Products
Voretigenneparvovec was approved in the United States in 2017, in the EU in 2018, and in many countries in 2020 as a concentrate and solvent for the preparation of a solution for injection (Luxturna).
Structure and properties
Voretigenneparvovec is the capsid of an adeno-associated viral vector serotype 2 (AAV2). It contains the cDNA of the human retinal pigment epithelium-specific 65 kDa protein (hRPE65).
Effects
Voretigenneparvovec (ATC S01XA27) is a gene therapy drug. The drug supplies retinal pigment epithelial cells with a copy of the gene (cDNA) encoding the human retinal pigment epithelium-specific 65-kilodalton protein (RPE65). The cDNA is not incorporated into the host cell genome. It remains outside the chromosomes in the nucleus.
Indications
For the treatment of patients with visual loss due to hereditary retinal dystrophy based on proven biallelic RPE65 mutations who have sufficient viable retinal cells.
Dosage
According to the SmPC. The drug is injected into the subretinal space. It must not be administered intravitreally.
Contraindications
- Hypersensitivity
- Ocular or periocular infection
- Active intraocular inflammation
Refer to the drug label for complete precautions.
Adverse effects
The most common possible adverse effects include local ocular side effects:
- Conjunctival redness, eye inflammation, eye irritation, eye pain.
- Cataract
- Increased intraocular pressure
- Retinal tear
- Corneal dent
- Macular hole, maculopathy
- Subretinal deposits
