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

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 Nameeculizumab
Brands1 brand available
Active Ingredienteculizumab
Drug ClassParoxysmal nocturnal hemoglobinuria
ManufacturerAlexion Pharmaceuticals
Dosage FormsIV infusion, 10mg/mL concentrate for solution
Medical CodeL04AA25
Orphan StatusYes — Orphan Drug
Cold ChainRequired
Lead Time14 days
Reg. StatusFDA Approved, EMA Approved
Clinical TrialNCT00122226
Countries9 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations10 Validated Nodes