Table 4.
Treatment-Emergent Adverse Events
| Pivotal patients with BBS and Alstrӧm syndrome (SAS) (N=38) | Pivotal patients with BBS (n=32) | |
|---|---|---|
| Overall treatment-emergent AEs | 38 (100) | 32 (100) |
| Treatment-emergent AEs occurring in ≥10% of patients | ||
| Skin hyperpigmentation | 23 (60·5) | 18 (56·3) |
| Injection site erythema | 18 (47·7) | 16 (50·0) |
| Injection site pruritus | 13 (34·2) | 11 (34·4) |
| Nausea | 13 (34·2) | 11 (34·4) |
| Injection site bruising | 11 (28·9) | 11 (34·4) |
| Injection site pain | 11 (28·9) | 10 (31·3) |
| Vomiting | 10 (26·3) | 9 (28·1) |
| Injection site induration | 9 (23·7) | 8 (25·0) |
| Diarrhea | 7 (18·4) | 7 (21·9) |
| Headache | 10 (26·3) | 9 (28·1) |
| Back pain | 4 (10·5) | 2 (6·3) |
| Cough | 4 (10·5) | 2 (6·3) |
| HDL cholesterol decrease | 4 (10·5) | 3 (9·4) |
| Injection site edema | 4 (10·5) | 4 (12·5) |
| Melanocytic nevus | 4 (10·5) | 4 (12·5) |
| Nasopharyngitis | 4 (10·5) | 3 (9·4) |
| Spontaneous penile erection | 4 (10·5) | 4 (12·5) |
| Abdominal pain | 4 (10·5) | 2 (6·3) |
| Treatment-related AEs | 37 (97·4) | 32 (100) |
| Serious AEs | 2 (5·3) | 2 (6·3) |
| Serious treatment-related AEs | 1 (2·6)a | 1 (3·1)a |
| AEs leading to study drug withdrawal | 5 (13·2) | 3 (9·4) |
| AEs leading to death | 0 | 0 |
Data are n (%) unless otherwise specified. AE, adverse event; BBS, Bardet-Biedl syndrome; HDL, high-density lipoprotein; SAS, safety analysis set.
a
One patient with BBS experienced an anaphylactic reaction to study drug (placebo).