Table 4.
Safety summary
| Risdiplam arm 0–12 months (n = 120)a [13] | Risdiplam arm 12–24 months (n = 120)b | Placebo arm/placebo 0–12 months (n = 60)a [13] | Placebo arm/risdiplam 12–24 months (n = 60)b | ||
|---|---|---|---|---|---|
| Patients with at least one AE, n (%) | 111 (92.5) | 110 (91.7) | 55 (91.7) | 48 (80.0) | |
| Total number of AEs | 789 | 506 | 354 | 242 | |
| Total number of deaths | 0 | 0 | 0 | 0 | |
| Total number of patients with at least one, n (%) | |||||
| AE with fatal outcome | 0 | 0 | 0 | 0 | |
| SAE | 24 (20.0) | 25 (20.8) | 11 (18.3) | 3 (5.0) | |
| SAE leading to withdrawal from treatment | 0 | 0 | 0 | 0 | |
| SAE leading to dose modification/interruption | 4 (3.3) | 4 (3.3) | 2 (3.3) | 0 | |
| Treatment-related SAE | 0 | 0 | 0 | 0 | |
| AE leading to withdrawal from treatment | 0 | 0 | 0 | 0 | |
| AE leading to dose modification/interruption | 8 (6.7) | 7 (5.8) | 2 (3.3) | 2 (3.3) | |
| Treatment-related AE | 16 (13.3) | 5 (4.2) | 6 (10.0) | 3 (5.0) | |
| Related AE leading to dose modification/interruption | 0 | 0 | 0 | 0 | |
| Grade 3–5 AE | 21 (17.5) | 19 (15.8) | 8 (13.3) | 3 (5.0) | |
| Most common AEs, n (number of patients [%]) | |||||
| Upper respiratory tract infection | 38 (31.7) | 19 (15.8) | 18 (30.0) | 10 (16.7) | |
| Nasopharyngitis | 31 (25.8) | 26 (21.7) | 15 (25.0) | 6 (10.0) | |
| Pyrexia | 25 (20.8) | 16 (13.3) | 10 (16.7) | 6 (10.0) | |
| Headache | 24 (20.0) | 12 (10.0) | 10 (16.7) | 10 (16.7) | |
| Diarrhea | 20 (16.7) | 9 (7.5) | 5 (8.3) | 6 (10.0) | |
| Vomiting | 17 (14.2) | 14 (11.7) | 14 (23.3) | 8 (13.3) | |
| Cough | 17 (14.2) | 12 (10.0) | 12 (20.0) | 5 (8.3) | |
| Most common SAEs, n (number of patients [%]) | |||||
| Pneumonia | 9 (7.5) | 8 (6.7) | 1 (1.7) | 0 (0) | |
AE adverse event, SAE serious AE
aMonth 12 data cut-off: 6 Sep 2019
bMonth 24 data cut-off: 30 Sep 2020