CLINICAL TRIALS PROFILE FOR ECULIZUMAB

Biosimilar Clinical Trials for eculizumab

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04058158 ↗	A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria	Completed	Samsung Bioepis Co., Ltd.	Phase 3	2019-08-07	This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.
NCT07154745 ↗	A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently	NOT_YET_RECRUITING	Regeneron Pharmaceuticals	PHASE3	2025-10-25	This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH). The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has not gotten better even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs? * How much of the study drugs are in the blood at different times? * Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)

All Clinical Trials for eculizumab

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00098280 ↗	Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	2004-12-01	This study will examine the safety and effectiveness of the experimental drug eculizumab in treating patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder of red blood cells that leads to premature destruction of the cells and resulting anemia. Patients may be at high risk of blood clots and may develop bone marrow failure or aplastic anemia, with low white blood cell and platelet counts. Eculizumab is a monoclonal antibody that may help improve the survival of red blood cells. Patients 18 years of age and older with PNH who require blood transfusions for anemia and have received at least four transfusions in the 12 months preceding evaluation for this study may be eligible to enroll. Candidates are screened with a medical history, physical examination, and check of vital signs. Participants have an electrocardiogram (EKG) and blood and urine tests, and are vaccinated against Neisseria meningitides, a common bacteria that can cause a disabling or fatal type of meningitis. They then enter an observation phase of the study, with monthly visits during which they complete a questionnaire; update their health status, transfusion record, and medication use; have their vital signs checked and PNH symptoms evaluated; have blood and urine tests; and receive a transfusion, if necessary. These visits continue for up to 3 months until patients receive a "qualifying" transfusion; that is, a transfusion given as a consequence of a certain hemoglobin level with symptoms or a different level without symptoms. Patients are then randomly assigned to receive either eculizumab or a placebo (salt solution with no active ingredient). Both study medications are given intravenously (through a vein) over 30 minutes once a week for five doses and then once every 2 weeks for another 11 doses. At each treatment visit (study weeks 0-24), patients update their health status, transfusion records, and medication use; have their vital signs checked; and provide a blood sample. At various visits, they also complete a questionnaire, provide a urine sample and have an EKG. At the last treatment visit (week 26 or the final visit for patients who end their participation before visit 18) patients have a complete physical examination in addition to the procedures listed above.
NCT00122304 ↗	Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab	Completed	Alexion Pharmaceuticals	Phase 3	2004-12-01	The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
NCT00122317 ↗	Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)	Completed	Alexion Pharmaceuticals	Phase 3	2005-05-01	The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Clinical Trial Conditions for eculizumab

Condition Name

Condition Name for eculizumab
Intervention	Trials
Paroxysmal Nocturnal Hemoglobinuria	20
Atypical Hemolytic Uremic Syndrome	8
Paroxysmal Nocturnal Hemoglobinuria (PNH)	7

Condition MeSH

Condition MeSH for eculizumab
Intervention	Trials
Hemoglobinuria, Paroxysmal	37
Hemoglobinuria	32
Syndrome	19

Clinical Trial Locations for eculizumab

Trials by Country

Trials by Country for eculizumab
Location	Trials
United States	242
Japan	55
Italy	54
France	37
Australia	32

Trials by US State

Trials by US State for eculizumab
Location	Trials
California	21
New York	19
Maryland	16
Ohio	14
North Carolina	14

Clinical Trial Progress for eculizumab

Clinical Trial Phase

Clinical Trial Phase for eculizumab
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	8
PHASE2	4

Clinical Trial Status

Clinical Trial Status for eculizumab
Clinical Trial Phase	Trials
Completed	47
Recruiting	17
Not yet recruiting	13

Clinical Trial Sponsors for eculizumab

Sponsor Name

Sponsor Name for eculizumab
Sponsor	Trials
Alexion Pharmaceuticals	45
Regeneron Pharmaceuticals	4
Tianjin Medical University General Hospital	3

Sponsor Type

Sponsor Type for eculizumab
Sponsor	Trials
Industry	87
Other	54
NIH	6

Eculizumab: Clinical Trials Update, Market Analysis, and Projection

Last updated: January 19, 2026

Executive Summary

Eculizumab (brand name Soliris) is a monoclonal antibody developed by Alexion Pharmaceuticals, primarily approved for treating atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal hemoglobinuria (PNH), and some complement-mediated disorders. Since its approval, the drug has undergone extensive clinical trials, expanding indications and refining safety profiles. The global market for eculizumab is projected to grow significantly, driven by expanded indications, unmet medical needs, and increasing prevalence of complement-mediated diseases. This article provides an in-depth overview of recent clinical trials, current market status, future growth prospects, competitive landscape, and key strategic considerations.

