纯合家族性高胆固醇血症市场－全球产业规模、份额、趋势、机会和预测，按药物类别、给药途径、技术、配销通路、地区和竞争细分，2020 年至 2030 年

2024 年全球纯合家族性高胆固醇血症市值为 8,267 万美元，预计到 2030 年将达到 1.0397 亿美元，年复合成长率(CAGR) 为 3.87%。该市场是更广泛的製药和医疗保健领域的一个重要部分，专注于解决被称为纯合家族性高胆固醇血症的罕见且严重的遗传性疾病。该公司致力于开发、生产和分销针对管理和治疗这种遗传疾病的治疗方案。

HoFH 是一种体染色体隐性遗传疾病，其特征是血液中低密度脂蛋白胆固醇 (LDL-C) 水平异常高，导致早期严重的心血管併发症。患有 HoFH 的人从年轻时就面临心臟病发作和其他心血管疾病的风险显着增加，需要终生接受专门的医疗护理。

近年来，在生物技术、遗传学和药理学突破的推动下，HoFH 市场取得了显着成长。製药业的领导者正在积极投资研发，以发现创新疗法，例如单株抗体、基因疗法和基于 RNA 的疗法，以更好地控制并可能治癒这种使人衰弱的疾病。这些新疗法有望大幅降低 LDL-C 水平并改善患者的生活品质。

关键市场驱动因素

主要市场挑战

主要市场趋势

这些进步凸显了 HoFH 市场的动态特性，在治疗和医疗保健服务的创新推动下，其具有巨大的成长潜力。

目录

第 1 章：产品概述

第二章：研究方法

第三章：执行摘要

第四章：全球纯合家族性高胆固醇血症市场展望

• 市场规模和预测
  • 按价值
• 市场占有率和预测
  • 依药物类别（他汀类药物、胆固醇吸收抑制剂、PCSK9 抑制剂、MTP 抑制剂、ANGPTL3 抑制剂）
  • 依给药途径（口服、肠胃外、鼻腔）
  • 依技术（CRISPR-Cas9、RNA干扰、奈米粒子疗法）
  • 按配销通路（医院药房、零售药房、网路药房）
  • 按地区
  • 按公司分类（2024）
• 市场地图
  • 按药物类别
  • 依给药途径
  • 依技术
  • 按配销通路
  • 按地区

第五章：亚太地区纯合家族性高胆固醇血症市场展望

• 市场规模和预测
  • 按价值
• 市场占有率和预测
  • 按药物类别
  • 依给药途径
  • 依技术
  • 按配销通路
  • 按国家/地区
• 亚太地区：国家分析
  • 中国
  • 印度
  • 澳洲
  • 日本
  • 韩国

第六章：欧洲纯合家族性高胆固醇血症市场展望

• 市场规模和预测
• 市场占有率和预测
• 欧洲：国家分析
  • 法国
  • 德国
  • 西班牙
  • 义大利
  • 英国

第七章：北美纯合家族性高胆固醇血症市场展望

• 市场规模和预测
• 市场占有率和预测
  • 药物类别
• 北美：国家分析
  • 美国
  • 墨西哥
  • 加拿大

第八章：南美洲纯合家族性高胆固醇血症市场展望

• 市场规模和预测
• 市场占有率和预测
• 南美洲：国家分析
  • 巴西
  • 阿根廷
  • 哥伦比亚

第九章：中东和非洲纯合家族性高胆固醇血症市场展望

• 市场规模和预测
• 市场占有率和预测
• MEA：国家分析
  • 南非
  • 沙乌地阿拉伯
  • 阿联酋

第十章：市场动态

• 驱动程式
• 挑战

第 11 章：市场趋势与发展

• 最新动态
• 产品发布
• 併购

第 12 章：全球纯合家族性高胆固醇血症市场：SWOT 分析

第 13 章：波特五力分析

• 产业竞争
• 新进入者的潜力
• 供应商的力量
• 顾客的力量
• 替代产品的威胁

第 14 章：竞争格局

• AstraZeneca PLC
• Viatris Inc.
• Teva Pharmaceutical Industries Ltd.
• Accord Healthcare
• Changzhou Pharmaceutical Factory
• Regeneron Pharmaceuticals, Inc.
• Amryt Pharma plc
• Amgen Inc.
• Organon Global Inc.
• CMP Pharma

第 15 章：策略建议

第16章调查会社について・免责事项

Global Homozygous Familial Hypercholesterolemia Market was valued at USD 82.67 million in 2024 and is projected to reach USD 103.97 million by 2030, growing at a compound annual growth rate (CAGR) of 3.87%. This market is a vital segment within the broader pharmaceutical and healthcare sectors, focused on addressing the rare and severe genetic disorder known as Homozygous Familial Hypercholesterolemia. It is dedicated to the development, production, and distribution of therapeutic solutions aimed at managing and treating this genetic condition.

