LUXTURNA(voretigene neparvovec-rzyl)intraocular suspension forsubretinal injection(十)
at Year 1. The horizontal lines with arrows represent themagnitude of the score change and its direction. Arrows pointing towards the right represent improvement. The top section shows theresults of the 21 subjects in the treatment group. The bottom section shows the results of the 10 subjects in the control group.
Subjects in each group are chronologically organized by age, with the youngest subject at the top and the oldest subject at the bottom.
Analysis of white light FST testing showed statistically significant improvement from Baseline to Year 1 in theLUXTURNA treatment group compared to the control group. The change in visual acuity from Baseline to
Year 1 was not significantly different between the LUXTURNA and control groups.
Figure 7 shows the effect of LUXTURNA over the two-year period in the LUXTURNA treatment group, aswell as the effect in the control group after crossing over to receive subretinal injection of LUXTURNA. Amedian MLMT score change of two was observed for the LUXTURNA treatment group at Day 30, and thiseffect was sustained over the remaining follow-up visits throughout the two-year period. For the control group,a median MLMT score change of 0 was observed at all four follow-up visits during the first year. However,after crossing-over to receive subretinal injection of LUXTURNA, the subjects in the control group showed asimilar response to LUXTURNA as compared to the subjects in the LUXTURNA treatment group.
Figure 7. MLMT Time-Course over Two Years: Using Both Eyes
Note for Figure 7: Each box represents the middle 50% of distribution of MLMT score change. Vertical dotted lines representadditional 25% above and below the box. The horizontal bar within each box represents the median. The dot within each boxrepresents the mean. The solid line connects the mean MLMT score changes over visits for the treatment group, including five visitsduring the first year and one visit at Year 2 (marked as x365). The dotted line connects the mean MLMT score change over visits forthe control group, including five visits during the first year without receiving LUXTURNA, and four visits within the second year(marked as x30, x90, x180, and x365) after cross-over at Year 1 to receive LUXTURNA.
16 HOW SUPPLIED/STORAGE AND HANDLING
Each carton of LUXTURNA (NDC 71394 – 415-01) contains one single-dose vial of the LUXTURNA (NDC71394 – 065-01, 0.5 mL extractable volume) and two vials of Diluent (NDC 71394 – 716-01, 1.7 mLextractable volume in each vial). LUXTURNA contains 5 x 1012 vector genomes (vg) per mL, requires a 1:10dilution prior to administration.
Store LUXTURNA and Diluent frozen at ≤ -65 °C.
Following thaw of the vials, store at room temperature. Store diluted LUXTURNA at room temperature [SeeDosage and Administration 2.2].
LUXTURNA is an adeno-associated virus vector-based gene therapy. Follow universal biohazard precautionsfor handling.
17 PATIENT COUNSELING INFORMATION
Advise patients and/or their caregivers of the following risks.
• Endophthalmitis and other eye infectionsSerious infection can occur inside of the eye and may lead to blindness.
In such cases, there is an urgentneed for management without delay. Advise patients to call their healthcare provider if they experience newfloaters, eye pain, or any change in vision.
• Permanent decline in visual acuityPermanent decline in visual acuity may occur following subretinal injection of LUXTURNA.
Advise patientsto contact their healthcare p |
