Voxelotor for Sickle Cell Disease Wins FDA Nod for Patients as Young as 4

Voxelotor has a novel mechanism of action. The first-in-class therapy regulates the affinity of hemoglobin for oxygen, resulting in a decrease in the concentration of deoxygenated sickle hemoglobin (HbS), which forms polymers. It’s hypothesized that because oxygenated HbS cannot polymerize, modifying HbS to increase the proportion of oxygenated to deoxygenated HbS in red blood cells would alter disease severity.

The FDA Friday approved the wider use of voxelotor (Oxbryta) in patients with sickle cell disease (SCD) as young as 4 years of age. In 2019, the oral polymerization inhibitor was approved by the FDA for adults and pediatric patients with SCD 12 years of age and older.