CLINICAL TRIALS PROFILE FOR SOLIRIS

Biosimilar Clinical Trials for SOLIRIS

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.

All Clinical Trials for SOLIRIS

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
NCT00670774 ↗	Dosing Regimen of Eculizumab Added to Conventional Treatment in Positive Cross Match Living Donor Kidney Transplant	Completed	Alexion Pharmaceuticals	Phase 1/Phase 2	2008-03-01	A strongly positive crossmatch has long been considered an absolute contraindication to kidney transplantation and most patients with anti-human leukocyte antigen (HLA) antibody never were able to receive a kidney transplant. Over the past decade, significant progress has been made in overcoming early antibody-mediated renal allograft injury. Our group has performed more than 200 such transplants providing the possibility of transplant to previously untransplantable patients. Despite our best efforts, transplantation in these patients is still complicated by a high rate of acute humoral rejection (AHR). Patients included in this study will be those who have demonstrable anti-HLA antibody specific for their living donor. It is our hypothesis that blockade of terminal complement activation at the time of transplant in combination with our current protocols will reduce the incidence of AHR in patients with anti-donor HLA antibody.
NCT00670774 ↗	Dosing Regimen of Eculizumab Added to Conventional Treatment in Positive Cross Match Living Donor Kidney Transplant	Completed	Mark Stegall	Phase 1/Phase 2	2008-03-01	A strongly positive crossmatch has long been considered an absolute contraindication to kidney transplantation and most patients with anti-human leukocyte antigen (HLA) antibody never were able to receive a kidney transplant. Over the past decade, significant progress has been made in overcoming early antibody-mediated renal allograft injury. Our group has performed more than 200 such transplants providing the possibility of transplant to previously untransplantable patients. Despite our best efforts, transplantation in these patients is still complicated by a high rate of acute humoral rejection (AHR). Patients included in this study will be those who have demonstrable anti-HLA antibody specific for their living donor. It is our hypothesis that blockade of terminal complement activation at the time of transplant in combination with our current protocols will reduce the incidence of AHR in patients with anti-donor HLA antibody.
NCT00727194 ↗	Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis	Terminated	Alexion Pharmaceuticals	Phase 2	2008-10-01	The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of patients with generalized myasthenia gravis despite treatment with various immunosuppressants, such as prednisone, methotrexate, Cellcept, cyclosporine, and cyclophosphamide, that are currently available.
NCT00867932 ↗	Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)	Completed	Alexion Pharmaceuticals	Phase 4	2009-10-02	The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.

Clinical Trial Conditions for SOLIRIS

Condition Name

Condition Name for SOLIRIS
Intervention	Trials
Paroxysmal Nocturnal Hemoglobinuria	8
Neuromyelitis Optica	3
Kidney Transplant	3
PNH	2

Condition MeSH

Condition MeSH for SOLIRIS
Intervention	Trials
Hemoglobinuria, Paroxysmal	14
Hemoglobinuria	14
Syndrome	5
Vascular Diseases	3

Clinical Trial Locations for SOLIRIS

Trials by Country

Trials by Country for SOLIRIS
Location	Trials
United States	143
Japan	20
Italy	17
Australia	16
Canada	14

Trials by US State

Trials by US State for SOLIRIS
Location	Trials
California	13
New York	11
Maryland	9
Florida	8
North Carolina	8

Clinical Trial Progress for SOLIRIS

Clinical Trial Phase

Clinical Trial Phase for SOLIRIS
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	9
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for SOLIRIS
Clinical Trial Phase	Trials
Completed	21
Terminated	8
Recruiting	7

Clinical Trial Sponsors for SOLIRIS

Sponsor Name

Sponsor Name for SOLIRIS
Sponsor	Trials
Alexion Pharmaceuticals	25
Apellis Pharmaceuticals, Inc.	3
Mayo Clinic	3

Sponsor Type

Sponsor Type for SOLIRIS
Sponsor	Trials
Industry	41
Other	26
NIH	3

Clinical Trials Update, Market Analysis, and Projection for SOLIRIS (Eculizumab)

Last updated: January 25, 2026

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Summary

SOLIRIS (eculizumab), developed by Alexion Pharmaceuticals (a subsidiary of AstraZeneca), is a monoclonal antibody targeting the complement protein C5. It is approved for rare complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Recent clinical trials, regulatory developments, and market dynamics suggest a focus on expanding therapeutic indications and geographic reach.

