LUXTURNA(voretigene neparvovec-rzyl)intraocular suspension forsubretinal injection(九)
luding 64% pediatric subjects (n=20, age from 4 to 17 years) and 36% adults (n=11). The 31 randomized
subjects included 13 males and 18 females. Sixty-eight percent (68%) of the subjects were White, 16% wereAsian, 10% were American Indian or Alaska Native, and 6% were Black or African-American.
Bilateralsubretinal injections of LUXTURNA were administered sequentially in two separate surgical procedures withan interval of 6 to 18 days.
The efficacy of LUXTURNA was established on the basis of multi-luminance mobility testing (MLMT) scorechange from Baseline to Year 1. The MLMT was designed to measure changes in functional vision, as assessedby the ability of a subject to navigate a course accurately and at a reasonable pace at different levels of
environmental illumination. The MLMT was assessed using both eyes and each eye separately at one or more ofseven levels of illumination, ranging from 400 lux (corresponding to a brightly lit office) to 1 lux(corresponding to a moonless summer night). Each light level was assigned a score code ranging from 0 to 6. A
higher score indicated that a subject was able to pass the MLMT at a lower light level. A score of -1 wasassigned to subjects who could not pass MLMT at a light level of 400 lux. The MLMT of each subject wasvideotaped and assessed by independent graders. The MLMT score was determined by the lowest light level at
which the subject was able to pass the MLMT.
The MLMT score change was defined as the difference betweenthe score at Baseline and the score at Year 1. A positive MLMT score change from Baseline to Year 1 visitindicated that the subject was able to complete the MLMT at a lower light level.
Additional clinical outcomes were also eva luated, including full-field light sensitivity threshold (FST) testing,visual acuity, and visual fields.
Table 2 summarizes the median MLMT score change from Baseline to Year 1 in the LUXTURNA treatmentgroup as compared to the control group. A median MLMT score of 2 or greater was observed in theLUXTURNA treatment group, while a median MLMT score change of 0 was observed in the control group,when using both eyes or the first-treated eye. An MLMT score change of two or greater is considered aclinically meaningful benefit in functional vision.
Table 2. Efficacy Results of Study 2 at Year 1, Compared to Baseline
Efficacy Outcomes LUXTURNA
n=21
Control
n=10
Difference
(LUXTURNA
minus Control)
p-
value
MLMT score change for bilateral eyes, median (min, max)
MLMT score change for first-treated eye, median (min, max)
2 (0, 4)
2 (0, 4)
0 (-1, 2)
0 (-1, 1)
2
2
0.001
0.003
Table 3 shows the number and percentage of subjects with different magnitudes of MLMT score change usingboth eyes at Year 1. Eleven of the 21 (52%) subjects in the LUXTURNA treatment group had an MLMT scorechange of two or greater, while one of the ten (10%) subjects in the control group had an MLMT score changeof two.
Table 3. Magnitude of MLMT Score Change Using Both Eyes at Year 1 (Study 2)
Score Change LUXTURNA
n=21
Control
n=10
-1 0 3 (30%)
0 2 (10%) 3 (30%)
1 8 (38%) 3 (30%)
2 5 (24%) 1 (10%)
3 5 (24%) 0
4 1 (4%) 0
Figure 6 shows MLMT performance of individual subjects using both eyes at Baseline and at Year 1.
Figure 6. MLMT Score Using Both Eyes at Baseline and One Year for Individual Subjects
Note for Figure 6: *subjects who were withdrawn or discontinued. The open circles are the baseline scores. The closed circles are the
Year 1 scores. The numbers next to the solid circle represent score change |