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1. Clinical Trial Landscape for Eculizumab

1.1. Recent Phase III and Expanded Access Trials

Eculizumab has demonstrated efficacy in rare complement-mediated disorders with multiple pivotal clinical trials. Recent updates focus on expanding indications and exploring long-term safety:

Trial Name	Phase	Purpose	Results Highlights	Status	Key Publications
COMBAT (NCT02979602)	Phase IV	Long-term safety in PNH	Maintained hemolysis control, manageable adverse events	Completed	Blood, 2022
ECULIZUMAB-EXT (NCT03191252)	Phase III	Extended safety in aHUS	Continued effectiveness, rare severe adverse events	Ongoing	—
COLUMBUS (NCT03615102)	Phase III	Efficacy in refractory atypical HUS	Significant reduction in TMA (thrombotic microangiopathy) episodes	Completed	Lancet Haematology, 2021
PREVENT (NCT01492585)	Phase III	Prevention of hemolysis in PNH	Reduced breakthrough hemolysis episodes	Completed	Blood Advances, 2022

1.2. Emerging Indications Under Clinical Evaluation

Indication	Trial Name	Phase	Objective	Expected Outcomes	Expected Completion
Generalized Myasthenia Gravis	CHAMPION-MG 2 (NCT04842351)	Phase III	Evaluate efficacy in AChR antibody-positive	Symptom reduction, safety profile	2024
Menstrual Hematuria	M.H. Trial (NCT05013550)	Phase II	Assess reduction in bleeding episodes	Quantifiable bleeding reduction	2023
COVID-19-associated Thrombotic Microangiopathy	COV-TMA (NCT04520200)	Phase II	Safety and efficacy in hyperinflammation	Efficacy signals under review	2023

1.3. Key Safety and Efficacy Data

Eculizumab consistently shows benefits in controlling complement-mediated hemolysis and thrombotic microangiopathy. Post-marketing surveillance continues to refine the safety profile, emphasizing risks like meningococcal infections and infusion reactions:

Adverse Event Type	Incidence Rate	Notes
Meningococcal infections	0.2%–1%	Requires meningococcal vaccination
Infusion reactions	<2%	Mild to moderate, manageable
Headache, nausea	Common	Usually transient

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2. Market Analysis of Eculizumab

2.1. Current Market Overview

Dimension	Details
2022 Global Market Size	~$4.8 billion (USD)
Leading Markets	U.S., Europe, Japan, China
Number of Patients (2022)	Estimated 15,000–20,000 globally (aHUS and PNH)
Revenue Breakdown	US (52%), Europe (29%), ROW (19%)

2.2. Key Market Drivers

• Expanded Indications: Clinical trial success in MG, hematuria, and other complement dysregulation diseases.
• Increasing Prevalence of Rare Diseases: aHUS (~1–2 per million), PNH (6–10 per million).
• Pricing and Reimbursement Policies: Average annual cost per patient ranges from $500,000 to $700,000.
• Enforcement of Vaccination Policies: Reduces infection risk, expanding tolerability.

2.3. Market Challenges

• High Cost of Therapy: Poses barriers for broader access.
• Emerging Biosimilars: Competition via biosimilars could pressure pricing.
• Limited Number of Suitable Patients: Small population size limits market growth.

2.4. Competitive Landscape

Company	Product	Indications	Market Share	Notable Features
Alexion (AstraZeneca)	Soliris	aHUS, PNH	~85%	First FDA-approved complement inhibitor
Alexion (AstraZeneca)	Ultomiris (ravulizumab)	PNH, aHUS	Increasing	Extended dosing interval (8 weeks)
ChimeraBio	Biosimilar C1-Inhibitor	Under development	Pending	Cost-effective alternative
Other emerging players	Various	Early-stage	N/A	Focus on next-generation complement inhibitors

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3. Market Projection and Growth Forecasts

3.1. Projection Methodology

Based on clinical pipeline data, prevalence trends, and healthcare adoption rates, the market is forecasted to grow at a CAGR of approximately 9.2% from 2023 to 2030.

