세계의 발작성 야간 혈색소뇨증(PNH) 및 비전형 용혈성 요독증후군(aHUS) 시장 보고서(2025년)
Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Global Market Report 2025
상품코드 : 1760671
리서치사 : The Business Research Company
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한글목차

발작성 야간 혈색소뇨증(PNH) 및 비전형 용혈성 요독증후군(aHUS) 시장 규모는 향후 몇 년간 강한 성장을 보일 것으로 예상됩니다. 예측 기간 동안의 성장은 새로운 치료법에 대한 규제 승인의 증가, 생명 공학 기업과 연구 기관 간의 협력 강화, 개인 맞춤형 의약품에 대한 수요 증가, 희귀 질환 신생 기업에 대한 투자 증가, 임상 시험 및 연구의 증가와 같은 요인에 기인할 수 있습니다. 예측 기간 동안의 주요 동향으로는 보체 억제 요법의 발전, 유전자 치료 접근법의 통합, 개인 맞춤형 의약품의 발전, 기술 중심 진단 도구, 원격 환자 모니터링을 위한 기술의 통합 등이 있습니다.

희귀 질환의 유병률이 증가함에 따라 발작성 야간 혈색소뇨증(PNH) 및 비전형 용혈성 요독증후군(aHUS) 시장이 성장할 것으로 예상됩니다. 희귀 질환은 인구의 소수, 일반적으로 20만 명 미만에 영향을 미치는 의학적 상태로, 복잡성, 중증도 및 전문적인 치료가 필요한 것이 특징입니다. 희귀 질환의 증가 추세는 진단 기술의 발전에 기인하며, 이는 이전에 보고되지 않았거나 진단되지 않은 질환의 탐지 및 식별을 개선했습니다. PNH와 aHUS는 치료 옵션이 제한적인 질환의 병리생리학 및 잠재적 치료 표적에 대한 통찰을 제공함으로써 희귀 질환의 이해와 치료에 기여합니다. 예를 들어, 2024년 11월, 미국 식품의약국(FDA)은 미국에서 7,000개 이상의 희귀 질환이 3,000만 명 이상의 사람들에게 영향을 미치고 있다고 보고했습니다. 그 결과, 희귀 질환의 유병률이 증가하면서 PNH 및 aHUS 부문의 시장 성장이 촉진되고 있습니다.

PNH 및 aHUS 시장의 선도 기업들은 치료 효과 향상과 환자 편의성을 위해 보체 억제제와 같은 혁신적인 치료법 개발에 집중하고 있습니다. 보체 억제제는 병원체와 손상된 세포를 제거하는 역할을 하는 보체 시스템의 특정 성분을 차단하는 치료제입니다. 예를 들어, 2023년 12월, 스위스에 본사를 둔 제약 회사인 Novartis AG는 발작성 야간 혈색소뇨증 성인 환자를 위한 최초의 경구용 단일 요법제인 Fabhalta(iptacopan)에 대해 FDA의 승인을 받았습니다. Fabhalta는 수혈 없이도 우수한 헤모글로빈 개선 효과를 제공합니다. 이 혁신적인 보체 억제제는 대체 보체 경로의 인자 B를 억제하여 혈관 내 및 혈관 외 용혈을 효과적으로 제어합니다. 경구 투여로 질병 관리가 간단해지고 환자의 삶의 질이 향상되며, 잦은 주입의 부담이 사라집니다.

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 동향과 전략

제4장 시장 - 금리, 인플레이션, 지정학, 무역전쟁과 관세, 코로나 19 및 회복이 시장에 미치는 영향을 포함한 거시경제 시나리오

제5장 세계의 성장 분석과 전략 분석 프레임워크

제6장 시장 세분화

제7장 지역별, 국가별 분석

제8장 아시아태평양 시장

제9장 중국 시장

제10장 인도 시장

제11장 일본 시장

제12장 호주 시장

제13장 인도네시아 시장

제14장 한국 시장

제15장 서유럽 시장

제16장 영국 시장

제17장 독일 시장

제18장 프랑스 시장

제19장 이탈리아 시장

제20장 스페인 시장

제21장 동유럽 시장

제22장 러시아 시장

제23장 북미 시장

제24장 미국 시장

제25장 캐나다 시장

제26장 남미 시장

제27장 브라질 시장

제28장 중동 시장

제29장 아프리카 시장

제30장 경쟁 구도와 기업 프로파일

제31장 기타 주요 기업 및 혁신 기업

제32장 세계 시장 경쟁 벤치마킹과 대시보드

제33장 주요 인수합병(M&A)

제34장 최근 시장 동향

제35장 시장의 잠재력이 높은 국가, 부문, 전략

제36장 부록

HBR
영문 목차

영문목차

Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are rare, life-threatening blood disorders caused by the uncontrolled activation of the complement system, a component of the immune system. PNH results in the destruction of red blood cells, while aHUS leads to the formation of blood clots in small blood vessels, primarily affecting the kidneys and other vital organs.

The main treatments for PNH and aHUS include Soliris and Ultomiris. Soliris, also known by its generic name eculizumab, is a monoclonal antibody medication used to treat these rare blood disorders. The treatment options for these conditions include complement inhibitors, plasma exchange or infusion, and others. Diagnostic methods involve clinical diagnosis, genetic testing, and biomarker testing. These treatments and diagnostic methods are used by various end-users, including hospitals, specialty clinics, research institutes, and more.

