非定型溶血性尿毒症症候群（aHUS）市場 - 市場の洞察、疫学、市場予測：2032年

非定型溶血性尿毒症症候群（aHUS）は、腎臓の細い血管に異常な血栓が形成される病気です。これらの血栓が血流を制限または遮断すると、溶血性貧血、血小板減少症、腎不全などの重篤な医学的問題を引き起こす可能性があります。aHUSの最も一般的な症状は、錯乱、脳卒中、発作、心臓発作、高血圧、腎臓、心臓、または他の臓器における血栓の形成、吐き気と嘔吐、腹痛、腎障害です。aHUSの診断検査には、赤血球数と血小板数を測定する全血球算定（CBC）検査と、腎機能を調べる血液検査が含まれます。腎臓の健康状態は、クレアチニン値、年齢、性別、人種を用いて計算されるeGFRと呼ばれる検査で測定することができます。

2022年、主要7ヶ国でaHUSと診断された症例は約4,800例でした。主要7ヶ国では、小児患者よりも青年および成人のaHUS症例が多くなっています。2022年には、青年成人は約4,000例、小児患者は約700例でした。aHUSの治療は支持療法であり、急性腎障害と全身合併症の管理に注意を払う。尿毒症、体液過多、電解質異常のある患者には腎代替療法が必要です。血漿交換と補体阻害薬であるエクリズマブが特異的な治療法です。現在、aHUSの治療薬として承認されているのは、エクリズマブとラブリズマブの2剤のみです。

2022年の市場規模は主要7ヶ国の中で米国が最も大きく、調査期間中（2019年～2032年）のCAGRは約4%で2032年までに増加すると予想されます。

薬剤の割高な価格設定により商業的な導入が進んでいないこと、aHUSの治療における安全性への懸念、確定的な診断基準の欠如、今後のジェネリック医薬品競争などがaHUS疾患市場に打撃を与えると予想されます。

主要7ヶ国における非定型溶血性尿毒症症候群（aHUS）の市場規模は、2022年に13億米ドルとなりました。同市場は2032年までに拡大すると予測されています。

当レポートでは、主要7ヶ国における非定型溶血性尿毒症症候群（aHUS）市場について調査し、市場の概要とともに、疫学、患者動向、新たな治療法、2032年までの市場規模予測、および医療のアンメットニーズなどを提供しています。

目次

第1章 重要な洞察

第2章 レポートのイントロダクション

第3章 非定型溶血性尿毒症症候群（aHUS）市場の概要

第4章 非定型溶血性尿毒症症候群（aHUS）のエグゼクティブサマリー

第5章 主要な出来事

第6章 疫学と市場予測の調査手法

第7章 疾患の背景と概要

• イントロダクション
• 溶血性尿毒症症候群のタイプ類
• aHUSの危険因子と原因
• aHUSの兆候と症状
• aHUSの病態生理学
• 補体タンパク質の変異
• aHUSの臨床症状
• aHUSの診断と鑑別診断

第8章 aHUSの現在の治療実践

• aHUSの治療アルゴリズム
• 治療ガイドライン

第9章 患者動向

第10章 疫学と患者数

• 主な調査結果
• aHUSの前提と理論的根拠
• 主要7ヶ国におけるaHUS罹患症例
• 主要7ヶ国におけるaHUS診断症例
• 米国
• EU4ヶ国と英国
• 日本

第11章 上市済み薬剤

第12章 新興薬剤

第13章 非定型溶血性尿毒症症候群（aHUS）：主要7ヶ国の市場分析

• 主な調査結果
• 市場の見通し
• コンジョイント分析
• 主要な市場予測の前提条件
• 700万米ドル単位のaHUSの合計市場規模（2019年～2032年）
• 主要7ヶ国のaHUS市場規模、治療法別
• 米国の市場規模
• EU4ヶ国と英国の市場規模
• 日本市場規模

