Atypical Hemolytic Uremic Syndrome Ahus Market Insight

DelveInsight’s ‘Atypical Hemolytic Uremic Syndrome (aHUS) - Market Insights, Epidemiology, and Market Forecast—2030’ report delivers an in-depth understanding of the Atypical Hemolytic Uremic Syndrome, historical and forecasted epidemiology as well as the Atypical Hemolytic Uremic Syndrome market trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

 

The Atypical Hemolytic Uremic Syndrome market report provides current treatment practices, emerging drugs, Atypical Hemolytic Uremic Syndrome market share of the individual therapies, current and forecasted Atypical Hemolytic Uremic Syndrome market size from 2018 to 2030 segmented by seven major markets. The Report also covers current Atypical Hemolytic Uremic Syndrome treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered        

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2018–2030

Atypical Hemolytic Uremic Syndrome (aHUS): Disease Understanding and Treatment Algorithm

Atypical Hemolytic Uremic Syndrome (aHUS) Overview

Atypical hemolytic uremic syndrome (aHUS) is a rare cause of thrombotic microangiopathy (TMA), characterized by microangiopathic hemolytic anemia, consumptive thrombocytopenia, and multisystem end organ involvement, most commonly affecting the kidney.

 

Most cases of aHUS are genetic, although some may be acquired due to autoantibodies or occur for unknown reasons (idiopathic). aHUS may become chronic, and affected individuals may experience repeated episodes of the disorder. Unlike individuals with typical HUS, who usually recover from the life-threatening initial episode and usually respond well to supportive treatment, individuals with aHUS are much more likely to develop serious chronic complications such as severe high blood pressure (hypertension) and kidney (renal) failure.

 

aHUS is a disease that primarily affects kidney function. This condition, which can occur at any age, causes abnormal blood clots (thrombi) to form in small blood vessels in the kidneys. These clots can cause serious medical problems if they restrict or block blood flow. It is characterized by three major features related to abnormal clotting: hemolytic anemia, thrombocytopenia, and kidney failure.

 

Hemolytic Uremic Syndrome (HUS) is one of the disorders that belong to the spectrum of thrombotic microangiopathy (TMA). TMAs are a group of life-threatening diseases characterized by endothelial dysfunction and the presence of thrombi in small blood vessels. As the thrombus forms, there is the consumption of platelets and mechanical disruption of erythrocytes, which leads to thrombocytopenia and microangiopathic hemolytic anemia. Vessel occlusion results in tissue ischemia and organ damage, mainly affecting the kidneys, although other organs can be involved.

 

Atypical Hemolytic Uremic Syndrome (aHUS) Diagnosis

Diagnosing aHUS is complicated by the fact that it is more difficult 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.

 

Most of the time, aHUS does not present with initial severe symptoms. The initial onset will most likely appear to be flu-like, with lethargic behavior, pale color, and a loss of appetite. It has been experienced that adult hospitals may often send the patient home with a flu diagnosis, while children’s hospitals may do a blood draw and conduct basic tests, such as Hemoglobin, Hematocrit, white blood cell counts, and platelet counts.

 

After initial blood tests, the hospital may conduct creatinine and BUN tests and may (or may not) reach an initial diagnosis of aHUS. The flu-like symptoms described above will continue to worsen when episodes are active. At this point, kidney function may begin to fall, often quite dramatically. Other organs sometimes experience problems in some cases. Quite often, seizures have been reported, along with other neurological issues. Sometimes gastronomical problems occur as well.

 

aHUS is a diagnosis of exclusion, ruling out (STEC) infection and other secondary causes of TMA , including (TTP). To exclude TTP, a blood test that measures the level of a protein called ADAMTS13 is used. The diagnostic tests for aHUS include blood tests (CBC and eGFR), specific test to differ from TTP, and genetic testing.

 

Atypical Hemolytic Uremic Syndrome (aHUS) Treatment

Treatment of aHUS 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.

Atypical Hemolytic Uremic Syndrome (aHUS) Epidemiology

The Atypical Hemolytic Uremic Syndrome epidemiology division provides insights about historical and current Atypical Hemolytic Uremic Syndrome patient pool and forecasted trends for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

 

Key Findings

In the year 2020, the total prevalent cases of Atypical Hemolytic Uremic Syndrome was 7.0 thousand cases in the 7MM which are expected to grow during the study period, i.e., 2018–2030.

