eculizumab
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Eculizumab is a humanized monoclonal antibody that inhibits terminal complement activation by binding to complement protein C5. It prevents the formation of the membrane attack complex and blocks complement-mediated cell destruction. While primarily indicated for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, it has shown efficacy in complement-mediated disorders including properdin deficiency complications.
Indications & Therapeutic Use
Paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis, neuromyelitis optica spectrum disorder, complement-mediated thrombotic microangiopathy
Linked Diseases:
hereditary elliptocytosis
D004612
hydrops fetalis
P56D015160
properdin deficiency
C537241
Global Availability (9 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
eculizumab
| Generic Name | eculizumab |
| Brands | 1 brand available |
| Active Ingredient | eculizumab |
| Drug Class | Paroxysmal nocturnal hemoglobinuria |
| Manufacturer | Alexion Pharmaceuticals |
| Dosage Forms | IV infusion, 10mg/mL concentrate for solution |
| Medical Code | L04AA25 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00122226 |
| Countries | 9 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations10 Validated Nodes