Voretigene Neparvovec

Voretigene Neparvovec Brand Name


What is Voretigene Neparvovec

Voretigene neparvovec is an adeno-associated virus vector-based gene therapy indicated for the treatment of children and adults with biallelic RPE65 mutation-associated retinal dystrophy, an inherited form of vision loss.

Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the U.S.

Mutations in the RPE65 gene lead to reduced or absent concentrations of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in impaired vision. Vision loss often begins during childhood or adolescence and ultimately progresses to complete blindness.

Voretigene neparvovec delivers a normal copy of the gene encoding RPE65 to cells of the retina, which then facilitates normal protein production to allow phototransduction and restoration of vision loss.

A short course of an oral corticosteroid is started prior to the administration of voretigene neparvovec to limit the potential immune reaction.

Voretigene neparvovec is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.


  • biallelic RPE65 mutation-associated retinal dystrophy

For the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy

NOTE: The FDA has designated voretigene neparvovec as an orphan drug for this indication.

Side Effects

  1. cataracts
  2. conjunctival hyperemia
  3. endophthalmitis
  4. ocular inflammation
  5. ocular irritation
  6. ocular pain
  7. retinal detachment
  8. retinal hemorrhage
  9. visual impairment

Monitoring Parameters

  • laboratory monitoring not necessary


  • breast-feeding
  • cataracts
  • increased intraocular pressure
  • ocular infection
  • pregnancy
  • requires an experienced clinician


There are no drug interactions associated with Voretigene Neparvovec products.


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