Overview
Ravulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. Ravulizumab was engineered from eculizumab, another complement inhibitor, to increase the duration of action and reduce the frequency of drug administration. It works by blocking terminal complement-mediated inflammation, cell activation, and cell lysis in blood disorders associated with the destruction of red blood cells, thrombosis, and impaired bone marrow function. Ravulizumab was first approved by the FDA on December 21, 2018, for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in children and adults. It was later approved by the European Commission on July 2, 2019, for the same indications. Ravulizumab is also used to treat myasthenia gravis. Ravulizumab is currently being investigated for the treatment of Coronavirus disease (COVID-19)-induced microvasculature injury and endothelial damage leading to thrombotic microangiopathy (TMA) causing acute kidney injury (AKI).
Background
Ravulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. Ravulizumab was engineered from eculizumab, another complement inhibitor, to increase the duration of action and reduce the frequency of drug administration. It works by blocking terminal complement-mediated inflammation, cell activation, and cell lysis in blood disorders associated with the destruction of red blood cells, thrombosis, and impaired bone marrow function. Ravulizumab was first approved by the FDA on December 21, 2018, for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in children and adults. It was later approved by the European Commission on July 2, 2019, for the same indications. Ravulizumab is also used to treat myasthenia gravis. Ravulizumab is currently being investigated for the treatment of Coronavirus disease (COVID-19)-induced microvasculature injury and endothelial damage leading to thrombotic microangiopathy (TMA) causing acute kidney injury (AKI).
Indication
Ravulizumab is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). It is also indicated for 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). However, the FDA advises against the use of ravulizumab for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Ravulizumab is also indicated for treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. The European Commission approved ravulizumab for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) in adults and children with a body weight of 10 kg or more with the following conditions: hemolysis with clinical symptoms indicative of high disease activity or clinically stable after having been treated with eculizumab for at least the past six months. Ravulizumab is also indicated for the treatment of hemolytic uremic syndrome (aHUS) in patients with a body weight of 10 kg or more who are either complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.
Associated Conditions
- Atypical Hemolytic Uremic Syndrome (aHUS)
- Generalized Myasthenia Gravis
- Paroxysmal Nocturnal Haemoglobinuria (PNH)
- Thrombotic Microangiopathies
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/06/17 | Phase 3 | Not yet recruiting | |||
2025/06/08 | Phase 4 | Not yet recruiting | |||
2025/05/13 | N/A | Not yet recruiting | |||
2025/04/03 | N/A | Recruiting | |||
2025/02/17 | Phase 3 | Recruiting | |||
2024/10/09 | N/A | Recruiting | |||
2024/08/30 | Phase 3 | Active, not recruiting | |||
2024/05/03 | N/A | Recruiting | |||
2024/03/27 | Phase 2 | Recruiting | |||
2024/03/15 | N/A | Recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | 25682-028 | INTRAVENOUS | 1100 mg in 11 mL | 3/28/2024 | |
| Alexion Pharmaceuticals Inc. | 25682-022 | INTRAVENOUS | 300 mg in 30 mL | 3/28/2024 | |
| Alexion Pharmaceuticals Inc. | 25682-025 | INTRAVENOUS | 300 mg in 3 mL | 3/28/2024 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 7/2/2019 | ||
Authorised | 7/2/2019 | ||
Authorised | 7/2/2019 | ||
Authorised | 7/2/2019 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| ULTOMIRIS CONCENTRATE FOR SOLUTION FOR INFUSION 100 MG/ML | SIN17026P | INFUSION, SOLUTION CONCENTRATE | 100 mg/mL | 6/19/2024 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| ULTOMIRIS ravulizumab rch 1100 mg in 11 mL (100 mg/mL) concentrated solution for injection by intravenous infusion vial | 336710 | Medicine | A | 3/23/2021 | |
| ULTOMIRIS ravulizumab rch 300 mg in 3 mL (100 mg/mL) concentrated solution for injection by intravenous infusion vial | 330566 | Medicine | A | 3/23/2021 | |
| ULTOMIRIS ravulizumab rch 10 mg/mL concentrated solution for intravenous infusion vial | 311926 | Medicine | A | 10/17/2019 |