Global Blood Therapeutics Seeks Approval of Voxelotor for Pediatric Sickle Cell Treatment

Next year, Global Blood Therapeutics Inc. (GBT) will seek expanded approval of the use of its treatment for sickle cell disease (SCD), voxelotor, in children ages 4 to 11 through the FDA’s accelerated approval pathway. Previously, the drug was approved in November 2019 for patients aged 12 and over.

The application will be supported by data from the ongoing phase II-a HOPE-KIDS 1 trial, a single-dose pharmacokinetic study of 155 pediatric patients with SCD.

“We’ve got a significant amount of data that really reinforces that the drug has a very similar profile for both safety and efficacy in this age group,” said Ted Love, President and CEO of GBT.

Voxelotor is a hemoglobin S polymerization inhibitor that prevents red blood cells from sickling, an abnormality that restricts oxygen flow in blood vessels.

Sources: GBT press release, June 8, 2020; Reuters, June 8, 2020;, February 18, 2020; FDA press release, November 25, 2019.