Lumasiran
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Lumasiran is an RNAi therapeutic targeting glycolate oxidase (GO) to reduce the production of oxalate in the liver. It is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels.
Indications & Therapeutic Use
Primary hyperoxaluria type 1
Global Availability (3 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — | |
| — | — | — | — | |
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Lumasiran
| Generic Name | Lumasiran |
| Brands | 1 brand available |
| Active Ingredient | Lumasiran |
| Drug Class | Primary hyperoxaluria type 1 |
| Manufacturer | Alnylam Pharmaceuticals |
| Dosage Forms | Subcutaneous injection |
| Medical Code | A16AX18 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT03681184 |
| Countries | 3 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations4 Validated Nodes