HoFH is an autosomal recessive disorder marked by abnormally high levels of low-density lipoprotein cholesterol (LDL-C) in the bloodstream, which leads to early and severe cardiovascular complications. Individuals affected by HoFH face significantly increased risks of heart attacks and other cardiovascular issues from a young age, necessitating lifelong, specialized medical care.

The HoFH market has seen significant growth in recent years, driven by breakthroughs in biotechnology, genetics, and pharmacology. Pharmaceutical leaders are actively investing in research and development to discover innovative therapies, such as monoclonal antibodies, gene therapies, and RNA-based treatments, to better manage and potentially cure this debilitating condition. These novel therapies show promise in dramatically lowering LDL-C levels and improving patients' quality of life.

Key Market Drivers

Advancements in Biotechnology and Genetics: Breakthroughs in biotechnology and genetics have been pivotal in propelling the growth of the HoFH market. Monoclonal antibodies like evinacumab (Evkeeza) have been instrumental in this progress. Approved by the U.S. FDA in February 2021, evinacumab inhibits angiopoietin-like 3 (ANGPTL3), a protein that obstructs lipid breakdown. By targeting ANGPTL3, evinacumab significantly reduces LDL-C levels in HoFH patients, decreasing the risk of cardiovascular complications. Clinical trials have confirmed its transformative potential in treating HoFH.

Key Market Challenges

High Development Costs: A major challenge facing the HoFH market is the high development costs associated with creating therapies for this ultra-rare genetic disorder. HoFH affects roughly 1 in a million individuals globally, resulting in a small and geographically dispersed patient population. This scarcity complicates clinical trial recruitment and raises costs related to patient participation and data collection. Additionally, the complex nature of HoFH requires extensive research and multi-phase clinical trials, further driving up development costs.

Key Market Trends

Telemedicine and Remote Monitoring: Telemedicine and remote monitoring technologies have become transformative in the management of HoFH, addressing the challenges posed by geographic and specialist access limitations. Telemedicine enables patients to consult with specialized healthcare providers remotely, ensuring timely and consistent care. Through telehealth platforms, healthcare professionals can monitor patient progress, suggest lifestyle changes, and adjust treatments as needed, reducing the need for in-person visits.

Furthermore, remote monitoring technologies, including wearable devices and mobile applications, allow patients to track their cholesterol levels, medication adherence, and overall health. This continuous monitoring provides healthcare providers with real-time data, allowing for prompt interventions and minimizing the risk of cardiovascular events.

These advancements highlight the dynamic nature of the HoFH market, with significant potential for growth driven by innovation in therapeutic treatments and healthcare delivery.

Key Market Players

• AstraZeneca PLC
• Viatris Inc.
• Teva Pharmaceutical Industries Ltd.
• Accord Healthcare
• Changzhou Pharmaceutical Factory
• Regeneron Pharmaceuticals, Inc.
• Amryt Pharma plc
• Amgen Inc.
• Organon Global Inc.
• CMP Pharma

Report Scope:

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

Homozygous Familial Hypercholesterolemia Market, By Drug Class:

• Statins
• Cholesterol Absorption Inhibitors
• PCSK9 Inhibitors
• MTP Inhibitors
• ANGPTL3 Inhibitors

Homozygous Familial Hypercholesterolemia Market, By Route of Administration:

• Oral
• Parenteral
• Nasal

Homozygous Familial Hypercholesterolemia Market, By Technology:

• CRISPR-Cas9
• RNA Interference
• Nanoparticle-Based Therapies

Homozygous Familial Hypercholesterolemia Market, By Distribution Channel:

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

Homozygous Familial Hypercholesterolemia 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 Homozygous Familial Hypercholesterolemia Market.

