.Pharmacolibrary.Drugs.S_SensoryOrgans.S01X_OtherOphthalmologicals.S01XA27_VoretigeneNeparvovec.VoretigeneNeparvovec .Pharmacolibrary.Drugs.S_SensoryOrgans.S01X_OtherOphthalmologicals.S01XA27_VoretigeneNeparvovec.VoretigeneNeparvovec| name: | VoretigeneNeparvovec | |
| ATC code: | S01XA27 | route: | subretinal |
| compartments: | 1 | |
| dosage: | 150000000000.0 | mg |
| volume of distribution: | 1 | L |
| clearance: | 0 | |
| other parameters in model implementation | ||
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.