LUXTURNA(voretigene neparvovec-rzyl)intraocular suspension forsubretinal injection(二)
hthalmitis
5.2 Permanent decline in visual acuity
5.3 Retinal abnormalities
5.4 Increased intraocular pressure
5.5 Expansion of intraocular air bubbles
5.6 Cataract
6 ADVERSE REACTIONS
6.1 Clinical Trials Experience
8 USE IN SPECIFIC POPULATIONS
8.1 Pregnancy
8.2 Lactation
8.3 Females and Males of Reproductive Potential
8.4 Pediatric Use
8.5 Geriatric Use
11 DESCRIPTION
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
12.2 Pharmacodynamics
12.3 Pharmacokinetics
13 NONCLINICAL TOXICOLOGY
13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
13.2 Animal Toxicology and/or Pharmacology
14 CLINICAL STUDIES
16 HOW SUPPLIED/STORAGE AND HANDLING
17 PATIENT COUNSELING INFORMATION
*Sections or subsections omitted from the full prescribing information are not
listed.
----------------------------------------------------
LUXTURNA (voretigene neparvovec-rzyl)
FULL PRESCRIBING INFORMATION
1 INDICATIONS AND USAGE
LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated forthe treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
Patients must have viable retinal cells as determined by the treating physician(s).
2 DOSAGE AND ADMINISTRATION
For subretinal injection only.
2.1 Dose
• The recommended dose of LUXTURNA for each eye is 1.5 x 1011 vector genomes (vg), administered bysubretinal 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 40mg/day) for a total of 7 days (starting 3 days before administration of LUXTURNA to the first eye), andfollowed by tapering the dose during the following 10 days. The same corticosteroid dosing regimenapplies for the administration of LUXTURNA to the second eye. 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 forthe first eye.
2.2 Preparation
Prepare LUXTURNA within 4 hours of administration using sterile technique under aseptic conditions in aClass II vertical laminar flow biological safety cabinet (BSC). Below is the list of items required for dilutionand administration syringe preparation:
• One single-dose vial of Luxturna
• Two vials of Diluent
• One 3-mL sterile syringe
• One 20G 1-inch sterile needle
• Three 1-mL sterile syringes
• Three 27G ½-inch sterile needles
• Two sterile syringe caps
• One 10-mL sterile empty glass vial
• One sterile utility drape
• One sterile plastic bag
• Two sterile labels for administration syringes
• One sterile plain label
• One sterile skin marker
Dilution of LUXTURNA
1. Thaw one single-dose vial of LUXTURNA and two vials of Diluent at room temperature.
2. Mix the contents of the thawed Diluent vials by gently inverting them approximately 5 times.
3. Inspect the Diluent vials. If particulates, cloudiness, or discoloration are visible, do not use the vial(s);new vial(s) of Diluent should be used.
4. Obtain a 3-mL sterile syringe, a 20G 1-inch sterile needle, and a |
