陣發性睡眠性血紅素尿症 (PNH) 治療市場、機會、成長動力、產業趨勢分析與預測，2024-2032 年

2023 年，全球陣發性睡眠性血紅素尿症 (PNH) 治療市值為 39 億美元，預計 2024 年至 2032 年複合年成長率為 11.2%。 PNH 盛行率的上升是該市場成長的主要驅動力，導致對專業診斷技術的需求增加。這些進步促進了更早、更精確的診斷，擴大了需要治療的患者族群。因此，隨著診斷技術的進步和對 PNH 認知的提高，加上治療方案的改進，PNH 患者的預期壽命有所延長。預期壽命的延長導致對長期治療解決方案的持續需求，進一步推動市場成長。

整個陣發性睡眠性血紅蛋白尿 (PNH) 治療行業根據治療類型、給藥途徑、年齡層、最終用途和地區進行分類。

全球 PNH 治療市場將治療方法分為補體抑制劑、鐵療法、免疫抑制劑、骨髓移植和其他方法。 2023年，補體抑制劑領域引領市場，營收達14億美元。補體抑制劑，包括依庫珠單抗和拉維珠單抗，已被證明可以有效抑制溶血、阻止血栓形成並提高 PNH 患者的生活品質。這些令人信服的臨床結果正在推動其日益普及，從而促進市場成長。補體抑制的研究進展催生了下一代抑制劑，其具有優越的較低給藥頻率、藥物動力學和更高的患者依從性，所有這些都促進了市場成長。

全球 PNH 治療市場區分口服和腸胃外給藥途徑。 2023年，注射途徑佔了58.5%的市場。靜脈注射可確保藥物立即進入血液，這是解決急性 PNH 發作的關鍵因素。透過注射或輸注給藥的單株抗體（如依庫珠單抗和拉維珠單抗）是生物製劑，體積太大且不穩定，無法口服吸收，這凸顯了市場對腸胃外製劑的傾斜。

到 2023 年，北美將佔據 46.6% 的佔有率，預計將保持其在全球 PNH 治療市場的主導地位。美國和加拿大對包括 PNH 在內的罕見疾病治療的優惠報銷政策減輕了患者的經濟負擔。此類政策提高了昂貴治療的機會。對依庫珠單抗 (Soliris) 和拉維珠單抗 (Ultomiris) 等優質藥物的保險支持進一步激勵患者尋求治療。美國 FDA 的快速通道地位和孤兒藥指定等監管機制加快了罕見疾病藥物（包括 PNH 藥物）的批准。此類激勵措施吸引了對 PNH 療法開發的製藥投資，加速了市場成長。

目錄

第 1 章：方法與範圍

第 2 章：執行摘要

第 3 章：產業洞察

• 產業生態系統分析
• 產業影響力
  • 成長動力
    • PNH 盛行率不斷上升
    • 改善醫療基礎設施
    • 治療方案的進步
    • 增加研發活動，推出 PNH 治療新療法
  • 產業陷阱與挑戰
    • 嚴格的法規核准
    • 治療費用高
• 成長潛力分析
• 監管環境
• 未來市場趨勢
• 波特的分析
• PESTEL分析

第 4 章：競爭格局

• 介紹
• 公司矩陣分析
• 主要市場參與者的競爭分析
• 競爭定位矩陣
• 戰略儀表板

第 5 章：市場估計與預測：按治療類型，2021 - 2032 年

• 主要趨勢
• 補體抑制劑
• 鐵療法
• 免疫抑制劑藥物
• 骨髓移植
• 其他治療類型

第 6 章：市場估計與預測：按給藥途徑，2021 - 2032 年

• 主要趨勢
• 口服
• 注射用

第 7 章：市場估計與預測：按年齡分類，2021 - 2032

• 主要趨勢
• 兒科
• 成人

第 8 章：市場估計與預測：按最終用途，2021 - 2032 年

• 主要趨勢
• 醫院
• 專科診所
• 家庭護理設置
• 其他最終用戶

第 9 章：市場估計與預測：按地區，2021 - 2032

• 主要趨勢
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 英國
  • 法國
  • 西班牙
  • 義大利
  • 荷蘭
  • 歐洲其他地區
• 亞太地區
  • 日本
  • 中國
  • 印度
  • 澳洲
  • 韓國
  • 亞太地區其他地區
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 拉丁美洲其他地區
• 中東和非洲
  • 南非
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國
  • 中東和非洲其他地區

第 10 章：公司簡介

• 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