Luxturna
Luxturna (voretigene neparvovec or voretigene neparvovec-rzyl) is a type of gene therapy used to treat retinal dystrophy. This is a rare condition caused by a change in the RPE65 gene and can lead to blindess. Luxturna (voretigene neparvovec) is given as a single dose by surgical injection into each eye by a healthcare provider who is trained to perform eye surgeries. Side effects include eye redness and cataracts.
What is Luxturna (voretigene neparvovec)?
What is Luxturna (voretigene neparvovec) used for?
- Retinal dystrophy in people ages 1 year of age and older with changes in the RPE65 gene
How Luxturna (voretigene neparvovec) works
Some people with retinal dystrophy have changes in the RPE65 genes, and this can lead to vision loss and blindness.
Luxturna (voretigene neparvovec) is a type of gene therapy. It works by delivering working copies of the RPE65 gene into the eye. The eye is then able to make the proteins needed for healthy vision.
Drug facts
More on Luxturna (voretigene neparvovec) essentials
Side effects of Luxturna (voretigene neparvovec)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Cataract (19%)
- Eye redness (up to 11%)
- Raised eye pressure (10%)
- Retinal tear (5%)
- Eye swelling (5%)
Less Common Side Effects
- Eye irritation
- Eye pain
- Other problems with the structures inside the eye
Luxturna (voretigene neparvovec) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Eye problems: floaters, eye pain, eye swelling, changes in vision, flashes of light
The following Luxturna (voretigene neparvovec) side effects have also been reported
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
Pros and cons of Luxturna (voretigene neparvovec)
Pros
- Each eye only needs one injection.
- Can be used in children as young as 1 year of age
- Can improve vision in as early as 1 month after treatment
Cons
- Must be given as an injection into each eye by a healthcare provider
- Only given at specific ocular gene therapy treatment centers
- Risk of damage to the retina
Pharmacist tips for Luxturna (voretigene neparvovec)
- Your provider will prescribe you an oral steroid to help ease swelling from the Luxturna (voretigene neparvovec) procedure. You'll start it 3 days before and continue it 7 days after the procedure. After that, your provider will help you stop the steroid safely by lowering the dose slowly over time.
- During the 24 hours after the Luxturna (voretigene neparvovec) procedure, it's important that you lie down to rest flat on your back as much as possible.
- After receiving each Luxturna (voretigene neparvovec) injection, avoid any changes in altitude, such as flying or scuba diving, until your provider tells you it's safe to do so. An air bubble might form in the eye from the injection, and doing these things while the air bubble is still there can cause vision loss. Check with your provider to make sure the air bubble is gone before doing these things.
- Some of the medication might leak out with your tears, so make sure to handle all of your dressings and tissues carefully for at least a week after each treatment procedure. Throw away your waste properly to prevent this medication from getting on other surfaces or other people. You can put your waste from dressings or tissues in a sealed bag before throwing it in the trash.
- Let your provider know right away if you have trouble seeing, such as blurry or cloudy vision. One of the more common side effects from Luxturna (voretigene neparvovec) is cataracts. Your provider can examine your eyes to see if you have cataracts that need to be treated, or if you have other vision-related side effects from the procedure.
- Ask your provider how often you need to come into the clinic for eye exams after the Luxturna (voretigene neparvovec) procedure. Because this procedure raises your risk for certain side effects, like retinal damage, it's important that your provider treat these early on to prevent worsening eyesight. Let your provider know as soon as possible if you experience eye pain, floaters, or changes in vision.
- It's not known if Luxturna (voretigene neparvovec) can cause harm to your baby during pregnancy or if it affects your breast milk. Before your treatment with Luxturna (voretigene neparvovec), let your provider know if you're pregnant, planning to become pregnant, or breastfeeding.
Common questions about Luxturna (voretigene neparvovec)
People in clinical studies for Luxturna (voretigene neparvovec) were given a vision test called the Multi-Luminance Mobility Test (MLMT). This test used an obstacle course with different levels of light to simulate everyday, real-world scenarios and environments, such as being in a well-lit room or walking around in a city at night. Studies show that one year after treatment, most people who received Luxturna (voretigene neparvovec) had improved vision and were able to complete the test at the lowest level of light.
Studies show that vision improved by 1 month after people were treated with Luxturna (voretigene neparvovec). This improvement in vision lasted for at least 4 years.
Luxturna (voretigene neparvovec) is given by a trained healthcare professional as an injection under the retina, which is the innermost layer of the eye. One eye is treated with Luxturna (voretigene neparvovec) at a time; both injections are typically separated by at least 6 days.
Luxturna (voretigene neparvovec) is made by Spark Therapeutics.
Luxturna (voretigene neparvovec) was approved by the FDA in 2017 as the first gene therapy for people with retinal dystrophy due to an abnormal RPE65 gene.
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Risks and warnings for Luxturna (voretigene neparvovec)
Luxturna (voretigene neparvovec) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Eye infection
With any surgical procedure on the eye or injection into the eye, including treatment with Luxturna (voretigene neparvovec), there's a potential risk of an eye infection. Let your provider know right away if you experience any swelling or signs of an eye infection, such as new floaters, eye pain, or vision changes.
Vision problems
Receiving Luxturna (voretigene neparvovec) can lead to vision problems, including vision loss, due to damage to certain parts of your eye, such as the retina. These vision problems can happen due to a tear in the area of the injection, thinning of the retina, separation of retinal layers, or bleeding in the retina. Let your provider know right away if you have any changes to your eyesight, and report any signs of blurry vision, flashes of light, or floaters.
Raised eye pressure
The Luxturna (voretigene neparvovec) injection can raise the pressure in your eye, which can lead to problems like vision loss or glaucoma if not caught and treated in time. Make sure to see your eye provider regular so your eyes can be examined. Your provider will monitor the pressure in your eye during your follow-up appointments.
Formation of air bubble in the eye
- Risk factors: Air travel | High altitude | Scuba diving
An air bubble might form in the eye and stay there for a week or more after the Luxturna (voretigene neparvovec) injection. During this time, avoid any changes in altitude, such as flying, traveling to high-elevation places, or scuba diving. These activities can cause permanent vision loss if they're done while the air bubble is still in your eye. Your provider can check to see if the air bubble has disappeared before you travel or carry out these activities.
Cataracts
Treatment with Luxturna (voretigene neparvovec) is associated with a higher risk of developing cataracts. It can also make cataracts that you already have worse. Let your provider know if you develop blurry or cloudy vision.
Luxturna (voretigene neparvovec) dosage
Typical dosage for Luxturna (voretigene neparvovec)
A single dose of Luxturna (voretigene neparvovec) contains 1.5 x 1011 vector genomes (vg).
Your provider will inject one dose of Luxturna (voretigene neparvovec) beneath the retina of one eye at a special treatment clinic. Then, they'll inject the medication into the other eye at least 6 days later.
