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Voretigene Neparvovec

Medically reviewed by Drugs.com. Last updated on Jul 9, 2020.

Pronunciation

(voe RET i jeen ne PAR voe vek)

Index Terms

  • Voretigene Neparvovec-rzyl

Dosage Forms

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

Suspension, Intraocular:

Luxturna: voretigene neparvovec-rzyl 5000000000000 VG/mL (0.5 mL)

Suspension, Intraocular [preservative free]:

Luxturna: voretigene neparvovec-rzyl 5000000000000 VG/mL (0.5 mL)

Brand Names: U.S.

  • Luxturna

Pharmacologic Category

  • Gene Therapy, Adeno-Associated Virus

Pharmacology

Voretigene neparvovec 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.

Absorption

Low

Use: Labeled Indications

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

Contraindications

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 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

Note: Beginning 3 days before administration of first dose in the first eye, initiate systemic oral corticosteroids equivalent to prednisone 1 mg/kg/day (maximum daily dose: 40 mg/day) and continue for a total of 7 days, then taper corticosteroid 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 regimen.

Retinal dystrophy: Children and Adolescents: Ophthalmic: Subretinal injection: 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); in trials, the youngest patients were 4 years of age.

Reconstitution

Prepare within 4 hours of administration. Thaw 1 single-dose vial of voretigene neparvovec 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 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.

Administration

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.

Storage

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%)

Warnings/Precautions

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

What is this drug used for?

• It is used to treat certain eye problems.

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Vision changes

• Eye pain

• Severe eye irritation

• Floater in the eye

• Seeing flashes of light

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

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