全球低磷酸血症市场:区域和国家分析和预测(2025-2035)
市场调查报告书
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1759271

全球低磷酸血症市场:区域和国家分析和预测(2025-2035)

Hypophosphatasia Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035

出版日期: | 出版商: BIS Research | 英文 100 Pages | 商品交期: 1-5个工作天内

价格

低磷酸血症 (HPP) 市场的主要成长要素之一是标靶治疗的需求不断增加,主要是酵素替代疗法 (ERT),例如 Strensiq (asfotase alfa)。

需求的成长归因于多种因素。首先,基因检测的进步使得能够早期发现HPP,从而能够及时干预并更好地管理疾病。快速诊断可达到早期治疗,进而改善患者的预后。此外,对罕见疾病的监管支持(孤儿药指定和各种奖励)正在推动治疗HPP等疾病的药物的开发和商业化。各国政府推出了优惠政策以促进新治疗方法的核准,从而改善了患者获得治疗的机会。此外,医疗服务提供者和患者对HPP的认识不断提高也支持了这种需求的成长。越来越多的患者被诊断出来并寻求有效治疗,这正在扩大市场。此外,专注于罕见疾病的专科治疗中心的发展改善了专科护理的可及性,使患者能够接受先进的治疗和综合管理。这些因素的结合为HPP治疗市场的发展和成长创造了有利环境,凸显了标靶治疗的关键作用。

然而,低磷酸血症市场仍面临许多挑战。其中最大的挑战之一是酵素替代疗法 (ERT) 的高昂成本,例如 Strensiq (asfotase alfa)。虽然这些治疗方法疗效显着,但价格昂贵,难以取得,尤其对于资源有限且保险不足的患者而言。治疗低磷酸血症 (HPP) 等慢性疾病的长期成本给患者和医疗保健系统都带来了沉重的经济负担。这些成本障碍可能导致治疗延误和护理品质低下,最终对患者的健康状况产生负面影响。此外,疾病的罕见性也为研发带来了障碍,使得替代治疗方法和低成本治疗方案难以出现。

全球HPP市场竞争激烈,几家大型企业推动创新和市场成长。阿斯特捷利康透过收购Alexion Pharmaceuticals在该领域发挥关键作用,为其提供Strensiq(asfotase alfa),这是首个也是唯一一个核准治疗重度HPP的酵素替代疗法。 Mereo BioPharma是另一家专注于罕见疾病的公司,在HPP领域的参与度有限但引人注目。安进是骨骼健康和代谢疾病领域的领导企业,目前持有针对HPP的特效疗法,但有潜力进入该领域。 BridgeBio Pharma透过其子公司QED Therapeutics推进基因治疗,虽然该公司主要专注于其他遗传疾病,但预计未来将利用其在罕见疾病方面的专业知识,开发HPP的潜在疗法。这些公司在推动治疗、满足未满足需求和改善患者预后方面发挥关键作用,使其成为快速成长的HPP市场的核心参与者。

本报告调查了全球低磷酸血症市场,并总结了主要趋势、市场影响因素分析、法律制度、临床试验趋势、市场规模趋势和预测、各个细分市场、地区/主要国家、竞争格局的详细分析以及主要企业的概况。

目录

执行摘要

第一章全球低磷酸酯酶症市场:产业展望

  • 市场趋势
  • 法律规范
  • 流行病学分析
  • 临床试验分析
  • 市场动态
    • 影响分析
    • 市场驱动因素
    • 市场挑战
    • 市场机会

2. 全球低磷酸血症市场(按地区)

  • 北美洲
    • 主要发现
    • 市场动态
    • 市场规模及预测
  • 欧洲
    • 主要发现
    • 市场动态
    • 市场规模及预测
  • 亚太地区
    • 主要发现
    • 市场动态
    • 市场规模及预测

3. 全球低磷酸血症市场:竞争格局与公司概况

  • 主要策略和发展
    • M&A
    • 协同活动
    • 业务扩展和资金筹措
    • 产品发布和核准
    • 其他活动
  • 公司简介
    • AstraZeneca
    • Mereo BioPharma
    • Amgen
    • Therachon (BridgeBio Pharma)

