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| Stem definition | Drug id | CAS RN |
|---|---|---|
| small interfering RNA including siRNA, miRNA and piRNA | 5766 | None |
| Molecule | Description |
|---|---|
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Synonyms:
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Fitusiran is a synthetic double-stranded small interfering RNA (siRNA) conjugated to a triantennary N-acetyl galactosamine (GalNAc) ligand targeting antithrombin (AT) mRNA. Fitusiran reduces AT production in the liver by causing degradation of AT mRNA through the RNA interference pathway.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| March 28, 2025 | FDA | Sanofi |
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| Source | Code | Description |
|---|---|---|
| ATC | B02BX12 | BLOOD AND BLOOD FORMING ORGANS ANTIHEMORRHAGICS VITAMIN K AND OTHER HEMOSTATICS Other systemic hemostatics |
| FDA CS | M0406100 | RNA, Small Interfering |
| FDA PE | N0000008852 | Decreased RNA Integrity |
| FDA EPC | N0000193848 | Small Interfering RNA |
| FDA EPC | N0000194148 | Antithrombin-directed RNA Interaction |
| FDA PE | N0000194154 | Increased RNA Degradation |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Reduce the frequency of bleeding episodes in patients with hemophilia A or B | indication | 90935002 |
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| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| Antithrombin-III | Secreted | DRUG LABEL | DRUG LABEL |
| ID | Source |
|---|---|
| 1499251-18-1 | SECONDARY_CAS_RN |
| CHEMBL4297754 | ChEMBL_ID |
| C000632624 | MESH_SUPPLEMENTAL_RECORD_UI |
| 13630 | IUPHAR_LIGAND_ID |
| DB15002 | DRUGBANK_ID |
| 4043893 | VANDF |
| C5139801 | UMLSCUI |
| CHEBI:757204 | CHEBI |
| 10177 | INN_ID |
| D11810 | KEGG_DRUG |
| 43958 | MMSL |
| d10268 | MMSL |
| 020041 | NDDF |
| SV9W47ZLE1 | UNII |
| 2709439 | RXNORM |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| Qfitlia | HUMAN PRESCRIPTION DRUG LABEL | 1 | 58468-0347 | INJECTION, SOLUTION | 20 mg | SUBCUTANEOUS | NDA | 32 sections |
| Qfitlia | HUMAN PRESCRIPTION DRUG LABEL | 1 | 58468-0348 | INJECTION, SOLUTION | 50 mg | SUBCUTANEOUS | NDA | 32 sections |