Luxturna

What is Luxturna used for?

Luxturna is a prescription medicine that is used to treat certain eye problems.

Description

LUXTURNA (voretigene neparvovec-rzyl) is a suspension of an adeno-associated virus vector-based gene therapy for subretinal injection. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. LUXTURNA is derived from naturally occurring adeno-associated virus using recombinant DNA techniques.

Each single-dose vial of LUXTURNA contains 5 x 1012 vector genomes (vg) per mL, and the excipients 180 mM sodium chloride, 10 mM sodium phosphate, and 0.001% Poloxamer 188 (pH 7.3), in a 0.5-mL extractable volume. LUXTURNA requires a 1:10 dilution prior to administration. After dilution, each dose of LUXTURNA consists of 1.5 x 1011 vg in a deliverable volume of 0.3 mL.

The Diluent, supplied in 1.7 mL extractable volume per vial in two 2-mL vials, is composed of sterile water containing 180 mM sodium chloride, 10 mM sodium phosphate, and 0.001% Poloxamer 188 (pH 7.3).

LUXTURNA may also contain residual components of HEK293 cells including DNA and protein and trace quantities of fetal bovine serum.

The product contains no preservative.

Mechanism of Action

LUXTURNA is designed to deliver a normal copy of the gene encoding the human retinoid isomerohydrolase RPE65 (RPE65) to cells of the retina in persons with reduced or absent levels of biologically active RPE65. The RPE65 is produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol, which subsequently forms the chromophore, 11-cis-retinal, during the visual (retinoid) cycle. The visual cycle is critical in phototransduction, which refers to the biological conversion of a photon of light into an electrical signal in the retina. Mutations in the RPE65 gene lead to reduced or absent levels of retinoid isomerohydrolase RPE65 activity, blocking the visual cycle and resulting in impairment of vision.

Before taking Luxturna, tell your doctor:

• If you are allergic to Luxturna; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

Children:

• If your child is younger than 12 months old. Do not give Luxturna to a child younger than 12 months old.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Luxturna with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take Luxturna?

• Tell all of your health care providers that you take Luxturna. This includes your doctors, nurses, pharmacists, and dentists.
• Use care when driving or doing other tasks that call for clear eyesight.
• Long-lasting change in eyesight may happen with Luxturna. If you have questions, talk with the doctor.
• Have your eye pressure and eyesight checked as you have been told by the doctor.
• You will need to avoid air travel, travel to high areas, or scuba diving for some time after getting Luxturna. Your doctor will tell you when you can do these things.
• This medicine may make cataracts worse or may raise the chance of new cataracts. Talk with the doctor.
• After getting Luxturna, tears may have Luxturna in them. You will need to take special care when handling and throwing away used dressings and other cleaning supplies with tears and nose discharge on them for 7 days after getting Luxturna. Throw them away in household waste in a sealed bag. If you have questions, talk with the doctor.
• Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using Luxturna while you are pregnant.
• Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.

How is Luxturna best taken?

Use Luxturna as ordered by your doctor. Read all information given to you. Follow all instructions closely.

• It is given as a shot into the eye.

What do I do if I miss a dose?

• Call your doctor to find out what to do.

What are the side effects of Luxturna that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
• Very bad infections or other eye problems can happen inside the eye. These could lead to loss of eyesight. Call your doctor right away if you have a change in eyesight, eye pain, or very bad eye irritation. Call your doctor right away if you see any new floaters or flashes of light.

What are some other side effects of Luxturna?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Eye irritation.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Luxturna?

• If you need to store Luxturna at home, talk with your doctor, nurse, or pharmacist about how to store it.

Label