Pfizer has announced the worldwide discontinuation of Oxbryta (voxelotor), its sickle cell disease treatment, previously priced at $125,000 per year. This decision follows a review of clinical trial data that raised serious safety concerns, including increased risk of complications and fatalities among patients.
Oxbryta, which received U.S. approval in 2019, was the first treatment targeting the underlying cause of sickle cell disease by preventing red blood cells from assuming an abnormal sickle shape. It aimed to reduce life-threatening complications such as vaso-occlusive crises (VOC). Initially approved for adults and children 12 and older, its use was later expanded to younger patients, receiving approval in over 35 countries.
However, recent trial data involving 236 patients revealed alarming results: eight deaths occurred in patients treated with Oxbryta, compared to two in the placebo group, alongside an increased incidence of VOC. Due to these safety concerns, Pfizer has decided to cease all clinical trials and discontinue the global availability of Oxbryta.