[8] It initially appeared to have an acceptable safety profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity noted in clinical trials.

[10] In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older.

[14] In September 2024, Pfizer announced a voluntary withdrawal of voxelotor from all global markets due to concerns regarding the potential for severe safety events, including fatalities.

The CHMP also cited observational studies which found a higher rate of painful vaso-occlusive crises during treatment with voxelotor than the subjects had before starting the medicine.

[24] In September 2024, Pfizer announced a voluntary withdrawal of voxelotor from all global markets due to concerns regarding the potential for severe safety events, including fatalities.