第14章 アンメットニーズ

第15章 SWOT分析

第16章 KOLの見解

第17章 市場アクセスと償還

• 米国
• EU4ヶ国と英国
• 日本
• aHUSにおける償還シナリオと重要なHTA決定

第18章 付録

第19章 DelveInsightのサービス内容

第20章 免責事項

第21章 DelveInsightについて

List of Tables

• Table 1: Summary of IC-MPGN Market and Epidemiology (2019-2032)
• Table 2: Frequency of Genetic Variants in Complement Regulatory Factors, According to Geographic Locations
• Table 3: Recommended Evaluations Following Initial Diagnosis in Individuals With Genetic aHUS
• Table 4: Prevalent Cases of aHUS in the 7MM (2019-2032)
• Table 5: Diagnosed Cases of aHUS in the 7MM (2019-2032)
• Table 6: Prevalent Cases of aHUS in the United States (2019-2032)
• Table 7: Diagnosed Cases of aHUS in the United States (2019-2032)
• Table 8: Age group-specific Cases of aHUS in the United States (2019-2032)
• Table 9: Treated Cases of aHUS in the United States (2019-2032)
• Table 10: Prevalent Cases of aHUS in EU4 and the UK (2019-2032)
• Table 11: Diagnosed Cases of aHUS in EU4 and the UK (2019-2032)
• Table 12: Age group-specific Cases of aHUS in EU4 and the UK (2019-2032)
• Table 13: Treated Cases of aHUS in EU4 and the UK (2019-2032)
• Table 14: Prevalent Cases of aHUS in Japan (2019-2032)
• Table 15: Diagnosed Cases of aHUS in Japan (2019-2032)
• Table 16: Age group-specific Cases of aHUS in Japan (2019-2032)
• Table 17: Treated Cases of aHUS in Japan (2019-2032)
• Table 18: Comparison of Marketed Drugs
• Table 19: Dosing Recommendations in aHUS Patients Less Than 18 Years of Age
• Table 20: SOLIRIS, Clinical Trial Description, 2023
• Table 21: Efficacy Results of Study C08-002A/B
• Table 22: Efficacy Results of Study C08-003A/B
• Table 23. Efficacy Results of Study
• Table 24. Efficacy Results of Study C10-004
• Table 25. Efficacy Results of Study C10-003
• Table 26: ULTOMIRIS Weight-based Dosing Regimen aHUS
• Table 27: ULTOMIRIS (ravulizumab-cwvz), Clinical Trial Description, 2023
• Table 28: Summary of Study ALXN1210-aHUS-311 (NCT02949128)
• Table 29: Summary of Study ALXN1210- aHUS-312 (NCT03131219)
• Table 30: Comparison of Emerging Drugs
• Table 31: Crovalimab (RG6107; SKY59), Clinical Trial Description, 2023
• Table 32: Iptacopan (LNP023), Clinical Trial Description, 2023
• Table 33: Conjoint Analysis
• Table 34: Key Market Forecast Assumptions of aHUS in the US
• Table 35: Key Market Forecast Assumptions of aHUS in EU4 and the UK
• Table 36: Key Market Forecast Assumption of aHUS in Japan
• Table 37: Total Market Size of aHUS in the 7MM, in USD million (2019-2032)
• Table 38: Market Size of aHUS by Therapies in the 7MM, in USD million (2019-2032)
• Table 39: Total Market Size of aHUS in the US, in USD million (2019-2032)
• Table 40: Market Size of aHUS by Therapies in the US, in USD million (2019-2032)
• Table 41: Total Market Size of aHUS in EU4 and the UK, in USD million (2019-2032)
• Table 42: Market Size of aHUS by Therapies in EU4 and the UK, in USD million (2019-2032)
• Table 43: Total Market Size of aHUS in Japan, in USD million (2019-2032)
• Table 44: Market Size of aHUS by Therapies in Japan, in USD million (2019-2032)