Projected revenues anticipate continued growth driven by increased adoption, pipeline expansion, and geographic expansion into emerging markets. This report synthesizes current clinical research, competitive landscape, and financial forecasts from 2023 to 2028.

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1. Clinical Trials Update for SOLIRIS

Trial Name	Phase	Indication	Status	Key Findings	Completion Date	Source
SOLIRIS in Adult PNH (NCT04649663)	Phase 3	PNH	Active, recruiting	Eculizumab shows sustained hemoglobin stabilization and reduced transfusions	Expected late 2023	[1]
Eculizumab in Complement-Mediated Myasthenia Gravis	Phase 3	MG	Completed; data under review	Significant improvement in muscle strength; favorable safety profile	2022	[2]
Preventing aHUS in Pediatric Patients (NCT04132566)	Phase 2/3	aHUS	Ongoing	Promising safety; reduction in thrombotic microangiopathy episodes	Expected 2024	[3]
Long-Term Efficacy in NMOSD (NCT04586052)	Phase 3	Neuromyelitis Optica Spectrum Disorder	Ongoing	Positive trends in relapse reduction; safety maintained	Expected 2024	[4]

Major ongoing studies focus on expanding indications beyond approved uses, including neurology (e.g., NMOSD), transplantation, and complement-mediated diseases.

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2. Market Overview and Competitive Landscape

Parameter	Details
Current Approved Indications	PNH, aHUS, generalized myasthenia gravis (gMG) with anti-AChR antibodies, neuromyelitis optica spectrum disorder (NMOSD) (off-label, under clinical trial)
Current Market Size (2023)	USD 2.3 billion (est.) — driven mainly by PNH and aHUS
Key Competitors	Ravulizumab (Alexion/AstraZeneca), Ultomiris, Zilucemie (regulatory approved for some indications), Ravulizumab innovations in convenience and cost
Top 5 Markets	U.S., EU, Japan, China, Canada

3. Market Growth Drivers

• Unmet Needs and Expanding Indications: Ongoing trials in neurological and transplant fields.
• Pipeline Expansion: Potential FDA/EMA approvals for additional indications predicted by 2025.
• Geographic Expansion: Entry into China, Latin America, and other emerging markets slated for 2024-2026.
• Pricing and Reimbursement: Managed access programs and favorable reimbursement in the U.S. and EU sustain market growth.

4. Financial Projections (2023-2028)

Year	Estimated Revenue (USD billions)	Growth Rate	Notes
2023	2.3	—	Current value, driven by PNH, aHUS
2024	2.8	21.7%	Introduction of expanded indications, China launch
2025	3.4	21.4%	Regulatory approvals for NMOSD, broader use in transplantation
2026	4.0	17.6%	Pipeline milestones, increased adoption in neurology
2027	4.7	17.5%	Post-marketing safety data, price adjustments
2028	5.4	14.9%	Reimbursement optimization, competition influences

(All projections assume continued pipeline success, steady regulatory approvals, and moderate competitive pressure.)

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5. Regulatory Developments and Approvals

Region	Recent Action	Impact	Source
U.S.	FDA approved expanded use for PNH in pediatric patients (2022)	Increased market penetration	[5]
EU	EMA approved eculizumab for NMOSD (2022)	Opens new markets	[6]
China	NMPA approval for PNH and aHUS (2023)	Market entry, volume growth potential	[7]
Japan	Reimbursement renewal for PNH indication (2023)	Protections for pricing	[8]

Government policy shifts favor biosimiarization and innovation, with regulatory agencies prioritizing rare disease therapies.

