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EFFICACY®The multi-luminance mobility test (MLMT) results

One of the best things I have ever seen after surgery was the stars. I never knew that they were little dots that twinkled.

– Misty, study participant

For illustrative purposes based on actual patient experience. Results will vary.

LUXTURNA improved functional vision based on a test that models activities of daily living1-3

Watch how LUXTURNA improved functional vision

At Year 1, participants treated with LUXTURNA demonstrated a statistically significant and clinically meaningful improvement in their ability to navigate at lower lux levels.1*

Efficacy outcomes1 LUXTURNA
n=21
Control
n=10
Difference
(LUXTURNA – control)
P value
MLMT score change for bilateral eyes,
median (min, max)
2 (0, 4) 0 (-1, 2) 2 0.001
MLMT score change for first-treated eye,
median (min, max)
2 (0, 4) 0 (-1, 1) 2 0.003

Using the MLMT to simulate everyday walking environments

The multi-luminance mobility test (MLMT) is a standardized, lab-based test in which participants were observed navigating a course with obstacles of varying height under different levels of illumination.1-3

BASELINE VISIT AT 1 LUX (FAIL)

1-YEAR VISIT AFTER LUXTURNA
ADMINISTRATION AT 1 LUX (PASS)

*The camera used automatically adjusts the level and temperature of light that it captures. Because of this feature, there may be slight variations in hue when filming at low light levels (eg, 1 lux). Both videos were filmed in low light environments. Note: This participant’s baseline passing level was 10 lux and 1-year passing level was 1 lux.

Lux levels were measured using a standardized and calibrated light meter.3

LUXTURNA’s demonstrated efficacy at year 11,2

55%of all participants had an MLMT score change of 2 or greater (16 out of 29)6*

0%

of participants (8 of 9) who subsequently crossed over passed at 1 lux 1 year posttreatment6

0%

of participants (13 of 20) in the intervention group successfully completed the MLMT at the lowest light level of 1 lux, while no participants in the control group passed at this level 1 year posttreatment2,6

*Intention-to-treat (ITT) population intervention (n=21) vs control (n=10).1

Improvements in functional vision have been sustained for 5 years—with observations ongoing.4,5 Follow-up will continue for up to 15 years postadministration.2,7

MLMT chart time-course over 5 years.

*Modified intention-to-treat (mITT) population.
Baseline (BL) represents last MLMT measurement before treatment with LUXTURNA.
Each box represents the middle 50% of distribution of MLMT score change. Vertical lines represent additional 25% above and below the box.
The horizontal bar within each box represents the median. The dot within each box represents the mean.

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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.

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. LUXTURNA [package insert]. Philadelphia, PA: Spark Therapeutics, Inc; 2017. 2. 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. 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. Data on file. MLMT time course over 4 years. September 2020. Spark Therapeutics, Inc. Philadelphia, PA. 5. Data on file. MT change score table. September 2020. Spark Therapeutics, Inc. Philadelphia, PA. 6. Data on file. CSR AAV2-hRPE65v2-301 Addendum 2016. January 12, 2017. Spark Therapeutics, Inc. Philadelphia, PA. 7. Safety and efficacy study in subjects with Leber congenital amaurosis. ClinicalTrials.gov. Updated December 13, 2021. Accessed January 25, 2022. https://clinicaltrials.gov/ct2/show/NCT00999609.