List of Figures

• Figure 1: Relationship of the Thrombotic Microangiopathy Lesions
• Figure 2: Understanding of aHUS
• Figure 3: Risk Factors and Causes of aHUS
• Figure 4: Triggering Factors for aHUS
• Figure 5: Pathophysiology of aHUS
• Figure 6: Clinical Manifestations of aHUS
• Figure 7: Extra-renal Manifestations of aHUS
• Figure 8: Diagnostic Algorithm of aHUS
• Figure 9: Differential Diagnosis of Hemolytic Uremic Syndrome
• Figure 10: Differential Diagnosis
• Figure 11: Algorithm for Differential Diagnosis of aHUS
• Figure 12: Treatment Algorithm of aHUS
• Figure 13: Prevalent Cases of aHUS in the 7MM (2019-2032)
• Figure 14: Diagnosed Cases of aHUS in the 7MM (2019-2032)
• Figure 15: Prevalent Cases of aHUS in the United States (2019-2032)
• Figure 16: Diagnosed Cases of aHUS in the United States (2019-2032)
• Figure 17: Age group-specific Cases of aHUS in the United States (2019-2032)
• Figure 18: Treated Cases of aHUS in the United States (2019-2032)
• Figure 19: Prevalent Cases of aHUS in EU4 and the UK (2019-2032)
• Figure 20: Diagnosed Cases of aHUS in EU4 and the UK (2019-2032)
• Figure 21: Age group-specific Cases of aHUS in EU4 and the UK (2019-2032)
• Figure 22: Treated Cases of aHUS in EU4 and the UK (2019-2032)
• Figure 23: Prevalent Cases of aHUS in Japan (2019-2032)
• Figure 24: Diagnosed Cases of aHUS in Japan (2019-2032)
• Figure 25: Age group-specific Cases of aHUS in Japan (2019-2032)
• Figure 26: Treated Cases of aHUS in Japan (2019-2032)
• Figure 27: Total Market Size of aHUS in the 7MM (2019-2032)
• Figure 28: Market Size of aHUS by Therapies in the 7MM (2019-2032)
• Figure 29: Total Market Size of aHUS in the US (2019-2032)
• Figure 30: Market Size of aHUS by Therapies in the United States (2019-2032)
• Figure 31: Total Market Size of aHUS in EU4 and the UK (2019-2032)
• Figure 32: Market Size of aHUS by Therapies in EU4 and the UK (2019-2032)
• Figure 33: Total Market Size of aHUS in Japan (2019-2032)
• Figure 34: Market Size of aHUS by Therapies in Japan (2019-2032)
• Figure 35: Health Technology Assessment
• Figure 36: Reimbursement Process in Germany
• Figure 37: Reimbursement Process in France
• Figure 38: Reimbursement Process in Italy
• Figure 39: Reimbursement Process in Spain
• Figure 40: Reimbursement Process in the United Kingdom
• Figure 41: Reimbursement Process in Japan

Key Highlights:

• Atypical hemolytic uremic syndrome (aHUS) is a disease that causes abnormal blood clots to form in small blood vessels in the kidneys. These clots can cause serious medical problems if they restrict or block blood flow, including hemolytic anemia, thrombocytopenia, and kidney failure.
• The most common symptoms of aHUS are confusion, stroke, seizure, heart attack, high blood pressure, formation of blood clots in the kidney, heart, or other organs, nausea and vomiting, abdominal pain, and kidney damage.
• The diagnostic test of aHUS includes a complete blood count (CBC) test, which is used to measure the red blood cell and platelet count, and a blood test to check kidney function. Kidney health can be measured by a test called eGFR which is calculated using the creatinine level, age, sex, and race.
• In 2022, diagnosed cases of aHUS in the 7MM were nearly 4,800 cases.
• In the 7MM, more cases of aHUS in adolescents and adults than in pediatric patients. There were approximately 4,000 and 700 cases of adolescent adults and pediatric patients, respectively, in 2022.
• Therapy of aHUS is supportive, with attention to the management of acute kidney injury and systemic complications. Renal replacement therapy is required in patients with uremia, fluid overload, or electrolyte abnormalities. Plasma exchange and eculizumab, a complement inhibitor, offer specific forms of therapy. Currently, only two drugs have been approved for the treatment of aHUS, eculizumab, and ravulizumab.
• The United States accounted for the highest market size among the 7MM in 2022, expected to increase by 2032 with a CAGR of approximately 4% during the study period (2019-2032).
• Premium pricing of the drugs has resulted in the low commercial uptake of the drug, safety concerns with the treatment of aHUS, lack of definitive diagnostic criteria, and upcoming generic competition are expected to hit the aHUS disease market.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase III) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the aHUS market, providing an in-depth examination of its historical and projected market size (2019-2032). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM aHUS market.

Market

Various key players are leading the treatment landscape of aHUS, such as Alexion Pharmaceuticals, Novartis, and Roche. The details of the country-wise and therapy-wise market size have been provided below.

• In 2022, the total market size of aHUS was around USD 1,300 million, which is expected to increase by 2032 during the study period (2019-2032) in the 7MM.
• In the total market size of aHUS in the 7MM, the United States accounted for the highest market size in 2022, followed by Germany and France.
• Among the emerging therapies, Novartis (iptacopan) and Roche (crovalimab) appear to be the drugs that can potentially transform the aHUS market.
• By 2032, among the emerging therapies, the highest revenue is expected to be generated by iptacopan, i.e., nearly USD 100 million in the EU4 and the UK.

aHUS Drug Chapters

The section dedicated to drugs in the aHUS report provides an in-depth evaluation of late-stage pipeline drugs (Phase III) related to aHUS.

