Drug Class
Gene therapy, Retinal gene therapy
Uses & Indications
Treatment of inherited retinal disease caused by RPE65 gene mutations, leading to vision loss
Storage Requirements
Store at -80°C (-112°F) or colder; do not store in a refrigerator or at room temperature; protect from light
Manufacturer & Packaging
Manufacturer: NOVARTIS EUROPHARM LIMITED, IRELAND
Package Size
1 vial of concentrate (2 mL cyclic olefin polymer vial) and 2 vials of solvent (2 mL cyclic olefin polymer vial)
Price & Supplier
Price in UAE: AED 1881560.50
Dosage Information
Adult Dose
1.5 x 10^11 vector genomes (5 trillion vector genomes/ml) administered as a single intraocular injection per affected eye
Pediatric Dose
1.5 x 10^11 vector genomes (5 trillion vector genomes/ml) administered as a single intraocular injection per affected eye; approved for patients aged 1 year and older
Side Effects
Intraocular inflammation, increased intraocular pressure, retinal detachment, cataract formation, visual disturbances