龐貝氏症治療市場 - 全球產業規模、佔有率、趨勢、機會和預測，按治療類型、給藥途徑、最終用戶、地區和競爭進行細分，2020-2030 年預測

2024 年全球龐貝氏症治療市場價值為 15 億美元，預計在預測期內將保持穩定成長，到 2030 年的複合年成長率為 4.40%。全球龐貝氏症治療市場是罕見疾病和孤兒藥產業中一個快速發展的領域，其驅動力來自科學進步和對創新療法的投資不斷增加。龐貝氏症，又稱 II 型肝醣累積病，是一種罕見的溶小體疾病，由酸性 α-葡萄糖苷酶 (GAA) 缺乏引起，導致肌肉組織中肝醣堆積。這會導致肌肉無力和嚴重的呼吸併發症，需要有針對性的治療介入。

市場主要受到酵素替代療法 (ERT) 的影響，例如 Myozyme (alglucosidase alfa)、Lumizyme 和較新的 Nexviazyme (avalglucosidase alfa)，它們可提高肝醣清除率並改善臨床效果。此外，2023 年 Pombiliti（cipaglucosidase alfa）和 Opfolda（miglustat）的批准擴大了患者的治療選擇，特別是那些對傳統 ERT 反應不佳的晚髮型龐貝病 (LOPD) 患者。

儘管取得了這些進步，但由於治療成本高且新生兒篩檢計畫有限，市場滲透率受到限制，導致診斷延遲和病情進展。然而，基因療法和下一代療法正在成為潛在的突破，正在進行的臨床試驗旨在提供更持久、更具成本效益的治療替代方案。

主要市場促進因素

提高疾病意識和診斷

主要市場挑戰

疾病認知有限且診斷較晚

主要市場趨勢

個人化醫療和精準治療

目錄

第 1 章：產品概述

第 2 章：研究方法

第3章：執行摘要

第4章：顧客之聲

第5章：龐貝氏症治療市場展望

• 市場規模和預測
  • 按價值
• 市場佔有率和預測
  • 依治療類型（藥物、酵素替代療法、物理治療、陪伴-高級替代療法）
  • 依給藥途徑（口服、腸胃外）
  • 按最終使用者（醫院和專科診所，其他）
  • 按地區
  • 按公司分類（2024）
• 市場地圖

第6章：北美龐貝氏症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 北美：國家分析
  • 加拿大
  • 墨西哥

第7章：歐洲龐貝氏症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 歐洲：國家分析
  • 英國
  • 義大利
  • 法國
  • 西班牙

第8章：亞太龐貝氏症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 亞太地區：國家分析
  • 印度
  • 日本
  • 韓國
  • 澳洲

第 9 章：南美洲龐貝氏症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• 南美洲：國家分析
  • 阿根廷
  • 哥倫比亞

第 10 章：中東和非洲龐貝氏症治療市場展望

• 市場規模和預測
• 市場佔有率和預測
• MEA：國家分析
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國

第 11 章：市場動態

• 驅動程式
• 挑戰

第 12 章：市場趨勢與發展

• 最新動態
• 產品發布
• 合併與收購

第 13 章：全球龐貝氏症治療市場：SWOT 分析

第 14 章：競爭格局

• Genzyme Corp
• Amicus Therapeutics Inc
• Valerion Therapeutics LLC
• Audentes Therapeutics Inc
• Actus Therapeutics Inc
• BioMarin Pharmaceutical Inc
• EpiVax inc
• Oxyrane Co Ltd.
• Sangamo BioSciences Inc
• Avrobio Inc
• Spark Therapeutics Inc.

第 15 章：策略建議

第16章調查會社について,免責事項

Global Pompe Disease Therapeutics Market was valued at USD 1.50 Billion in 2024 and is anticipated to project steady growth in the forecast period with a CAGR of 4.40% through 2030. The Global Pompe Disease Therapeutics Market is a rapidly evolving segment within the rare disease and orphan drug industry, driven by scientific advancements and increasing investment in innovative therapies. Pompe disease, or glycogen storage disease type II, is a rare lysosomal disorder caused by the deficiency of acid alpha-glucosidase (GAA), leading to glycogen accumulation in muscle tissues. This results in progressive muscle weakness and severe respiratory complications, necessitating targeted therapeutic interventions.

