The Medication(s) listed below have been approved by the Food & Drug Administration (FDA) as orphan products for treatment of this condition. There may be other products available
that are not considered orphan products.
Voretigene neparvovec-rzyl (Brand name: Luxturna)
- Manufactured by
Spark Therapeutics, Inc
FDA-approved indication: 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 determined by a treating physician.
National Library of Medicine Drug Information Portal