Emerging therapies for Atypical Hemolytic Uremic Syndrome (aHUS), including Crovalimab (RG6107; SKY59), FABHALTA (iptacopan), and others, are expected to drive significant growth in the aHUS market in the coming years.
DelveInsight has released a new report, “Atypical Hemolytic Uremic Syndrome – Market Insights, Epidemiology, and Market Forecast 2034”, providing comprehensive insights into aHUS, including historical and projected epidemiology, as well as market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Discover about the Atypical Hemolytic Uremic Syndrome market report
During the ERS Congress 2025 in September 2025, several biotechnology and pharmaceutical companies shared notable advancements in pulmonary and cardiopulmonary research:
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In September 2025, Diagonal Therapeutics presented new preclinical data highlighting the potential of its lead clustering antibody, DIAG723, as a disease-modifying therapy for pulmonary arterial hypertension (PAH).
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In September 2025, a study revealed that babies raised with dogs may have a reduced risk of developing childhood asthma.
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In September 2025, Savara shared data from the Phase 3 IMPALA-2 trial of Molgramostim Inhalation Solution for patients with autoimmune pulmonary alveolar proteinosis (aPAP).
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In September 2025, AllRock Bio, Inc. reported encouraging Phase 1 results for ROC-101, a first-in-class oral pan-ROCK inhibitor for cardiopulmonary and fibrotic diseases.
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In September 2025, Gossamer Bio, Inc., in collaboration with Chiesi Group, announced five scientific presentations on seralutinib for PAH and PH-ILD.
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In September 2025, Boehringer Ingelheim presented pooled analyses from the Phase III FIBRONEER™ program on nerandomilast, an investigational oral PDE4B inhibitor, showing a nominally significant reduction in mortality risk across IPF and PPF.
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In September 2025, Kaia Health launched a pilot program of Kaia Breathe, a digital pulmonary rehabilitation solution for COPD patients, in partnership with MedImprove.
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In September 2025, aTyr Pharma, Inc. shared additional findings from the Phase 3 EFZO-FIT™ study of efzofitimod in 268 patients with pulmonary sarcoidosis, a key form of interstitial lung disease.
Some of the key facts of the Atypical Hemolytic Uremic Syndrome Market Report:
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In 2023, the United States accounted for the largest share of the Atypical Hemolytic Uremic Syndrome (aHUS) market among the seven major markets (7MM), with the market expected to grow at a compound annual growth rate (CAGR) of around 4% through 2034. aHUS is a rare disorder characterized by abnormal blood clot formation in small vessels, primarily affecting the kidneys, and can lead to hemolytic anemia, thrombocytopenia, and kidney failure.
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Patients often present with symptoms such as confusion, seizures, strokes, high blood pressure, and organ damage. While aHUS can occur at any age, it is more common in adolescents and adults. In 2023, approximately 4,800 cases were diagnosed across the 7MM, including around 4,000 in adolescents and adults and 700 in children. Unlike typical HUS, which is usually triggered by infections like E. coli, aHUS is often associated with genetic mutations that require a precipitating event—such as infection, pregnancy, cancer, or certain medications—to activate the disease.
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Currently, only two FDA-approved treatments—eculizumab and ravulizumab, both from Alexion Pharmaceuticals (acquired by AstraZeneca in 2021)—are available. While effective, these therapies are limited by high costs and safety concerns. Novartis’s iptacopan (LNP023), an oral therapy in Phase III trials, is expected to provide a more convenient option, with projected sales exceeding USD 500 million by 2034.
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Challenges in the aHUS market include high drug prices, limited diagnostic clarity, safety concerns, and potential generic competition, which could impact growth in the coming years. Key companies shaping the aHUS treatment landscape include Alexion Pharmaceuticals, Novartis, and Roche.
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According to DelveInsight’s patient-based forecasting model, the total aHUS market across the 7MM was valued at approximately USD 1.3 billion in 2023, with substantial growth anticipated over the forecast period driven by new therapy launches. In 2023, the U.S. held about 70% of the total market value, followed by Germany and France.
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Recent developments include the April 2025 launch of Epysqli (eculizumab‑aagh), a Soliris biosimilar from Teva and Samsung Bioepis, available in the U.S. for aHUS (as well as PNH and gMG) at a 30% lower cost. Iptacopan (Fabhalta), although not yet FDA-approved for aHUS, has received multiple approvals for related complement-mediated kidney diseases, most recently for C3 glomerulopathy in March 2025. Additionally, the FDA issued draft guidance in May 2025 regarding iptacopan hydrochloride, marking progress toward potential approval pathways for anti-complement therapies.
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Key aHUS companies, including Alexion Pharmaceuticals, Omeros Corporation, Novartis Pharmaceuticals, Hoffmann-La Roche, Chugai Pharmaceutical, and others, are actively developing new therapies to enhance the treatment landscape. Promising pipeline candidates include Crovalimab (RG6107; SKY59), FABHALTA (iptacopan), and additional emerging therapies.
