatment options for these rare genetic disorders. Key developments include the introduction of enzyme replacement therapies (ERTs) that replace deficient enzymes, thereby mitigating disease progression and improving quality of life for MPS patients. Additionally, ongoing research into novel treatments such as substrate reduction therapies (SRTs) and gene therapies holds promise for addressing underlying biochemical defects more effectively. These advancements enhance therapeutic efficacy and broaden the market by offering new options for patients previously underserved by traditional treatments.

Opportunities

• Growing Supportive Regulatory Environment

Regulatory agencies, such as the FDA in the U.S. and the EMA in Europe, often provide incentives and pathways for orphan drug designation, fast-track approvals, and priority review status for therapies targeting rare diseases such as MPS. These regulatory incentives reduce the time and cost of bringing new treatments to market, encouraging pharmaceutical companies to invest in research and development. Additionally, regulatory support ensures that MPS patients have timely access to innovative therapies, fostering market growth and improving patient outcomes by addressing significant unmet medical needs in rare disease management.

• Rising Healthcare Expenditure

Rising healthcare expenditure impacts access to advanced diagnostic tools, therapeutic options, and comprehensive patient care. Increased healthcare funding enables healthcare systems to invest in specialized infrastructure, such as genetic testing facilities and multidisciplinary clinics, essential for early detection and management of MPS. Moreover, higher expenditure supports research and development initiatives focused on novel therapies, including enzyme replacement therapies (ERTs), gene therapies, and supportive care measures, which are crucial for improving outcomes and quality of life for MPS patients.

Restraints/Challenges

• High Treatment Costs

Therapies such as enzyme replacement therapies (ERTs) and emerging gene therapies are costly to develop, manufacture, and administer, often leading to substantial financial burdens for healthcare systems, patients, and insurers. The small patient populations for each MPS subtype further exacerbate this issue by limiting economies of scale that could potentially lower treatment costs. Moreover, the lifelong nature of MPS treatment necessitates ongoing financial commitment, placing strain on families and healthcare budgets.

• Complexity of Treating Mucopolysaccharidosis (MPS)

MPS disorders are characterized by a wide spectrum of symptoms affecting multiple organ systems, requiring a multidisciplinary approach to management. Treatment often involves lifelong therapies such as enzyme replacement therapies (ERTs), substrate reduction therapies (SRTs), and in some cases, hematopoietic stem cell transplantation or emerging gene therapies. Coordinating these treatments and managing their side effects can be challenging for healthcare providers and burdensome for patients and caregivers. Moreover, the high costs associated with these complex treatment regimens add financial strain, limiting accessibility and adoption, particularly in regions with limited healthcare resources.

本市场报告详细介绍了最新发展、贸易法规、进出口分析、生产分析、价值链优化、市场份额、国内和本地市场参与者的影响，分析了新兴收入领域的机会、市场法规的变化、战略市场增长分析、市场规模、类别市场增长、应用领域和主导地位、产品批准、产品发布、地域扩展、市场技术创新。如需获取更多市场信息，请联系 Data Bridge Market Research 获取分析师简报，我们的团队将帮助您做出明智的市场决策，实现市场增长。

粘多糖贮积症市场范围

市场根据治疗、疾病类型、给药途径和最终用户进行细分。这些细分市场之间的增长情况将帮助您分析行业中增长缓慢的细分市场，并为用户提供有价值的市场概览和市场洞察，帮助他们做出战略决策，确定核心市场应用。

治疗

• 酶替代疗法
• 干细胞治疗
  • 骨髓移植
  • 脐带血移植

疾病类型

• 粘多糖贮积症 I 型
• 粘多糖贮积症 II 型
• IV A型粘多糖贮积症
• 粘多糖贮积症 VI 型
• 粘多糖贮积症 VII 型
• 其他的

给药途径

• 静脉
• 脑室内 (ICV)

最终用户

• 医院
• 专科诊所
• 其他的

粘多糖贮积症市场区域分析/见解

对市场进行分析，并按国家、治疗、疾病、给药途径和最终用户提供市场规模信息，如上所述。

市场报告涉及的国家包括美国、加拿大、墨西哥、巴西、阿根廷、秘鲁、南美洲其他地区、德国、法国、英国、荷兰、瑞士、比利时、俄罗斯、意大利、西班牙、土耳其、匈牙利、立陶宛、奥地利、爱尔兰、挪威、波兰、欧洲其他地区、中国、日本、印度、韩国、新加坡、马来西亚、澳大利亚、泰国、印度尼西亚、菲律宾、越南、亚太其他地区、沙特阿拉伯、阿联酋、埃及、以色列、科威特、南非、中东和非洲其他地区。

由于监管机构批准的新产品数量增加，北美占据了市场主导地位。这一趋势增强了治疗选择，更有效地满足了未满足的医疗需求。该地区受益于强大的监管框架，这些框架简化了审批流程，促进了创新和新疗法的快速商业化。此外，北美强大的医疗基础设施和研究生态系统吸引了大量投资，推动了药物开发的进步，并确保各个治疗领域的患者能够及时获得尖端治疗。

由于技术进步和医疗支出增加，亚太地区预计将在市场上占据主导地位。随着亚太地区各国继续投资医疗基础设施并采用先进的医疗技术，MPS 等罕见疾病的诊断和治疗预计将显著改善。医疗服务提供者和患者对遗传疾病的认识不断提高，再加上政府提高医疗可及性的举措，进一步助长了这一趋势。总体而言，这些因素使亚太地区在可预见的未来成为 MPS 市场的关键增长区域。

报告的国家部分还提供了影响单个市场因素和国内市场监管变化，这些因素和变化会影响市场的当前和未来趋势。下游和上游价值链分析、技术趋势和波特五力分析、案例研究等数据点是用于预测单个国家市场情景的一些指标。此外，在提供国家数据的预测分析时，还考虑了全球品牌的存在和可用性以及它们因来自本地和国内品牌的大量或稀缺竞争而面临的挑战、国内关税和贸易路线的影响。

医疗保健基础设施增长安装基础和新技术渗透

该市场还为您提供每个国家/地区资本设备医疗支出增长、市场上不同类型产品的安装基数、使用生命线曲线的技术影响以及医疗监管情景的变化及其对市场的影响的详细市场分析。数据适用于 2016-2021 年的历史时期。

竞争格局和粘多糖贮积症市场分析

市场竞争格局按竞争对手提供详细信息。详细信息包括公司概况、公司财务状况、产生的收入、市场潜力、研发投资、新市场计划、全球影响力、生产基地和设施、生产能力、公司优势和劣势、产品发布、产品宽度和广度、应用主导地位。以上提供的数据点仅与公司对市场的关注有关。

市场上的一些主要参与者包括：

• 武田药品工业株式会社 (日本)
• BioMarin (美国)
• Ultragenyx Pharmaceutical（美国）
• Sarepta Therapeutics（美国）
• Inventiva（法国）
• GC Pharma（韩国）
• JCR Pharmaceuticals Co Ltd.（日本）
• REGENXBIO Inc.（美国）
• Sangamo Therapeutics（美国）
• Denali Therapeutics（美国）
• Bioasis Technologies Inc.（加拿大）
• Paradigm Biopharmaceuticals Ltd.（澳大利亚）

SKU-59294