Kim Smith-Whitley, MD: New Treatment for Children with Sickle Cell Disease

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Voxelotor study results show it’s just as effective in children as adults and adolescents.

Global Blood Therapeutics (GBT) recently announced that voxelotor (Oxbryta®) is currently undergoing the approval process with the US Food and Drug Administration (FDA) for treatment of sickle cell disease in children aged 4-11.

According to a study conducted by Kim Smith-Whitley, MD, Executive Vice President and Head of Research & Development, GBT, voxelotor was found to be just as effective in children as adults and adolescents.

In an interview with HCPLive®, Smith-Whitley spoke about the importance of treating sickle cell patients as early as possible.

“So, this is an opportunity to hopefully, over time, see individuals who are younger with sickle cell disease, who may not have chronic organ damage yet, have access to something that will lead to improvements in hemoglobin and hopefully long term improvements in their disease,” she said. “And those studies are underway.”

Voxelotor uses weight-based dosing with dispersible tablets in patients with sickle cell disease aged 4-11. The safety profile for this age group was consistent with the existing safety profile in adult and adolescent patients. These results show voxelotor as a potential strategy for early mitigation of the morbidity and mortality associated with sickle cell disease in pediatric patients.

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.