Voretigene Neparvovec

Voretigene Neparvovec is a prescription medication indicated for confirmed biallelic RPE65 mutation–associated retinal dystrophy.

• Voretigene Neparvovec is available under the following different brand names: Luxturna, Voretigene Neparvovec-rzyl

Common side effects of Voretigene Neparvovec include:

• eye redness
• cataracts
• increased intraocular pressure
• retinal tear
• dellen (thinning of the corneal stroma)
• macular hole
• subretinal deposits
• eye inflammation
• eye irritation
• eye pain
• maculopathy (wrinkling on the surface of the macula)

Serious side effects of Voretigene Neparvovec include:

• hives
• difficult breathing
• swelling of the face, lips, tongue, or throat
• new or worsening vision problems
• eye pain
• flashes of light or "floaters" in the vision, seeing halos around lights
• signs of eye infection--eye pain or swelling, headache, increased sensitivity to light, crusting or drainage, vision loss

Rare side effects of Voretigene Neparvovec include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Suspension for subretinal injection

• 0.5 mL extractable volume per 2-mL vial
• Supplied concentration (ie, 5 × 1012 vector genomes [vg]/mL) requires a 1:10 dilution before administration

Retinal dystrophy

Adult dosage

• Dose per eye: 1.5 × 1011 vg by subretinal injection in a total volume of 0.3 mL
• Give subretinal administration to each eye on separate days within a close interval but at least 6 days apart
• Systemic oral corticosteroids
  • Start systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day (not to exceed 40 mg/day) for a total of 7 days
  • Initiate corticosteroid dosing regimen 3 days before administration to each eye; then taper dose during the next 10 days
  • Same corticosteroid dosing regimen applies for the administration of voretigene neparvovex-rzyl to the second eye
• Taper corticosteroids
  • If the corticosteroid taper following the first eye is not complete 3 days before the planned administration to the second eye, then the corticosteroid regimen for the second eye replaces the taper for the first eye

Pediatric dosage

• Children younger than 1 year: Safety and efficacy not established
• Children older than 1 year
  • Dose per eye: 1.5 × 1011 vg by subretinal injection in a total volume of 0.3 mL
  • Give subretinal administration to each eye on separate days within a close interval but at least 6 days apart
• See also Administration
• Systemic oral corticosteroids
  • Start systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day (not to exceed 40 mg/day) for a total of 7 days
  • Initiate corticosteroid dosing regimen 3 days before administration to each eye; then taper dose during the next 10 days
  • The same corticosteroid dosing regimen applies for the administration of voretigene neparvovex-rzyl to the second eye
• Taper corticosteroids
  • If the corticosteroid taper following the first eye is not complete 3 days before the planned administration to the second eye, then the corticosteroid regimen for the second eye replaces the taper for the first eye

Dosage Considerations – Should be Given as Follows:

• See "Dosages"

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, healthcare provider, or pharmacist first.

• Voretigene Neparvovec has no noted severe interactions with any other drugs
• Voretigene Neparvovec has no noted serious interactions with any other drugs
• Voretigene Neparvovec has no noted moderate interactions with any other drugs
• Voretigene Neparvovec has no noted minor interactions with any other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See "What Are Side Effects Associated with Using Voretigene Neparvovec?"

Long-Term Effects

• See "What Are Side Effects Associated with Using Voretigene Neparvovec?"

Cautions

• Endophthalmitis may occur following any intraocular surgical procedure or injection; monitor and advise any signs or symptoms of infection or inflammation without delay
• Permanent decline in visual acuity may occur following subretinal injection; monitor for visual disturbances
• Retinal abnormalities (eg, macular holes, foveal thinning, loss of foveal function, foveal dehiscence, retinal hemorrhage) may occur; monitor and manage retinal abnormalities appropriately; do not administer voretigene neparvovex-rzyl near the fovea
• During or following vitrectomy, retinal abnormalities (eg, retinal tears, epiretinal membrane, retinal detachment) may occur; monitor during and following injection to permit early treatment of these retinal abnormalities; advise patients to report any signs or symptoms of retinal tears and/or detachment without delay
• Increased intraocular pressure may occur
  • Avoid air travel, travel to high elevation, or scuba diving until the air bubble formed following administration has completely dissipated from the eye; may take above 1 week or more following injection for the air bubble to dissipate; changes in altitude, while the air bubble is still present, can result in irreversible vision loss
  • Subretinal injection of voretigene neparvovex-rzyl, especially vitrectomy surgery, is associated with an increased incidence of cataract development and/or progression

Pregnancy and Lactation

• Well-controlled human and animal reproductive studies with voretigene neparvovex-rzyl have not been conducted in pregnant women
• No nonclinical or clinical studies were performed to evaluate the effect of voretigene neparvovex-rzyl on fertility
• Lactation
  • No information regarding the presence of voretigene neparvovex-rzyl in human milk, the effects on the breastfed infant, or milk production is available
  • Development and health benefits of breastfeeding should be considered along with the mother's clinical need for voretigene neparvovex-rzyl and any potential adverse effects on the breastfed infant from voretigene neparvovex-rzyl