全球低磷酸血症市場：區域和國家分析和預測（2025-2035）

低磷酸血症 (HPP) 市場的主要成長要素之一是標靶治療的需求不斷增加，主要是酵素替代療法 (ERT)，例如 Strensiq (asfotase alfa)。

需求的成長歸因於多種因素。首先，基因檢測的進步使得能夠早期發現HPP，從而能夠及時干預並更好地管理疾病。快速診斷可達到早期治療，進而改善患者的預後。此外，對罕見疾病的監管支持（孤兒藥指定和各種獎勵）正在推動治療HPP等疾病的藥物的開發和商業化。各國政府推出了優惠政策以促進新治療方法的核准，從而改善了患者獲得治療的機會。此外，醫療服務提供者和患者對HPP的認知不斷提高也支持了這種需求的成長。越來越多的患者被診斷出來並尋求有效治療，這正在擴大市場。此外，專注於罕見疾病的專科治療中心的發展改善了專科護理的可及性，使患者能夠接受先進的治療和綜合管理。這些因素的結合為HPP治療市場的發展和成長創造了有利環境，凸顯了標靶治療的關鍵作用。

然而，低磷酸血症市場仍面臨許多挑戰。其中最大的挑戰之一是酵素替代療法 (ERT) 的高昂成本，例如 Strensiq (asfotase alfa)。雖然這些治療方法療效顯著，但價格昂貴，難以取得，尤其對於資源有限且保險不足的患者而言。治療低磷酸血症 (HPP) 等慢性疾病的長期成本給患者和醫療保健系統都帶來了沉重的經濟負擔。這些成本障礙可能導致治療延誤和護理品質低下，最終對患者的健康狀況產生負面影響。此外，疾病的罕見性也為研發帶來了障礙，使得替代治療方法和低成本治療方案難以出現。

全球HPP市場競爭激烈，幾家大型企業推動創新和市場成長。阿斯特捷利康透過收購Alexion Pharmaceuticals在該領域發揮關鍵作用，為其提供Strensiq（asfotase alfa），這是首個也是唯一一個核准治療重度HPP的酵素替代療法。 Mereo BioPharma是另一家專注於罕見疾病的公司，在HPP領域的參與度有限但引人注目。安進是骨骼健康和代謝疾病領域的領導企業，目前持有針對HPP的特效療法，但有潛力進入該領域。 BridgeBio Pharma透過其子公司QED Therapeutics推進基因治療，雖然該公司主要專注於其他遺傳疾病，但預計未來將利用其在罕見疾病方面的專業知識，開發HPP的潛在療法。這些公司在推動治療、滿足未滿足需求和改善患者預後方面發揮關鍵作用，使其成為快速成長的HPP市場的核心參與者。

本報告調查了全球低磷酸血症市場，並總結了主要趨勢、市場影響因素分析、法律制度、臨床試驗趨勢、市場規模趨勢和預測、各個細分市場、地區/主要國家、競爭格局的詳細分析以及主要企業的概況。

目錄

執行摘要

第1章全球低磷酸酯酶症市場：產業展望

• 市場趨勢
• 法律規範
• 流行病學分析
• 臨床試驗分析
• 市場動態
  • 影響分析
  • 市場促進因素
  • 市場挑戰
  • 市場機會

2. 全球低磷酸血症市場（按地區）

• 北美洲
  • 主要發現
  • 市場動態
  • 市場規模及預測
• 歐洲
  • 主要發現
  • 市場動態
  • 市場規模及預測
• 亞太地區
  • 主要發現
  • 市場動態
  • 市場規模及預測

3. 全球低磷酸血症市場：競爭格局與公司概況

• 主要策略和發展
  • M&A
  • 協同活動
  • 業務擴展和資金籌措
  • 產品發布和核准
  • 其他活動
• 公司簡介
  • AstraZeneca
  • Mereo BioPharma
  • Amgen
  • Therachon (BridgeBio Pharma)

第4章調查方法

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