.Pharmacolibrary.Drugs.N_NervousSystem.N07X_OtherNervousSystemDrugs.N07XX12_Patisiran.Patisiran .Pharmacolibrary.Drugs.N_NervousSystem.N07X_OtherNervousSystemDrugs.N07XX12_Patisiran.Patisiran| name: | Patisiran |
| ATC code: | N07XX12 | route: | intravenous |
| n-compartments | 3 |
Patisiran is an RNA interference (RNAi) therapeutic agent approved for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It works by silencing the transthyretin (TTR) gene, thereby reducing the production of mutant and wild-type TTR protein which forms amyloid deposits.
Pharmacokinetic parameters reported in adult patients with hereditary transthyretin-mediated amyloidosis; values pertain to patisiran lipid nanoparticle (LNP) formulation after intravenous administration.
Suhr, OB, et al., & Adams, D (2015). Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet journal of rare diseases 10 109–None. DOI:10.1186/s13023-015-0326-6 PUBMED:https://pubmed.ncbi.nlm.nih.gov/26338094