Description and Brand Names

Drug information provided by: IBM Micromedex


Voretigene neparvovec-rzyl injection is used to treat retinal dystrophy (a rare form of inherited loss of vision) with confirmed biallelic RPE65 mutation.

This medicine must be given only by or under the direct supervision of your doctor.

Before Using

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:


Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.


Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of voretigene neparvovec-rzyl injection in children 12 months of age and older. Use is not recommended in children younger than 12 months of age.


Appropriate studies have not been performed on the relationship of age to the effects of geriatric-specific problems that would limit the usefulness of voretigene neparvovec-rzylin injection in the elderly. Safety and efficacy have not been established.

Drug Interactions

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other Interactions

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Proper Use

Your doctor will give you this medicine through a needle into your retina (part of the eye). It is to be injected into each eye separately at least 6 days apart.

You may be given an oral steroid medicine before, during, and after treatment with voretigene neparvovec-rzyl in the first eye and to be repeated with treatment of your other eye.

Your doctor may want you to lie down as much as possible for 24 hours after treatment.


It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly and to check for unwanted effects.

Endophthalmitis or inflammation of the inside of the eye may occur after eye injection or surgery. Check with your doctor right away at the first signs of an infection: blurred vision or other changes in vision, eye pain, redness of the eye, sensitivity of the eye to light, or tearing.

This medicine may cause air bubbles to form inside the eye. Avoid air travel, traveling to places with high elevations, or scuba diving for at least 1 week.

Retinal problems (eg, a tear or hole, bleeding) may occur with this medicine. Check with your doctor right away if you see floaters or light flashes in your line of vision, decreased vision, or any other change in vision.

This medicine may increase your risk of cataracts and cause an increase of pressure in the eyes. Check with your doctor if you have blindness, blurred vision, or decreased vision.

Be careful when handling your clothes or other materials that have been wet by your tears or nose secretions. This medicine may still be present for up to 7 days after treatment.

Side Effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

  1. Blindness
  2. blurred vision
  3. change in vision
  4. decreased vision
  5. increase in blood flow to the whites of the eyes
  6. loss of vision

Less common

  1. Eye inflammation, pain, redness, irritation, or swelling
  2. headache
  3. seeing floaters or light flashes
  4. tearing of the eyes

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.