第四章调查方法

Product Code: BHL3076SA

Global Hypophosphatasia Market, Analysis and Forecast: 2025-2035

Hypophosphatasia is a rare genetic disorder caused by a deficiency in the enzyme alkaline phosphatase, which is essential for bone and tooth mineralization. This deficiency leads to weakened bones, skeletal deformities, and premature loss of teeth. The severity of hypophosphatasia varies, with the perinatal and infantile forms being life-threatening, while the childhood and adult forms are milder but still cause symptoms such as fractures, bone pain, and growth delays. Early diagnosis, often through blood tests and genetic testing, is crucial for effective treatment. The primary treatment for hypophosphatasia is enzyme replacement therapy, with Strensiq (asfotase alfa) being the most common option. This therapy helps restore enzyme levels, improving bone health and reducing fractures. While there is no cure for hypophosphatasia, advancements in treatment have significantly improved the quality of life for individuals with this condition, especially when diagnosed and treated early.

One of the key drivers of the Hypophosphatasia market is the increasing demand for targeted therapies, particularly enzyme replacement therapies (ERTs) like Strensiq (asfotase alfa). This growing demand is fueled by several factors. Advancements in genetic testing have enabled earlier detection of HPP, allowing for timely interventions and better disease management. The ability to diagnose HPP more quickly leads to improved patient outcomes through early treatment. Additionally, regulatory support for rare diseases, including orphan drug designations and incentives, has encouraged the development and commercialization of therapies for conditions like HPP. Governments have implemented favorable policies that facilitate the approval of new treatments, making them more accessible to patients. Increased disease awareness among healthcare providers and patients has further contributed to this demand, as more people are diagnosed and seek effective treatment options. Furthermore, the availability of specialized care centers dedicated to rare diseases has improved access to expert care, ensuring that patients receive advanced treatments and comprehensive management for HPP. Together, these factors have created a more favorable environment for the development and growth of the HPP treatment market, emphasizing the crucial role of targeted therapies in managing this rare and complex disease.

Despite the growth of the Hypophosphatasia market, several challenges continue to hinder its progress. One of the primary challenges is the high cost of treatment, particularly for enzyme replacement therapies (ERTs) such Strensiq (asfotase alfa). These therapies, while highly effective, are expensive, making access to treatment difficult for many patients, especially in regions with limited healthcare resources or without comprehensive insurance coverage. The high cost of long-term treatment for chronic conditions such hypophosphatasia can place a significant financial burden on both patients and healthcare systems. This barrier to access can result in delayed treatments or suboptimal care, ultimately impacting the outcomes for individuals with hypophosphatasia. Additionally, the rarity of the disease means that research and development in this field are limited, making it harder to introduce alternative therapies or lower-cost options.

The global Hypophosphatasia market is highly competitive, with several leading companies driving innovation and market growth. AstraZeneca, through its acquisition of Alexion Pharmaceuticals, is a major player, offering Strensiq (asfotase alfa), the first and only enzyme replacement therapy approved for severe forms of HYPOPHOSPHATASIA. Mereo BioPharma is another key player, with its focus on rare diseases, although its involvement in hypophosphatasia has been limited. Amgen, a leader in bone health and metabolic diseases, has the potential to expand its portfolio into the hypophosphatasia space, despite not currently offering a specific treatment for the condition. BridgeBio Pharma, through its subsidiary QED Therapeutics, is advancing genetic therapies, and while its primary focus is on other genetic diseases, its expertise in rare disorders could lead to future developments for hypophosphatasia. These companies are crucial in advancing treatments, addressing unmet medical needs, and enhancing patient outcomes in the rapidly growing hypophosphatasia market.

Market Segmentation:

Segmentation 1: by Region

  • North America
  • Europe
  • Asia-Pacific

The global Hypophosphatasia market is undergoing significant transformation, fueled by emerging trends that are enhancing treatment options and patient outcomes. Key developments include the introduction of next-generation enzyme replacement therapies (ERTs), such as ALXN1850 (efzimfotase alfa) by AstraZeneca, which offer improved dosing schedules and greater bioavailability compared to earlier treatments such Strensiq (asfotase alfa).