The market is primarily shaped by enzyme replacement therapies (ERTs) such as Myozyme (alglucosidase alfa), Lumizyme, and the more recent Nexviazyme (avalglucosidase alfa), which offer improved glycogen clearance and clinical outcomes. Additionally, the approval of Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) in 2023 has expanded treatment options for patients, particularly those with late-onset Pompe disease (LOPD) who exhibit suboptimal responses to conventional ERTs.

Despite these advancements, market penetration is constrained by high treatment costs and limited newborn screening programs, leading to delayed diagnoses and disease progression. However, gene therapy and next-generation therapeutics are emerging as potential breakthroughs, with ongoing clinical trials aiming to provide more durable and cost-effective treatment alternatives.

Key Market Drivers

Increased Disease Awareness and Diagnosis

Pompe disease, often considered a rare or orphan disease, historically suffered from a lack of recognition and understanding. Pompe disease is a complex, multisystem genetic disorder with a prevalence of approximately 1 in 40,000 individuals in the United States. Increasing awareness of this rare condition is crucial, as early diagnosis and timely intervention can significantly improve patient outcomes and quality of life. Efforts to enhance disease recognition are being driven by medical institutions, patient advocacy groups, and biotech companies focused on expanding access to diagnostics and treatment. Newborn screening programs, physician education initiatives, and targeted awareness campaigns are playing a pivotal role in identifying patients earlier and facilitating access to advanced therapies. Patients faced delayed or misdiagnoses, leading to prolonged suffering and a dearth of effective treatments. Advocacy groups dedicated to Pompe disease have played a pivotal role in driving disease awareness. These organizations tirelessly work to raise public awareness, educate healthcare professionals, and provide support to affected individuals and their families. By leveraging social media, organizing awareness campaigns, and collaborating with healthcare institutions, these groups have succeeded in putting Pompe disease on the radar. The evolution of diagnostic technologies is another catalyst for increased disease awareness. Cutting-edge tools such as genetic testing and biomarker identification have revolutionized the diagnostic process. Physicians can now identify Pompe disease more accurately and swiftly, enabling timely intervention and treatment initiation. This, in turn, has elevated the importance of early diagnosis in improving patient outcomes.

Key Market Challenges

Limited Disease Awareness and Late Diagnosis

One of the foremost challenges in the Pompe Disease Therapeutics Market is the limited awareness of the disease itself, leading to delayed or missed diagnoses. Pompe disease is considered a rare condition, making it less well-known among healthcare professionals and the general public. This lack of awareness often results in late-stage diagnoses, which can significantly impact treatment outcomes.

Pompe disease's rarity, with an estimated incidence of 1 in 40,000 to 1 in 300,000 births, contributes to its obscurity. As healthcare professionals encounter rare diseases less frequently, the likelihood of prompt recognition and diagnosis diminishes. Pompe disease presents with symptoms that can overlap with those of other more common conditions, such as muscle weakness and respiratory problems. This lack of distinctiveness can lead to misdiagnoses or a failure to consider Pompe disease as a potential cause.

Key Market Trends

Personalized Medicine and Precision Therapies

One of the most significant trends in the Pompe Disease Therapeutics Market is the shift towards personalized medicine and precision therapies. Traditional treatment approaches often took a one-size-fits-all approach, but advancements in genetics and diagnostics have paved the way for tailored treatments that address the specific genetic mutations of individual patients. Advances in genetic research have provided insights into the precise genetic mutations that cause Pompe disease. This knowledge allows for the development of therapies that target the specific genetic defects in individual patients, maximizing treatment efficacy. Pharmaceutical companies are investing in innovative approaches, such as gene therapy and RNA-based therapies, that can be customized to target specific genetic mutations. These therapies hold the potential to correct the underlying genetic causes of Pompe disease. Advanced diagnostic tools, including genetic testing and biomarker identification, enable healthcare providers to identify the specific genetic mutations in Pompe disease patients. This information guides treatment decisions, ensuring that therapies are tailored to each patient's unique genetic profile.

Key Market Players

• Genzyme Corp
• Amicus Therapeutics Inc
• Valerion Therapeutics LLC
• Audentes Therapeutics Inc
• Actus Therapeutics Inc
• BioMarin Pharmaceutical Inc
• EpiVax inc
• Oxyrane Co Ltd.
• Sangamo BioSciences Inc
• Avrobio Inc
• Spark Therapeutics Inc

Report Scope:

In this report, the Global Pompe Disease Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Pompe Disease Therapeutics Market, By Treatment Type:

• Drugs
• Enzyme Replacement Therapy
• Physical Therapy
• Chaperone-Advanced Replacement Therapy

Pompe Disease Therapeutics Market, By Route of Administration:

• Oral
• Parenteral

Pompe Disease Therapeutics Market, By End User:

• Hospitals & Speciality Clinics
• Other

Pompe Disease Therapeutics Market, By Region:

• North America
  • United States
  • Canada
  • Mexico
• Europe
  • France
  • United Kingdom
  • Italy
  • Germany
  • Spain
• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • South Korea
• South America
  • Brazil
  • Argentina
  • Colombia
• Middle East & Africa
  • South Africa
  • Saudi Arabia
  • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Pompe Disease Therapeutics Market.

Available Customizations:

Global Pompe Disease Therapeutics market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

• Detailed analysis and profiling of additional market players (up to five).

Table of Contents

1. Product Overview

• 1.1. Market Definition
• 1.2. Scope of the Market
  • 1.2.1. Markets Covered
  • 1.2.2. Years Considered for Study
  • 1.2.3. Key Market Segmentations

2. Research Methodology

• 2.1. Objective of the Study
• 2.2. Baseline Methodology
• 2.3. Key Industry Partners
• 2.4. Major Association and Secondary Sources
• 2.5. Forecasting Methodology
• 2.6. Data Triangulation & Validation
• 2.7. Assumptions and Limitations

3. Executive Summary

• 3.1. Overview of the Market
• 3.2. Overview of Key Market Segmentations
• 3.3. Overview of Key Market Players
• 3.4. Overview of Key Regions/Countries
• 3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Pompe Disease Therapeutics Market Outlook

• 5.1. Market Size & Forecast
  • 5.1.1. By Value
• 5.2. Market Share & Forecast
  • 5.2.1. By Treatment Type (Drugs, Enzyme Replacement Therapy, Physical Therapy, Chaperone-Advanced Replacement Therapy)
  • 5.2.2. By Route of Administration (Oral, Parenteral)
  • 5.2.3. By End User (Hospitals & Speciality Clinics, Other)
  • 5.2.4. By Region
  • 5.2.5. By Company (2024)
• 5.3. Market Map

6. North America Pompe Disease Therapeutics Market Outlook

• 6.1. Market Size & Forecast
  • 6.1.1. By Value
• 6.2. Market Share & Forecast
  • 6.2.1. By Treatment Type
  • 6.2.2. By Route of Administration
  • 6.2.3. By End User
  • 6.2.4. By Country
• 6.3. North America: Country Analysis
  • 6.3.1. United States Pompe Disease Therapeutics Market Outlook
    • 6.3.1.1. Market Size & Forecast
      • 6.3.1.1.1. By Value
    • 6.3.1.2. Market Share & Forecast
      • 6.3.1.2.1. By Treatment Type
      • 6.3.1.2.2. By Route of Administration
      • 6.3.1.2.3. By End User
  • 6.3.2. Canada Pompe Disease Therapeutics Market Outlook
    • 6.3.2.1. Market Size & Forecast
      • 6.3.2.1.1. By Value
    • 6.3.2.2. Market Share & Forecast
      • 6.3.2.2.1. By Treatment Type
      • 6.3.2.2.2. By Route of Administration
      • 6.3.2.2.3. By End User
  • 6.3.3. Mexico Pompe Disease Therapeutics Market Outlook
    • 6.3.3.1. Market Size & Forecast
      • 6.3.3.1.1. By Value
    • 6.3.3.2. Market Share & Forecast
      • 6.3.3.2.1. By Treatment Type
      • 6.3.3.2.2. By Route of Administration
      • 6.3.3.2.3. By End User

7. Europe Pompe Disease Therapeutics Market Outlook

• 7.1. Market Size & Forecast
  • 7.1.1. By Value
• 7.2. Market Share & Forecast
  • 7.2.1. By Treatment Type
  • 7.2.2. By Route of Administration
  • 7.2.3. By End User
  • 7.2.4. By Country
• 7.3. Europe: Country Analysis
  • 7.3.1. Germany Pompe Disease Therapeutics Market Outlook
    • 7.3.1.1. Market Size & Forecast
      • 7.3.1.1.1. By Value
    • 7.3.1.2. Market Share & Forecast
      • 7.3.1.2.1. By Treatment Type
      • 7.3.1.2.2. By Route of Administration
      • 7.3.1.2.3. By End User
  • 7.3.2. United Kingdom Pompe Disease Therapeutics Market Outlook
    • 7.3.2.1. Market Size & Forecast
      • 7.3.2.1.1. By Value
    • 7.3.2.2. Market Share & Forecast
      • 7.3.2.2.1. By Treatment Type
      • 7.3.2.2.2. By Route of Administration
      • 7.3.2.2.3. By End User
  • 7.3.3. Italy Pompe Disease Therapeutics Market Outlook
    • 7.3.3.1. Market Size & Forecast
      • 7.3.3.1.1. By Value
    • 7.3.3.2. Market Share & Forecast
      • 7.3.3.2.1. By Treatment Type
      • 7.3.3.2.2. By Route of Administration
      • 7.3.3.2.3. By End User
  • 7.3.4. France Pompe Disease Therapeutics Market Outlook
    • 7.3.4.1. Market Size & Forecast
      • 7.3.4.1.1. By Value
    • 7.3.4.2. Market Share & Forecast
      • 7.3.4.2.1. By Treatment Type
      • 7.3.4.2.2. By Route of Administration
      • 7.3.4.2.3. By End User
  • 7.3.5. Spain Pompe Disease Therapeutics Market Outlook
    • 7.3.5.1. Market Size & Forecast
      • 7.3.5.1.1. By Value
    • 7.3.5.2. Market Share & Forecast
      • 7.3.5.2.1. By Treatment Type
      • 7.3.5.2.2. By Route of Administration
      • 7.3.5.2.3. By End User

8. Asia-Pacific Pompe Disease Therapeutics Market Outlook

• 8.1. Market Size & Forecast
  • 8.1.1. By Value
• 8.2. Market Share & Forecast
  • 8.2.1. By Treatment Type
  • 8.2.2. By Route of Administration
  • 8.2.3. By End User
  • 8.2.4. By Country
• 8.3. Asia-Pacific: Country Analysis
  • 8.3.1. China Pompe Disease Therapeutics Market Outlook
    • 8.3.1.1. Market Size & Forecast
      • 8.3.1.1.1. By Value
    • 8.3.1.2. Market Share & Forecast
      • 8.3.1.2.1. By Treatment Type
      • 8.3.1.2.2. By Route of Administration
      • 8.3.1.2.3. By End User
  • 8.3.2. India Pompe Disease Therapeutics Market Outlook
    • 8.3.2.1. Market Size & Forecast
      • 8.3.2.1.1. By Value
    • 8.3.2.2. Market Share & Forecast
      • 8.3.2.2.1. By Treatment Type
      • 8.3.2.2.2. By Route of Administration
      • 8.3.2.2.3. By End User
  • 8.3.3. Japan Pompe Disease Therapeutics Market Outlook
    • 8.3.3.1. Market Size & Forecast
      • 8.3.3.1.1. By Value
    • 8.3.3.2. Market Share & Forecast
      • 8.3.3.2.1. By Treatment Type
      • 8.3.3.2.2. By Route of Administration
      • 8.3.3.2.3. By End User
  • 8.3.4. South Korea Pompe Disease Therapeutics Market Outlook
    • 8.3.4.1. Market Size & Forecast
      • 8.3.4.1.1. By Value
    • 8.3.4.2. Market Share & Forecast
      • 8.3.4.2.1. By Treatment Type
      • 8.3.4.2.2. By Route of Administration
      • 8.3.4.2.3. By End User
  • 8.3.5. Australia Pompe Disease Therapeutics Market Outlook
    • 8.3.5.1. Market Size & Forecast
      • 8.3.5.1.1. By Value
    • 8.3.5.2. Market Share & Forecast
      • 8.3.5.2.1. By Treatment Type
      • 8.3.5.2.2. By Route of Administration
      • 8.3.5.2.3. By End User