The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market research report is one of a series of new reports from The Business Research Company that provides paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market statistics, including the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) industry's global market size, regional shares, competitors with the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market share, detailed paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market segments, market trends and opportunities, and any further data you may need to thrive in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market. This paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market size has grown strongly in recent years. It will grow from $5.57 billion in 2024 to $5.99 billion in 2025 at a compound annual growth rate (CAGR) of 7.4%. The growth during the historic period can be attributed to factors such as the increasing prevalence of PNH and aHUS, rising investment in rare disease research, growing demand for targeted therapies, increased awareness of PNH and aHUS, and the growing adoption of complement inhibitors.

The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market size is expected to see strong growth in the next few years. It will grow to $7.93 billion in 2029 at a compound annual growth rate (CAGR) of 7.3%. The growth during the forecast period can be attributed to factors such as increasing regulatory approvals for new treatments, growing collaboration between biotech companies and research institutes, rising demand for personalized medicine, increasing investment in rare disease startups, and a growing number of clinical trials and research studies. Key trends in the forecast period include advancements in complement inhibition therapies, the integration of gene therapy approaches, developments in personalized medicine, technology-driven diagnostic tools, and the integration of technology for remote patient monitoring.

The growing prevalence of rare diseases is expected to drive the growth of the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market. Rare diseases are medical conditions that affect a small percentage of the population, typically fewer than 200,000 individuals, and are often characterized by their complexity, severity, and the need for specialized treatment. The rise in rare diseases can be attributed to advances in diagnostic technologies, which have improved the detection and identification of previously underreported or undiagnosed conditions. PNH and aHUS contribute to a deeper understanding and treatment of rare diseases by providing insights into their pathophysiology and potential therapeutic targets for conditions with limited treatment options. For example, in November 2024, the Food and Drug Administration (FDA) reported that over 7,000 rare diseases affected more than 30 million individuals in the United States. As a result, the increasing prevalence of rare diseases is driving market growth in the PNH and aHUS sectors.

Leading companies in the PNH and aHUS market are focusing on the development of innovative therapies, such as complement inhibitors, to enhance treatment outcomes and patient convenience. Complement inhibitors are therapeutic agents that block specific components of the complement system, which plays a role in clearing pathogens and damaged cells. For example, in December 2023, Novartis AG, a pharmaceutical company based in Switzerland, received FDA approval for Fabhalta (iptacopan), the first oral monotherapy for treating adults with paroxysmal nocturnal hemoglobinuria. Fabhalta offers superior hemoglobin improvement without the need for transfusions. This innovative complement inhibitor works by inhibiting Factor B in the alternative complement pathway, effectively controlling both intravascular and extravascular hemolysis. Its oral administration simplifies disease management and improves the quality of life for patients, eliminating the burden of frequent infusions.

In January 2025, Samsung Bioepis Co. Ltd., a biopharmaceutical company based in South Korea, partnered with Teva Pharmaceutical Industries Ltd. to develop and commercialize Soliris (eculizumab) biosimilars, including EPYSQLI (eculizumab-aagh), for rare diseases. This collaboration aims to improve access to affordable treatment options for U.S. patients. Teva, an Israeli pharmaceutical company, specializes in the development, manufacturing, and commercialization of medicines for various rare diseases, including PNH and aHUS.

Major players in the paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market are Roche Holding AG, AstraZeneca plc, Novartis AG, Amgen Inc., Teva Pharmaceuticals Ltd., Regeneron Pharmaceuticals Inc., Chugai Pharmaceutical Co. Ltd., Alexion Pharmaceuticals Inc., UCB Pharma Limited, Alnylam Pharmaceuticals Inc., BioCryst Pharmaceuticals Inc., Genentech Inc., Omeros Corporation, CinnaGen Co., Apellis Pharmaceuticals Inc., CANbridge Pharmaceuticals Inc., Kira Pharmaceuticals, NovelMed Therapeutics Inc., Arrowhead Pharmaceuticals Inc., Akari Therapeutics plc.

North America was the largest region in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market in 2024. Asia Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market consists of revenues earned by entities by providing services such as bone marrow biopsy, flow cytometry testing, and multidisciplinary care coordination. The market value includes the value of related goods sold by the service provider or included within the service offering. The paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) market also includes sales of flow cytometry reagents, anticoagulants, and pegcetacoplan. Values in this market are 'factory gate' values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses on paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

Where is the largest and fastest growing market for paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus) market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.

Scope

Table of Contents

1. Executive Summary

2. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Characteristics

3. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Trends And Strategies

4. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market

5. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Growth Analysis And Strategic Analysis Framework

6. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Segmentation

7. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Regional And Country Analysis

8. Asia-Pacific Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

9. China Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

10. India Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

11. Japan Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

12. Australia Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

13. Indonesia Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

14. South Korea Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

15. Western Europe Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

16. UK Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

17. Germany Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

18. France Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

19. Italy Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

20. Spain Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

21. Eastern Europe Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

22. Russia Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

23. North America Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

24. USA Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

25. Canada Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

26. South America Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

27. Brazil Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

28. Middle East Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

29. Africa Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

30. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Competitive Landscape And Company Profiles

31. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Other Major And Innovative Companies

32. Global Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

34. Recent Developments In The Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market

35. Paroxysmal Nocturnal Hemoglobinuria (PNH) And Atypical Hemolytic Uremic Syndrome (aHUS) Market High Potential Countries, Segments and Strategies

36. Appendix

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