Available Customizations:

Global Homozygous Familial Hypercholesterolemia 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. Global Homozygous Familial Hypercholesterolemia Market Outlook

• 4.1. Market Size & Forecast
  • 4.1.1. By Value
• 4.2. Market Share & Forecast
  • 4.2.1. By Drug Class (Statins, Cholesterol Absorption Inhibitors, PCSK9 Inhibitors, MTP Inhibitors, ANGPTL3 Inhibitors)
  • 4.2.2. By Route of Administration (Oral, Parenteral, Nasal)
  • 4.2.3. By Technology (CRISPR-Cas9, RNA Interference, Nanoparticle-Based Therapies)
  • 4.2.4. By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies)
  • 4.2.5. By Region
  • 4.2.6. By Company (2024)
• 4.3. Market Map
  • 4.3.1. By Drug Class
  • 4.3.2. By Route of Administration
  • 4.3.3. By Technology
  • 4.3.4. By Distribution Channel
  • 4.3.5. By Region

5. Asia Pacific Homozygous Familial Hypercholesterolemia Market Outlook

• 5.1. Market Size & Forecast
  • 5.1.1. By Value
• 5.2. Market Share & Forecast
  • 5.2.1. By Drug Class
  • 5.2.2. By Route of Administration
  • 5.2.3. By Technology
  • 5.2.4. By Distribution Channel
  • 5.2.5. By Country
• 5.3. Asia Pacific: Country Analysis
  • 5.3.1. China Homozygous Familial Hypercholesterolemia Market Outlook
    • 5.3.1.1. Market Size & Forecast
      • 5.3.1.1.1. By Value
    • 5.3.1.2. Market Share & Forecast
      • 5.3.1.2.1. By Drug Class
      • 5.3.1.2.2. By Route of Administration
      • 5.3.1.2.3. By Technology
      • 5.3.1.2.4. By Distribution Channel
  • 5.3.2. India Homozygous Familial Hypercholesterolemia Market Outlook
    • 5.3.2.1. Market Size & Forecast
      • 5.3.2.1.1. By Value
    • 5.3.2.2. Market Share & Forecast
      • 5.3.2.2.1. By Drug Class
      • 5.3.2.2.2. By Route of Administration
      • 5.3.2.2.3. By Technology
      • 5.3.2.2.4. By Distribution Channel
  • 5.3.3. Australia Homozygous Familial Hypercholesterolemia Market Outlook
    • 5.3.3.1. Market Size & Forecast
      • 5.3.3.1.1. By Value
    • 5.3.3.2. Market Share & Forecast
      • 5.3.3.2.1. By Drug Class
      • 5.3.3.2.2. By Route of Administration
      • 5.3.3.2.3. By Technology
      • 5.3.3.2.4. By Distribution Channel
  • 5.3.4. Japan Homozygous Familial Hypercholesterolemia Market Outlook
    • 5.3.4.1. Market Size & Forecast
      • 5.3.4.1.1. By Value
    • 5.3.4.2. Market Share & Forecast
      • 5.3.4.2.1. By Drug Class
      • 5.3.4.2.2. By Route of Administration
      • 5.3.4.2.3. By Technology
      • 5.3.4.2.4. By Distribution Channel
  • 5.3.5. South Korea Homozygous Familial Hypercholesterolemia Market Outlook
    • 5.3.5.1. Market Size & Forecast
      • 5.3.5.1.1. By Value
    • 5.3.5.2. Market Share & Forecast
      • 5.3.5.2.1. By Drug Class
      • 5.3.5.2.2. By Route of Administration
      • 5.3.5.2.3. By Technology
      • 5.3.5.2.4. By Distribution Channel

6. Europe Homozygous Familial Hypercholesterolemia Market Outlook

• 6.1. Market Size & Forecast
  • 6.1.1. By Value
• 6.2. Market Share & Forecast
  • 6.2.1. By Drug Class
  • 6.2.2. By Route of Administration
  • 6.2.3. By Technology
  • 6.2.4. By Distribution Channel
  • 6.2.5. By Country
• 6.3. Europe: Country Analysis
  • 6.3.1. France Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 6.3.1.2.2. By Route of Administration
      • 6.3.1.2.3. By Technology
      • 6.3.1.2.4. By Distribution Channel
  • 6.3.2. Germany Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 6.3.2.2.2. By Route of Administration
      • 6.3.2.2.3. By Technology
      • 6.3.2.2.4. By Distribution Channel
  • 6.3.3. Spain Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 6.3.3.2.2. By Route of Administration
      • 6.3.3.2.3. By Technology
      • 6.3.3.2.4. By Distribution Channel
  • 6.3.4. Italy Homozygous Familial Hypercholesterolemia Market Outlook
    • 6.3.4.1. Market Size & Forecast
      • 6.3.4.1.1. By Value
    • 6.3.4.2. Market Share & Forecast
      • 6.3.4.2.1. By Drug Class
      • 6.3.4.2.2. By Route of Administration
      • 6.3.4.2.3. By Technology
      • 6.3.4.2.4. By Distribution Channel
  • 6.3.5. United Kingdom Homozygous Familial Hypercholesterolemia Market Outlook
    • 6.3.5.1. Market Size & Forecast
      • 6.3.5.1.1. By Value
    • 6.3.5.2. Market Share & Forecast
      • 6.3.5.2.1. By Drug Class
      • 6.3.5.2.2. By Route of Administration
      • 6.3.5.2.3. By Technology
      • 6.3.5.2.4. By Distribution Channel

