In 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for OXBRYTA tablets for the treatment of Sickle Cell Disease (SCD) in adults and children ages 12 years and older. In December 2021, the FDA expanded the approved use of OXBRYTA for the treatment of SCD in patients 4 years of age and older in the U.S. OXBRYTA was the first FDA approved drug to address underlying issue of the SCD by helping red blood cells (RBC) retain a normal shape which prevents cells from clumping/sticking together inside the blood vessels and assists with treatment of chronic anemia.
Pfizer announced on September 25, 2024, that they are voluntarily withdrawing all lots of OXBRYTA (voxelotor) and discontinuing all active clinical trials and expanded access programs worldwide. “Pfizer’s decision is based on the totality of clinical data that now indicates the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population. The data suggest an imbalance in vaso-occlusive crises and fatal events which require further assessment. Pfizer has notified regulatory authorities about these findings and its decision to voluntarily withdraw OXBRYTA from the market and discontinue distribution and clinical studies while further reviewing the available data and investigating the findings.” https://www.pfizer.com/news/press-release/press-release-detail/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease