Fitusiran
Orphan Drug Cold Chain RequiredFDA Approved
Description
Fitusiran is a subcutaneous small-interfering RNA therapy designed to lower antithrombin and rebalance hemostasis in hemophilia A or B. It is a specialty rare-disease biologic requiring careful handling and is part of the newer RNAi wave in hematology. The product has attracted attention because it aims to reduce bleeding in patients with or without inhibitors.
Indications & Therapeutic Use
hemophilia A, hemophilia B
Global Availability (1 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Fitusiran
| Generic Name | fitusiran |
| Brands | 1 brand available |
| Active Ingredient | fitusiran |
| Drug Class | hemophilia A |
| Manufacturer | Sanofi |
| Dosage Forms | Injection, subcutaneous |
| Medical Code | B02BX |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT03754790 |
| Countries | 1 countries |
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes