For: Biallelic RPE65-Mediated Inherited Retinal Disease
Luxturna (voretigene neparvovec) is an adeno-associated viral (AAV) vector gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). Each carton of LUXTURNA (NDC 71394 – 415-01) contains one single-dose vial of the LUXTURNA (NDC 71394 – 065-01, 0.5 mL extractable volume) and two vials of Diluent (NDC 71394 – 716-01, 1.7 mL extractable volume in each vial). LUXTURNA contains 5 x 1012 vector genomes (vg) per mL, requires a 1:10 dilution prior to administration.
Store LUXTURNA and Diluent frozen at ? -65 °C.
Following thaw of the vials, store at room temperature. Store diluted LUXTURNA at room temperature [See Dosage and Administration 2.2].
LUXTURNA is an adeno-associated virus vector-based gene therapy. Follow universal biohazard precautions for handling. These highlights do not include all the information needed to use LUXTURNA safely and effectively. See full prescribing information for LUXTURNA.
LUXTURNA (voretigene neparvovec-rzyl) intraocular suspension for subretinal injection
Initial U.S. Approval: 2017
INDICATIONS AND USAGE
LUXTURNA 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). (1)
DOSAGE AND ADMINISTRATION
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. (2.1)
Perform subretinal administration of LUXTURNA to each eye on separate days within a close interval, but no fewer than 6 days apart. (2.1)
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. (2.1)
DOSAGE FORMS AND STRENGTHS
LUXTURNA is a suspension for subretinal injection, supplied in a 0.5 mL extractable volume in a single-dose 2 mL vial for a single administration in one eye. The supplied concentration (5x1012vg/mL) requires a 1:10 dilution prior to administration. The Diluent is supplied in two single-use 2-mL vials. (3)
WARNINGS AND PRECAUTIONS
Endophthalmitis: Use proper aseptic injection technique and monitor for signs and symptoms of infection. (5.1)
Permanent decline in visual acuity: Monitor for visual disturbances. (5.2)
Retinal abnormalities: Monitor for macular abnormalities, retinal tears or breaks. Do not inject in the immediate vicinity of the fovea. (5.3)
Increased intraocular pressure: Monitor and manage intraocular pressure elevations. (5.4)
Expansion of intraocular air bubbles: Air travel and/or scuba diving is not recommended until any intraocular air bubbles have been absorbed. (5.5)
Cataract: Subretinal injection of LUXTURNA may result in cataract formation or increase in the rate of cataract progression. (5.6)
The most common adverse reactions (incidence ? 5%) in the clinical trials 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). (6)
To report SUSPECTED ADVERSE REACTIONS, contact Spark Therapeutics, Inc. at 1-855-SPARKTX, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
USE IN SPECIFIC POPULATIONS
Pediatric use: Use in infants under 12 months of age is not recommended because of potential dilution or loss of LUXTURNA after administration due to the active retinal cells proliferation occurring in this age group. (8.4)
See 17 for PATIENT COUNSELING INFORMATION.