 

The disease epidemiology covered in the report provides historical as well as forecasted Atypical Hemolytic Uremic Syndrome epidemiology [segmented as Total Prevalent Cases of Atypical hemolytic uremic syndrome, Total Diagnosed Cases of Atypical hemolytic uremic syndrome, Total Age group-specific Cases of Atypical hemolytic uremic syndrome, and Total Treated Cases of Atypical hemolytic uremic syndrome] in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2018 to 2030.

Country Wise- Atypical Hemolytic Uremic Syndrome Epidemiology

  • Estimates show that the highest cases of Atypical Hemolytic Uremic Syndrome in the 7MM were in the United States, followed by Germany, France, Italy, Spain, the United Kingdom, and Japan in 2020.
  • In the United States, the total number of prevalent cases of Atypical Hemolytic Uremic Syndrome was 3.5 thousand cases in the year 2020 which are expected to grow during the study period, i.e., 2018–2030.
  • In the year 2020, the total prevalent cases of Atypical Hemolytic Uremic Syndrome were 3.1 thousand cases in EU-5 which are expected to grow during the study period, i.e., 2018–2030.
  • In Japan, the total number of prevalent cases of Atypical Hemolytic Uremic Syndrome was 452 cases in the year 2020 which are expected to grow during the study period, i.e., 2018–2030.

Atypical Hemolytic Uremic Syndrome (aHUS) Drug Chapters

The drug chapter segment of the Atypical Hemolytic Uremic Syndrome report encloses the detailed analysis of Atypical Hemolytic Uremic Syndrome marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Atypical Hemolytic Uremic Syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

 

Atypical Hemolytic Uremic Syndrome Emerging Drugs

 

Iptacopan/LNP023 (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. The AP acts as an amplification loop of complement activation and contributes to various human diseases. Inhibition of factor B is being exploited as a mechanism to treat complement-mediated conditions such as C3 glomerulopathy (glomerulonephritis, including IgA nephropathy), paroxysmal nocturnal hemoglobinuria, and aHUS. Upon administration, Iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb). This limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders.

 

Crovalimab/RG6107 (Hoffmann-La Roche/Chugai Pharmaceutical)

Crovalimab (RG6107, SKY59) is a humanized complement inhibitor C5 monoclonal antibody discovered by Chugai using recycling antibody technology. By blocking the cleavage of C5 to C5a and C5b, it is expected to inhibit complement activation, which is the cause of several diseases. As the complement system is a key innate immune defense mechanism, the company plans to study the potential of this antibody in a broader range of complement-mediated diseases. This drug is engineered with Sequential Monoclonal Antibody Recycling Technology (SMART) that improves the drug’s half-life and facilitates subcutaneous dosing while still achieving complete C5 inhibition C5 inhibition. Crovalimab binds to the C5 β-chain and prevents cleavage of the wild-type and SNP C5 by the C5 convertase. In addition, it uniquely inhibits C5b6 deposition on membranes, further limiting membrane attack complex-mediated tissue damage. SMART has led to limited C5 accumulation and increased C5 binding capacity in nonhuman primates.

Note: Detailed emerging therapies assessment will be provided in the final report.

Atypical Hemolytic Uremic Syndrome (aHUS) Market Outlook

Atypical HUS patients are much more likely to develop complications. These complications may be a recurrence of initial symptoms, kidney failure, seizures, neurological problems or other multi-organ involvement, no response to supportive treatments, return of atypical HUS even after a kidney transplant occurs, severe hypertension (high blood pressure).

 

In Japan, the diagnostic criteria for aHUS were defined in the 2013 clinical guides of the Japanese Society of Nephrology and Japan Pediatric Society. These criteria were then revised in 2015 clinical guides, to account for recent findings and the emerging international consensus on aHUS.

 

Treatment of aHUS 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.

 

Historically, plasma infusion or plasma exchange (PI/PE) was the standard of care by supplying the functional complement inhibitory molecules. But the outcomes of patients treated with PI/PE remained unsatisfactory as nearly 70% of patients with complement genetic variants died or reached ESRD during the first episode of aHUS or within 3 years. Nevertheless, PI/PE is still used until thrombotic thrombocytopenic purpura (TTP) is discarded, when eculizumab is not available, and in anti-CFH antibodies aHUS. Liver transplantation has been proposed for aHUS caused by genetic variants in complement proteins synthesized in the liver (CFH, CFI, FB, and C3), mainly if the patient also needs a kidney transplant.

 

As per the study of Blaha et al. (2018), therapy with plasma exchange and plasma infusions (PE/PI) are widely available across the world, including in resource-limited countries. Also, even though PE/PI have been the standard of care for aHUS, they do not address the underlying cause of complement dysfunction. PE is an effective form of acute management that enables removal of antibodies and other proteins; supportive data from clinical trials is, however, lacking.

 

Key Findings

The Atypical Hemolytic Uremic Syndrome (aHUS) market size in the 7MM is expected to change during the study period 2018–2030. The therapeutic market of Atypical Hemolytic Uremic Syndrome in the seven major markets is expected to increase during the study period (2018–2030) with a CAGR of 3.3%. According to the estimates, the highest market size of Atypical Hemolytic Uremic Syndrome is found in the United States followed by Germany.

 

The United States Market Outlook

The total market size of Atypical Hemolytic Uremic Syndrome therapies in the United States is expected to increase with a CAGR of 3.9% in the study period (2018–2030).

 

EU-5 Countries: Market Outlook

The total market size of Atypical Hemolytic Uremic Syndrome therapies in EU-5 countries is expected to increase with a CAGR of 2.2% in the study period (2018–2030).

 

Japan Market Outlook

The total market size of Atypical Hemolytic Uremic Syndrome therapies in Japan is expected to increase with a CAGR of 1.3% in the study period (2018–2030).

Atypical Hemolytic Uremic Syndrome (aHUS) Pipeline Development Activities

The drugs which are in pipeline include:

  • Iptacopan/LNP023 (Novartis Pharmaceuticals)
  • Crovalimab/RG6107 (Hoffmann-La Roche/Chugai Pharmaceutical)

Note: Detailed emerging therapies assessment will be provided in the final report.

Analyst Commentary

  • Due to lack of approved treatment options in aHUS, currently Soliris and Ultomiris are the preferred choice of treatment. Entry of Iptacopan (LNP 023) is expected to impact the aHUS market dynamics significantly.
  • Limited pipeline activity in the aHUS space can be observed and the possible hurdles might be rarity of disorder, data scarcity on the latest epidemiology studies across the geographies and the lack of definite diagnosis criteria.
  • Among the emerging therapies, Novartis’ Iptacopan (LNP023) and Roche’s Crovalimab (RG6107; SKY59) appears to be the only drugs which have the potential to transform the aHUS market owing to impressive clinical data.

Access and Reimbursement Scenario in Atypical Hemolytic Uremic Syndrome Therapies

Currently, eculizumab and ravulizumab are the only licensed therapy for aHUS in the 7MM. They are effective in all patients with aHUS whether or not a complement mutation has been identified and whether the patient has a native or transplanted kidney. Consensus guidelines recommend eculizumab’s early use in all patients with aHUS for optimal outcomes.

 

The summary of product characteristics for eculizumab by the NICE recommends that the treatment is to continue for a patient’s lifetime unless discontinuation of treatment is clinically indicated. Patients under 18 years weighing 40 kg or more are treated in line with the adult dosing recommendations. Pediatric patients weighing below 40 kg have their dose adjusted by body weight. Eculizumab costs GBP 3,150 per 30 mL (10 mg/mL) vial (excluding VAT; British national formulary, online July 2014). The total cost of eculizumab per adult was estimated to be about GBP 340,200 (initial and maintenance treatment) in the first year of treatment and about GBP 327,600 for 1 year of treatment on the recommended maintenance dose.

 

Eculizumab is currently commissioned, through an interim commissioning policy, by NHS England in line with the Clinical Commissioning Policy Statement: Eculizumab for atypical hemolytic uremic syndrome for:

  • New patients with aHUS (defined to include those with a functioning kidney)
  • Existing patients who are on dialysis and are suitable for a kidney transplant
  • The policy is currently administered through an interim national aHUS service at the Newcastle Upon Tyne Hospitals NHS Foundation Trust.

 

Ravulizumab is also recommended by the NICE, within its marketing authorization, as an option for treating atypical hemolytic uremic syndrome (aHUS) in people weighing 10 kg or more:

  • Who has not had a complement inhibitor before
  • Whose disease has responded to at least 3 months of eculizumab treatment

 

It is recommended if only the company provides ravulizumab according to the commercial arrangement.

