Table 4.

TEAEs occurring in 10% or more of patients

Voxelotor/placebo, mg/dPooled placebo
5007007009001000All
Days28289090 d to 6 mo2828-90
N101267*641*14
Headache, n (%)4 (40)5 (42)1 (17)2 (29)4 (67)16 (39)8 (57)
Back pain, n (%)2 (20)3 (25)02 (29)2 (33)9 (22)2 (14)
Pain, n (%)1 (10)4 (33)02 (29)1 (17)8 (20)4 (29)
Pain in extremity, n (%)1 (10)1 (8)1 (17)2 (29)05 (12)0
Diarrhea, n (%)02 (17)02 (14)2 (33)6 (15)0
Cough, n (%)02 (17)2 (33)01 (17)5 (12)0
Rash, n (%)001 (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).