Take your first step

Confirming mutations in both copies of the RPE65 gene is the first step to determine if you may be appropriate for treatment with LUXTURNA^®.

Your healthcare professional can help

Based on your current diagnosis and symptoms, your healthcare professional can discuss genetic testing options with you.

Learn more

Taking your next steps

Here is an overview of what will occur before, during, and after treatment:

Once your healthcare professional confirms you have mutations in both copies of the RPE65 gene, you will discuss if LUXTURNA may be right for you.
You may fill out an enrollment and consent form with your healthcare professional to enroll you into Spark Therapeutics Generation Patient Services^®.*

Enrollment in Spark Therapeutics Generation Patient Services is voluntary and patients are not required to enroll in order to receive treatment with LUXTURNA.

Get to know Spark Therapeutics Generation Patient Services

*
Participation in Spark Therapeutics Generation Patient Services is voluntary. You may choose to participate in all, some, or none of the services offered. Participating or deciding not to participate in these services will have no effect on your ability to get treatment or the nature of your treatment or care. Generation Patient Services does not provide medical advice. You should always rely on the direction of your healthcare professional for treatment and care.
Spark Therapeutics Generation Patient Services will coordinate an ocular gene therapy treatment center visit where a retina specialist will confirm that you have enough remaining retinal cells.
Your surgery will be scheduled and you will return to the treatment center on two separate occasions for treatment, one for each eye.
After your treatment, you may resume follow-up care with your original healthcare professional or continue to see your treatment center specialist.

Get help with genetic testing

Use the Specialist Locator to find a healthcare professional in your area of the United States who can discuss genetic testing for RPE65 mutations and can provide more information on inherited retinal diseases (IRDs).

Find a specialist

Discover patient resources for LUXTURNA

Indication

LUXTURNA (voretigene neparvovec-rzyl) is a prescription gene therapy product used for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene, which can only be confirmed through genetic testing. You must also have enough remaining cells in your retina (the thin layer of tissue in the back of your eyes) as determined by your healthcare professional.

Important Safety Information for LUXTURNA

The following serious side effects may occur during or after the administration of LUXTURNA:

Eye infections, including a serious infection inside of the eye called endophthalmitis, that may lead to blindness.
Permanent decline in visual acuity, or the sharpness of central vision.
Changes in the retina (the thin layer of tissue in the back of the eye) that can lead to vision loss including:
- development of a hole, thinning, or loss of function of the retina, separation of the layers in the center of the retina, loss of retinal cells and the choroid (the layer of blood vessels that lines the back of the eye), or bleeding in the retina.
- breaks in or wrinkling on the surface of the retina or detachment of the retina.
Increased pressure inside of the eye. You should follow-up with your healthcare professional as instructed to detect and treat any increased pressure in the eye as this may cause blindness.
Expansion of the air bubble formed in the eye after administration of LUXTURNA. You should avoid air travel, travel to high elevations, or scuba diving until your healthcare professional has told you that the air bubble formed in the eye following administration of LUXTURNA has disappeared. Engaging in these activities while the air bubble is present can cause permanent vision loss.
Formation or worsening of cataract (clouding of the lens inside of the eye).

Tell your healthcare professional right away if you have any of the following symptoms of these serious side effects:

Seeing floaters (specks that float about in your field of vision)
Pain in the eye
Any change in vision including decreased vision or blurred vision
Seeing flashes of light

The following are the most common side effects that may occur with LUXTURNA:

Redness of the eye
Cataract (clouding of the lens inside of the eye)
Increased pressure inside of the eye
Breaks in the retina
Dellen (thinning of the clear layer in the front of the eye)
Development of a hole in the center of the retina
Subretinal deposits (deposits under the retina)
Eye swelling, irritation or pain
Wrinkling on the surface of the center of the retina

Treatment with LUXTURNA is not recommended for patients younger than 12 months of age because the retina is still growing, which may affect how LUXTURNA works.

Because small quantities of LUXTURNA may be in your tears for a short period of time, for the first 7 days after administration of LUXTURNA, place any waste material from dressings, tears and nasal secretions in sealed bags prior to disposal.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You may also report side effects to Spark Therapeutics at 1-855-SPARKTX (1-855-772-7589).

This information does not take the place of talking to your healthcare professional about your medical condition or treatment. If you have questions about LUXTURNA after reading this information, ask your healthcare professional.

Please see the US Full Prescribing Information for LUXTURNA.