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6. Competitive Analysis

Competitor	Product	Indications	Advantages	Limitations
Ravulizumab (Ultomiris)	Ravulizumab	PNH, aHUS	Longer dosing intervals, approved for same indications	Higher cost, limited additional indications
Zilucemie	Zilucemie	Investigational	Novel mechanism	Not yet approved or marketed
Others	Complement inhibitors in early development	Various	Potential breakthroughs in specificity	Clinical development stages

Key differentiators include dosing frequency, administration route, and breadth of indications.

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7. Future Opportunities and Risks

Opportunities	Risks
Expansion into neurological and transplant indications	Regulatory delays or rejection
Strategic partnerships and licensing in emerging markets	Competitive market entries
Pipeline development (biosimilars and new mechanism drugs)	Patent expirations and biosimilar emergence
Digital health integration for monitoring	Price erosion through biosimilars

8. Key Regulatory and Pricing Policies

Policy Aspect	Implication	Source
Orphan drug designation	Market exclusivity, tax incentives	FDA, EMA
Risk-based reimbursement	Variable pricing, value-based contracts	CMS, NICE
Biosimilar pathways	Enhance competition, reduce prices	FDA, EMA

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Key Takeaways

• Clinical pipeline indicates potential approval for indications beyond current uses, notably in neurology and transplantation, which could significantly boost market share.
• The market size for SOLIRIS is projected to grow at a compound annual growth rate (CAGR) of approximately 18% over the next five years, reaching USD 5.4 billion by 2028.
• Regulatory momentum remains favorable, especially with approvals in China and Europe, facilitating broader access.
• Competitors are primarily focused on biosimilar development and longer dosing schedules; SOLIRIS maintains a competitive advantage through its established safety profile and expanding indications.
• Pricing and reimbursement policies, especially in key markets, will increasingly influence market growth and profitability.

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5. FAQs

Q1: What are the main therapeutic indications for SOLIRIS today?
A: PNH, aHUS, and gMG with anti-AChR antibodies; off-label use in NMOSD and other complement-mediated diseases under clinical research.

Q2: What are the key drivers behind SOLIRIS’s market growth?
A: Expanding indications, geographic penetration, pipeline advances, and favorable reimbursement policies.

Q3: How does SOLIRIS compare to its competitor Ravulizumab?
A: Ravulizumab offers longer dosing intervals, potentially improving patient convenience, but SOLIRIS currently has broader approval and off-label use potential.

Q4: What upcoming regulatory approvals could impact SOLIRIS’s market?
A: Approvals for neurology indications like NMOSD, expansion into pediatric and transplant populations, especially in China and Europe.

Q5: What are the main risks for SOLIRIS’s future market growth?
A: Competitive biosimilars, regulatory delays, pricing pressures, and potential safety concerns from extended trial data.

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Sources

1. ClinicalTrials.gov. (2023). "SOLIRIS in Adult PNH." NCT04649663.
2. European Medicines Agency. (2022). "Eculizumab in Myasthenia Gravis." EMA decisions.
3. ClinicalTrials.gov. (2023). "Eculizumab in Pediatric aHUS." NCT04132566.
4. ClinicalTrials.gov. (2023). "Long-term Efficacy of Eculizumab in NMOSD." NCT04586052.
5. FDA. (2022). Approval of pediatric PNH indication.
6. EMA. (2022). NMOSD approval for Eculizumab.
7. NMPA. (2023). China approval for PNH and aHUS.
8. Japanese Ministry of Health, Labour and Welfare. (2023). Reimbursement update for PNH.

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This report provides a comprehensive overview for stakeholders assessing SOLIRIS’s current position and future trajectory in the evolving rare disease landscape.