Inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations

Active Ingredient: Voretigene neparvovec

Indication for Voretigene neparvovec

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 - 65 years old)

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:

1.5 x 10ยนยน vg

Route of admnistration

Intraocular

Defined daily dose

0.3 - 0.3 mL

Dosage regimen

From 0.3 To 0.3 mL once every day

Detailed description

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.

Immunomodulatory regimen

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-operative3 days prior to voretigene neparvovec administrationPrednisone (or equivalent) 1 mg/kg/day (maximum of 40 mg/day)
Post-operative4 days (including the day of administration)Prednisone (or equivalent) 1 mg/kg/day (maximum of 40 mg/day)
Followed by 5 daysPrednisone (or equivalent) 0.5 mg/kg/day (maximum of 20 mg/day)
Followed by 5 days of one dose every other dayPrednisone (or equivalent) 0.5 mg/kg every other day (maximum of 20 mg/day)

Dosage considerations

Subretinal use.

Active ingredient

Voretigene neparvovec

Voretigene neparvovec results to transduction of retinal pigment epithelial cells with a cDNA encoding normal human RPE65 protein (gene augmentation therapy), providing the potential to restore the visual cycle in patients with vision loss due to inherited retinal dystrophy.

Read more about Voretigene neparvovec

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