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Drug

Eculizumab

Approved · FDA / EMA
Emerging researchLate-stage developmentRising momentum

Also known as Bkemv, Epysqli, Soliris.

RxNorm591781UNIIA3ULP0F556
9
Research papers
8
Active clinical trials
50
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Label change: ECULIZUMAB (BLA125166)

Regulatory2026-06-30FDA

Supplemental approval: ECULIZUMAB-AAGH (BLA761340)

Regulatory2026-06-29FDA

Supplemental approval: ECULIZUMAB-AEEB (BLA761333)

Regulatory2026-06-29FDA

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Clinical trial2026-06-24Results expected Q1 2030 · ClinicalTrials.gov

Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Clinical trial2026-06-01Results posted · ClinicalTrials.gov

Supplemental approval: ECULIZUMAB (BLA125166)

Regulatory2026-06-01FDA

A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

Clinical trial2026-04-30Results expected Q4 2026 · ClinicalTrials.gov

A Phase III, Randomized, Open-label, Active-controlled, Multicenter Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

Clinical trial2026-04-28Results posted · ClinicalTrials.gov

Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study.

Research2019-08-03Journal of the neurological sciences

Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Research2019-03-23Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Research2019-03-08Muscle & nerve

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.

Research2017-10-20Howard JF Jr · The Lancet. Neurology

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Eculizumab
Aliases & brands
BkemvEpysqliSoliris
RxNorm CUI
591781
UNII
A3ULP0F556
Regulatory jurisdictions
emafda

Regulatory timeline

50 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Approval / market entry confirmed across FDA and EMA (2007-03-16 → 2007-06-20)
Approval / market entry confirmed across FDA and EMA (2023-04-19 → 2025-04-18)
Earliest approval
2007-03-16
Latest approval
2026-06-29
Authorities
FDA · EMA
Total events
50
fdaU.S. Food and Drug Administration· 47 events
2026-06-30Label change
Label change: ECULIZUMAB (BLA125166)
Evidence ↗
2026-06-29Supplemental approval
Supplemental approval: ECULIZUMAB-AEEB (BLA761333)
Evidence ↗
2026-06-29Supplemental approval
Supplemental approval: ECULIZUMAB-AAGH (BLA761340)
Evidence ↗
emaEuropean Medicines Agency· 3 events
2023-05-26ApprovalMulti-authority
Approval: Epysqli (EMA)
Indication: Epysqli is indicated in adults and children for the treatment of:- Paroxysmal nocturnal haemoglobinuria (PNH).Evidence of clinical benefit is demonstrated in patients with… Show full indication

Epysqli is indicated in adults and children for the treatment of:- Paroxysmal nocturnal haemoglobinuria (PNH).Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history.- Atypical haemolytic uremic syndrome (aHUS).

Evidence ↗
2023-04-19ApprovalMulti-authority
Approval: Bekemv (EMA)
Indication: Bekemv is indicated in adults and children for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with… Show full indication

Bekemv is indicated in adults and children for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history (see section 5.1).

Evidence ↗
2007-06-20Accelerated approvalMulti-authority
Accelerated approval: Soliris (EMA)
Indication: Soliris is indicated in adults and children for the treatment of: Paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with… Show full indication

Soliris is indicated in adults and children for the treatment of: Paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history (see section 5.1).  Atypical haemolytic uremic syndrome (aHUS). Soliris is indicated in adults for the treatment of: Refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive (see section 5.1). Neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease.

