Resources

Brochures and forms

Patient enrollment form

Use this form to enroll in Spark Therapeutics Generation Patient ServicesSM (not for patients at ocular gene therapy treatment centers) and to schedule consultations.

Enrollment is not required to receive LUXTURNA treatment.

Download PDF

Patient and caregiver insurance guide

Navigate health insurance options and treatment support with this helpful guide.

Download PDF

Work/school time-off request information

Guidance on how to request time off from work or school for a medical leave of absence

Download PDF for workDownload PDF for school

Mentee brochure

Learn how the Mentor Program connects people who are considering treatment with people who have already been treated with LUXTURNA® (voretigene neparvovec-rzyl).

Download PDF

Patient – frequently asked questions

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.

Brochures and forms

Referring physician guide

This guide provides support for your practice with the patient consultation process for LUXTURNA® (voretigene neparvovec-rzyl).

Download PDF

Enrollment form

Use this form to enroll your patients in the Spark Therapeutics Generation Patient ServicesSM program. It also allows Generation Patient Services to look into the patient’s insurance and next steps for treatment. Make sure to have the patient complete and include the patient authorization.

Download PDF

Statement of Medical Necessity

This form is completed by the ocular gene therapy treatment center when a patient has had a consultation and meets the eligibility requirements for LUXTURNA. This form allows Spark® Therapeutics to start a benefits investigation. It also enrolls a patient in Spark Therapeutics Generation Patient Services if the patient is not yet enrolled. Make sure to have the patient complete and include the patient authorization.

Download PDF

LUXTURNA (voretigene neparvovec-rzyl) reimbursement guide for ocular gene therapy treatment centers

This guide provides information to your ocular gene therapy treatment center to help facilitate the LUXTURNA reimbursement process.

Download PDF

Mentee brochure

Learn more about eligibility and how to get your patients started with a mentor.

Download PDF

HCP– frequently asked questions

What is Spark Therapeutics Generation Patient Services? What assistance can they provide my patient?

Generation Patient Services is a program sponsored by Spark® Therapeutics for patients who have confirmed biallelic RPE65 gene mutations and may be candidates for treatment with LUXTURNA® (voretigene neparvovec-rzyl). Our goal is to provide your patients with personalized assistance throughout the patient’s journey.

The Spark Therapeutics Generation Patient Services support team will:

  • Contact your patient within 1 to 2 business days after receiving the completed enrollment form
  • Provide your patient with a caring support team, from confirmed diagnosis through postsurgery follow-up
  • Ensure a single point of contact to help your patient navigate insurance coverage and get connected with financial assistance resources as needed
  • Help to coordinate your patient’s ocular gene therapy treatment center visits
  • Answer nonmedical questions your patient 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 your patients are not required to enroll in order to receive treatment with LUXTURNA.

Why do my patients need to use an ocular gene therapy treatment center?

Spark Therapeutics carefully selected ocular gene therapy treatment centers to support patient care. Each ocular gene therapy treatment center was selected based on specific criteria and is staffed with retinal specialists, nurses, and other healthcare professionals who have experience treating patients with inherited retinal diseases and have been educated on administering LUXTURNA.

Some of the criteria for ocular gene therapy treatment center selection include:

  • Specialized scientific or clinical experience relevant to the administration of LUXTURNA
  • The ability to properly receive, store, and prepare LUXTURNA
  • A commitment to pharmacy and surgical education
How is an ocular gene therapy treatment center selected for my patient?

If you decide that LUXTURNA is right for your patient, several factors will be considered in helping the patient determine their ocular gene therapy treatment center: your patient’s insurance coverage, in an effort to minimize out-of-pocket costs; your patient’s and the ocular gene therapy treatment center's schedules; and your and your patient's preference, among other considerations.

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

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

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

Getting insurance approval for LUXTURNA may involve several steps. With your patient’s consent, Spark Therapeutics Generation Patient Services will contact your patient’s 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 a patient is eligible for treatment with LUXTURNA, they will begin the request for treatment approval, known as prior authorization. The Generation Patient Services team will be there to set expectations about the process and will inform your patient of any updates related to coverage and prior authorization along the way.

How can I determine if LUXTURNA is right for my patient?

LUXTURNA is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation–associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

To learn more about genetic testing, click here.

Does my patient need to enroll in 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. Your patient 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 their ability to get treatment or the nature of their treatment or care. Generation Patient Services does not provide medical advice.

How do I get my patient started on LUXTURNA?

Once a completed enrollment form and patient authorization have been submitted, a member of the Generation Patient Services team will reach out to your patient and schedule a consultation at an ocular gene therapy treatment center if they have confirmed biallelic RPE65 mutation–associated retinal dystrophy. A prescribing physician at the ocular gene therapy treatment center will determine if your patient has viable retinal cells and their eligibility for LUXTURNA. If it’s been determined that your patient is eligible, LUXTURNA will be ordered directly from the ocular gene therapy treatment center.

Where are the ocular gene therapy treatment centers that are administering LUXTURNA?