Atypical Hemolytic Uremic Syndrome Overview
Atypical Hemolytic Uremic Syndrome (aHUS) is the most prevalent form of central nervous system (CNS) tumor, arising from glial cells. These tumors are highly invasive, often infiltrating surrounding brain tissue. aHUS is generally classified into three main types based on cellular characteristics: astrocytomas, ependymomas, and oligodendrogliomas. The tumors develop due to genetic mutations accumulating in glial stem or progenitor cells, leading to uncontrolled cell proliferation.
aHUS tumors are graded from I to IV, with glioblastoma (GBM, Grade IV) being the most aggressive, and pilocytic astrocytomas (Grade I) being the least malignant. Frequently mutated genes include tumor suppressors such as TP53 and PTEN, growth-regulating genes like BRAF, and metabolism-related genes including IDH1.
Atypical Hemolytic Uremic Syndrome Market Outlook
The introduction of complement C5 inhibitors has significantly improved outcomes and quality of life for patients with atypical hemolytic uremic syndrome (aHUS). The primary therapies available across the seven major markets (7MM) are SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), both developed by Alexion Pharmaceuticals, which was acquired by AstraZeneca in 2021.
A common strategy is to transition patients from eculizumab to ravulizumab due to its less frequent dosing schedule. This switch not only reduces the financial and logistical burden of regular infusions but also enhances patient convenience and overall quality of life.
Despite their efficacy and safety, the high cost of these therapies limits broader accessibility. Therefore, there is a strong need for more affordable and effective treatment options, especially for managing severe aHUS cases.
In summary, while several promising therapies are in development, it is still too early to predict their market availability. Nevertheless, the outlook for the aHUS treatment landscape is optimistic, with anticipated advancements driven by increased healthcare investment and innovation, likely improving patient care in the coming years.
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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
Scope of the Atypical Hemolytic Uremic Syndrome Market Report
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Study Period: 2020-2034
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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
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Key Atypical Hemolytic Uremic Syndrome Companies: Alexion Pharmaceuticals, Omeros Corporation, Novartis Pharmaceuticals, Hoffmann-La Roche, Chugai Pharmaceutical, and others
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Atypical Hemolytic Uremic Syndrome Therapies: Crovalimab (RG6107; SKY59), FABHALTA (iptacopan), and others.
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Atypical Hemolytic Uremic Syndrome Therapeutic Assessment: Atypical Hemolytic Uremic Syndrome current marketed and Atypical Hemolytic Uremic Syndrome emerging therapies
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Atypical Hemolytic Uremic Syndrome Market Dynamics: Atypical Hemolytic Uremic Syndrome market drivers and Atypical Hemolytic Uremic Syndrome market barriers
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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
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Atypical Hemolytic Uremic Syndrome Unmet Needs, KOL’s views, Analyst’s views, Atypical Hemolytic Uremic Syndrome Market Access and Reimbursement
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Key benefits of the Atypical Hemolytic Uremic Syndrome Market Report:
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Atypical Hemolytic Uremic Syndrome market report covers a descriptive overview and comprehensive insight of the Atypical Hemolytic Uremic Syndrome Epidemiology and Atypical Hemolytic Uremic Syndrome market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
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The Atypical Hemolytic Uremic Syndrome market report provides insights into the current and emerging therapies.
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The Atypical Hemolytic Uremic Syndrome market report provides a global historical and forecasted market covering drug outreach in 7MM.
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The Atypical Hemolytic Uremic Syndrome market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Atypical Hemolytic Uremic Syndrome market.
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Table of Contents
1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Atypical Hemolytic Uremic Syndrome Patient Share (%) Overview at a Glance
5. Atypical Hemolytic Uremic Syndrome Market Overview at a Glance
6. Atypical Hemolytic Uremic Syndrome Disease Background and Overview
7. Atypical Hemolytic Uremic Syndrome Epidemiology and Patient Population
8. Country-Specific Patient Population of Atypical Hemolytic Uremic Syndrome
9. Atypical Hemolytic Uremic Syndrome Current Treatment and Medical Practices
10. Unmet Needs
11. Atypical Hemolytic Uremic Syndrome Emerging Therapies
12. Atypical Hemolytic Uremic Syndrome Market Outlook
13. Country-Wise Atypical Hemolytic Uremic Syndrome Market Analysis (2020–2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Atypical Hemolytic Uremic Syndrome Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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Atypical Hemolytic Uremic Syndrome Pipeline Insights, DelveInsight
“Atypical Hemolytic Uremic Syndrome Pipeline Insight, 2024” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Atypical Hemolytic Uremic Syndrome market. A detailed picture of the Atypical Hemolytic Uremic Syndrome pipeline landscape is provided, which includes the disease overview and Atypical Hemolytic Uremic Syndrome treatment guidelines.
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