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EFFICACYVisual function and functional vision

Actual patient and caregiver.

I couldn’t be happier for my little sister because she’s showing me a whole new perspective of her personality.

– Sister of Monroe, actual patient

Actual patient and caregiver.

Vision loss means functional loss1

Vision loss affects much more than the eye—a loss of visual function translates to a loss of functional vision.1

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Visual function

Structural and functional changes at the organ level1

For example, rod dysfunction results in night blindness2

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Functional vision

Vision-related quality of life, including skills, activities of daily living, and social participation1

For example, night blindness limits individuals’ ability to navigate in dimly lit environments, such as a crosswalk at night2

The primary endpoint integrated measurements of visual function1,3

The endpoint combines aspects of vision loss into a single measure that quantified what study participants experience on a daily basis—their functional vision.1,3

Visual functionAdditional clinical outcomes
Functional visionPrimary endpoint
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Full-field light sensitivity threshold (FST) testing

Measured light sensitivity of the entire visual field4-6

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Visual acuity (VA) testing

Measured sharpness or clarity of vision1,5-7

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Visual field (VF) testing

Goldmann kinetic perimetry measured in sum total degrees; Humphrey static perimetry measured macula threshold values4-6

Bilateral MLMT

The MLMT measured participants’ ability to navigate the course at different real-world levels of illumination, which depends on light sensitivity, VA, and VF.3,6

Study description:

The randomized, controlled, open-label, phase 3 trial included 31 patients, aged 4-44 years. Patients were eligible to participate if they were 3 years of age or older, had a genetic diagnosis of biallelic RPE65 mutations, sufficient viable retinal cells, VA of 20/60 or worse in both eyes and/or VF less than 20 degrees, and were able to perform the MLMT within the luminance range evaluated, but unable to pass at the lowest light level (darkest). Patients were randomized into control (n=10) and intervention (n=21) groups. Prior to intervention, 2 patients discontinued the trial, resulting in an intent-to-treat (ITT) population (n=31) and a modified intent-to-treat population (mITT) (n=29). The intervention group received the first injection of LUXTURNA (first eye) and within 6-18 days later received the second injection (second eye). Placebo injections were not given to patients in the control group. Patients receiving LUXTURNA followed a perioperative immunomodulatory regimen to reduce risks related to immune response. Efficacy endpoints were assessed 1 year after the second injection. At 1 year, the control group crossed over and followed the same treatment schedule as the intervention group.4,5

An impact on visual function results in functional improvement1,3,6

At Year 1, LUXTURNA® demonstrated statistically significant improvements in FST and VF and a nonstatistically significant improvement in VA4

>100-fold improvement in light sensitivity4,5

A statistically significant difference in FST monocular averaged over both eyes of -2.11 log10 (cd.s/m2) (95% CI: -3.19, -1.04) between groups4,5*†

8-letter improvement in VA4,5

A nonstatistically significant difference in VA monocular averaged over both eyes of -0.16 LogMAR (95% CI: -0.41, 0.08) in the intervention group, compared with 0.01 LogMAR in the control group4,5*‡

Nearly doubled VF in sum total degrees4,5§

A statistically significant difference of 378.7 sum total degrees between groups (95% CI: 145.5, 612.0)4,5*||

Increased VF by nearly 8 dB4,5

A statistically significant difference in macula sensitivity thresholds of 7.9 dB between groups (95% CI: 3.5, 12.2)4,5*

dB = decibels.
*ITT population: intervention (n=21) vs control (n=10).4
§For the intervention group alone.4
||Post hoc P=0.0059, Goldmann test.4
Post hoc P=0.0005, Humphrey test.4

Do you have a patient with biallelic RPE65 gene mutations who is ready for LUXTURNA?

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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%).


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.


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.


1. Colenbrander A. Visual standards: aspects and ranges of vision loss with emphasis on population surveys. Report prepared for the International Council of Ophthalmology, 29th International Congress of Ophthalmology; Sydney, Australia: April 20, 2002. Accessed January 21, 2022. 2. National Eye Institute, National Institutes of Health. Facts about retinitis pigmentosa. Updated July 10, 2019. Accessed January 21, 2022. 3. Chung DC, McCague S, Yu Z-F, et al. Novel mobility test to assess functional vision in patients with inherited retinal dystrophies. Clin Experiment Ophthalmol. 2018;46(3):247-259. 4. Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390(10097):849-860. doi:10.1016/S0140-6736(17)31868-8. 5. Data on file. CSR AAV2-hRPE65v2-301:57-59,414,416. December 13, 2016. Spark Therapeutics, Inc. Philadelphia, PA. 6. LUXTURNA [package insert]. Philadelphia, PA: Spark Therapeutics, Inc; 2022. 7. American Optometric Association. Visual acuity: what is 20/20 vision? Accessed January 21, 2022.