Fitusiran
Medication
From Wikipedia, the free encyclopedia
Fitusiran, sold under the brand name Qfitlia, is a medication used for the treatment of hemophilia.[1] It is an antithrombin-directed small interfering ribonucleic acid.[1] It is given by subcutaneous injection.[1] Fitusiran reduces the amount of a protein called antithrombin.[2]
| Clinical data | |
|---|---|
| Trade names | Qfitlia |
| Other names | ALN-AT3SC |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625058 |
| License data | |
| Routes of administration | Subcutaneous |
| Drug class | Anthithrombin production inhibitor |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C520H636F21N175Na43O309P43S6 |
| Molar mass | 17193.48 g·mol−1 |
The most common side effects include viral infection, common cold symptoms (nasopharyngitis) and bacterial infection.[2]
Fitusiran was approved for medical use in the United States in March 2025.[2]
Medical uses
Adverse effects
The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal).[2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran.[2]
History
The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B.[2] In one study, participants had inhibitory antibodies to coagulation factor VIII or coagulation factor IX and previously received on-demand treatment with medicines known as "bypassing agents" for bleeding.[2] In the second study, participants did not have inhibitory antibodies to coagulation factor VIII or coagulation factor IX and previously received on-demand treatment with clotting factor concentrates.[2] In the two randomized trials, participants received either a fixed dose of fitusiran monthly or their usual on-demand treatment (bypassing agents or clotting factor concentrates) as needed for nine months.[2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants.[2]
The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. The FDA granted the approval of Qfitlia to Sanofi.
