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GETTING STARTEDThe patient journey

Actual patient.

I feel much more confident coaching night games now, seeing exactly what’s going on in the field.

– Shawn, patient who received LUXTURNA®

Actual patient.

Your patient’s treatment journey starts with you

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1FIND
2Test
3COnnect
4Follow up
1FIND

FIND

Identify appropriate patients for genetic testing, which may include patients diagnosed with Leber congenital amaurosis (LCA), retinitis pigmentosa (RP), or other inherited retinal diseases (IRDs), or who have symptoms consistent with the clinical manifestations of biallelic RPE65 mutation–associated retinal dystrophy.1-7

Ready to identify patients for genetic testing? Genetically test your patients

2Test

Test

  • Confirm your patient has biallelic RPE65 gene mutations by ordering a genetic test.
  • If needed, diagnostic specialists are available to provide more information on genetic testing.

Learn more about genetic testing

3COnnect

COnnect

Spark Therapeutics Generation Patient ServicesSM

  • Patients who have confirmed biallelic RPE65 gene mutations are eligible to enroll in Spark Therapeutics Generation Patient Services.*
  • Enrollment in Spark Therapeutics Generation Patient Services is voluntary and not required to receive treatment with LUXTURNA.

Download our enrollment form

Ocular gene therapy treatment center

  • Patients will travel to their designated treatment center to confirm retinal viability and then again for the administration of LUXTURNA.
  • Spark Therapeutics Generation Patient Services can help connect enrolled patients to a treatment center. Alternatively, you or your patients can contact a treatment center.

Explore treatment centers

*Participation in Spark Therapeutics Generation Patient ServicesSM is voluntary. 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 your patient’s ability to get treatment or the nature of your patient’s treatment or care. Generation Patient Services does not provide medical advice.

4Follow up

Follow up

After the procedure, you may work with the specialist at the treatment center to handle follow-up care, or you may resume follow-up care on your own.

LUXTURNA JOURNEYS

Monroe’s mother and sister talk about the LUXTURNA treatment process

LUXTURNA helped Shawn continue doing the things he loves

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IMPORTANT SAFETY INFORMATION FOR LUXTURNA

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, 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.

References:

1. Cideciyan AV. Leber congential amaurosis due to RPE65 mutations and its treatment gene therapy. Prog Retin Eye Res. 2010;29(5):398-427. doi:10.1016/j.preteyeres.2010.04.002. 2. Felius J, Thompson DA, Khan NW, et al. Clinical course and visual function in a family with mutations in the RPE65 gene. Arch Ophthalmol. 2002;120(1):55-61. doi:10.1001/archopht.120.1.55. 3. LUXTURNA [package insert]. Philadelphia, PA: Spark Therapeutics, Inc; 2017. 4. Morimura H, Fishman GA, Grover SA, Fulton AB, Berson EL, Dryja TP. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis. Proc Natl Acad Sci U S A. 1998;95(6):3088-3093. doi:10.1073/pnas.95.6.3088. 5. Chung DC, Bertelsen M, Lorenz B, et al. The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene. Am J Ophthalmol. 2019 Mar;199:58-70. doi: 10.1016/j.ajo.2018.09.024. 6. Weleber RG, Michaelides M, Trzupek KM, Stover NB, Stone EM. The phenotype of Severe Early Childhood Onset Retinal Dystrophy (SECORD) from mutation of RPE65 and differentiation from Leber congenital amaurosis. Invest Ophthalmol Vis Sci. 2011;52(1):292-302. doi:10.1167/iovs.10-6106. 7. Nash BM, Wright DC, Grigg JR, Bennetts B, Jamieson RV. Retinal dystrophies, genomic applications in diagnosis and prospects for therapy. Transl Pediatr. 2015;4(2):139-163. doi:10.3978/j.issn.2224-4336.2015.04.03.