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.
NCT00122330 ↗	Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients	Completed	Alexion Pharmaceuticals	Phase 3	2004-10-01	The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
NCT00130000 ↗	Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 3	2005-08-09	This study will evaluate the safety and effectiveness of the experimental drug eculizumab for treating paroxysmal nocturnal hemoglobinuria (PNH), a disorder that can cause premature destruction of red blood cells. PNH may result in anemia requiring blood transfusions. Patients may be at high risk of life-threatening blood clots in their veins and may have urine discoloration, stomach pain, difficulty swallowing, tiredness, and poor quality of life. Men may have problems getting or maintaining an erection. Eculizumab is a monoclonal antibody that may improve the survival of red blood cells in patients with PNH. Patients 18 years of age and older who have been diagnosed with PNH for more than 6 months, who have active disease, and who require blood transfusions may be eligible for this study. Each candidate is screened with a physical examination, electrocardiogram, blood and urine tests, and a questionnaire for information on how PNH affects the patient physically, socially, emotionally, and functionally. Participants receive infusions of eculizumab through a needle in a vein once a week for five doses and then every two weeks for another 24 doses. All patients are vaccinated against Neisseria meningitides, a bacteria that can cause symptoms, possibly including life-threatening meningitis, in susceptible people, including people who take eculizumab. At every treatment visit, patients update their health status, transfusion record, and medication use; review their laboratory results from the preceding visit; have vital signs measured; and provide a blood sample for laboratory tests. At selected visits, they also provide a urine sample, have a repeat electrocardiogram, and complete a questionnaire. At the final treatment visit, participants have a complete physical examination, in addition to the routine procedures.
NCT00438789 ↗	The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol	Approved for marketing	Alexion Pharmaceuticals		1969-12-31	The primary objective is to provide access to eculizumab for PNH patient pending commercial availability.
NCT00485576 ↗	Safety and Efficacy Study of Eculizumab in Patients With Mild Allergic Asthma	Completed	Alexion Pharmaceuticals	Phase 2	2007-09-01	This study is to designed to test the role of complement in the late asthmatic response to allergen challenge in mild asthma, as an indicator of the possible role of complement in the broader asthmatic population.

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
Hemoglobinuria, Paroxysmal	5

Condition MeSH

Condition MeSH for eculizumab
Intervention	Trials
Hemoglobinuria, Paroxysmal	37
Hemoglobinuria	32
Syndrome	19
Hemolysis	18

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 Market Projection

Last updated: April 20, 2026

What is the current status of clinical trials involving eculizumab?

Eculizumab (marketed as Soliris by Alexion Pharmaceuticals) continues to be evaluated in multiple clinical trials across rare disease indications and emerging therapeutic areas. Current trials aim to expand its use in complement-mediated disorders beyond paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

Active and Pending Clinical Trials (as of Q1 2023)

• Indications Investigated:

  • Myasthenia gravis (MG)
  • Neuromyelitis optica spectrum disorder (NMOSD)
  • Generalized myasthenia gravis
  • Refractory COVID-19 with cytokine storm

• Number and Phase:

  • 12 clinical trials: 7 Phase III, 4 Phase II, 1 Phase I

• Key Trials:

  • ClinicalTrials.gov identifiers:
  • NCT04571457: Efficacy of eculizumab in refractory generalized MG (Phase III)
  • NCT04459742: Use in NMOSD (Phase III)
  • NCT04680042: COVID-19 cytokine storm management (Phase II)

• Outcomes:

  • Early trials report promising reductions in disease activity and cytokine levels.
  • Confirmatory Phase III trials are ongoing, with interim data suggesting clinical benefit in selected patient populations.

Regulatory Status

• Approved in multiple jurisdictions for PNH, aHUS, and refractory generalized MG.
• Expanded access programs exist for investigational indications.
• European Medicines Agency (EMA) granted orphan designation for additional autoimmune diseases under development.

How mature is the market for eculizumab?

The eculizumab market is characterized by high exclusivity and limited competition but faces challenges due to its high cost and emergence of biosimilars.

Market Size and Revenue (2022)

Category	Data
Global revenue	$3.2 billion
Market share in complement inhibitors	90% (dominant biologic)
Number of approved indications	3 (PNH, aHUS, generalized MG)

Key Market Dynamics

• Pricing: In the US, list price exceeds $500,000 annually per patient.
• Patient Population:
  • PNH: Estimated 3,000 patients globally.
  • aHUS: Approximately 1,500 patients.
  • MG: An estimated 1,200 patients in the US.
• Market Penetration:
  • Nearly full penetration in developed markets.
  • Limited adoption in emerging economies due to cost and healthcare infrastructure.

Competition Landscape

• Biosimilars:
  • Several biosimilar candidates in late-stage development.
  • Expected to launch in Europe by 2025, potentially reducing prices.
• Alternative Therapies:
  • Ravulizumab (Ultomiris) by Alexion, offering longer dosing intervals.
  • Experimental agents targeting complement pathways via different mechanisms.

What is the market projection for eculizumab over the next decade?

Projected Revenue

Year	Global Revenue (billion USD)	Growth Rate (%)	Drivers
2023	$3.2	0%	Stable but plateauing market
2025	$3.1	-3%	Biosimilar competition begins
2030	$2.5	-10%	Increased biosimilar penetration, new indications emerge

Key Factors Influencing Market Trends

• Biosimilar Entry: Introduction of biosimilars expected to erode revenue by 15-20% annually after launch.
• Expansion into Autoimmune Diseases: Ongoing trials could unlock new revenue streams if successful.
• Reimbursement Policies: Cost containment measures and value-based pricing will influence adoption rates.
• Regulatory Approvals:
  • Additional indications could expand the market.
  • Delays or failure in clinical trials could limit growth.

Long-term Outlook

• The market may decline as biosimilars gain market share.
• Revenue stabilization might occur if new indications receive approval and demonstrate significant clinical benefits.
• Innovation in complement inhibition strategies may challenge eculizumab's dominance.

Key Takeaways

• Eculizumab remains a critical drug for complement-mediated rare diseases with a current revenue of about $3.2 billion.
• Its clinical pipeline targets expanded indications, notably autoimmune neurological disorders.
• Biosimilar development is the primary risk to its long-term market share, with European biosimilars expected around 2025.
• Market growth is expected to decline gradually over the next decade, driven by biosimilar competition and reimbursement policies.
• Continued clinical success and regulatory approvals for new indications could stabilize or extend market relevance.

Frequently Asked Questions

1. What diseases does eculizumab currently treat?
   Eculizumab is approved for PNH, aHUS, and refractory generalized myasthenia gravis.

2. Are biosimilars a threat to eculizumab’s market?
   Yes. Biosimilars in late-stage development could reduce prices and market share, especially in Europe where biosimilar approval is imminent.

3. What is the primary driver for eculizumab’s market decline?
   Biosimilar entry and subsequent price erosion.

4. Has eculizumab demonstrated efficacy in other diseases?
   Clinical trials show promise in autoimmune and inflammatory conditions, but regulatory approvals are pending.

5. What factors could extend eculizumab’s market life?
   Approval of new indications and successful clinical trial outcomes could sustain or grow its market share.

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References

1. ClinicalTrials.gov. (2023). Eculizumab clinical trials. Retrieved from https://clinicaltrials.gov
2. IQVIA Institute. (2022). The Global Use of Medicines in 2022.
3. European Medicines Agency. (2022). Eculizumab approval updates.
4. Alexion Pharmaceuticals. (2023). Soliris product monograph.
5. MarketWatch. (2023). Biologic drugs market analysis.