Additionally, advancements in genetic testing have led to earlier and more accurate diagnoses, enabling personalized treatment plans that are tailored to individual patient profiles. The increasing adoption of precision medicine is further driving the development of therapies that target the underlying genetic causes of hypophosphatasia, moving beyond symptom management to potential curative approaches. Furthermore, global initiatives aimed at expanding healthcare infrastructure and providing financial support for rare disease treatments are improving access to care in underserved regions, thereby broadening the patient base and fostering market growth.

Table of Contents

Executive Summary

Scope and Definition

Market/Product Definition

Inclusion and Exclusion

Key Questions Answered

Analysis and Forecast Note

1. Global Hypophosphatasia Market: Industry Outlook

  • 1.1 Introduction
  • 1.2 Market Trends
  • 1.3 Regulatory Framework
  • 1.4 Epidemiology Analysis
  • 1.5 Clinical Trial Analysis
  • 1.6 Market Dynamics
    • 1.6.1 Impact Analysis
    • 1.6.2 Market Drivers
    • 1.6.3 Market Challenges
    • 1.6.4 Market Opportunities

2. Global Hypophosphatasia Market (Region), ($Billion), 2023-2035

  • 2.1 North America
    • 2.1.1 Key Findings
    • 2.1.2 Market Dynamics
    • 2.1.3 Market Sizing and Forecast
      • 2.1.3.1 North America Hypophosphatasia Market, by Country
        • 2.1.3.1.1 U.S.
  • 2.2 Europe
    • 2.2.1 Key Findings
    • 2.2.2 Market Dynamics
    • 2.2.3 Market Sizing and Forecast
      • 2.2.3.1 Europe Hypophosphatasia Market, by Country
        • 2.2.3.1.1 Germany
        • 2.2.3.1.2 U.K.
        • 2.2.3.1.3 France
        • 2.2.3.1.4 Italy
  • 2.3 Asia Pacific
    • 2.3.1 Key Findings
    • 2.3.2 Market Dynamics
    • 2.3.3 Market Sizing and Forecast
      • 2.3.3.1 Asia Pacific Hypophosphatasia Market, by Country
        • 2.3.3.1.1 China
        • 2.3.3.1.2 Japan

3. Global Hypophosphatasia Market: Competitive Landscape and Company Profiles

  • 3.1 Key Strategies and Development
    • 3.1.1 Mergers and Acquisitions
    • 3.1.2 Synergistic Activities
    • 3.1.3 Business Expansions and Funding
    • 3.1.4 Product Launches and Approvals
    • 3.1.5 Other Activities
  • 3.2 Company Profiles
    • 3.2.1 AstraZeneca
      • 3.2.1.1 Overview
      • 3.2.1.2 Top Products / Product Portfolio
      • 3.2.1.3 Top Competitors
      • 3.2.1.4 Target Customers/End-Users
      • 3.2.1.5 Key Personnel
      • 3.2.1.6 Analyst View
    • 3.2.2 Mereo BioPharma
      • 3.2.2.1 Overview
      • 3.2.2.2 Top Products / Product Portfolio
      • 3.2.2.3 Top Competitors
      • 3.2.2.4 Target Customers/End-Users
      • 3.2.2.5 Key Personnel
      • 3.2.2.6 Analyst View
    • 3.2.3 Amgen
      • 3.2.3.1 Overview
      • 3.2.3.2 Top Products / Product Portfolio
      • 3.2.3.3 Top Competitors
      • 3.2.3.4 Target Customers/End-Users
      • 3.2.3.5 Key Personnel
      • 3.2.3.6 Analyst View
    • 3.2.4 Therachon (BridgeBio Pharma)
      • 3.2.4.1 Overview
      • 3.2.4.2 Top Products / Product Portfolio
      • 3.2.4.3 Top Competitors
      • 3.2.4.4 Target Customers/End-Users
      • 3.2.4.5 Key Personnel
      • 3.2.4.6 Analyst View

4. Research Methodology

List of Figures

  • Figure: Global Hypophosphatasia Market (by Region), $Billion, 2024 and 2035
  • Figure: Global Hypophosphatasia Market Key Trends, Analysis

List of Tables

  • Table: Global Hypophosphatasia Market Dynamics, Impact Analysis
  • Table: Global Hypophosphatasia Market (by Region), $Billion, 2024-2035