Patisiran
Orphan Drug Cold Chain RequiredFDA Approved
Contents
Description
Patisiran is a double-stranded small interfering RNA (siRNA) that targets transthyretin (TTR) mRNA. It is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Indications & Therapeutic Use
hATTR amyloidosis
Global Availability (6 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Patisiran
| Generic Name | Patisiran |
| Brands | 1 brand available |
| Active Ingredient | Patisiran |
| Drug Class | hATTR amyloidosis |
| Manufacturer | Alnylam Pharmaceuticals |
| Dosage Forms | IV infusion, 2 mg/mL |
| Medical Code | C05BB07 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT01960348 |
| Countries | 6 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations7 Validated Nodes