See below for a list of centers:
Alkek Eye Center, Baylor College of Medicine Jamail Specialty Care Center – Houston, TX
Bascom Palmer Eye Institute, University of Miami Health System – Miami, FL
Boston Children’s Hospital – Boston, MA
Children’s Hospital of Philadelphia – Philadelphia, PA
Cincinnati Children’s Hospital – Cincinnati, OH
Kellogg Eye Center, University of Michigan – Ann Arbor, MI
Massachusetts Eye and Ear – Boston, MA
OHSU Casey Eye Institute – Portland, OR
Scheie Eye Institute Penn Presbyterian – Philadelphia, PA
UAB Callahan Eye – Birmingham, AL
University of Iowa Hospitals and Clinics – Iowa City, IA
UPMC Eye Center - Pittsburgh, PA
University of Rochester Medical Center, Flaum Eye Institute – Rochester, NY
The Vision Center at Children’s Hospital Los Angeles – Los Angeles, CA

Ocular gene therapy treatment centers

Alkek Eye Center, Baylor College of Medicine Jamail Specialty Care Center

Houston, TX 77030

Bascom Palmer Eye Institute, University of Miami Health System

Miami, FL 33136

Boston Children’s Hospital

Boston, MA 02115

Children’s Hospital of Philadelphia

Philadelphia, PA 19104

Cincinnati Children’s Hospital

Cincinnati, OH 45229

Kellogg Eye Center, University of Michigan

Ann Arbor, MI 48105

Massachusetts Eye and Ear

Boston, MA 02114

OHSU Casey Eye Institute

Portland, OR 97239

Scheie Eye Institute Penn Presbyterian

Philadelphia, PA 19104

UAB Callahan Eye

Birmingham, AL 35233

University of Iowa Hospitals and Clinics

Iowa City, IA 52242

UPMC Eye Center

Pittsburgh, PA 15213

University of Rochester Medical Center, Flaum Eye Institute

Rochester, NY 14642

The Vision Center at Children’s Hospital Los Angeles

Los Angeles, CA 90027

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.

IMPORTANT SAFETY INFORMATION FOR LUXTURNA (voretigene neparvovec-rzyl)

Warnings and Precautions

  • Endophthalmitis may occur following any intraocular surgical procedure or injection. Use proper aseptic injection technique when administering LUXTURNA, and monitor for and advise patients to report any signs or symptoms of infection or inflammation to permit early treatment of any infection.
  • Permanent decline in visual acuity may occur following subretinal injection of LUXTURNA. Monitor patients for visual disturbances.
  • Retinal abnormalities may occur during or following the subretinal injection of LUXTURNA, including macular holes, foveal thinning, loss of foveal function, foveal dehiscence, chorioretinal atrophy, and retinal hemorrhage. Monitor and manage these retinal abnormalities appropriately. Do not administer LUXTURNA in the immediate vicinity of the fovea. Retinal abnormalities may occur during or following vitrectomy, including retinal tears, epiretinal membrane, or retinal detachment. Monitor patients during and following the injection to permit early treatment of these retinal abnormalities. Advise patients to report any signs or symptoms of retinal tears and/or detachment without delay.
  • Increased intraocular pressure may occur after subretinal injection of LUXTURNA. Monitor and manage intraocular pressure appropriately.
  • Expansion of intraocular air bubbles Instruct patients to avoid air travel, travel to high elevations or scuba diving until the air bubble formed following administration of LUXTURNA has completely dissipated from the eye. It may take one week or more following injection for the air bubble to dissipate. A change in altitude while the air bubble is still present can result in irreversible vision loss. Verify the dissipation of the air bubble through ophthalmic examination.
  • Cataract Subretinal injection of LUXTURNA, especially vitrectomy surgery, is associated with an increased incidence of cataract development and/or progression.

Adverse Reactions

  • In clinical studies, ocular adverse reactions occurred in 66% of study participants (57% of injected eyes), and may have been related to LUXTURNA, the subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products.
  • The most common adverse reactions (incidence ≥ 5% of study participants) were conjunctival hyperemia (22%), cataract (20%), increased intraocular pressure (15%), retinal tear (10%), dellen (thinning of the corneal stroma) (7%), macular hole (7%), subretinal deposits (7%), eye inflammation (5%), eye irritation (5%), eye pain (5%), and maculopathy (wrinkling on the surface of the macula) (5%).

Immunogenicity

Immune reactions and extra-ocular exposure to LUXTURNA in clinical studies were mild. No clinically significant cytotoxic T-cell response to either AAV2 or RPE65 has been observed. In clinical studies, the interval between the subretinal injections into the two eyes ranged from 7 to 14 days and 1.7 to 4.6 years. Study participants received systemic corticosteroids before and after subretinal injection of LUXTURNA to each eye, which may have decreased the potential immune reaction to either AAV2 or RPE65.

Pediatric Use

Treatment with LUXTURNA is not recommended for patients younger than 12 months of age, because the retinal cells are still undergoing cell proliferation, and LUXTURNA would potentially be diluted or lost during the cell proliferation. The safety and efficacy of LUXTURNA have been established in pediatric patients. There were no significant differences in safety between the different age subgroups.

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.

INDICATION

LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Patients must have viable retinal cells as determined by the treating physicians.

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