.Pharmacolibrary.Drugs.ATC.S.S01XA27 .Pharmacolibrary.Drugs.ATC.S.S01XA27| name: | VoretigeneNeparvovec |
| ATC code: | S01XA27 | route: | subretinal |
| n-compartments | 1 |
Voretigene neparvovec is a gene therapy product used for the treatment of inherited retinal dystrophy due to confirmed biallelic RPE65 mutations, approved for use in several regions including the US and EU. It delivers a functional copy of the RPE65 gene via an adeno-associated virus vector to retinal cells to improve vision.
No traditional pharmacokinetic parameters (such as plasma concentration profiles) are reported, as voretigene neparvovec is a gene therapy vector administered directly into the subretinal space, with primarily local retinal action and minimal systemic distribution. Clinical pharmacokinetic studies are not applicable.