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Voretigene Neparvovec Injection

Voretigene neparvovec injection treats inherited retinal disease (IRD) due to mutations in both copies of the RPE65 gene. It’s a type of gene therapy your provider gives you in one dose through a needle in your retina.

What is this medication?

VORETIGENE NEPARVOVEC (vor ET i jeen ne PAR voe vek) treats a genetic condition that affects the retina and causes vision loss. It works by helping your body make a protein that keeps the cells that detect light in the retina healthy. This helps prevent worsening symptoms. It may also improve vision. It is a gene therapy.

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This medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

COMMON BRAND NAME(S): LUXTURNA

What should I tell my care team before I take this medication?

They need to know if you have any of these conditions:

  • An unusual or allergic reaction to voretigene neparvovec, other medications, foods, dyes, or preservatives
  • Pregnant or trying to get pregnant
  • Breastfeeding

How should I use this medication?

This medication is injected into the eye. It is given by your care team in a hospital or clinic setting.

Talk to your care team about the use of this medication in children. While it may be given to children as young as 1 year for selected conditions, precautions do apply.

Overdosage: If you think you have taken too much of this medicine contact a poison control center or emergency room at once.

NOTE: This medicine is only for you. Do not share this medicine with others.

What if I miss a dose?

Keep appointments for follow-up doses. It is important not to miss your dose. Call your care team if you are not able to keep your appointment.

What may interact with this medication?

Interactions are not expected.

This list may not describe all possible interactions. Give your health care provider a list of all the medicines, herbs, non-prescription drugs, or dietary supplements you use. Also tell them if you smoke, drink alcohol, or use illegal drugs. Some items may interact with your medicine.

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What should I watch for while using this medication?

Your condition will be monitored carefully while you are receiving this medication.

Your vision may be tested before and after treatment. Tell your care team right away if you have any change in your eyesight.

An air bubble forms in the eye during this treatment. A change in elevation while this air bubble is still there can cause serious eye injury. Avoid air travel, travel to high elevations, and scuba diving until your care team tells you it is safe.

After gene therapy, it is important to avoid contact with the treated person's tears for 7 days. By avoiding contact, the gene therapy will not affect others by accident. Wash hands with soap and water after coming into contact with tears and waste from dressings or nasal secretions. Use disposable gloves when handling. Place waste items in a sealed bag. Get rid of the bag in the regular trash.

What side effects may I notice from receiving this medication?

Side effects that you should report to your care team as soon as possible:

  • Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
  • Blurry vision, vision loss, seeing wavy or bent objects or blind spots with dark, light, or flashing spots
  • Change in vision
  • Eye pain, redness, irritation, or discharge with blurry or decreased vision
  • Small spots floating in vision
  • Sudden eye pain or change in vision such as blurry vision, seeing halos around lights, vision loss

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

  • Eye irritation or itching
  • Eye pain
  • Eye redness

This list may not describe all possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Where should I keep my medication?

This medication is given in a hospital or clinic. It will not be stored at home.

NOTE: This sheet is a summary. It may not cover all possible information. If you have questions about this medicine, talk to your doctor, pharmacist, or health care provider.

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Note: Introduction and Additional Common Questions written and medically approved by Cleveland Clinic professionals.

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