voretigene neparvovec-rzyl 1.5x10exp11 vg/0.3 mL(final)subretinal susp


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Medication name

Generic name:
Voretigene neparvovec - intraocular

(voe-RET-i-jeen ne-PAR-voe-vek)

Brand name(s)


This medication is used to treat a certain inherited eye condition (retinal dystrophy). Voretigene neparvovec is made from a certain virus that has been specially prepared. The virus is used to carry a certain human gene (RPE65) into cells. You will receive this treatment only after tests by your doctor have found that this gene does not work the way it should in your cells. This type of treatment is known as gene therapy. Treatment may improve symptoms of retinal dystrophy, such as decreased vision, loss of side vision, or poor vision at night.

How to use

Your doctor may prescribe medication (such as prednisone) for you to take by mouth daily, starting 3 days before treatment to each eye, and continuing for 2 weeks after the injections. Carefully follow your doctor's directions for taking your medication(s).

This medication is prepared and given by injection into the affected eye(s) by a health care professional. Your doctor will treat each eye on separate days, at least 6 days apart. The eye is numbed before the injection. After the injection, you will remain in the doctor's office for a while, and your eye(s) and vision will be monitored.

After leaving the hospital or clinic, try to rest lying down as much as possible for 24 hours.

During your procedure, an air bubble will form in your eye. This will slowly shrink over 1 week or longer. Do not fly in an airplane, travel to high altitudes, or scuba dive until you are told the air bubble is gone. A change in altitude while the air bubble is in your eye may cause permanent vision loss.

Small amounts of virus may shed from your tears and nose for a short time after the injection. Ask your doctor how to handle and dispose of waste material from dressings, tears, and your nose for up to 7 days after the injection.

Tell your doctor if your condition does not get better or if it gets worse.

Side effects

Mild discomfort or increased tears may occur. If either of these effects lasts or gets worse, tell your doctor or pharmacist promptly.

Remember that this medication has been prescribed because your doctor has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.

This medication may rarely increase your risk for developing certain serious eye conditions (such as cataracts, endophthalmitis, retinal tear/detachment). Tell your doctor right away if you notice any of these symptoms in the affected eye(s):

  • pain
  • vision changes (such as decreased vision, blurred vision, flashes of light, seeing new floaters)

A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including:

  • rash
  • itching/swelling (especially of the face/tongue/throat)
  • severe dizziness
  • trouble breathing

This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.

In the US -

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.

In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.


See also How to Use section.

Before receiving this medication, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.

Before using this medication, tell your doctor or pharmacist your medical history, especially of:

  • other eye conditions (such as cataracts, glaucoma)

This drug may cause blurred vision. Do not drive, use machinery, or do anything that needs clear vision until you can do it safely.

Do not fly in an airplane, travel to high altitudes, or scuba dive until your doctor says you may do so.

Before having surgery, tell your doctor or dentist about all the products you use (including prescription drugs, nonprescription drugs, and herbal products).

During pregnancy, this medication should be used only when clearly needed. Discuss the risks and benefits with your doctor.

It is unknown if this medication passes into breast milk. Consult your doctor before breastfeeding.

Drug interactions

Drug interactions may change how your medications work or increase your risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with your doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without your doctor's approval.


If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call their local poison control center at 1-800-222-1222. Canada residents can call a provincial poison control center.


Medical tests (such as eye exams) should be done while you are using this medication. Keep all medical appointments. Consult your doctor for more details.

Missed dose

It is important to get each dose of this medication as scheduled. If you miss a dose, ask your doctor right away for a new dosing schedule.


Not applicable. This medication is given in a hospital or doctor's office and will not be stored at home.

Important note

HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.

Information last revised March 2024.

Selected from NATIONAL DRUG DATA FILE (NDDF) data included with permission and copyrighted by First Databank, Inc., 2019. This copyrighted material has been downloaded from a licensed data provider.

The above information is intended to supplement, not substitute for, the expertise and judgment of your health care professional. You should consult your health care professional before taking any drug, changing your diet, or commencing or discontinuing any course of treatment.