← Search & browse all drugs

voretigene neparvovec

S01XA27

Forms & Strengths

Intravitreal suspension: 5 x 10^12 vector genomes (vg)/mL in a 0.5 mL extractable volume
Diluent: 0.3 mL extractable volume in a separate vial

Adult Dosing

Single dose of 1.5 x 10^11 vector genomes (vg) administered subretinally per eye
Administered in each eye on separate days with at least 6 days between surgeries
Systemic corticosteroid premedication required: prednisone 1 mg/kg/day (max 40 mg/day) starting 3 days prior, tapering after completion

Pediatric Dosing

Same as adult dosing: 1.5 x 10^11 vg per eye administered subretinally
Applicable to pediatric patients diagnosed with confirmed biallelic RPE65 mutation-associated retinal dystrophy
Systemic corticosteroid weight-based regimen required as premedication

Indications

Treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy
Patients must have sufficient viable retinal cells as determined by the treating ophthalmologist

Mechanism of Action

A recombinant adeno-associated virus serotype 2 (AAV2) vector designed to deliver a normal human RPE65 gene
Restores RPE65 protein activity in the retinal pigment epithelium to normalize the visual cycle in deficient patients

Contraindications

Intraocular or periocular infection
Severe intraocular inflammation
Known hypersensitivity to voretigene neparvovec or any of its components

Adverse Reactions

Procedural complications: subretinal deposit, increased intraocular pressure, macular hole
Ocular events: chorioretinal atrophy, cataract, vessel narrowing, retinopathy, eye inflammation, corneal thinning
Conjunctival hemorrhage, dry eye, eye pain, endophthalmitis

Drug Interactions

No formal drug-drug interaction studies have been performed
Concurrent use of hepatotoxic agents should be monitored given systemic exposure of viral vectors
Potential interactions with systemic immunosuppressive agents used during corticosteroid taper

Curated Content: Needs Vetting Before Put to Clinical Use