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Luxturna®

GoodRx

Voretigene Neparvovec-rzyl · intraocular injection

Injections & Implants

Indication
LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).
Contraindications
None.
Available Forms
5 x 10¹² vg/mL suspension (requires 1:10 dilution), 0.5 mL in a 2-mL single-dose vial, with two 1.7-mL diluent vials
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. The visual cycle is critical in phototransduction, which refers to the biological conversion of a photon of light into an electrical signal in the retina.
Dosage
For subretinal injection only. The recommended dose of LUXTURNA for each eye is 1.5 x 10¹¹ vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL. Perform subretinal administration of LUXTURNA to each eye on separate days within a close interval, but no fewer than 6 days apart. Recommend systemic oral corticosteroids equivalent to prednisone at 1 mg/kg/day (maximum of 40 mg/day) for a total of 7 days (starting 3 days before administration of LUXTURNA to each eye), and followed by a tapering dose during the next 10 days. If the corticosteroid taper following LUXTURNA administration to the first eye is not complete three days prior to the planned LUXTURNA administration to the second eye, then the corticosteroid regimen for the second eye replaces the taper for the first eye. Prepare LUXTURNA within 4 hours of administration.
Warnings & Precautions
  • Endophthalmitis: Endophthalmitis may occur following any intraocular surgical procedure or injection. Use proper aseptic injection technique when administering LUXTURNA. Following the injection, monitor patients to permit early treatment of any infection. Advise patients to report any signs or symptoms of infection or inflammation without delay.
  • Permanent Decline in Visual Acuity: Permanent decline in visual acuity may occur following subretinal injection of LUXTURNA. Monitor patients for visual disturbances.
  • Retinal Abnormalities: Retinal abnormalities may occur during or following the subretinal injection of LUXTURNA, including macular holes, foveal thinning, loss of foveal function, foveal dehiscence, chorioretinal atrophy, and retinal hemorrhage. Monitor and manage these retinal abnormalities appropriately. Do not administer LUXTURNA in the immediate vicinity of the fovea.
  • Retinal Tears and/or Detachment: Retinal abnormalities may occur during or following vitrectomy including retinal tears, epiretinal membrane, or retinal detachment. Monitor patients during and following the 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: Increased intraocular pressure may occur after subretinal injection of LUXTURNA. Monitor and manage intraocular pressure appropriately.
  • Expansion of Intraocular Air Bubbles: Instruct patients to avoid air travel, travel to high elevations or scuba diving until the air bubble formed following administration of LUXTURNA has completely dissipated from the eye. It may take one week or more following injection for the air bubble to dissipate. A change in altitude while the air bubble is still present can result in irreversible vision loss. Verify the dissipation of the air bubble through ophthalmic examination.
  • Cataract: Subretinal injection of LUXTURNA, especially vitrectomy surgery, is associated with an increased incidence of cataract development and/or progression.
Common Side Effects
The most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula).
Pregnancy & Lactation
Pregnancy: Adequate and well-controlled studies with LUXTURNA have not been conducted in pregnant women. Animal reproductive studies have not been conducted with LUXTURNA.
Lactation: There is no information regarding the presence of LUXTURNA in human milk, the effects on the breastfed infant, or the effects on milk production.
Pediatric Use
Treatment with LUXTURNA is not recommended for patients younger than 12 months of age, because the retinal cells are still undergoing cell proliferation, and LUXTURNA would potentially be diluted or lost during cell proliferation. The safety and efficacy of LUXTURNA have been established in pediatric patients.