Table 2.
Overall summary of TEAEs (safety population)
| Patients, n (%) | Cipa + mig group (N = 85)a |
Switch group (N = 37)b |
Total patients treated with cipa + mig (N = 122) |
|---|---|---|---|
| TEAE | 84 (98.8) | 36 (97.3) | 120 (98.4) |
| TEAE leading to study drug discontinuation | 3 (3.5) | 2 (5.4) | 5 (4.1) |
| Treatment-related TEAE | 37 (43.5) | 15 (40.5) | 52 (42.6) |
| Treatment-related TEAE leading to study drug discontinuation | 3 (3.5)c | 2 (5.4)d | 5 (4.1) |
| TESAE | 14 (16.5) | 6 (16.2) | 20 (16.4) |
| TESAE leading to study drug discontinuation | 1 (1.2) | 2 (5.4) | 3 (2.5) |
| Treatment-related TESAE | 1 (1.2) | 2 (5.4) | 3 (2.5) |
| Treatment-related TESAE leading to study drug discontinuation | 1 (1.2) | 2 (5.4) | 3 (2.5) |
| TESAE leading to death | 0 | 0 | 0 |
| IAR | 27 (31.8) | 10 (27.0) | 37 (30.3) |
| Treatment-related TEAE by preferred term occurring in ≥ 2 patients, n (%) | |||
| Headache | 11 (12.9) | 4 (10.8) | 15 (12.3) |
| Diarrhea | 7 (8.2) | 2 (5.4) | 9 (7.4) |
| Pyrexia | 6 (7.1) | 1 (2.7) | 7 (5.7) |
| Fatigue | 5 (5.9) | 2 (5.4) | 7 (5.7) |
| Nausea | 5 (5.9) | 2 (5.4) | 7 (5.7) |
| Dizziness | 4 (4.7) | 0 (0.0) | 4 (3.3) |
| Pruritus | 3 (3.5) | 1 (2.7) | 4 (3.3) |
| Urticaria | 2 (2.4) | 3 (8.1) | 5 (4.1) |
| Somnolence | 1 (1.2) | 2 (5.4) | 3 (2.5) |
| Abdominal pain upper | 2 (2.4) | 1 (2.7) | 3 (2.5) |
| Abdominal distension | 3 (3.5) | 0 (0.0) | 3 (2.5) |
| Abdominal pain | 0 (0.0) | 2 (5.4) | 2 (1.6) |
A TEAE was defined as any adverse event that started on or after the first dose of study drug. Any AE that occurred after 30 days from last dose of study drug in PROPEL and before the first dose of study drug in the OLE was not counted as treatment emergent. A treatment-related TEAE was defined as TEAE with a definite, probable, or possible relationship to study drug as judged by the investigator
AE adverse event; cipa + mig cipaglucosidase alfa + miglustat; IAR infusion-associated reaction; TEAE treatment-emergent adverse event; TESAE treatment-emergent serious adverse event
aIncludes data from patients treated with cipa + mig in PROPEL who may or may not have continued cipa + mig in the OLE, including data from both PROPEL and the OLE
bIncludes data from the OLE only
cTwo patients discontinued treatment during PROPEL due to anaphylactoid reaction and chills, respectively, and one patient discontinued treatment during the OLE due to urticaria
dTwo patients discontinued from the OLE due to urticaria and hypotension, and anaphylaxis, respectively