Voxelotor is the first and only FDA-approved medication for children with sickle cell disease.
Phase 2 data from Global Blood Therapeutic's HOPE-KIDS 1 trial found that voxelotor (Oxbryta) improves hemoglobin (Hb) levels and markers of hemolysis and has the potential to mitigate sickle cell disease-related complications based on its mechanism as a Sickle hemoglobin (HbS) polymerization inhibitor.
These data were recently published in Pediatric Blood & Cancer. The featured phase 2A open-label, single and multiple-dose study assessed the efficacy, safety, and pharmacokinetics of voxelotor in pediatric patients with sickle cell disease.
Voxelotor was the first and only medication for sickle cell disease in children approved by the US Food and Drug Administration (FDA) in December 2021. The sickle hemoglobin polymerization inhibitor is now recommended for patients aged 4 and older.
According to the data, a cohort of patients between the ages of 4-11 who received voxelotor for up to 48 weeks displayed improved hemoglobin levels along with markers of hemolysis. Related complications can also potentially be mitigated.
Inestigators found no new safety signals to be concerned about and the therapy was well tolerated in trial patients. The phase 2 research has been presented at multiple meetings including 2021 European Hematology Association Congress, and the 2021 49th Annual Sickle Cell Disease Association of America National Convention.