TEAEs occurring in 10% or more of patients
| . | Voxelotor/placebo, mg/d . | Pooled placebo . | |||||
|---|---|---|---|---|---|---|---|
| 500 . | 700 . | 700 . | 900 . | 1000 . | All . | ||
| Days | 28 | 28 | 90 | 90 d to 6 mo | 28 | 28-90 | |
| N | 10 | 12 | 6 | 7* | 6 | 41* | 14 |
| Headache, n (%) | 4 (40) | 5 (42) | 1 (17) | 2 (29) | 4 (67) | 16 (39) | 8 (57) |
| Back pain, n (%) | 2 (20) | 3 (25) | 0 | 2 (29) | 2 (33) | 9 (22) | 2 (14) |
| Pain, n (%) | 1 (10) | 4 (33) | 0 | 2 (29) | 1 (17) | 8 (20) | 4 (29) |
| Pain in extremity, n (%) | 1 (10) | 1 (8) | 1 (17) | 2 (29) | 0 | 5 (12) | 0 |
| Diarrhea,† n (%) | 0 | 2 (17) | 0 | 2 (14) | 2 (33) | 6 (15) | 0 |
| Cough, n (%) | 0 | 2 (17) | 2 (33) | 0 | 1 (17) | 5 (12) | 0 |
| Rash,‡ n (%) | 0 | 0 | 1 (17) | 1 (14) | 3 (50) | 5 (12) | 1 (7) |
| Sickle cell anemia with crisis,§ n (%) | 2 (20) | 2 (17) | 2 (33) | 2 (29) | 1 (17) | 9 (22) | 1 (7) |
| . | Voxelotor/placebo, mg/d . | Pooled placebo . | |||||
|---|---|---|---|---|---|---|---|
| 500 . | 700 . | 700 . | 900 . | 1000 . | All . | ||
| Days | 28 | 28 | 90 | 90 d to 6 mo | 28 | 28-90 | |
| N | 10 | 12 | 6 | 7* | 6 | 41* | 14 |
| Headache, n (%) | 4 (40) | 5 (42) | 1 (17) | 2 (29) | 4 (67) | 16 (39) | 8 (57) |
| Back pain, n (%) | 2 (20) | 3 (25) | 0 | 2 (29) | 2 (33) | 9 (22) | 2 (14) |
| Pain, n (%) | 1 (10) | 4 (33) | 0 | 2 (29) | 1 (17) | 8 (20) | 4 (29) |
| Pain in extremity, n (%) | 1 (10) | 1 (8) | 1 (17) | 2 (29) | 0 | 5 (12) | 0 |
| Diarrhea,† n (%) | 0 | 2 (17) | 0 | 2 (14) | 2 (33) | 6 (15) | 0 |
| Cough, n (%) | 0 | 2 (17) | 2 (33) | 0 | 1 (17) | 5 (12) | 0 |
| Rash,‡ n (%) | 0 | 0 | 1 (17) | 1 (14) | 3 (50) | 5 (12) | 1 (7) |
| Sickle cell anemia with crisis,§ n (%) | 2 (20) | 2 (17) | 2 (33) | 2 (29) | 1 (17) | 9 (22) | 1 (7) |
Includes 1 patient who received placebo in GBT440-001 and transitioned to voxelotor 900 mg in the extension study.
Grade 1; all resolved with continued dosing.
Two patients had treatment-related rashes (preferred terms of rash and rash papular): 1 in the voxelotor 1000-mg group and 1 in the voxelotor 900-mg group. Other TEAEs of rash were not consistent with drug rashes.
Also referred to as VOC. Events occurred off-treatment (during posttreatment follow-up; n = 9) or after a dose hold/dose reduction (voxelotor 900-mg group; n = 1).