 

According to DeCoy et al. (2019), the cost of a single 30 mL/300 mg vial of eculizumab, once labeled as the most expensive drug worldwide by Forbes, was around USD 6830 or GBP 3150 with an annual cost of treatment being more than USD 500,000 per year approximately. This cost of treatment for the patients decreases to approximately USD 490,000 after the discounts given for the drug. The high price and limited availability of the drug limit its use, especially in the developing world. Even in developed countries like the UK, reimbursement limitations pose challenges to both patients and physicians and restrict access to this life-saving drug. The biweekly hospitalization for the administration of eculizumab during the maintenance phase of treatment further adds to the rising healthcare-associated financial burden. “A balance between pharmaceutical companies’ incentives and societal budgetary constraints” must be maintained to ensure the timely and regular availability of orphan drugs like eculizumab.

Note: Detailed HTA assessment will be provided in the final report.

KOL-Views

To keep up with current market trends, we take KOLs and SME’s opinion working in the Atypical Hemolytic Uremic Syndrome domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Atypical Hemolytic Uremic Syndrome market trends. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Atypical Hemolytic Uremic Syndrome Market by using various Competitive Intelligence tools that includes – SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

  • The report covers the descriptive overview of Atypical Hemolytic Uremic Syndrome, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
  • Comprehensive insight has been provided into the Atypical Hemolytic Uremic Syndrome epidemiology and treatment in the 7MM.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Atypical Hemolytic Uremic Syndrome is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of the Atypical Hemolytic Uremic Syndrome market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Atypical Hemolytic Uremic Syndrome market.

Report Highlights

  • In the coming years, the Atypical Hemolytic Uremic Syndrome market is set to change due to the upcoming therapies with novel route of administrations which are under investigation and ongoing research in the aHUS; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Atypical Hemolytic Uremic Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Major players are involved in developing therapies for Atypical Hemolytic Uremic Syndrome. The launch of emerging therapies will significantly impact the Atypical Hemolytic Uremic Syndrome market.
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Atypical Hemolytic Uremic Syndrome.
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Atypical Hemolytic Uremic Syndrome Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Atypical Hemolytic Uremic Syndrome Pipeline Analysis
  • Atypical Hemolytic Uremic Syndrome Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Atypical Hemolytic Uremic Syndrome Report Key Strengths

  • 10 Years Forecast
  • 7MM Coverage
  • Atypical Hemolytic Uremic Syndrome Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

Atypical Hemolytic Uremic Syndrome Report Assessment

  • SWOT Analysis
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Conjoint Analysis
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Atypical Hemolytic Uremic Syndrome Market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the Atypical Hemolytic Uremic Syndrome total market size as well as market size by therapies across the 7MM during the study period (2018–2030)?
  • What are the key findings of the market across the 7MM and which country will have the largest Atypical Hemolytic Uremic Syndrome market size during the study period (2018–2030)?
  • At what CAGR, the Atypical Hemolytic Uremic Syndrome market is expected to grow in the 7MM during the study period (2018–2030)?
  • What would be the Atypical Hemolytic Uremic Syndrome market outlook across the 7MM during the study period (2018–2030)?
  • What would be the Atypical Hemolytic Uremic Syndrome market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
  • Atypical Hemolytic Uremic Syndrome patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
  • How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration, and frequencies based on their clinical trial results?
  • Among the emerging therapies, what are the potential therapies which are expected to disrupt the Atypical Hemolytic Uremic Syndrome market?

 

Epidemiology Insights:

  • What are the disease risks, burdens, and unmet needs of the Atypical Hemolytic Uremic Syndrome?
  • What is the historical Atypical Hemolytic Uremic Syndrome patient pool in the seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
  • What would be the forecasted patient pool of Atypical Hemolytic Uremic Syndrome in the 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan?
  • What will be the growth opportunities in the 7MM concerning the patient population about Atypical Hemolytic Uremic Syndrome?
  • Out of all the 7MM countries, which country would have the highest prevalent population of Atypical Hemolytic Uremic Syndrome during the study period (2018–2030)?
  • At what CAGR the population is expected to grow in the 7MM during the study period (2018–2030)?
  • What are the various recent and upcoming events which are expected to improve the diagnosis of Atypical Hemolytic Uremic Syndrome?

