Eculizumab

Eculizumab, the active ingredient in SOLIRIS, is a monoclonal antibody that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b-9. SOLIRIS inhibits terminal complement-mediated intravascular hemolysis in PNH patients and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS.

• SOLIRIS is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. ( 1.1 ) the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

• SOLIRIS is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . SOLIRIS is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection.contraindicated

For intravenous infusion only; recommended dosage for: PNH: ( 2.2 ) aHUS, gMG, and NMOSD in adults: ( 2.3 ) aHUS and gMG in pediatric patients: ( 2.4 ) 2.1 Recommended Vaccination and Prophylaxis for Meningococcal Infection Vaccinate patients against meningococcal infection (serogroups A, C, W, Y and B) according to current ACIP recommendations at least 2 weeks prior to initiation of SOLIRIS [see Warnings and Precautions (5.1) ] . If urgent SOLIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. Healthcare providers who prescribe SOLIRIS must enroll in the ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2) ] . 2.2 Recommended Dosage for Adults – PNH The recommended dosage of SOLIRIS for the treatment of PNH in patients 18 years of age and older is administered as an intravenous infusion [see Dosage and Administration (2.7) ] as follows: 600 mg weekly for the first 4 weeks, followed by 900 mg for the fifth dose 1 week later, then 900 mg every 2 weeks thereafter. Administer SOLIRIS at the recommended dosage regimen time points, or within two days of these time points [see Warnings and Precautions (5.4) ] .

Use caution when administering SOLIRIS to patients with any other systemic infection. ( 5.3 ) Infusion-Related Reactions: Monitor patients during infusion, interrupt for reactions, and institute appropriate supportive measures. ( 5.6 ) 5.1 Serious Meningococcal Infections SOLIRIS, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of SOLIRIS treatment is contraindicated in patients with unresolved serious Neisseria meningitidis infection. Complete or update meningococcal vaccination (for serogroups A, C, W, Y and B) at least 2 weeks prior to administration of the first dose of SOLIRIS, according to current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations, considering the duration of therapy with SOLIRIS. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent SOLIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible.

The following serious adverse reactions are discussed in greater detail in other sections of the labeling: Serious Meningococcal Infections [see Warnings and Precautions (5.1) ] Other Infections [see Warnings and Precautions (5.3) ] Monitoring Disease Manifestations after SOLIRIS Discontinuation [see Warnings and Precautions (5.4) ] Thrombosis Prevention and Management [see Warnings and Precautions (5.5) ] Infusion-Related Reactions [see Warnings and Precautions (5.6) ] The most frequently reported adverse reactions in the PNH randomized trial (≥10% overall and greater than placebo) are: headache, nasopharyngitis, back pain, and nausea. ( 6.1 ) The most frequently reported adverse reactions in aHUS single arm prospective trials (≥20%) are: headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, pyrexia. ( 6.1 ) The most frequently reported adverse reaction in the gMG placebo-controlled clinical trial (≥10%) in adult patients is musculoskeletal pain. ( 6.1 ) The most frequently reported adverse reactions in the NMOSD placebo- controlled trial (≥10%) are: upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

8.1 Pregnancy Risk Summary Limited data on outcomes of pregnancies that have occurred following SOLIRIS use in pregnant women have not identified a concern for specific adverse developmental outcomes ( see Data ). There are risks to the mother and fetus associated with untreated paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in pregnancy ( see Clinical Considerations ). Animal studies using a mouse analogue of the SOLIRIS molecule (murine anti-C5 antibody) showed increased rates of developmental abnormalities and an increased rate of dead and moribund offspring at doses 2-8 times the human dose ( see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Clinical Considerations Disease-associated maternal and/or fetal/neonatal risk PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages and increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery.

Metabolism

Following intravenous maintenance doses of 900 mg once every 2 weeks in patients with PNH, the week 26 observed mean ± SD serum eculizumab maximum concentration (C max ) was 194 ± 76 mcg/mL and the trough concentration (C trough ) was 97 ± 60 mcg/mL. Following intravenous maintenance doses of 1200 mg once every 2 weeks in patients with aHUS, the week 26 observed mean ± SD C trough was 242 ± 101 mcg/mL.

• Mar 16, 2007BLABLA125166Alexion Pharm
• May 28, 2024BLABLA761333Amgen Inc
• Jul 19, 2024BLABLA761340Samsung Bioepis Co Ltd

1. 1Fatigue5,84511%
2. 2Off Label Use4,6638.7%
3. 3Haemoglobin Decreased3,9047.3%
4. 4Headache3,4636.5%
5. 5Death2,6234.9%
6. 6Pyrexia2,3984.5%
7. 7Dyspnoea2,1624.1%
8. 8Asthenia2,0063.8%
9. 9Nausea1,9833.7%
10. 10Drug Ineffective1,9063.6%
11. 11Haemolysis1,8553.5%
12. 12Platelet Count Decreased1,8203.4%
13. 13Malaise1,7853.3%
14. 14Blood Lactate Dehydrogenase Increased1,5813.0%
15. 15Abdominal Pain1,5372.9%

The 10 most recently updated of 152 ClinicalTrials.gov registrations naming Eculizumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies
  Withdrawn · Phase 3 · Interventional · 0 enrolled · University Hospital, Rouen
  NCT05702996updated 2026-06-09
• Danicopan Early Access Program
  Available · Expanded access · Alexion Pharmaceuticals, Inc.
  NCT05982938updated 2026-06-04
• Eculizumab For the Acute Attack of Neuromyelitis Optica Spectrum Disorder
  Active not recruiting · Phase 2 · Interventional · 110 enrolled · Tianjin Medical University General Hospital
  NCT07184840updated 2026-06-03
• A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
  Recruiting · Observational · 80 enrolled · Novartis Pharmaceuticals
  NCT06931691updated 2026-06-01
• Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
  Completed · Phase 3 · Interventional · 25 enrolled · Alexion Pharmaceuticals, Inc.
  NCT05886244updated 2026-06-01
• Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 59 enrolled · Hoffmann-La Roche
  NCT03157635updated 2026-05-14
• Modified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder
  Recruiting · Phase 3 · Interventional · 198 enrolled · Tianjin Medical University General Hospital
  NCT07420296updated 2026-05-14
• Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
  Completed · Observational · 120 enrolled · Alexion Pharmaceuticals, Inc.
  NCT05274633updated 2026-05-07
• Eculizumab in Shiga-toxin Related Hemolytic and Uremic Syndrome Pediatric Patients - ECULISHU
  Completed · Phase 3 · Interventional · 100 enrolled · University Hospital, Toulouse
  NCT02205541updated 2026-05-04
• Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx
  Completed · Phase 3 · Interventional · 64 enrolled · Hansa Biopharma AB
  NCT04935177updated 2026-04-30

Frequently asked questions

How does Eculizumab work?

Eculizumab, the active ingredient in SOLIRIS, is a monoclonal antibody that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b-9. SOLIRIS inhibits terminal complement-mediated intravascular hemolysis in PNH patients and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS.

What is Eculizumab used for?

According to FDA labeling, Eculizumab carries indications including: SOLIRIS is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. ( 1.1 ) the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Eculizumab?

Eculizumab is classified as Complement inhibitors, Complement Inhibitor, Complement Inhibitors.

What are the brand names for Eculizumab?

Eculizumab is marketed under brand names including Bkemv, Epysqli, Soliris.

What are the contraindications for Eculizumab?

Eculizumab labeling lists contraindications including: SOLIRIS is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . SOLIRIS is contraindicated for initiation in patients with unresolved serious Neisseria meningitidis infection.. Always consult the full prescribing information and a clinician.