Fitusiran is a small interfering RNA used for routine prophylaxis of bleeding episodes in hemophilia A or B, with or without factor VIII or IX inhibitors. Fitusiran is an antithrombin-directed double-stranded small interfering ribonucleic acid (siRNA) that is covalently linked to a ligand containing a triantennary N-acetylgalactosamine (GalNAc) moiety.
- Mechanism
- Curator reviewed · 3 references
Hemophilia A and B arise from factor VIII (FVIII) or FIX deficiency, respectively, leading to insufficient thrombin production and increased risk for bleeding events. Fitusiran causes degradation of antithrombin (AT) messenger RNA (mRNA) through RNA interference, reducing plasma AT levels and promoting clot formation.
- Primary indication
- Fitusiran is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII...Curator reviewed · 4 structured indications
- First approval
- United States, 2025
- Also known as
- SMALL INTERFERING RNA (SIRNA) INHIBITING ANTITHROMBIN LIVER PRODUCTION: DUPLEX OF ((2S,4R)-1-(1-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-16,16-BIS((3-((3-(5-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)PENTANAMIDO)PROPYL)AMINO)-3-OXOPR
- Code names
- ALN-57213 · ALN-AT3SC · SAR439774
- Brand names
Resolves to
What you can answer from here — as of September 12, 2026
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