The Atypical Hemolytic Uremic Syndrome market report also offers comprehensive insights into the Atypical Hemolytic Uremic Syndrome market size, share, Atypical Hemolytic Uremic Syndrome epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Atypical Hemolytic Uremic Syndrome market size growth forward.
Some of the key highlights from the Atypical Hemolytic Uremic Syndrome Market Insights Report:
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Several key pharmaceutical companies, including Alexion Pharmaceuticals, Omeros Corporation, Novartis Pharmaceuticals, Hoffmann-La Roche, Chugai Pharmaceutical, and others, are developing novel products to improve the Atypical Hemolytic Uremic Syndrome treatment outlook.
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In April 2024, Hoffmann-La Roche revealed the results of a Phase III, multicenter, single-arm study assessing the efficacy, safety, pharmacokinetics, and pharmacodynamics of Crovalimab in pediatric patients with atypical hemolytic uremic syndrome (aHUS).
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In February 2024, Alexion Pharmaceuticals, Inc. announced the results of a prospective, single-arm, multicenter study evaluating the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in complement inhibitor treatment-naïve pediatric and adult patients with aHUS in China.
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In 2023, the United States had the largest market share for Atypical Hemolytic Uremic Syndrome (aHUS) among the 7MM, and this market is expected to grow with a compound annual growth rate (CAGR) of approximately 4% by 2034.
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Atypical hemolytic uremic syndrome (aHUS) is a condition that causes abnormal blood clot formation in the small blood vessels of the kidneys, which can lead to serious health problems like hemolytic anemia, thrombocytopenia, and kidney failure. Common symptoms include confusion, stroke, seizures, heart attack, high blood pressure, and kidney damage.
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There are two types of HUS: Typical HUS, caused by pathogens like E. coli, and atypical HUS, which is often linked to genetic mutations, but can also be triggered by illnesses or other unknown causes. Most aHUS cases are genetic, although some can be acquired through autoantibodies or idiopathic causes. The condition can become chronic, leading to complications such as hypertension and kidney failure.
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The disease is related to gene mutations and triggering events, like pregnancy, infections, cancers, and certain medications. The clinical manifestations of aHUS can involve various organ systems, including renal, hematologic, respiratory, ocular, skin, CNS, cardiovascular, and GI systems.
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Diagnosis includes a complete blood count (CBC) to measure red blood cell and platelet counts, and an estimated glomerular filtration rate (eGFR) to assess kidney function.
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In 2023, there were nearly 4,800 diagnosed cases of aHUS in the 7MM, with the majority of cases being in adults and adolescents (around 4,000 cases) compared to pediatric cases (approximately 700 cases).
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Currently, only eculizumab and ravulizumab are approved for treatment, both developed by Alexion Pharmaceuticals, which was acquired by AstraZeneca in 2021. Iptacopan (LNP023), an oral drug by Novartis, is in Phase III trials and could become a preferred treatment due to its convenient oral administration. It is expected to generate over USD 500 million by 2034.
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Challenges in the aHUS market include the high cost of medications, safety concerns, the lack of clear diagnostic criteria, and emerging generic competition, which may impact market uptake.
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As per DelveInsight analysis, the Atypical Hemolytic Uremic Syndrome market is anticipated to witness growth at a considerable CAGR
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Atypical Hemolytic Uremic Syndrome Overview
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare disease characterized by the destruction of red blood cells (hemolytic anemia), low platelet count (thrombocytopenia), and acute kidney failure (uremia), which impairs the kidneys’ ability to filter waste. Hemolytic anemia occurs when red blood cells rupture as they pass through clots in small blood vessels, leading to anemia if the body cannot replace them quickly enough. This condition can result in kidney damage, and approximately half of those affected develop end-stage renal disease (ESRD).
Diagnosing aHUS can be challenging, especially without a family history of the disease. The diagnosis is based on the presence of hemolytic anemia, low platelet count, and kidney dysfunction.
Treatment focuses on managing acute kidney injury and related complications. Patients with severe anemia may require packed red blood cell transfusions, while platelet transfusions are only needed in severe cases of thrombocytopenia. Maintaining fluid and electrolyte balance is crucial to address the effects of aHUS, and nephrotoxic medications should be avoided. Hypertension must be managed, and renal replacement therapy may be necessary for patients with kidney failure. Specific therapies, including plasma exchange and complement inhibitors like eculizumab or ravulizumab, are used to target the underlying mechanisms of the disease.
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Atypical Hemolytic Uremic Syndrome Epidemiology Insights
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DelveInsight’s epidemiology model estimates that there were approximately 4,800 diagnosed cases of aHUS across the 7MM in 2023, with this number expected to rise throughout the forecast period.
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The United States had the highest number of aHUS cases, with nearly 3,500 cases reported in 2023, and this figure is projected to increase by 2034.
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Among the 7MM, adolescents and adults made up about 80% of the diagnosed cases in 2023.