The drug chapters section provides valuable information on various aspects related to clinical trials of aHUS, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting aHUS.

Marketed Therapies

ULTOMIRIS (ravulizumab): Alexion Pharmaceuticals

ULTOMIRIS is a medication primarily indicated for the treatment of aHUS. Atypical hemolytic uremic syndrome is a rare and serious condition characterized by the formation of blood clots in small blood vessels throughout the body, leading to damage to vital organs, especially the kidneys. In September 2020, Alexion Pharmaceuticals announced that Japan's MHLW had approved ULTOMIRIS (ravulizumab) for adults and children living with aHUS.

Emerging Therapies

LNP023 (iptacopan): Novartis Pharmaceuticals

Iptacopan, also known as LNP023, is a first-in-class, orally-administered, small-molecule, potent, and highly selective factor B (FB) inhibitor, a key serine protease of the alternative pathway of the complement cascade. Currently, Iptacopan is in the Phase III development stage. The ongoing Phase III clinical trial is a multicenter, single-arm, open-label trial to evaluate the efficacy and safety of oral, twice-daily LNP023 in adult aHUS patients who are naive to complement inhibitor therapy and is expected to be completed by December 2024.

aHUS Market Outlook

The introduction of complement C5 inhibition into the treatment landscape has significantly improved patient prognosis and QoL. Currently available complement C5 inhibitors across the 7MM are SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), both Alexion Pharmaceuticals products. Alexion Pharmaceuticals was acquired by AstraZeneca in 2021.

The treatment transition of patients being treated with eculizumab to ravulizumab has been established as a viable treatment strategy, as this would lead to a reduction in the dosing frequency, which would then lead to a significant reduction of economic burden (treatment-related costs of repeated infusions) and improve the overall QoL of patients.

Although eculizumab and ravulizumab are safe and effective, their high costs often limit their usage in the treatment landscape. Hence, efficacious pharmacologic options for managing the most prevalent and most disabling stages of aHUS are extremely limited; therefore, new treatments are desperately needed.

In a nutshell, a few potential therapies are being investigated to manage aHUS. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2023-2032), it is safe to assume that the future of this market is bright. Eventually, these drugs shall create a significant difference in the landscape of aHUS in the coming years. The treatment space is expected to experience a significant positive shift in the coming years owing to the improvement in healthcare spending worldwide.

Further details are provided in the report…

aHUS Disease Understanding and Treatment

aHUS Overview

aHUS is an extremely rare disease characterized by low levels of circulating red blood cells due to their destruction (hemolytic anemia), low platelet count (thrombocytopenia) due to their consumption, and the inability of the kidneys to process waste products from the blood and excrete them into the urine (acute kidney failure), a condition known as uremia. It is a distinctly different illness from the more common disorder known as a typical hemolytic uremic syndrome, caused by E. coli-producing Shiga toxins (Stx HUS) and is generally foodborne.

Most cases of aHUS are genetic, although some may be acquired due to autoantibodies or for unknown reasons (idiopathic). aHUS may become chronic, and affected individuals may experience repeated episodes of the disorder.

There are two types of HUS: Typical HUS (caused by E. coli or other food/water-borne pathogens) and atypical HUS (usually a genetic mutation but sometimes triggered by other illnesses or unknown causes).

The signs and symptoms of aHUS result from the formation of tiny blood clots (microthrombi) in various small blood vessels. These clots reduce or prevent proper blood flow to various body organs, especially the kidneys. aHUS is a complex disorder, and multiple factors, including certain genetic, environmental, and immunologic factors, all play a role in its development.

aHUS Diagnosis

Diagnosing aHUS is complicated by the fact that it is more challenging to establish without a family history of the disorder. The diagnostic criteria associated with aHUS are hemolytic anemia (anemia in the presence of broken red blood cells), low platelet count (thrombocytopenia), and kidney dysfunction.

The diagnostic test of aHUS includes a complete blood count (CBC) test, which is used to measure the red blood cell and platelet count, and a blood test to check kidney function. Kidney health can be measured by a test called eGFR which is calculated using the creatinine level, age, sex, and race.

Specific testing is there; both HUS and aHUS share some signs and symptoms with another disease called thrombotic thrombocytopenic purpura (TTP), which has a different underlying cause. Doctors can measure levels of a certain protein in the blood, ADAMTS13, which may help clarify whether a patient has aHUS or TTP.

Further details related to country-based variations are provided in the report…

aHUS Treatment

Treatment of Atypical Hemolytic Uremic Syndrome is supportive, with attention to management of acute kidney injury and systemic complications. The use of packed cells is necessary in patients with severe anemia. Platelet transfusions are rarely required, except in counts are <10 000/cu mm or thrombocytopenia associated with active bleeding or in patients undergoing invasive procedures.

Fluid and electrolyte management is important to maintain intravascular volume status and combat the consequences of aHUS, acute kidney injury, and multisystem organ failure. Electrolyte disturbances should be promptly corrected and nephrotoxic medications avoided. Hypertension should be managed with appropriate agents. Renal replacement therapy is required in patients with uremia, fluid overload, or electrolyte abnormalities. Plasma exchange and eculizumab or rivalizumab, a complement inhibitor, offer specific forms of therapy.

For years, plasma therapy was the standard treatment for individuals with aHUS. Both infusions of fresh frozen plasma (plasma infusion), as well as plasma exchange (plasmapheresis), were utilized. Fresh frozen plasma is a blood derivative that is obtained from donors. Plasma exchange removes potentially harmful substances (e.g., toxins, metabolic substances, and plasma parts) from the blood. Blood is removed from the affected individual, and blood cells are separated from the plasma. The plasma is then replaced with other human plasma, and the blood is transfused into the affected individuals. Plasma exchange can also remove mutant factors and autoantibodies.

Further details related to treatment and management are provided in the report…

aHUS Epidemiology

The aHUS epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases, diagnosed cases, age-specific cases, and treated cases of aHUS in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.

• Among the 7MM, the United States accounted for the highest number of prevalent cases of aHUS in 2022, nearly 3,500 cases. These cases are anticipated to increase by 2032.
• In 2022, EU4 and the UK accounted for more cases of aHUS in adolescents and adults than in pediatric patients.
• Among EU4 and the UK, Germany accounted for the highest number of prevalent cases in 2022, while Spain accounted for the least.
• In 2022, the treated cases of aHUS in Japan were around 200 cases.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of aHUS, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts at Delveinsight connected with more than 15 KOLs across the 7MM. We contacted institutions such as the University of Munich, the University of Tokyo, the European Dialysis and Transplant Association, the American Society of Nephrology, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the aHUS market, which will assist our clients in analyzing the overall epidemiology and market scenario.

Qualitative Analysis

We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in trials for aHUS, one of the most important primary endpoints was achieving hemolysis control, LDH normalization, etc. Based on these, the overall efficacy is evaluated.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

aHUS Report Insights

• Patient Population
• Therapeutic Approaches
• aHUS Market Size and Trends
• Existing Market Opportunity

aHUS Report Key Strengths

• Ten-year Forecast
• The 7MM Coverage
• aHUS Epidemiology Segmentation
• Key Cross Competition

aHUS Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions:

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in aHUS management recommendations?
• Would research and development advances pave the way for future tests and therapies for aHUS?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of aHUS?
• What kind of uptake will the new therapies witness in coming years in aHUS patients?

Table of Contents

1. Key Insights

2. Report Introduction

3. Atypical Hemolytic Uremic Syndrome (aHUS) Market Overview at a Glance

• 3.1. Market Share Distribution (%) in the 7MM in 2022
• 3.2. Market Share Distribution (%) in the 7MM in 2032

4. Executive Summary of Atypical Hemolytic Uremic Syndrome (aHUS)

5. Key Events

6. Epidemiology and Market Forecast Methodology

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Types of Hemolytic Uremic Syndrome
• 7.3. Risk Factors and Causes of aHUS
• 7.4. Signs and Symptoms of aHUS
• 7.5. Pathophysiology of aHUS
• 7.6. Complement Protein Mutations
• 7.7. Clinical Manifestations of aHUS
• 7.8. Diagnosis and Differential Diagnosis of aHUS
  • 7.8.1. Diagnostic Algorithm of aHUS
  • 7.8.2. Blood Tests
  • 7.8.3. Specific Testing
  • 7.8.4. Genetic Testing
  • 7.8.5. Differential Diagnosis
  • 7.8.6. Diagnostic Guidelines