 

Current Treatment Scenario and Emerging Therapies:

  • What are the current options for the treatment of Atypical Hemolytic Uremic Syndrome?
  • What are the current treatment guidelines for the treatment of Atypical Hemolytic Uremic Syndrome in the US, Europe, and Japan?
  • How many companies are developing therapies for the treatment of Atypical Hemolytic Uremic Syndrome?
  • How many therapies are developed by each company for the treatment of Atypical Hemolytic Uremic Syndrome?
  • How many emerging therapies are in the mid-stage and late stages of development for the treatment of Atypical Hemolytic Uremic Syndrome?
  • What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Atypical Hemolytic Uremic Syndrome therapies?
  • What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Atypical Hemolytic Uremic Syndrome and their status?
  • What are the key designations that have been granted for the emerging therapies for Atypical Hemolytic Uremic Syndrome?
  • What is the global historical and forecasted market of Atypical Hemolytic Uremic Syndrome?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Atypical Hemolytic Uremic Syndrome market.
  • To understand the future market competition in the Atypical Hemolytic Uremic Syndrome market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Atypical Hemolytic Uremic Syndrome in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for the Atypical Hemolytic Uremic Syndrome market.
  • To understand the future market competition in the Atypical Hemolytic Uremic Syndrome market.

1. Key Insights

2. Report Introduction

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

3.1. Market Share (%) Distribution of aHUS in 2018

3.2. Market Share (%) Distribution of aHUS in 2030

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

4.1. Key Events

5. Epidemiology and Market Methodology

6. Disease Background and Overview

6.1. Introduction

6.2. Types of Hemolytic Uremic Syndrome

6.3. Risk Factors and Causes of Atypical Hemolytic Uremic Syndrome

6.4. Signs and Symptoms of Atypical Hemolytic Uremic Syndrome

6.5. Pathophysiology of Atypical Hemolytic Uremic Syndrome

6.6. Complement Protein Mutations

6.7. Clinical Manifestations of Atypical Hemolytic Uremic Syndrome

7. Diagnosis and Differential Diagnosis of Atypical Hemolytic Uremic Syndrome

7.1. Diagnostic Algorithm of Atypical Hemolytic Uremic Syndrome

7.2. Blood Tests

7.3. Specific Testing

7.4. Genetic Testing

7.5. Differential Diagnosis

8. Diagnostic Guidelines

8.1. The Joint Committee of the Japanese Society of Nephrology and the Japan Pediatric Society

8.2. Clinical Practice Guidelines for the management of atypical Hemolytic Uremic Syndrome in the United Kingdom

9. Current Treatment Practices of Atypical Hemolytic Uremic Syndrome

9.1. Treatment Algorithm of Atypical hemolytic uremic syndrome

9.1.1. Plasma Exchange

9.1.2. Eculizumab

9.1.3. Ravulizumab

9.1.4. Kidney Transplant

9.1.5. Liver transplantation

10. Treatment Guidelines

10.1. The European Rare Kidney Disease Reference Network guidelines to the management of atypical hemolytic uremic syndrome in children

