阵发性睡眠性血红素尿症 (PNH) 治疗市场、机会、成长动力、产业趋势分析与预测，2024-2032 年

2023 年，全球阵发性睡眠性血红素尿症 (PNH) 治疗市值为 39 亿美元，预计 2024 年至 2032 年复合年增长率为 11.2%。 PNH 盛行率的上升是该市场成长的主要驱动力，导致对专业诊断技术的需求增加。这些进步促进了更早、更精确的诊断，扩大了需要治疗的患者族群。因此，随着诊断技术的进步和对 PNH 认识的提高，加上治疗方案的改进，PNH 患者的预期寿命有所延长。预期寿命的延长导致对长期治疗解决方案的持续需求，进一步推动市场成长。

整个阵发性睡眠性血红蛋白尿 (PNH) 治疗行业根据治疗类型、给药途径、年龄组、最终用途和地区进行分类。

全球 PNH 治疗市场将治疗方法分为补体抑制剂、铁疗法、免疫抑制剂、骨髓移植和其他方法。 2023年，补体抑制剂领域引领市场，营收达14亿美元。补体抑制剂，包括依库珠单抗和拉维珠单抗，已被证明可以有效抑制溶血、阻止血栓形成并提高 PNH 患者的生活品质。这些令人信服的临床结果正在推动其日益普及，从而促进市场成长。补体抑制的研究进展催生了下一代抑制剂，其具有优越的较低给药频率、药物动力学和更高的患者依从性，所有这些都促进了市场成长。

全球 PNH 治疗市场区分口服和肠胃外给药途径。 2023年，注射途径占了58.5%的市场。静脉注射可确保药物立即进入血液，这是解决急性 PNH 发作的关键因素。透过注射或输注给药的单株抗体（如依库珠单抗和拉维珠单抗）是生物製剂，体积太大且不稳定，无法口服吸收，这凸显了市场对肠胃外製剂的倾斜。

到 2023 年，北美将占据 46.6% 的份额，预计将保持其在全球 PNH 治疗市场的主导地位。美国和加拿大对包括 PNH 在内的罕见疾病治疗的优惠报销政策减轻了患者的经济负担。此类政策提高了昂贵治疗的机会。对依库珠单抗 (Soliris) 和拉维珠单抗 (Ultomiris) 等优质药物的保险支持进一步激励患者寻求治疗。美国 FDA 的快速通道地位和孤儿药指定等监管机制加快了罕见疾病药物（包括 PNH 药物）的批准。此类激励措施吸引了对 PNH 疗法开发的製药投资，加速了市场成长。

目录

第 1 章：方法与范围

第 2 章：执行摘要

第 3 章：产业洞察

• 产业生态系统分析
• 产业影响力
  • 成长动力
    • PNH 盛行率不断上升
    • 改善医疗基础设施
    • 治疗方案的进步
    • 增加研发活动，推出 PNH 治疗新疗法
  • 产业陷阱与挑战
    • 严格的监管审批
    • 治疗费用高
• 成长潜力分析
• 监管环境
• 未来市场趋势
• 波特的分析
• PESTEL分析

第 4 章：竞争格局

• 介绍
• 公司矩阵分析
• 主要市场参与者的竞争分析
• 竞争定位矩阵
• 战略仪表板

第 5 章：市场估计与预测：按治疗类型，2021 - 2032 年

• 主要趋势
• 补体抑制剂
• 铁疗法
• 免疫抑制剂药物
• 骨髓移植
• 其他治疗类型

第 6 章：市场估计与预测：按给药途径，2021 - 2032 年

• 主要趋势
• 口服
• 注射用

第 7 章：市场估计与预测：按年龄划分，2021 - 2032

• 主要趋势
• 儿科
• 成人

第 8 章：市场估计与预测：按最终用途，2021 - 2032 年

• 主要趋势
• 医院
• 专科诊所
• 家庭护理设置
• 其他最终用户

第 9 章：市场估计与预测：按地区，2021 - 2032

• 主要趋势
• 北美洲
  • 我们
  • 加拿大
• 欧洲
  • 德国
  • 英国
  • 法国
  • 西班牙
  • 义大利
  • 荷兰
  • 欧洲其他地区
• 亚太地区
  • 日本
  • 中国
  • 印度
  • 澳洲
  • 韩国
  • 亚太地区其他地区
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 拉丁美洲其他地区
• 中东和非洲
  • 南非
  • 沙乌地阿拉伯
  • 阿联酋
  • 中东和非洲其他地区

第 10 章：公司简介

• Amgen Inc.
• Apellis Pharmaceuticals, Inc.
• AstraZeneca Plc
• Children's National Hospital
• F. Hoffmann-La Roche Ltd.
• Memorial Sloan Kettering Cancer Center
• Novartis AG
• Pfizer, Inc.
• Regeneron Pharmaceuticals Inc.
• Teva Pharmaceuticals Industries Ltd.
• UCLA Health
• University of Rochester Medical Center

The Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment market was valued at USD 3.9 billion in 2023 and is projected to indicate a CAGR of 11.2% from 2024-2032. The rising prevalence of PNH is a primary driver of this market growth, leading to heightened demand for specialized diagnostic techniques. These advancements have facilitated earlier and more precise diagnoses, expanding the patient population in need of treatment. Consequently, as diagnostic techniques advance and awareness of PNH grows, coupled with improved treatment options, the life expectancy of PNH patients has risen. This uptick in life expectancy has resulted in a sustained demand for long-term treatment solutions, further propelling market growth.

