A recent clinical study analysis of Oxbryta (voxelotor), globally withdrawn in 2024, was submitted to the FDA with the intent of getting voxelotor back on the market.
Sickle cell disease (SCD) is a disorder in which red blood cells become “sickle”-shaped and can not move easily. As a result, blood vessels can be damaged and/or block blood flow throughout the body. This can cause a plethora of serious problems such as stroke, retinopathy, infections, and pain crises.
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with SCD, designed to increase hemoglobin’s affinity for oxygen and inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia. The treatment was granted accelerated approval for the treatment of SCD in adults and children ages 12 years and older by the US Food and Drug Administration (FDA) in 2019 and expanded for patients 4 years of age and older in 2021.
In September 2024, voxelotor was globally withdrawn from all markets and active voxelotor clinical trials and expanded access programs were discontinued. This decision was based on an imbalance in vaso-occlusive crises and fatal events observed at the time of withdrawal, risks that outweighed the overall benefit of treatment.
Since the withdrawal, final clinical trial, registry, and post-marketing safety data has been under review. Recently, the data was submitted to the FDA with the intent of getting voxelotor back on the market.
Plain language summaries of the two US registry studies (RETRO and PROSPECT) published in Blood Advances are in preparation. Results from these studies illustrate no new safety signals in patients prescribed voxelotor and expand on the effectiveness of the treatment in improving hemoglobin and hemolysis markers. Further data from randomized clinical trials of voxelotor (HOPE KIDS 2 and RESOLVE) are expected to be published in peer-reviewed medical journals this year as well.
To learn more about SCD and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/