• Miscellaneous ocular agents

Luxturna Generic Name & Formulations

General Description

Voretigene neparvovec-rzyl 5x10^12 vector genomes (vg); per mL; susp for subretinal inj after dilution; preservative-free.

Pharmacological Class

Adeno-associated virus vector-based gene therapy.

How Supplied

Single-dose vial (0.5mL)—1 (w. diluent)

Generic Availability


Mechanism of Action

Luxturna is designed to deliver a normal copy of the gene encoding the human retinoid isomerohydrolase RPE65 (RPE65) to cells of the retina in persons with reduced or absent levels of biologically active RPE65. The RPE65 is produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol, which subsequently forms the chromophore, 11-cis-retinal, during the visual (retinoid) cycle. 

Luxturna Indications


Treatment of biallelic RPE65 mutation-associated retinal dystrophy.

Luxturna Dosage and Administration

Adults and Children

Must have viable retinal cells as determined by physician. Give as subretinal inj. <12mos: not recommended. ≥12mos: 1.5x1011 vg (total 0.3mL) for each eye; separate injs to each eye by ≥6 days. Start oral corticosteroid regimen 3 days prior to Luxturna inj in first eye: give equivalent to prednisone 1mg/kg/day (max 40mg/day) for 7 days and taper dose for the next 10 days; apply same regimen for second eye.

Luxturna Contraindications

Not Applicable

Luxturna Boxed Warnings

Not Applicable

Luxturna Warnings/Precautions


Use aseptic technique. Do not inj in immediate vinicity of fovea. Monitor for infection, inflammation, visual disturbances, retinal abnormalities, intraocular pressure; manage appropriately. Avoid changes in altitude (eg, air travel, high elevations, scuba diving) until intraocular air bubble has completely dissipated. Elderly: not established. Pregnancy. Nursing mothers.

Luxturna Pharmacokinetics

See Literature

Luxturna Interactions

Not Applicable

Luxturna Adverse Reactions

Adverse Reactions

Conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen, macular hole, subretinal deposits, eye inflammation/irritation/pain, maculopathy; endophthalmitis.

Luxturna Clinical Trials

See Literature

Luxturna Note

Not Applicable

Luxturna Patient Counseling

See Literature