9. South America Pompe Disease Therapeutics Market Outlook

• 9.1. Market Size & Forecast
  • 9.1.1. By Value
• 9.2. Market Share & Forecast
  • 9.2.1. By Treatment Type
  • 9.2.2. By Route of Administration
  • 9.2.3. By End User
  • 9.2.4. By Country
• 9.3. South America: Country Analysis
  • 9.3.1. Brazil Pompe Disease Therapeutics Market Outlook
    • 9.3.1.1. Market Size & Forecast
      • 9.3.1.1.1. By Value
    • 9.3.1.2. Market Share & Forecast
      • 9.3.1.2.1. By Treatment Type
      • 9.3.1.2.2. By Route of Administration
      • 9.3.1.2.3. By End User
  • 9.3.2. Argentina Pompe Disease Therapeutics Market Outlook
    • 9.3.2.1. Market Size & Forecast
      • 9.3.2.1.1. By Value
    • 9.3.2.2. Market Share & Forecast
      • 9.3.2.2.1. By Treatment Type
      • 9.3.2.2.2. By Route of Administration
      • 9.3.2.2.3. By End User
  • 9.3.3. Colombia Pompe Disease Therapeutics Market Outlook
    • 9.3.3.1. Market Size & Forecast
      • 9.3.3.1.1. By Value
    • 9.3.3.2. Market Share & Forecast
      • 9.3.3.2.1. By Treatment Type
      • 9.3.3.2.2. By Route of Administration
      • 9.3.3.2.3. By End User

10. Middle East and Africa Pompe Disease Therapeutics Market Outlook

• 10.1. Market Size & Forecast
  • 10.1.1. By Value
• 10.2. Market Share & Forecast
  • 10.2.1. By Treatment Type
  • 10.2.2. By Route of Administration
  • 10.2.3. By End User
  • 10.2.4. By Country
• 10.3. MEA: Country Analysis
  • 10.3.1. South Africa Pompe Disease Therapeutics Market Outlook
    • 10.3.1.1. Market Size & Forecast
      • 10.3.1.1.1. By Value
    • 10.3.1.2. Market Share & Forecast
      • 10.3.1.2.1. By Treatment Type
      • 10.3.1.2.2. By Route of Administration
      • 10.3.1.2.3. By End User
  • 10.3.2. Saudi Arabia Pompe Disease Therapeutics Market Outlook
    • 10.3.2.1. Market Size & Forecast
      • 10.3.2.1.1. By Value
    • 10.3.2.2. Market Share & Forecast
      • 10.3.2.2.1. By Treatment Type
      • 10.3.2.2.2. By Route of Administration
      • 10.3.2.2.3. By End User
  • 10.3.3. UAE Pompe Disease Therapeutics Market Outlook
    • 10.3.3.1. Market Size & Forecast
      • 10.3.3.1.1. By Value
    • 10.3.3.2. Market Share & Forecast
      • 10.3.3.2.1. By Treatment Type
      • 10.3.3.2.2. By Route of Administration
      • 10.3.3.2.3. By End User

11. Market Dynamics

• 11.1. Drivers
• 11.2. Challenges

12. Market Trends & Developments

• 12.1. Recent Developments
• 12.2. Product Launches
• 12.3. Mergers & Acquisitions

13. Global Pompe Disease Therapeutics Market: SWOT Analysis

14. Competitive Landscape

• 14.1. Genzyme Corp
  • 14.1.1. Business Overview
  • 14.1.2. Product & Service Offerings
  • 14.1.3. Recent Developments
  • 14.1.4. Financials (If Listed)
  • 14.1.5. Key Personnel
  • 14.1.6. SWOT Analysis
• 14.2. Amicus Therapeutics Inc
• 14.3. Valerion Therapeutics LLC
• 14.4. Audentes Therapeutics Inc
• 14.5. Actus Therapeutics Inc
• 14.6. BioMarin Pharmaceutical Inc
• 14.7. EpiVax inc
• 14.8. Oxyrane Co Ltd.
• 14.9. Sangamo BioSciences Inc
• 14.10.Avrobio Inc
• 14.11.Spark Therapeutics Inc.

15. Strategic Recommendations

16. About Us & Disclaimer