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Voretigene Neparvovec-rzyl

Medically reviewed on Nov 15, 2018


(voe RET i jeen ne PAR voe vek)

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Suspension, Intraocular:

Luxturna: (0.5 mL)

Suspension, Intraocular [preservative free]:

Luxturna: (0.5 mL)

Brand Names: U.S.

  • Luxturna

Pharmacologic Category

  • Gene Therapy, Adeno-Associated Virus


Voretigene neparvovec-rzyl is an adeno-associated virus vector-based gene therapy that delivers a normal copy of the gene encoding human retinal pigment epithelial 65 kDa protein (RPE65) to retinal cells thus augmenting reduced or absent levels of biologically active RPE65. The RPE65 gene mutations lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and ultimately impairing vision.



Use: Labeled Indications

Retinal dystrophy: Treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy


There are no contraindications listed in the manufacturer's labeling

Dosing: Adult

Retinal dystrophy: Subretinal: 1.5 x 1011 vector genomes in a total volume of 0.3 mL injected into each eye on separate days within a close interval (no fewer than 6 days apart)

Concomitant medications: Systemic oral corticosteroids equivalent to prednisone 1 mg/kg/day (maximum: 40 mg/day) for a total of 7 days (starting 3 days before administration of voretigene neparvovec-rzyl to the first eye), and followed by tapering the dose during the following 10 days. The same corticosteroid dosing regimen applies for administration to the second eye. If the corticosteroid taper to the first eye is not complete 3 days before planned administration to the second eye, the corticosteroid regimen for the second eye replaces the taper for the first eye.

Dosing: Geriatric

Refer to adult dosing.

Dosing: Pediatric

Retinal dystrophy: Children ≥12 months of age and Adolescents: Refer to adult dosing.

Dosing: Renal Impairment

There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, no adjustment expected due to minimal systemic absorption.

Dosing: Hepatic Impairment

There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, no adjustment expected due to minimal systemic absorption.


Prepare within 4 hours of administration. Thaw 1 single-dose vial of voretigene neparvovec-rzyl and 2 vials of diluent at room temperature. Mix all vials by gently inverting about 5 times; transfer 2.7 mL of diluent (using both thawed diluent vials) to the empty 10 mL glass vial using the 3 mL syringe with 20-gauge 1-inch needle. Draw 0.3 mL from the voretigene neparvovec-rzyl vial into a 1 mL syringe with a 27-gauge 1/2 -inch needle, transfer to the 10 mL glass vial containing the diluent, and gently invert about 5 times to mix. Once diluted, 2 operators are required for transfer of the contents of the 10 mL glass vial into each of 2 sterile 1 mL syringes. Refer to manufacturer's labeling for additional information.


Subretinal: For subretinal injection only. Apply a topical broad spectrum microbiocide to the conjunctiva, cornea, and eyelids prior to surgery. Inject along the superior vascular arcade, at least 2 mm distal to the center of the fovea, avoiding direct contact with the retinal vasculature or areas of pathologic features (such as dense atrophy or intraretinal migration). Inject small amount of product slowly until an initial subretinal bleb is observed; inject remaining volume slowly until the total 0.3 mL is delivered. Refer to manufacturer's labeling for additional detailed information.

Following injection, initiate supine head positioning immediately. Rest in a supine position as much as possible for the next 24 hours.


Store intact vial and diluent frozen at ≤ -65°C (≤ -85°F). Store thawed vials and diluted solution at room temperature.

Drug Interactions

There are no known significant interactions.

Adverse Reactions

>10%: Ophthalmic: Conjunctival hyperemia (≤22%), cataract (≤20%; including progression), increased intraocular pressure (≤15%)

1% to 10%: Ophthalmic: Retinal pigment epithelium tear (≤10%), corneal thinning (≤7%), eye disease (macular hole: ≤7%), retinal deposits (subretinal: ≤7%), eye irritation (≤5%), eye pain (≤5%), maculopathy (surface wrinkling: ≤5%), ophthalmic inflammation (≤5%), endophthalmitis (≤2%), retinal changes (foveal thinning and loss of foveal function: ≤2%), retinal hemorrhage (≤2%), retinopathy (foveal dehiscence [separation of the retinal layers in the center of the macula]: ≤2%)


Concerns related to adverse effects:

• Cataracts: Subretinal injection, especially vitrectomy surgery, may increase risk of cataract development or progression.

• Endophthalmitis: May occur following intraocular surgical procedure or injection; use proper aseptic injection techniques. Monitor for signs or symptoms of infection or inflammation.

• Increased intraocular pressure (IOP): Following subretinal injection, IOP may increase; monitor for IOP.

• Retinal abnormalities: Retinal abnormalities may occur during or after subretinal injection or vitrectomy, including macular holes, foveal thinning, loss of foveal function, foveal dehiscence, retinal hemorrhage, retinal tears, epiretinal membrane, and retinal detachment; monitor for macular abnormalities, retinal tears or detachment. Avoid administration in immediate vicinity of fovea.

• Vision loss: Expansion of intraocular air bubble may lead to irreversible vision loss; avoid air travel, travel to high elevations, and scuba diving until air bubble has completely dissipated (may take 1 week or more following injection); verify air bubble dissipation through ophthalmic examination. Permanent decline in visual acuity may occur; monitor for visual disturbances.

Monitoring Parameters

Increase in intraocular pressure; retinal abnormalities (eg, macular holes, foveal thinning, loss of foveal function, foveal dehiscence, retinal hemorrhage, retinal tears, epiretinal membrane or retinal detachment) during and after injection; signs/symptoms of infection or inflammation; visual disturbance

Pregnancy Considerations

Animal reproduction studies have not been conducted.

Patient Education

• Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)

• Have patient report immediately to prescriber vision changes, eye pain, severe eye irritation, floater in the eye, or seeing flashes of light (HCAHPS).

• Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.

Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for health care professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience, and judgment in diagnosing, treating, and advising patients.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.