Table 4. Summary of Safety.
| Parameter, n (%) | Treatment Arm | |
|---|---|---|
| Ataluren N = 120 |
Placebo N = 118 |
|
| ≥1 Treatment emergent adverse event | 118 (98.3%) | 115 (97.5%) |
| ≥1 Treatment emergent serious adverse event | 45 (37.5%) | 48 (40.7%) |
| Adverse events by severity | ||
| Grade 1 (mild) | 18 (15.0%) | 20 (16.9%) |
| Grade 2 (moderate) | 81 (67.5%) | 65 (55.1%) |
| Grade 3 (severe) | 19 (15.8%) | 30 (25.4%) |
| Grade 4 (life-threatening) | 0 | 0 |
| Grade 4 (death) | 0 | 0 |
| Discontinued treatment due to adverse event | 8 (6.7%) | 3 (2.5%) |
| Treatment emergent adverse events leading to discontinued treatmenta | ||
| Congenital, familial and genetic disorders | 1 (0.8%) | 0 |
| Cystic fibrosis-related diabetes | 1 (0.8%) | 0 |
| Gastrointestinal disorders | 2 (1.7%) | 2 (1.7%) |
| Abdominal pain | 0 | 1 (0.8%) |
| Abdominal pain upper | 0 | 1 (0.8%) |
| Diarrhoea | 1 (0.8%) | 0 |
| Pancreatitis | 1 (0.8%) | 0 |
| General disorders and administration site conditions | 1 (0.8%) | 0 |
| Pyrexia | 1 (0.8%) | 0 |
| Infections and infestations | 1 (0.8%) | 0 |
| Urinary tract infection | 1 (0.8%) | 0 |
| Nervous system disorders | 0 | 1 (0.8%) |
| Headache | 0 | 1 (0.8%) |
| Renal and urinary disorders | 5 (4.2%) | 0 |
| Hypercreatininaemia | 2 (1.7%) | 0 |
| Nephrolithiasis | 1 (0.8%) | 0 |
| Renal failure | 2 (1.7%) | 0 |
| Respiratory, thoracic and mediastinal disorders | 1 (0.8%) | 1 (0.8%) |
| Pulmonary exacerbation | 1 (0.8%) | 0 |
| Haemoptysis | 0 | 1 (0.8%) |
| Skin and subcutaneous tissue disorders | 2 (1.7%) | 0 |
| Rash | 2 (1.7%) | 0 |
a
Adverse events leading to study drug discontinuation are displayed alphabetically by MedDRA System Organ Class and from highest to lowest incidence across both treatment arms within each System Organ Class. Patients may have had more than one adverse event.