10.2. Clinical Practice Guidelines for the management of atypical Hemolytic Uremic Syndrome in the United Kingdom

10.3. National Institute for Health and Research

11. Conclusion

12. Epidemiology and Patient Population

12.1. Key Findings

12.2. Epidemiology of Atypical Hemolytic Uremia Syndrome

12.3. Epidemiology Scenario

12.3.1. Total Prevalent Cases of Atypical Hemolytic Uremia Syndrome

12.3.2. Total Diagnosed Cases of Atypical Hemolytic Uremia Syndrome

12.3.3. Total Age group-specific Cases of Atypical Hemolytic Uremia Syndrome

12.3.4. Total Treated Cases of Atypical Hemolytic Uremia Syndrome

13. Patient Journey

14. Key Endpoints in Atypical Hemolytic Uremic Syndrome (aHUS) Clinical Trials

15. Marketed Therapies

15.1. Soliris (Eculizumab): Alexion Pharmaceuticals

15.1.1. Drug Description

15.1.2. Regulatory Milestones

15.1.3. Other Developmental Activities

15.1.4. Pivotal Clinical Trial

15.2. Ultomiris (Ravulizumab): Alexion Pharmaceuticals

15.2.1. Drug Description

15.2.2. Regulatory Milestones

15.2.3. Other Developmental Activities

15.2.4. Pivotal Clinical Trial

15.2.5. Ongoing Current Pipeline Activity

16. Emerging Therapies

16.1. Narsoplimab (OMS721): Omeros Corporation

16.1.1. Product Description

16.1.2. Other Developmental Activities

16.1.3. Clinical Developmental Activities

16.1.4. Safety and Efficacy

16.2. Iptacopan (LNP 023): Novartis Pharmaceuticals

16.2.1. Product Description

16.2.2. Clinical Developmental Activities

16.3. Crovalimab (RG6107; SKY59): Hoffmann-La Roche/Chugai Pharmaceutical

16.3.1. Product Description

16.3.2. Clinical Developmental Activities

17. Atypical Hemolytic Uremic Syndrome: 7 Major Market Analysis

17.1. Key Findings

17.2. Market Outlook

17.3. Market Size of Atypical Hemolytic Uremic Syndrome

17.3.1. Total Market Size of Atypical Hemolytic Uremic Syndrome

17.3.2. Market Size of Atypical Hemolytic Uremic Syndrome by Therapies

18. Market Access and Reimbursement

18.1. Key HTA decisions for aHUS

18.2. Reimbursement

18.3. Patient Access Programs

19. KOL Views

20. Market Drivers

21. Market Barriers

22. SWOT Analysis

23. Unmet Needs

24. Appendix

24.1. Bibliography

24.2. Report Methodology

24.3. DelveInsight Capabilities

25. Disclaimer

26. About DelveInsight

List of Table

Table 1: Summary of Atypical Hemolytic Uremic Syndrome (aHUS) Disease, Market, Epidemiology, and Key Events (2018–2030)

Table 2: Frequency of Genetic Variants in Complement Regulatory Factors, According to Geographic Locations

Table 3: Total Prevalent Cases of Atypical Hemolytic Uremia Syndrome (2018–2030)

Table 4: Total Diagnosed Cases of Atypical Hemolytic Uremia Syndrome (2018–2030)

Table 5: Total Age group-specific Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Table 6: Total Treated Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Table 7: Dosing Recommendations in aHUS Patients Less Than 18 Years of Age

Table 8: Ultomiris Weight-based Dosing Regimen aHUS

Table 9: Ultomiris (Ravulizumab), Clinical Trial Description, 2021

Table 10: Narsoplimab (OMS721), Clinical Trial Description, 2021

Table 11: Iptacopan (LNP023), Clinical Trial Description, 2021

Table 12: Crovalimab (RG6107; SKY59), Clinical Trial Description, 2021

Table 13: 7MM Market Size of Atypical Hemolytic Uremic Syndrome in USD Million (2018–2030)

Table 14: 7MM Market Size of Atypical Hemolytic Uremic Syndrome by Therapies in USD Million (2018–2030)

Table 15: Key HTA Decisions

List of Figures

Figure 1: Epidemiology and Market Methodology

Figure 2: Understanding of Atypical Hemolytic Uremic Syndrome

Figure 3: Risk Factors and Causes of Atypical Hemolytic Uremic Syndrome

Figure 4: Triggering Factors for Atypical Hemolytic Uremic Syndrome

Figure 5: Pathophysiology of Atypical Hemolytic Uremic Syndrome

Figure 6:  Clinical Manifestations of Atypical Hemolytic Uremic Syndrome

Figure 7: Extra-renal Manifestations of Atypical Hemolytic Uremic Syndrome

Figure 8: Diagnostic Algorithm of Atypical Hemolytic Uremic Syndrome

Figure 9: Differential Diagnosis of Hemolytic Uremic Syndrome

Figure 10: Differential Diagnosis

Figure 11: Treatment Algorithm of atypical HUS

Figure 12: Blockade of terminal complement activation by Eculizumab

Figure 13: Total Prevalent Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Figure 14: Total Diagnosed Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Figure 15: Total Age group-specific Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Figure 16: Total Age group-specific Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Figure 17: Total Treated Cases of Atypical Hemolytic Uremia Syndrome in the 7MM (2018–2030)

Figure 18: Market Size of Atypical Hemolytic Uremic Syndrome in the 7MM, USD Million (2018–2030)

Figure 19: 7MM Market Size of Atypical Hemolytic Uremic Syndrome by Therapies in USD Million (2018–2030)

Alexion Pharmaceuticals
Omeros Corporation
Novartis Pharmaceuticals
Hoffmann-La Roche
Chugai Pharmaceutical

 

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