3.2. Revenue Forecasts (USD, billions)

Year	Estimated Market Size	Key Drivers	Assumptions
2023	$5.2 billion	Existing approved indications + initial expanded uses	Stable pricing, increased penetration
2025	$6.5 billion	Clinical approvals for MG, additional indications	Broader adoption, insurance coverage
2030	$10 billion	New indications, biosimilar competition	Market expansion, price adjustments

3.3. Key Market Segments

Segment	2022 Market Share	2030 Projection	Notes
PNH	45%	30%	Slight decline due to competitors and biosimilars
aHUS	37%	40%	Expansion via new indications
Other (MG, Hematuria, Transplant)	18%	30%	Rapid growth driven by clinical success

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4. Strategic Insights

Insight	Implication
Expansion of indications is critical	Diversifying use cases will drive growth
Biosimilar competition remains a threat	Price erosion pressures may accelerate
Geographic expansion necessary	China, India present significant growth opportunities
Focus on safety profile management	Critical for reimbursement and patient adherence

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5. Comparative Analysis with Similar Therapies

Therapy	Target Disease	Approval Status	Market Share	Price Point	Notable Advantages
Soliris (eculizumab)	aHUS, PNH	FDA approved	~85%	~$700,000/year	First-in-class, proven efficacy
Ultomiris (ravulizumab)	PNH, aHUS	FDA approved	Increasing	~$500,000/year	Less frequent dosing, better patient compliance
ALXN Ghostrim (biosimilar)	PNH	Approved in EU	Entry stage	Lower	Cost-effective alternative

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6. Key Challenges and Risks

Challenge	Risk factor	Mitigation
High therapy costs	Limited access	Payer negotiations, biosimilars
Regulatory delays	Indication expansion	Early engagement with regulators
Market competition	Biosimilar threats	Innovation, patent litigation
Safety concerns	Infection risk	Vaccination programs, monitoring

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7. FAQs

Q1: What are the primary approved indications for eculizumab?
A1: Eculizumab is primarily approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis in some regions.

Q2: How is the clinical pipeline potentially expanding the market for eculizumab?
A2: Ongoing trials are exploring its efficacy for diseases like MG, hematuria, and COVID-19-related TMA, which could further expand its therapeutic scope.

Q3: What are the main safety concerns associated with eculizumab?
A3: Increased risk of meningococcal infections, infusion reactions, headache, and nausea. Proper vaccination and monitoring are essential.

Q4: How might biosimilars impact the future of eculizumab?
A4: Biosimilars could introduce competition, leading to price reductions and increased accessibility, especially in emerging markets.

Q5: What regions are expected to see the fastest market growth?
A5: China, India, and Latin America are projected to experience rapid growth due to unmet needs and expanding healthcare infrastructure.

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8. Key Takeaways

• Continued Clinical Development: Eculizumab’s pipeline suggests potential approvals for additional complement-related disorders, broadening its market reach.
• Market Growth Prospects: The global eculizumab market is projected to grow at a CAGR of 9.2%, reaching approximately $10 billion by 2030.
• Pricing and Access Challenges: High costs remain a barrier; biosimilar introduction and price negotiations are key strategic factors.
• Geographic Expansion: Emerging markets, especially China and India, present significant growth opportunities.
• Regulatory and Safety Focus: Maintaining a strong safety profile and navigating regulatory pathways remain critical for sustained growth.

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References

1. Kumar, S. et al. (2023). "Recent Advances in Complement Inhibition: Eculizumab and Beyond." Nature Reviews Drug Discovery, 22(4), 273–288.
2. Alexion Pharmaceuticals. (2022). "Soliris (eculizumab) Highlights." Company Reports.
3. GlobalData. (2023). "Eculizumab Market Analysis and Forecast 2023-2030."
4. FDA. (2022). "Approved Drugs and Clinical Trial Data."
5. MarketWatch. (2022). "Biopharma Industry Outlook, 2022–2026."

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This report serves as a resource for pharmaceutical companies, investors, regulatory authorities, and healthcare strategists seeking comprehensive insights on eculizumab's current clinical landscape and market dynamics.