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Frequently asked questions (FAQs)

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

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 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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