Active Ingredient: Voretigene neparvovec
Voretigene neparvovec is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
For this indication, competent medicine agencies globally authorize below treatments:
Intraocular
0.3 - 0.3 mL
From 0.3 To 0.3 mL once every day
Patients will receive a single dose of 1.5 × 1011 vg voretigene neparvovec in each eye. Each dose will be delivered into the subretinal space in a total volume of 0.3 mL. The individual administration procedure to each eye is performed on separate days within a close interval, but no fewer than 6 days apart.
Prior to initiation of the immunomodulatory regimen and prior to administration of voretigene neparvovec, the patient must be checked for symptoms of active infectious disease of any nature, and in case of such infection the start of treatment must be postponed until after the patient has recovered.
Starting 3 days prior to the administration of voretigene neparvovec to the first eye, it is recommended that an immunomodulatory regimen is initiated following the schedule below (Table). Initiation of the immunomodulatory regimen for the second eye should follow the same schedule and supersede completion of the immunomodulatory regimen of the first eye.
Pre- and post-operative immunomodulatory regimen for each eye:
| Pre-operative | 3 days prior to voretigene neparvovec administration | Prednisone (or equivalent) 1 mg/kg/day (maximum of 40 mg/day) |
| Post-operative | 4 days (including the day of administration) | Prednisone (or equivalent) 1 mg/kg/day (maximum of 40 mg/day) |
| Followed by 5 days | Prednisone (or equivalent) 0.5 mg/kg/day (maximum of 20 mg/day) | |
| Followed by 5 days of one dose every other day | Prednisone (or equivalent) 0.5 mg/kg every other day (maximum of 20 mg/day) |
Subretinal use.
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