.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX16_Givosiran.Givosiran .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX16_Givosiran.Givosiran| name: | Givosiran |
| ATC code: | A16AX16 | route: | subcutaneous |
| n-compartments | 1 |
Givosiran is a small interfering RNA (siRNA) therapeutic aimed at the treatment of acute hepatic porphyrias (AHP). It reduces the production of aminolevulinic acid synthase 1 (ALAS1) in the liver, thereby lowering toxic heme intermediates. It is approved and marketed under the brand name Givlaari.
Pharmacokinetic profile in adults with acute hepatic porphyria following subcutaneous administration.
Melch, M, et al., & Robbie, GJ (2023). Population Pharmacokinetic Analysis of the RNAi Therapeutic Givosiran in Patients with Acute Hepatic Porphyria. Clinical pharmacokinetics 62(1) 89–99. DOI:10.1007/s40262-022-01197-0 PUBMED:https://pubmed.ncbi.nlm.nih.gov/36633811
Jeon, JY, et al., & Mitra, A (2022). Pharmacokinetic and Pharmacodynamic Modeling of siRNA Therapeutics - a Minireview. Pharmaceutical research 39(8) 1749–1759. DOI:10.1007/s11095-022-03333-8 PUBMED:https://pubmed.ncbi.nlm.nih.gov/35819583