Overview
11 DESCRIPTION LUXTURNA (voretigene neparvovec-rzyl) is a suspension of an adeno-associated virus vector-based gene therapy for subretinal injection. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. LUXTURNA is derived from naturally occurring adeno-associated virus using recombinant DNA techniques.
Mechanism of Action
12 CLINICAL PHARMACOLOGY 12.1 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.
Indications
- 1 INDICATIONS AND USAGE 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). 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 )
Common Doses
- 0.3 mL
- 1 mg
- 40 mg
- 20 G
Dosage
2 DOSAGE AND ADMINISTRATION For subretinal injection only. The recommended dose of LUXTURNA for each eye is 1.5 x 10 11 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.
Contraindications
- 4 CONTRAINDICATIONS None. None.
Side Effects
- 6 ADVERSE REACTIONS 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). 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...
Interactions
- No major interactions listed on the FDA label. Always consult a healthcare professional.