Version May 2024
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.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, no adjustment expected due to minimal systemic absorption.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, no adjustment expected due to minimal systemic absorption.
Refer to adult dosing.
(For additional information see "Voretigene neparvovec: Pediatric drug information")
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.
All patients: There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, dosage adjustment unlikely due to low systemic absorption.
All patients: There are no dosage adjustments provided in the manufacturer's labeling (has not been studied); however, dosage adjustment unlikely due to low systemic absorption.
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Adverse reactions reported in children, adolescents, and adults.
>10%: Ophthalmic: Cataract (≤20%), conjunctival hyperemia (≤22%), increased intraocular pressure (≤15%)
1% to 10%: Ophthalmic: Corneal thinning (≤7%), endophthalmitis (≤2%), eye disease (macular hole: ≤7%), eye irritation (≤5%), eye pain (≤5%), maculopathy (surface wrinkling: ≤5%), ophthalmic inflammation (≤5%), retinal changes (foveal thinning and loss of foveal function: ≤2%), retinal deposits (subretinal: ≤7%), retinal hemorrhage (≤2%), retinal pigment epithelium tear (≤10%), retinopathy (foveal dehiscence [separation of the retinal layers in the center of the macula]: ≤2%)
Postmarketing: Ophthalmic: Retinal disturbance (chorioretinal atrophy)
There are no contraindications listed in the manufacturer's US labeling.
Canadian labeling: Hypersensitivity to voretigene neparvovec or any component of the formulation; ocular or periocular infection; active intraocular inflammation.
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.
Use in infants is not recommended due to ongoing retinal cell proliferation, which may dilute or cause loss of drug. In trials, the youngest patients included were 4 years old.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Suspension, Intraocular:
Luxturna: Voretigene neparvovec-rzyl 5 trillion VG/mL (0.5 mL)
Suspension, Intraocular [preservative free]:
Luxturna: Voretigene neparvovec-rzyl 5 trillion VG/mL (0.5 mL)
No
Suspension (Luxturna Intraocular)
5000000000000VG/ML (per 0.5 mL): $510,000.00
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution, Intraocular:
Luxturna: 5000000000000 VG/ML (1 ea)
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.
Ophthalmic: For administration in surgical suite only by physician experienced in performing intraocular surgery. Apply a topical broad spectrum microbiocide to the conjunctiva, cornea, and eyelids prior to surgery.
Subretinal injection: Attach sterile syringe to the provided extension tube (not to exceed 15.2 cm in length, 1.4 mm in diameter) and subretinal injection cannula; depress plunger slowly to 0.3 mL mark to prime tubing/cannula and eliminate any air bubbles. After appropriate preparation of the eye, 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.
Retinal dystrophy: Treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy in patients with viable retinal cells as determined by treating health care provider.
None known.
There are no known significant interactions.
Animal reproduction studies have not been conducted.
It is not known if voretigene neparvovec is present in breast milk. According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and benefits of treatment to the mother.
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
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
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