Table 3.
Adverse events observed in the JEWELFISH population
| Previous treatment | All patients (N = 173)b |
||||
|---|---|---|---|---|---|
| Olesoxime (n = 70) |
MOONFISH studya (n = 13) |
Nusinersen (n = 76) |
Onasemnogene abeparvovec (n = 14) |
||
| Patients with at least one AE, n (%) | 63 (90) | 12 (92) | 71 (93) | 13 (93) | 159 (92) |
| Total number of AEs | 357 | 66 | 450 | 50 | 923 |
| Total number of deaths | 0 | 0 | 0 | 0 | 0 |
| Total number of patients with at least one, n (%) | |||||
| SAE | 8 (11) | 3 (23) | 11 (15) | 2 (14) | 24 (14) |
| Treatment-related SAE | 1 (1)c | 0 | 0 | 0 | 1 (1)c |
| SAE leading to dose modification/interruption | 2 (3) | 1 (8) | 3 (4) | 0 | 6 (4) |
| AE leading to withdrawal from treatment | 0 | 0 | 1 (1)d | 0 | 1 (1)d |
| Treatment-related AE | 8 (11) | 6 (46) | 19 (25) | 0 | 33 (19) |
| Related AE leading to withdrawal from treatment | 0 | 0 | 0 | 0 | 0 |
| Most common AEse n (number of patients [%]) | |||||
| Upper respiratory tract infection | 14 (20) | 0 | 14 (18) | 2 (14) | 30 (17) |
| Pyrexia | 8 (11) | 1 (8) | 17 (22) | 4 (29) | 30 (17) |
| Headache | 12 (17) | 1 (8) | 15 (20) | 0 | 28 (16) |
| Nausea | 5 (7) | 0 | 14 (18) | 1 (7) | 20 (12) |
| Diarrhea | 4 (6) | 0 | 14 (18) | 1 (7) | 19 (11) |
| Nasopharyngitis | 6 (9) | 2 (15) | 7 (9) | 2 (14) | 17 (10) |
| Vomiting | 6 (9) | 1 (8) | 5 (7) | 2 (14) | 14 (8) |
| Most common SAEsf n (number of patients [%]) | |||||
| Pneumonia | 1 (1) | 0 | 2 (3) | 1 (7) | 4 (2) |
| Lower respiratory tract infection | 2 (3) | 0 | 1 (1) | 0 | 3 (2) |
| Upper respiratory tract infection | 0 | 0 | 3 (4) | 0 | 3 (2) |
| Respiratory failure | 0 | 0 | 3 (4) | 0 | 3 (2) |
As follow-up duration is different between groups, the overall rate of AEs and SAEs cannot be compared. Multiple occurrences of the same AE in one individual are counted only once except for the “Total number of AEs” row, for which multiple occurrences of the same AE are counted separately
AE adverse event, SAE serious AE
aThree patients who were previously enrolled in the MOONFISH study (NCT02240355) received placebo treatment and were never switched to RG7800
bOne patient withdrew from the study at baseline and therefore 173 patients received risdiplam
cAn SAE of supraventricular tachycardia was considered related to risdiplam treatment by the Investigator (in the context of hypoxia) and resolved with ongoing treatment with risdiplam
dIrritable bowel syndrome and panic attack, which were unrelated to risdiplam, led to the withdrawal of one patient who was previously treated with nusinersen
eAEs reported in ≥ 14 patients
fSAEs reported in ≥ 3 patients. Includes AEs with onset from first dose of study drug up to the clinical cutoff date (29 January 2021)