Table 3.
Summary of SPR1NT results and other SMA studies and cohortsa
| Onasemnogene abeparvovec | Nusinersen | ||||||
|---|---|---|---|---|---|---|---|
| Symptomatic patients | Presymptomatic children | Presymptomatic children | |||||
| PNCR7 | STR1VE-US13 | STR1VE-EU14 | SPR1NT two-copy cohort | SPR1NT three-copy cohort | NURTUREb two-copy cohort23 | NURTUREb three-copy cohort23 | |
| Intention-to-treat population, n | 23 | 22 | 32 | 14 | 15 | 15 | 10 |
| SMN2 copies | 2 | 2 | 2 | 2 | 3 | 2 | 3 |
| Median (range) age at diagnosis, days | N/A | 67 (56–126)c | 76 (26–156) | 8 (1–14) | 8 (2–26) | N/A | N/A |
| Median (range) age at infusion, days | N/A | 105 (15–177) | 123 (54–180) | 21 (8–34) | 32 (9–43) | 19 (8–41) | 23 (3–42) |
| Baseline median (range) CHOP INTEND | 32.5 (31–33)d | 33.5 (18–52) | 28.0 (14–55) | 48.5 (28–57) | N/A | 45.0 (25–60) | 53.5 (40–60) |
| Baseline median (range) CMAP amplitude, mVe | 0.3 (0.04–1.1) | N/A | N/A | 3.9 (2.1–6.1) | 4.1 (2.7–7.0) | 3.2 (1.1–9.7) | 4.0(0.2–7.0) |
| Sitting independently by 18 months, n (%)f | 0 | 14 (64) | 14 (44) | 14 (100) | N/A | N/A | N/A |
| Sitting independently by 24 months, n (%)f | 0 | N/A | N/A | N/A | 14 (93) | 15 (100) | 10 (100) |
| Standing independently by 18 months of age, n (%)f | 0 | 1 (5) | 1 (3) | 11 (79) | N/A | N/A | N/A |
| Standing independently by 24 months of age, n (%)f | 0 | N/A | N/A | N/A | 15 (100) | 9 (60) | 10 (100) |
| Walking independently by 18 months, n (%)f | 0 | 1 (5) | 1 (3) | 9 (64) | N/A | N/A | N/A |
| Walking independently by 24 months, n (%)f | 0 | N/A | N/A | N/A | 14 (93) | 9 (60) | 10 (100) |
| Alive without permanent ventilation at 18 months, n (%)f | 6 (26)g | 20 (91) | 31 (97) | 14 (100) | 15 (100) | 15 (100) | 10 (100) |
N/A, not available.
aThere are no published head-to-head studies of onasemnogene abeparvovec and nusinersen. Differences in trial design, including primary endpoints, how endpoints were measured, and eligibility criteria, make direct comparison of results from these studies infeasible. The PNCR measured CHOP INTEND, NURTURE measured WHO and HINE-2 criteria, and STR1VE-US and STR1VE-EU measured WHO criteria and CHOP INTEND.
bNURTURE results represent interim analysis at data cut of 29 March 2019. At the time of this analysis, the median age of the infants was 34.8 months (25.7–45.4)23.
cMedian (range) is reported as the interquartile range.
dValues indicate median (interquartile range) obtained for patients with symptom onset <3 months of age, a group that included seven patients with two SMN2 copies and one patient with three SMN2 copies.
eUlnar CMAP amplitude recorded from the abductor digiti minimi muscle at baseline for the PNCR study (n = 34 patients with SMA type 1; n = 23, two SMN2 copies and n = 9, three SMN2 copies) and peroneal CMAP amplitude recorded from the tibialis anterior muscle for the SPR1NT and NURTURE studies.
fMilestones were evaluated over different observation periods between studies, and included 18 months for STR1VE-US, STR1VE-EU, and the SPR1NT two-copy cohort, 24 months for the SPR1NT three-copy cohort, and a median follow-up time of 35 months for NURTURE.
gSurvival without permanent ventilation at 14 months.