Patient resources

Patient support overview

Spark Therapeutics Generation Patient Services^®

If you have confirmed mutations in both copies of the RPE65 gene and have discussed treatment with your healthcare professional, you are eligible* to enroll in Spark Therapeutics Generation Patient Services.

The goal of Spark Therapeutics Generation Patient Services is to provide support that’s customized for eligible* patients. While patients are encouraged to use Spark Therapeutics Generation Patient Services, enrollment is voluntary and is not required to receive treatment with LUXTURNA^®.

Spark Therapeutics Generation Patient Services can be a great resource for you but does not provide medical advice. You should always talk to your healthcare professional about any medical questions you may have.

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. Spark Therapeutics Generation Patient Services does not provide medical advice. You should always rely on the direction of your healthcare professional for treatment and care.

Your support team can help by:

Providing a caring support team from confirmed diagnosis through postsurgery follow-up

Ensuring a single point of contact to help you navigate insurance coverage and connect you with financial assistance resources as needed

Helping to coordinate your visits to the treatment center

Answering any nonmedical questions you may have along the way

Visit MySparkGeneration to learn more

Locate a specialist who can help with genetic testing and provide more information on inherited retinal diseases (IRDs).

Find a specialist

FAQs

You may ask your healthcare professional if LUXTURNA is right for you if you have an IRD due to mutations in both copies of your RPE65 gene and enough remaining cells in your retina (as determined by a healthcare professional).

LUXTURNA is a one-time gene therapy for patients 12 months of age and older. LUXTURNA is not recommended for patients younger than 12 months of age because the retina is still growing, which may affect how LUXTURNA works.

Talk to your healthcare professional to see if LUXTURNA could be right for you. LUXTURNA is a prescription gene therapy product used for the treatment of people with an IRD 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.

Haven’t had a genetic test? You can find a specialist who can help with testing and provide more information on IRDs.

Learn more

LUXTURNA is administered by retina specialists who have experience treating patients with IRDs and have received education on administering LUXTURNA. After the first eye is treated, the second eye will be treated at least six days later. For more insight into the treatment process, watch Monroe’s experience in this video.

Yes, LUXTURNA can be used to treat children 12 months of age and older who have an IRD due to mutations in both copies of the RPE65 gene, and enough remaining cells in 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.

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, decreased thickness of the retina 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).

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

Redness of the eye
Cataract (clouding of the lens inside 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

You should discuss any side effects with your healthcare professional.

Please see additional Important Safety Information for LUXTURNA.

Yes, your caregiver may reach out to Spark Therapeutics with any questions or concerns, or for more information go to mysparkgeneration.com.

Evaluating whether you will receive insurance approval for LUXTURNA may involve several steps. Get in touch with Spark^® Therapeutics to request more information about eligibility, coverage, and the approval process.

For more information about financial resources, call Spark Therapeutics Generation Patient Services^® toll-free at 1-833-SPARK-PS (1-833-772-7577).

Yes, there may be resources to assist you with out-of-pocket costs. It is important to note that eligibility for this support varies depending on the type of insurance you have. Your support team at Spark^® Therapeutics can help you navigate the process and determine which resources work best for you.

Get in touch

Spark Therapeutics Generation Patient Services aims to provide support that’s customized for eligible patients. While patients are encouraged to use Spark Therapeutics Generation Patient Services, enrollment is voluntary and is not required to receive treatment with LUXTURNA^®.

Visit mysparkgeneration.com to learn more.

Links and downloads

Patient and caregiver brochure

Use this brochure to gain more information about LUXTURNA and whether it might be right for you or someone you care for.

Download PDF

LUXTURNA Mentee Program brochure

Curious about the treatment journey with LUXTURNA? Use this brochure to learn more about the Mentee Program.

Download PDF

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.