TABLE 2.
Adverse events (AEs)#
| Parent Study 445-102 ¶ | Study 445-105 Week 144 interim analysis | |||||
|
Placebo
(n=201) Mean exposure 23.7 weeks |
ELX/TEZ/IVA
(n=202) Mean exposure 23.6 weeks |
ELX/TEZ/IVA
(n=506) Mean exposure 151.1 weeks |
||||
| Participants, n (%) | Events per 100 participant-years | Participants, n (%) | Events per 100 participant-years | Participants, n (%) | Events per 100 participant-years | |
| Any AE | 193 (96.0) | 1287.96 | 188 (93.1) | 1096.01 | 500 (98.8) | 586.55 |
| AEs by maximum severity | ||||||
| Mild | 53 (26.4) | NA | 67 (33.2) | NA | 83 (16.4) | NA |
| Moderate | 125 (62.2) | NA | 102 (50.5) | NA | 305 (60.3) | NA |
| Severe | 14 (7.0) | NA | 19 (9.4) | NA | 106 (20.9) | NA |
| Life threatening | 1 (0.5) | NA | 0 (0) | NA | 6 (1.2) | NA |
| Serious AEs | 42 (20.9) | 67.05 | 28 (13.9) | 36.93 | 154 (30.4) | 22.42 |
| AEs leading to treatment discontinuation | 0 (0) | 0 | 2 (1.0) | 2.99 | 14 (2.8) | 1.82 |
| AEs leading to death | 0 (0) | 0 | 0 (0) | 0 | 1 (0.2)+ | 0.06 |
| AEs leading to treatment interruption | 10 (5.0) | 14.01 | 19 (9.4) | 25.95 | 49 (9.7) | 6.20 |
| Most common AEs § | ||||||
| Infective pulmonary exacerbation of cystic fibrosis | 95 (47.3) | 181.13 | 44 (21.8) | 64.88 | 225 (44.5) | 37.40 |
| Cough | 77 (38.3) | 113.08 | 34 (16.8) | 38.93 | 212 (41.9) | 30.63 |
| Headache | 30 (14.9) | 42.03 | 35 (17.3) | 48.91 | 166 (32.8) | 18.29 |
| Oropharyngeal pain | 25 (12.4) | 26.02 | 20 (9.9) | 26.95 | 146 (28.9) | 16.85 |
| Nasopharyngitis | 26 (12.9) | 34.03 | 22 (10.9) | 29.95 | 135 (26.7) | 17.04 |
| Pyrexia | 19 (9.5) | 25.02 | 17 (8.4) | 17.97 | 134 (26.5) | 12.59 |
| Sputum increased | 39 (19.4) | 47.03 | 40 (19.8) | 46.91 | 120 (23.7) | 12.09 |
| Upper respiratory tract infection | 22 (10.9) | 26.02 | 24 (11.9) | 29.95 | 111 (21.9) | 11.65 |
| Nasal congestion | 15 (7.5) | 18.01 | 19 (9.4) | 20.96 | 106 (20.9) | 10.08 |
| Fatigue | 20 (10.0) | 22.02 | 9 (4.5) | 8.98 | 104 (20.6) | 11.21 |
| COVID-19 | 0 (0) | 0 | 0 (0) | 0 | 99 (19.6) | 7.70 |
| Nausea | 14 (7.0) | 17.01 | 16 (7.9) | 15.97 | 84 (16.6) | 7.77 |
| Diarrhoea | 14 (7.0) | 23.02 | 26 (12.9) | 31.94 | 80 (15.8) | 6.64 |
| Haemoptysis | 28 (13.9) | 42.03 | 11 (5.4) | 11.98 | 78 (15.4) | 12.03 |
| Vaccination complication | 0 (0) | 0 | 0 (0) | 0 | 78 (15.4) | 9.52 |
Data are presented as n (%). ELX/TEZ/IVA: elexacaftor/tezacaftor/ivacaftor; COVID-19: coronavirus disease 2019; NA: not applicable. #: a participant with multiple events within a category was counted only once in that category; ¶: the safety profile of ELX/TEZ/IVA was based on the 24-week, placebo-controlled, F508del–minimal function parent study; +: there was one death in the 144-week interim analysis of Study 445-105 which was due to accidental oxycodone toxicity and was not considered to be related to study drug; §: the most common AEs that occurred in ≥15% of participants in Study 445-102 or the interim analysis of Study 445-105 (listing is according to the preferred term; Medical Dictionary for Regulatory Activities version 24.1 (www.meddra.org)).