Kim Smith-Whitley, MD: Voxelotor and Sickle Cell Disease

In an interview with HCPLive®, Kim Smith-Whitley, MD, Executive Vice President and Head of Research & Development, Global Blood Therapeutics (GBT), spoke passionately about her research on voxelotor (Oxbryta) for children with sickle cell disease.

“We have really started to just explore the possibilities for intervening and improving the health of those with sickle cell,” she said.

GBT announced that the US Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for filing and review. The company is seeking accelerated approval for voxelotor for the treatment of sickle cell disease in children aged 4-11.

Smith-Whitley conducted a study that supported the supplemental application. The results found that the effectiveness and the safety profile of voxelotor as treatment for sickle cell disease is consistent in children aged 4-11, as already observed in adolescents and adults.

As a world-renowned hematologist with more than 30 years experience, Smith-Whitley’s research has focused on sickle cell disease, specifically pulmonary complications, transfusion-related complications and health care utilization.

According to Smith-Whitley, while much progress has been made there’s still a lot of work to do regarding sickle cell treatment.

“One of the reasons I’m so passionate about it is because this community is so overlooked,” Smith-Whitley said.

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.