The FDA has issued a warning to patients and healthcare professionals about the voluntary withdrawal of Oxbryta (voxelotor) from the market due to safety concerns. Pfizer, the manufacturer of Oxbryta, is ceasing distribution and discontinuing all clinical trials and access programs for the medication. This decision follows data from post marketing clinical trials and real-world studies that revealed a higher rate of vaso-occlusive crises (painful blockages in blood flow caused by sickled red blood cells) and more deaths in patients taking Oxbryta compared to those on placebo.
The FDA is currently conducting a safety review of this data, and further findings will be communicated if necessary. Oxbryta was initially approved in 2019 under the FDA’s accelerated approval pathway for the treatment of sickle cell disease in patients 12 years and older, with expanded approval in 2021 for younger patients aged 4 to 11. While the medication was expected to offer benefits to patients with this serious disease, the risks now outweigh the benefits.
Healthcare professionals are advised to stop prescribing Oxbryta, and patients should consult their doctors about alternative treatment options. The FDA highlights that there are other approved therapies available for sickle cell disease.