The overall paroxysmal nocturnal hemoglobinuria (PNH) treatment industry is classified based on treatment type, route of administration, age group, end-use, and region.

The global PNH treatment market categorizes treatments into complement inhibitors, iron therapy, immunosuppressants, bone marrow transplants, and other methods. In 2023, the complement inhibitors segment led the market, generating revenues of USD 1.4 billion. Complement inhibitors, including eculizumab and ravulizumab, have proven effective in curbing hemolysis, thwarting blood clots, and enhancing the quality of life for PNH patients. These compelling clinical outcomes are fueling their rising adoption, thus bolstering market growth. Research advancements in complement inhibition have birthed next-gen inhibitors, boasting superior less frequent dosing, pharmacokinetics, and heightened patient compliance, all of which amplify market growth.

The global PNH treatment market distinguishes between oral and parenteral routes of administration. In 2023, the parenteral route commanded a dominant 58.5% market share. Intravenous administration ensures medications enter the bloodstream instantly, a crucial factor for addressing acute PNH episodes. Monoclonal antibodies like eculizumab and ravulizumab, administered via injections or infusions, are biologics too large and unstable for oral absorption, underscoring the market's tilt towards parenteral formulations.

North America, with a commanding 46.6% share in 2023, is poised to maintain its dominance in the global PNH treatment market. Favorable reimbursement policies in the U.S. and Canada for rare disease treatments, including PNH, alleviate financial burdens on patients. Such policies enhance accessibility to costly therapies. Insurance backing for premium drugs like eculizumab (Soliris) and ravulizumab (Ultomiris) further motivates patients to pursue treatment. Regulatory mechanisms like Fast Track status and Orphan Drug Designation by the U.S. FDA expedite approvals for rare disease drugs, including those for PNH. Such incentives attract pharmaceutical investments in PNH therapy development, accelerating market growth.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definitions
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Base estimates and calculations
  • 1.3.1 Base year calculation
  • 1.3.2 Key trends for market estimation
• 1.4 Forecast model
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
  • 1.5.2 Data mining sources

Chapter 2 Executive Summary

• 2.1 Industry 360º synopsis

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Growing prevalence of PNH
    • 3.2.1.2 Improved healthcare infrastructure
    • 3.2.1.3 Advancement in treatment options
    • 3.2.1.4 Increasing R&D activities to introduce new therapies for PNH treatment
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 Stringent regulatory approvals
    • 3.2.2.2 High cost of treatment
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
• 3.5 Future market trends
• 3.6 Porter's analysis
• 3.7 PESTEL analysis

Chapter 4 Competitive Landscape, 2023

• 4.1 Introduction
• 4.2 Company matrix analysis
• 4.3 Competitive analysis of major market players
• 4.4 Competitive positioning matrix
• 4.5 Strategic dashboard

Chapter 5 Market Estimates and Forecast, By Treatment Type, 2021 - 2032 ($ Million)

• 5.1 Key trends
• 5.2 Complement inhibitors
• 5.3 Iron therapy
• 5.4 Immunosuppressants drugs
• 5.5 Bone marrow transplantation
• 5.6 Other treatment types

Chapter 6 Market Estimates and Forecast, By Route of administration, 2021 - 2032 ($ Million)

• 6.1 Key trends
• 6.2 Oral
• 6.3 Parenteral

Chapter 7 Market Estimates and Forecast, By Age Group, 2021 - 2032 ($ Million)

• 7.1 Key trends
• 7.2 Pediatric
• 7.3 Adult

Chapter 8 Market Estimates and Forecast, By End-use, 2021 - 2032 ($ Million)

• 8.1 Key trends
• 8.2 Hospitals
• 8.3 Specialty clinics
• 8.4 Homecare settings
• 8.5 Other end-users

Chapter 9 Market Estimates and Forecast, By Region, 2021 - 2032 ($ Million)

• 9.1 Key trends
• 9.2 North America
  • 9.2.1 U.S.
  • 9.2.2 Canada
• 9.3 Europe
  • 9.3.1 Germany
  • 9.3.2 UK
  • 9.3.3 France
  • 9.3.4 Spain
  • 9.3.5 Italy
  • 9.3.6 Netherlands
  • 9.3.7 Rest of Europe
• 9.4 Asia Pacific
  • 9.4.1 Japan
  • 9.4.2 China
  • 9.4.3 India
  • 9.4.4 Australia
  • 9.4.5 South Korea
  • 9.4.6 Rest of Asia Pacific
• 9.5 Latin America
  • 9.5.1 Brazil
  • 9.5.2 Mexico
  • 9.5.3 Rest of Latin America
• 9.6 Middle East and Africa
  • 9.6.1 South Africa
  • 9.6.2 Saudi Arabia
  • 9.6.3 UAE
  • 9.6.4 Rest of Middle East and Africa

Chapter 10 Company Profiles

• 10.1 Amgen Inc.
• 10.2 Apellis Pharmaceuticals, Inc.
• 10.3 AstraZeneca Plc
• 10.4 Children's National Hospital
• 10.5 F. Hoffmann-La Roche Ltd.
• 10.6 Memorial Sloan Kettering Cancer Center
• 10.7 Novartis AG
• 10.8 Pfizer, Inc.
• 10.9 Regeneron Pharmaceuticals Inc.
• 10.10 Teva Pharmaceuticals Industries Ltd.
• 10.11 UCLA Health
• 10.12 University of Rochester Medical Center