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Voxelotor has the potential to decrease long-term problems in pediatric sickle cell disease.
Kim Smith-Whitley, MD, Executive Vice President and Head of Research & Development, Global Blood Therapeutics (GBT), discussed the role of voxelotor (Oxbryta) in sickle cell disease treatment during an interview with HCPLive®.
“I think that having another option, like Oxbryta, with its sickle hemoglobin polymerization inhibition, we will be able to hopefully decrease those long-term problems in those young children with sickle cell,” Smith-Whitley said, “and make it possible for them to go into the adult world healthier, where we know at that time the complications associated with sickle cell disease increase.”
GBT announced that their supplemental New Drug Application (sNDA) has been accepted by the US Food and Drug Administration (FDA) to review voxelotor as treatment for sickle cell disease in children aged 4-11. Global Blood Therapeutics also submitted a New Drug Application (NDA) seeking approval for a new age-appropriate dispersible tablet dosage form of voxelotor which would be suitable for pediatric patients.
The applications were supported by results of a study conducted by Smith-Whitley that found that voxelotor is just as effective in treating sickle cell disease in children aged 4-11 as it is in adolescents and adults. The safety profile of voxelotor was found to be consistent among this age group as well.
The study, “Safety and Efficacy of Voxelotor in Pediatric Patients With Sickle Cell Disease Aged 4-11 Years: Results From the Phase 2a HOPE-KIDS 1 Study”, was presented at European Hematology Association Congress 2021.