Atypical Hemolytic Uremic Syndrome Epidemiology Segmentation
DelveInsight’s Atypical Hemolytic Uremic Syndrome market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Atypical Hemolytic Uremic Syndrome historical patient pools and forecasted Atypical Hemolytic Uremic Syndrome patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Atypical Hemolytic Uremic Syndrome Market report proffers epidemiological analysis for the study period 2020-32 in the 7MM segmented into:
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Atypical Hemolytic Uremic Syndrome Prevalence
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Age-Specific Atypical Hemolytic Uremic Syndrome Prevalence
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Gender-Specific Atypical Hemolytic Uremic Syndrome Prevalence
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Diagnosed and Treatable Cases of Atypical Hemolytic Uremic Syndrome
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Atypical Hemolytic Uremic Syndrome Market Insights:
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Several major companies, including Alexion Pharmaceuticals, Novartis, and Roche, are leading the Atypical Hemolytic Uremic Syndrome (aHUS) treatment landscape. The market details, broken down by country and therapy, are outlined below.
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According to DelveInsight’s patient-based forecasting model, the total aHUS market size in the 7MM was approximately USD 1,300 million in 2023, and significant growth is anticipated during the forecast period due to the introduction of new therapies.
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In 2023, the United States accounted for the largest share of the aHUS market in the 7MM, approximately 70%, followed by Germany and France.
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Among emerging therapies for aHUS, Novartis’ iptacopan and Roche’s crovalimab are expected to play a pivotal role in transforming the market. By 2034, iptacopan is projected to generate the highest revenue among emerging treatments, with nearly USD 100 million expected in the EU4 and the UK.
Atypical Hemolytic Uremic Syndrome Market Outlook
The introduction of complement C5 inhibition has significantly enhanced patient outcomes and quality of life (QoL) in the treatment of Atypical Hemolytic Uremic Syndrome (aHUS). The available complement C5 inhibitors in the 7MM are SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), both products of Alexion Pharmaceuticals, which was acquired by AstraZeneca in 2021.
Switching patients from eculizumab to ravulizumab has proven to be an effective treatment strategy. This transition reduces the frequency of dosing, resulting in lower treatment-related costs and improved patient QoL.
Despite their proven safety and effectiveness, the high cost of eculizumab and ravulizumab limits their accessibility in the treatment landscape. As a result, there is a significant need for more effective pharmacological options, especially for managing the more severe and disabling stages of aHUS.
While several promising therapies are under investigation for aHUS, it is too early to confirm which will enter the market during the forecast period (2024–2034). However, it is clear that the future of this market holds promise. These new treatments are expected to bring substantial improvements to the aHUS treatment landscape in the coming years, driven by increasing global healthcare spending.
Atypical Hemolytic Uremic Syndrome Marketed Drugs
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ULTOMIRIS (ravulizumab): Alexion Pharmaceuticals
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SOLIRIS (eculizumab): Alexion Pharmaceuticals
Atypical Hemolytic Uremic Syndrome Emerging Drugs
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Crovalimab (RG6107; SKY59): Hoffmann-La Roche/Chugai Pharmaceutical
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FABHALTA (iptacopan): Novartis Pharmaceuticals
Atypical Hemolytic Uremic Syndrome Key Companies
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Alexion Pharmaceuticals, Omeros Corporation, Novartis Pharmaceuticals, Hoffmann-La Roche, Chugai Pharmaceutical, and others
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Scope of the Atypical Hemolytic Uremic Syndrome Market Report:
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11 Years Forecast
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7MM Coverage
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Descriptive overview of Atypical Hemolytic Uremic Syndrome, causes, signs and symptoms, diagnosis, treatment
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Comprehensive insight into Atypical Hemolytic Uremic Syndrome epidemiology in the 7MM
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Atypical Hemolytic Uremic Syndrome marketed and emerging therapies
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Atypical Hemolytic Uremic Syndrome companies
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Atypical Hemolytic Uremic Syndrome market drivers and barriers
Table of Contents:
1 Atypical Hemolytic Uremic Syndrome Market Key Comprehensive Insights
2 Atypical Hemolytic Uremic Syndrome Market Report Introduction
3 Competitive Intelligence Analysis for Atypical Hemolytic Uremic Syndrome
4 Atypical Hemolytic Uremic Syndrome Market Analysis Overview at a Glance
5 Executive Summary of Atypical Hemolytic Uremic Syndrome
6 Atypical Hemolytic Uremic Syndrome Epidemiology and Market Methodology
7 Atypical Hemolytic Uremic Syndrome Epidemiology and Patient Population
8 Atypical Hemolytic Uremic Syndrome Patient Journey
9 Atypical Hemolytic Uremic Syndrome Treatment Algorithm, Atypical Hemolytic Uremic Syndrome Current Treatment, and Medical Practices
10 Key Endpoints in Atypical Hemolytic Uremic Syndrome Clinical Trials
11 Atypical Hemolytic Uremic Syndrome Marketed Therapies
12 Atypical Hemolytic Uremic Syndrome Emerging Therapies
13 Atypical Hemolytic Uremic Syndrome: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Atypical Hemolytic Uremic Syndrome
16 Atypical Hemolytic Uremic Syndrome Market Key Opinion Leaders Reviews
18 Atypical Hemolytic Uremic Syndrome Market Drivers
19 Atypical Hemolytic Uremic Syndrome Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
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