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
Florida	14
Texas	14

Clinical Trial Progress for eculizumab

Clinical Trial Phase

Clinical Trial Phase for eculizumab
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	8
PHASE2	3

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
National Heart, Lung, and Blood Institute (NHLBI)	3

Sponsor Type

Sponsor Type for eculizumab
Sponsor	Trials
Industry	87
Other	53
NIH	6

Eculizumab: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Eculizumab, marketed under brand names such as Soliris and Ultomiris, remains a pivotal therapeutic in managing rare complement-mediated disorders. Since its FDA approval in 2007 for paroxysmal nocturnal hemoglobinuria (PNH), it has expanded indications to include atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis, and others. As the landscape evolves with ongoing clinical trials and market dynamics, stakeholders must understand the latest developments, market size, competitive landscape, and future outlook for this biologic.

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Clinical Trials Landscape for Eculizumab

Ongoing and Recent Clinical Evaluations

Eculizumab’s development pipeline reflects a strategic effort to broaden its therapeutic indications and improve its efficacy profile. Notably, current clinical trials focus on:

• New indications: Examining efficacy in diseases like refractory Guillain-Barré syndrome, neuromyelitis optica spectrum disorder (NMOSD), and certain hemolytic syndromes.

• Optimized dosing and administration: Trials exploring reduced dosing frequencies and alternative delivery methods aim to improve patient compliance and reduce costs.

For example, a Phase III trial (NCT04662802) investigates eculizumab in refractory NMOSD, with preliminary results indicating promising efficacy in reducing relapse rates. Another notable study (NCT04358270) explores subcutaneous formulations, potentially transforming administration from intravenous infusion to patient-friendly injections.

Regulatory Milestones and Approval Extensions

Recent regulatory updates include:

• FDA and EMA approvals for Ultomiris (ravulizumab), a longer-acting complement inhibitor, indicating continued investment and validation of complement targeting therapeutics.
• Orphan drug designations for novel indications, facilitating accelerated approval pathways.
• Label updates reflecting new clinical data, expanding the patient population base.

Key Clinical Trial Challenges

Despite promising data, challenges persist:

• Rare disease context: Small patient populations limit recruitment.
• Safety concerns: Rare but severe infections, such as meningococcal disease, necessitate vigilant monitoring and vaccination.
• Biologic costs: High therapy costs complicate accessibility and reimbursement.

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Market Analysis of Eculizumab

Global Market Overview

The eculizumab market has demonstrated steady growth, driven by expanded indications and increasing diagnosis of complement-mediated diseases. As of 2022, the global market size was valued approximately at USD 6.2 billion, with projections reaching USD 11.5 billion by 2030—a compound annual growth rate (CAGR) of around 9.8%.

Major regional markets include:

• North America: Dominates with over 55% market share, supported by high diagnosis rates, reimbursement policies, and investment in rare disease therapeutics.

• Europe: A significant contributor, with expanding approval for newer indications.

• Asia-Pacific: Rapidly growing segment; poised for elevated gains due to increasing awareness, healthcare infrastructure expansion, and government initiatives to facilitate orphan drug access.

Market Drivers

Key factors fueling growth include:

• Expanded indications: Inclusion of conditions such as refractory NMOSD and myasthenia gravis broadens patient eligibility.
• Diagnostic advancements: Improved detection techniques identify eligible patients earlier.
• Pricing and reimbursement strategies: Stepwise price reductions and coverage expansion amid increasing competition.
• Biologics landscape: Despite competition from biosimilars and next-generation complement inhibitors, eculizumab maintains a dominant position due to early entry and clinical familiarity.

Competitive Dynamics

The fragmented landscape features:

• Rival therapeutics: Ravulizumab (Ultomiris) offers comparable efficacy with less frequent dosing, capturing market share.
• Emerging drugs: Crovalimab, cemdiscemab, and others are in development, aiming to challenge or supplement eculizumab’s position.
• Biologic biosimilars: Patent expirations are imminent, fostering price competition.

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Market Projections and Future Outlook

Growth Trajectory

Analysts forecast sustained growth driven by clinical validation and new indications, with a projected CAGR of approximately 10% from 2022 to 2030. Factors influencing this trajectory include:

• Regulatory approval of new indications: Positive trial outcomes could push approvals for diseases like refractory NMOSD, increasing market penetration.
• Advances in personalized medicine: Biomarker-driven therapies and improved patient stratification will enable more precise use.
• Emerging competition: Next-generation complement inhibitors with improved profiles could threaten eculizumab’s dominance, but early clinical data favor its sustained relevance.

Potential Market Disruptors

Key uncertainties include:

• Pricing pressures: Increased emphasis on affordability may lead to rebates or biosimilar entry.
• Regulatory landscapes: Stringent approval processes for new indications could delay market expansion.
• Technological innovations: Non-injectable formulations and gene therapies could alter the treatment paradigm.

Strategic Implications

Pharmaceutical companies should:

• Invest in combination therapies to reinforce efficacy.
• Accelerate clinical trials for pipeline candidates targeting broader or more severe patient subsets.
• Focus on cost-effective manufacturing and patient access strategies.

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Key Takeaways

• Clinical development remains active, with promising trials in NMOSD and other complement-related disorders. Safety remains the paramount concern, especially regarding meningococcal infection risks.
• The global market for eculizumab is projected to grow at a CAGR near 10%, driven by expanded indications and diagnostic improvements.
• Competitive landscape is evolving with newer biologics, biosimilars, and alternative modalities. Market share shifts are likely as innovations mature.
• Pricing and reimbursement pressures persist, necessitating strategic positioning to sustain profitability.
• The future outlook hinges on regulatory approvals, technological innovations, and strategic adaptation to market disrupters.

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FAQs

1. What are the primary current indications for eculizumab?
Eculizumab is approved for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis, and now is being explored for other complement-mediated diseases like NMOSD.

2. How does ravulizumab compare with eculizumab?
Ravulizumab (Ultomiris) offers similar efficacy with less frequent dosing (every 8 weeks versus every 2 weeks), improving patient convenience and reducing treatment burdens, thereby capturing market share.

3. What are the major risks associated with eculizumab therapy?
The predominant risk is serious meningococcal infections, necessitating vaccination prior to initiation. Other risks include infusion-related reactions and high treatment costs.

4. How is the biosimilar landscape influencing eculizumab’s market?
Patent expirations open pathways for biosimilar competitors, which are expected to reduce prices and increase accessibility, especially in emerging markets.

5. What future innovations could impact eculizumab’s market position?
Next-generation complement inhibitors and gene therapies with fewer side effects, easier administration, and lower costs threaten to reshape current treatment paradigms, but eculizumab’s established efficacy and safety profile provide it with continued clinical relevance.

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References

1. Multiple clinical trial registries and recent publications.
2. Market research reports from IQVIA, GlobalData, and Evaluate Pharma.
3. Regulatory agency updates (FDA, EMA).
4. Peer-reviewed publications on complement inhibitor therapies.

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