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

Visual function

Structural and functional changes at the organ level¹

For example, rod dysfunction results in night blindness²

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 function^1,3

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

Full-field light sensitivity threshold (FST) testing

Measured light sensitivity of the entire visual field^4-6

Visual acuity (VA) testing

Measured sharpness or clarity of vision^1,5-7

Visual field (VF) testing

Goldmann kinetic perimetry measured in sum total degrees; Humphrey static perimetry measured macula threshold values^4-6

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 improvement^1,3,6

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

>100-fold improvement in light sensitivity^4,5

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 groups^4,5*†

8-letter improvement in VA^4,5

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 group^4,5*‡

Nearly doubled VF in sum total degrees^4,5§

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 dB^4,5

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
^†P=0.0004.⁴
^‡P=0.17.⁴
^§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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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, 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.

EFFICACYVisual function and functional vision

Vision loss means functional loss¹

Visual function

Functional vision

The primary endpoint integrated measurements of visual function^1,3

Full-field light sensitivity threshold (FST) testing

Visual acuity (VA) testing

Visual field (VF) testing

Bilateral MLMT^®

An impact on visual function results in functional improvement^1,3,6

>100-fold improvement in light sensitivity^4,5

8-letter improvement in VA^4,5

Nearly doubled VF in sum total degrees^4,5§

Increased VF by nearly 8 dB^4,5

Stay informed by registering for updates

IMPORTANT SAFETY INFORMATION FOR LUXTURNA^®

Warnings and Precautions

Adverse Reactions

Immunogenicity

Pediatric Use

INDICATION

EFFICACYVisual function and functional vision

Vision loss means functional loss1

Visual function

Functional vision

The primary endpoint integrated measurements of visual function1,3

Full-field light sensitivity threshold (FST) testing

Visual acuity (VA) testing

Visual field (VF) testing

Bilateral MLMT®

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

>100-fold improvement in light sensitivity4,5

8-letter improvement in VA4,5

Nearly doubled VF in sum total degrees4,5§

Increased VF by nearly 8 dB4,5

Stay informed by registering for updates

IMPORTANT SAFETY INFORMATION FOR LUXTURNA®

Warnings and Precautions

Adverse Reactions

Immunogenicity

Pediatric Use

INDICATION

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Vision loss means functional loss¹

The primary endpoint integrated measurements of visual function^1,3

Bilateral MLMT^®

An impact on visual function results in functional improvement^1,3,6

>100-fold improvement in light sensitivity^4,5

8-letter improvement in VA^4,5

Nearly doubled VF in sum total degrees^4,5§

Increased VF by nearly 8 dB^4,5

IMPORTANT SAFETY INFORMATION FOR LUXTURNA^®

Stay informed by registering for updates about LUXTURNA^®