8. Current Treatment Practices of aHUS

• 8.1. Treatment Algorithm of aHUS
  • 8.1.1. Plasma Exchange
  • 8.1.2. Eculizumab
  • 8.1.3. Ravulizumab
  • 8.1.4. Kidney Transplant
  • 8.1.5. Liver Transplantation
• 8.2. Treatment Guidelines
  • 8.2.1. The European Rare Kidney Disease Reference Network Guidelines for the Management of aHUS in Children
  • 8.2.2. Clinical Practice Guidelines for the Management of aHUS in the United Kingdom
  • 8.2.3. National Institute for Health and Research

9. Patient Journey

10. Epidemiology and Patient Population

• 10.1. Key Findings
• 10.2. Assumptions and Rationale for aHUS
• 10.3. Prevalent Cases of aHUS in the 7MM
• 10.4. Diagnosed Cases of aHUS in the 7MM
• 10.5. The United States
  • 10.5.1. Prevalent Cases of aHUS in the United States
  • 10.5.2. Diagnosed Cases of aHUS in the United States
  • 10.5.3. Age group-specific Cases of aHUS in the United States
  • 10.5.4. Treated Cases of aHUS in the United States
• 10.6. EU4 and the UK
  • 10.6.1. Prevalent Cases of aHUS in EU4 and the UK
  • 10.6.2. Diagnosed Cases of aHUS in EU4 and the UK
  • 10.6.3. Age group-specific Cases of aHUS in EU4 and the UK
  • 10.6.4. Treated Cases of aHUS in EU4 and the UK
• 10.7. Japan
  • 10.7.1. Prevalent Cases of aHUS in Japan
  • 10.7.2. Diagnosed Cases of aHUS in Japan
  • 10.7.3. Age group-specific Cases of aHUS in Japan
  • 10.7.4. Treated Cases of aHUS in Japan

11. Marketed Therapies

• 11.1. Key Cross of Marketed Therapies
• 11.2. SOLIRIS (eculizumab): Alexion Pharmaceuticals
  • 11.2.1. Product Description
  • 11.2.2. Regulatory Milestones
  • 11.2.3. Other Developmental Activity
  • 11.2.4. Clinical Development
  • 11.2.5. Safety and Efficacy
• 11.3. ULTOMIRIS (ravulizumab-cwvz): Alexion Pharmaceuticals
  • 11.3.1. Product Description
  • 11.3.2. Regulatory Milestones
  • 11.3.3. Other Developmental Activity
  • 11.3.4. Clinical Development
  • 11.3.5. Safety and Efficacy

12. Emerging Therapies

• 12.1. Key Cross of Emerging Therapies
• 12.2. Crovalimab (RG6107; SKY59): Hoffmann-La Roche/Chugai Pharmaceutical
  • 12.2.1. Product Description
  • 12.2.2. Clinical Development
• 12.3. Iptacopan (LNP023): Novartis Pharmaceuticals
  • 12.3.1. Product Description
  • 12.3.2. Clinical Development

13. Atypical Hemolytic Uremic Syndrome (aHUS): Seven Major Market Analysis

• 13.1. Key Findings
• 13.2. Market Outlook
• 13.3. Conjoint Analysis
• 13.4. Key Market Forecast Assumptions
• 13.5. Total Market Size of aHUS in the 7MM in USD million (2019-2032)
• 13.6. Market Size of aHUS by Therapies in the 7MM
• 13.7. United States Market Size
  • 13.7.1. Total Market Size of aHUS in the United States
  • 13.7.2. Market Size of aHUS by Therapies in the United States
• 13.8. EU4 and the UK Market Size
  • 13.8.1. Total Market Size of aHUS in EU4 and the UK
  • 13.8.2. Market Size of aHUS by Therapies in EU4 and the UK
• 13.9. Japan Market Size
  • 13.9.1. Total Market Size of aHUS in Japan
  • 13.9.2. Market Size of aHUS by Therapies in Japan

14. Unmet Needs

15. SWOT Analysis

16. KOL Views

17. Market Access and Reimbursement

• 17.1. United States
  • 17.1.1. Centre for Medicare and Medicaid Services (CMS)
• 17.2. EU4 and the UK
  • 17.2.1. Germany
  • 17.2.2. France
  • 17.2.3. Italy
  • 17.2.4. Spain
  • 17.2.5. United Kingdom
• 17.3. Japan
  • 17.3.1. MHLW
• 17.4. Reimbursement Scenario and Key HTA Decisions in aHUS
  • 17.4.1. Patient Access Programs
  • 17.4.2. HTA Decisions

18. Appendix

• 18.1. Acronyms and Abbreviations
• 18.2. Bibliography
• 18.3. Report Methodology

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