Evidence ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

90 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
90
registered trials across all phases
LATEST COMPLETION 2025
8
Active studies
4
Recruiting
43
Late-stage (III+)
62
Completed
20
Discontinued
PHASE DISTRIBUTIONn = 90
Early Phase 11Phase 15Phase 1 / 27Phase 234Phase 2 / 33Phase 336Phase 44

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Not yet recruitingPH IVMassachusetts General Hospitaln=5402030

Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

CompletedPH IVAlexion Pharmaceuticals, Inc.n=182022

Multicentric, Prospective Open-label Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With Atypical Hemolytic Uremic Syndrome (aHUS)

CompletedPH IVNantes University Hospitaln=582019

An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria

CompletedPH IVAlexion Pharmaceuticals, Inc.n=72011

A Phase III, Randomized, Open-label, Active-controlled, Multicenter Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamic and Efficacy of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

Active, not recruitingPH IIIHoffmann-La Rochen=1902027

Recent completions

Trials that read out recently, adding to the completed evidence base.

An Open-label, Single-arm, Multi-center Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Participants With Refractory Generalized Myasthenia Gravis (gMG)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=152025

A Phase III, Multicenter, Randomized, Open Label, Active-Controlled Study to Evaluate the Efficacy and Safety of HSK39297 Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Naive to Complement Inhibitor Therapy

CompletedPH IIIHaisco Pharmaceutical Group Co., Ltd.n=732025

A Single Center, Open-label, Single Arm Phase II Trial of the Efficacy and Safety of Complement C5 Monoclonal Antibody in the Treatment of Anti-glomerular Basement Membrane Disease

CompletedPH IIPeking University First Hospitaln=162025

A Multicenter, Randomized, Open-label, Active-comparator Controlled Phase III Study to Evaluate the Efficacy and Safety of HRS-5965 Capsule in Patients With Paroxysmal Nocturnal Hemoglobinuria

CompletedPH IIIChengdu Suncadia Medicine Co., Ltd.n=762025

An Open-label, Controlled, Randomized Phase 3 Trial Evaluating 12-month Kidney Function in Highly Sensitized (cPRA ≥99.9%) Kidney Tx Patients With Positive XM Against a Deceased Donor, Comparing Desensitization Using Imlifidase With SoC

CompletedPH IIIHansa Biopharma ABn=642025

Prospective, Single-Arm, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment-Naïve Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=252025

Research activity

9 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20112019
Most influential

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.

The Lancet. Neurology · 2017 · 587 cites

Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study.

The Lancet. Neurology · 2013 · 284 cites

Eculizumab for dense deposit disease and C3 glomerulonephritis.

Clinical journal of the American Society of Nephrology : CJASN · 2012 · 261 cites

Systemic complement inhibition with eculizumab for geographic atrophy in age-related macular degeneration: the COMPLETE study.

Ophthalmology · 2014 · 246 cites

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Muscle & nerve · 2019 · 186 cites

Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial.

International journal of hematology · 2011 · 45 cites
Recent papers

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Muscle & nerve · 2019 · 186 cites

Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2019 · 42 cites

Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study.

Journal of the neurological sciences · 2019 · 18 cites

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.

The Lancet. Neurology · 2017 · 587 cites

Systemic complement inhibition with eculizumab for geographic atrophy in age-related macular degeneration: the COMPLETE study.

Ophthalmology · 2014 · 246 cites

Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: an open-label pilot study.

The Lancet. Neurology · 2013 · 284 cites
Major research themes4
Activities of Daily Living1Outcome Assessment, Health Care1Quality of Life1Severity of Illness Index1
Journals, researchers & institutions
Top journals
  • International journal of hematology2
  • The Lancet. Neurology2
  • Clinical journal of the American Society of Nephrology : CJASN1
  • Journal of the neurological sciences1
  • Muscle & nerve1
  • Ophthalmology1
Leading researchers
  • Wang JJ4
  • Howard JF Jr3
  • Mantegazza R3
  • Murai H3
  • O'Brien F3
  • Utsugisawa K3
  • Andersen H2
  • Ando K2
Leading institutions
free-text, unnormalised
  • Hanamaki General Hospital3
  • International University of Health and Welfare3
  • Aarhus University Hospital2
  • Foundation of the Carlo Besta Neurological Institute2
  • Hospital Sant Pau2
  • Osaka University Hospital2
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.