Ravulizumab

Ravulizumab-cwvz is a terminal complement inhibitor that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the membrane attack complex [MAC or C5b-9]) thus preventing MAC formation. ULTOMIRIS inhibits terminal complement-mediated intravascular hemolysis in patients with PNH and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS.

• ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

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

See Full Prescribing Information for instructions on dosage, preparation, and administration. ( 2.1 , 2.2 , 2.3 , 2.4 , 2.5 ) Dilute ULTOMIRIS before use. ( 2.5 ) Only administer as an intravenous infusion through a 0.2 or 0.22 micron filter. ( 2.5 ) 2.1 Important Dosage Information ULTOMIRIS is intended to be administered only as an intravenous infusion in adult or pediatric patients one month of age and older. 2.2 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 ULTOMIRIS [see Warnings and Precautions (5.1) ] . If urgent ULTOMIRIS 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 ULTOMIRIS must enroll in the ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2) ] .

Other Infections: Use caution when administering ULTOMIRIS to patients with any other systemic infection. ( 5.3 ) Infusion-Related Reactions: Monitor during infusion, interrupt for reactions, and institute appropriate supportive measures. ( 5.6 ) 5.1 Serious Meningococcal Infections ULTOMIRIS, 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 ULTOMIRIS 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 ULTOMIRIS, according to current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of ULTOMIRIS therapy. 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 ULTOMIRIS 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 clinically significant 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) ] Infusion-Related Reactions [see Warnings and Precautions (5.6) ] Most common adverse reactions in patients with PNH (incidence ≥ 10%) were upper respiratory tract infection and headache. ( 6.1 ) Most common adverse reactions in patients with aHUS (incidence ≥ 20%) were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension, and pyrexia. ( 6.1 ) Most common adverse reactions in adult patients with gMG (incidence ≥ 10%) were diarrhea and upper respiratory tract infection. ( 6.1 ) Most common adverse reactions in adult patients with NMOSD (incidence ≥ 10%) were COVID-19, headache, back pain, arthralgia, and urinary tract infection. ( 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. 6.1 Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ULTOMIRIS during pregnancy. Healthcare providers and patients may call 1-833-793-0563 or go to www.UltomirisPregnancyStudy.com to enroll in or to obtain information about the registry. Risk Summary There are no available data on ULTOMIRIS use in pregnant women to inform a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with untreated PNH and aHUS in pregnancy (see Clinical Considerations ) . Animal studies using a mouse analogue of the ravulizumab-cwvz molecule (murine anti-mouse complement component 5 [C5] antibody) showed increased rates of developmental abnormalities and an increased rate of dead and moribund offspring at doses 0.8-2.2 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 defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

Metabolism

Following ULTOMIRIS treatment, ravulizumab-cwvz pharmacokinetics increase proportionally over a dose range of 200 to 5400 mg. Ravulizumab-cwvz C max and C trough parameters are presented in Table 14, Table 15, Table 16, and Table 17.

• Dec 21, 2018BLABLA761108Alexion Pharm

1. 1Fatigue1,65514%
2. 2Asthenia8246.7%
3. 3Headache7125.8%
4. 4Drug Ineffective6725.5%
5. 5Off Label Use5314.3%
6. 6Dyspnoea4703.8%
7. 7Death4693.8%
8. 8Muscular Weakness4683.8%
9. 9Myasthenia Gravis4283.5%
10. 10Malaise4263.5%
11. 11Haemoglobin Decreased3833.1%
12. 12Pain3783.1%
13. 13Back Pain3522.9%
14. 14Feeling Abnormal3432.8%
15. 15Diplopia3282.7%

The 10 most recently updated of 62 ClinicalTrials.gov registrations naming Ravulizumab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Real-life Treatment Outcomes of Ravulizumab in PNH
  Active not recruiting · Observational · 64 enrolled · AstraZeneca
  NCT06633536updated 2026-06-12
• Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
  Completed · Phase 3 · Interventional · 18 enrolled · Alexion Pharmaceuticals, Inc.
  NCT06578949updated 2026-06-05
• Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)
  Not yet recruiting · Phase 3 · Interventional · 19 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07557420updated 2026-06-04
• Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)
  Not yet recruiting · Phase 3 · Interventional · 20 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07596784updated 2026-05-19
• Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis
  Recruiting · Observational · 50 enrolled · AstraZeneca
  NCT06909253updated 2026-05-15
• Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
  Completed · Observational · 120 enrolled · Alexion Pharmaceuticals, Inc.
  NCT05274633updated 2026-05-07
• A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
  Active not recruiting · Phase 3 · Interventional · 202 enrolled · Regeneron Pharmaceuticals
  NCT05133531updated 2026-04-30
• Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders
  Recruiting · Phase 2 · Interventional · 14 enrolled · Mayo Clinic
  NCT06333652updated 2026-04-28
• Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation
  Recruiting · Phase 3 · Interventional · 450 enrolled · Alexion Pharmaceuticals, Inc.
  NCT06830798updated 2026-04-28
• Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
  Recruiting · Phase 4 · Interventional · 20 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07308574updated 2026-04-20

Frequently asked questions

How does Ravulizumab work?

Ravulizumab-cwvz is a terminal complement inhibitor that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the membrane attack complex [MAC or C5b-9]) thus preventing MAC formation. ULTOMIRIS inhibits terminal complement-mediated intravascular hemolysis in patients with PNH and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS.

What is Ravulizumab used for?

According to FDA labeling, Ravulizumab carries indications including: ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Ravulizumab?

Ravulizumab is classified as Complement inhibitors, Complement Inhibitor, Complement Inhibitors, Decreased Protein Modification.

What are the brand names for Ravulizumab?

Ravulizumab is marketed under brand names including Ultomiris.

What are the contraindications for Ravulizumab?

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