Ravulizumab
Orphan Drug Cold Chain RequiredFDA Approved
Description
Ravulizumab is a long-acting C5 complement inhibitor used across multiple rare complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. It is a major specialty biologic because of its extended dosing interval and broad orphan footprint across hematology, nephrology, and neurology. The product is widely tracked in global access discussions due to its high-cost biologic profile and infusion-center requirements.
Indications & Therapeutic Use
paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis, neuromyelitis optica spectrum disorder
Linked Diseases:
acute lung injury
D055371
Paroxysmal nocturnal hemoglobinuria
3A70.0D010975
atypical hemolytic uremic syndrome
D59.3D065766
Neuromyelitis Optica Spectrum Disorder
G36.0D009471
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Ravulizumab
| Generic Name | ravulizumab |
| Brands | 1 brand available |
| Active Ingredient | ravulizumab |
| Drug Class | paroxysmal nocturnal hemoglobinuria |
| Manufacturer | Alexion Pharmaceuticals |
| Dosage Forms | Concentrate for solution for infusion |
| Medical Code | L04AJ |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT04119050 |
| Countries | 5 countries |
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes