Patient supportPatient FAQs

Frequently asked questions (FAQs)

What is Spark Therapeutics Generation Patient ServicesSM? What assistance do they provide?

Generation Patient Services is a program sponsored by Spark® Therapeutics for patients who have mutations in both copies of the RPE65 gene and may be candidates for treatment with LUXTURNA® (voretigene neparvovec-rzyl). Our goal is to provide personalized support throughout your journey.

We 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 ocular gene therapy treatment center
  • Answering any nonmedical questions you may have along the way

Because of legal requirements, not all services and resources are available to patients with government insurance.

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

How do I enroll in Spark Therapeutics Generation Patient Services?

Enrollment in Spark Therapeutics Generation Patient Services is open to patients with an inherited retinal disease due to mutations in both copies of the RPE65 gene, which can only be confirmed by genetic testing. Once confirmed, you may enroll by completing an enrollment form.

Once an enrollment form is submitted, a member of the Generation Patient Services team will contact you to assist in scheduling a consultation at an ocular gene therapy treatment center and explain other services and resources that may be available to you.

Why do I have to use an ocular gene therapy treatment center?

LUXTURNA is only administered at designated ocular gene therapy treatment centers. Each ocular gene therapy treatment center was selected based on specific criteria that support appropriate patient care. Every ocular gene therapy treatment center is staffed with retinal specialists, nurses, and other professionals who have experience treating patients with inherited retinal diseases and have received education on administering LUXTURNA.

How is my ocular gene therapy treatment center selected?

If your healthcare professional decides that LUXTURNA is right for you, you should consider selecting an ocular gene therapy treatment center based on your insurance coverage, in an effort to minimize your out-of-pocket costs; your schedule; and your preference, among other considerations.

Are financial assistance resources available to help with my out-of-pocket expenses?

Yes, there are resources available to assist you with out-of-pocket costs. Eligibility for this support varies depending on the type of insurance you have. Your Spark Therapeutics Generation Patient Services support team can help determine which resources are most appropriate and help you get connected.

Will my insurance cover LUXTURNA® (voretigene neparvovec-rzyl)?

Understanding whether you will receive insurance approval for LUXTURNA may involve several steps. With your consent, Spark Therapeutics Generation Patient Services will contact your insurance company and request information on coverage and the approval process. Once a prescribing physician at an ocular gene therapy treatment center has determined that you are eligible for treatment with LUXTURNA, they will begin the request for treatment approval, known as prior authorization. Your Generation Patient Services team will be there to set expectations about the process and will inform you of any updates related to coverage and prior authorization along the way.

Is LUXTURNA right for me? How do I get started on LUXTURNA?

LUXTURNA is a prescription gene therapy product used for the treatment of people with an 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. If you haven’t had genetic testing and you would like to find a specialist who can help with genetic testing and provide more information on inherited retinal diseases, please visit LUXTURNA.com.

Talk to your healthcare professional to see if LUXTURNA could be right for you. You should always rely on the direction of your healthcare team for treatment and care.

How do I learn more about LUXTURNA?

To learn more about LUXTURNA, please speak with your healthcare professional or visit LUXTURNA.com.

Do I have to enroll with Spark Therapeutics Generation Patient Services to obtain treatment?

Participation in Spark Therapeutics Generation Patient Services is voluntary and is not required to get treatment with LUXTURNA. 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.

LUXTURNA is a prescription gene therapy product. What is gene therapy and how does it work?

To learn more about gene therapy and LUXTURNA, please ask your healthcare professional or visit LUXTURNA.com.

After my healthcare professional and I have decided that treatment with LUXTURNA is appropriate for me, how long does the process take to get treatment?

Not every treatment journey is the same and can depend on a number of things, such as your insurance carrier and ocular gene therapy treatment center scheduling. To guide you throughout this process, your Spark Therapeutics Generation Patient Services team can help answer your questions and connect you to the proper resources.

Please keep in mind, 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.

IMPORTANT SAFETY INFORMATION FOR LUXTURNA (voretigene neparvovec-rzyl)

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.

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.

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