7. North America Homozygous Familial Hypercholesterolemia Market Outlook

• 7.1. Market Size & Forecast
  • 7.1.1. By Value
• 7.2. Market Share & Forecast
  • 7.2.1. By Route of Administration
  • 7.2.2. Drug Class
  • 7.2.3. By Distribution Channel
  • 7.2.4. By Technology
  • 7.2.5. By Country
• 7.3. North America: Country Analysis
  • 7.3.1. United States Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 7.3.1.2.2. By Route of Administration
      • 7.3.1.2.3. By Technology
      • 7.3.1.2.4. By Distribution Channel
  • 7.3.2. Mexico Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 7.3.2.2.2. By Route of Administration
      • 7.3.2.2.3. By Technology
      • 7.3.2.2.4. By Distribution Channel
  • 7.3.3. Canada Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 7.3.3.2.2. By Route of Administration
      • 7.3.3.2.3. By Technology
      • 7.3.3.2.4. By Distribution Channel

8. South America Homozygous Familial Hypercholesterolemia Market Outlook

• 8.1. Market Size & Forecast
  • 8.1.1. By Value
• 8.2. Market Share & Forecast
  • 8.2.1. By Drug Class
  • 8.2.2. By Route of Administration
  • 8.2.3. By Distribution Channel
  • 8.2.4. By Country
• 8.3. South America: Country Analysis
  • 8.3.1. Brazil Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 8.3.1.2.2. By Route of Administration
      • 8.3.1.2.3. By Technology
      • 8.3.1.2.4. By Distribution Channel
  • 8.3.2. Argentina Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 8.3.2.2.2. By Route of Administration
      • 8.3.2.2.3. By Technology
      • 8.3.2.2.4. By Distribution Channel
  • 8.3.3. Colombia Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 8.3.3.2.2. By Route of Administration
      • 8.3.3.2.3. By Technology
      • 8.3.3.2.4. By Distribution Channel

9. Middle East and Africa Homozygous Familial Hypercholesterolemia Market Outlook

• 9.1. Market Size & Forecast
  • 9.1.1. By Value
• 9.2. Market Share & Forecast
  • 9.2.1. By Drug Class
  • 9.2.2. By Drug Type
  • 9.2.3. By Technology
  • 9.2.4. By Distribution Channel
  • 9.2.5. By Country
• 9.3. MEA: Country Analysis
  • 9.3.1. South Africa Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 9.3.1.2.2. By Route of Administration
      • 9.3.1.2.3. By Technology
      • 9.3.1.2.4. By Distribution Channel
  • 9.3.2. Saudi Arabia Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 9.3.2.2.2. By Drug Type
      • 9.3.2.2.3. By Technology
      • 9.3.2.2.4. By Distribution Channel
  • 9.3.3. UAE Homozygous Familial Hypercholesterolemia 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 Drug Class
      • 9.3.3.2.2. By Route of Administration
      • 9.3.3.2.3. By Technology
      • 9.3.3.2.4. By Distribution Channel

10. Market Dynamics

• 10.1. Drivers
• 10.2. Challenges

11. Market Trends & Developments

• 11.1. Recent Developments
• 11.2. Product Launches
• 11.3. Mergers & Acquisitions

12. Global Homozygous Familial Hypercholesterolemia Market: SWOT Analysis

13. Porter's Five Forces Analysis

• 13.1. Competition in the Industry
• 13.2. Potential of New Entrants
• 13.3. Power of Suppliers
• 13.4. Power of Customers
• 13.5. Threat of Substitute Product

14. Competitive Landscape

• 14.1. AstraZeneca PLC
  • 14.1.1. Business Overview
  • 14.1.2. Company Snapshot
  • 14.1.3. Products & Services
  • 14.1.4. Current Capacity Analysis
  • 14.1.5. Financials (In case of listed)
  • 14.1.6. Recent Developments
  • 14.1.7. SWOT Analysis
• 14.2. Viatris Inc.
• 14.3. Teva Pharmaceutical Industries Ltd.
• 14.4. Accord Healthcare
• 14.5. Changzhou Pharmaceutical Factory
• 14.6. Regeneron Pharmaceuticals, Inc.
• 14.7. Amryt Pharma plc
• 14.8. Amgen Inc.
• 14.9. Organon Global Inc.
• 14.10. CMP Pharma

15. Strategic Recommendations

16. About Us & Disclaimer