Voxelotor Granted Priority Review for Children With Sickle Cell Disease

The U.S. Food and Drug Administration (FDA) has accepted Global Blood Therapeutics’ (GBT’s) supplemental New Drug Application (sNDA) seeking accelerated approval for voxelotor for the treatment of children ages 4 to 11 with sickle cell disease (SCD). Voxelotor is a once-daily oral therapy that modulates hemoglobin (Hb) affinity for oxygen by binding to and stabilizing Hb S.

Additionally, the FDA has accepted GBT’s New Drug Application (NDA) for a new dispersible tablet dosage form of voxelotor appropriate for pediatric patients. The 300 mg tablets include grape flavoring, are intended to be dispersed in room-temperature clear drinks for ease of swallowing, and allow for weight-based dosing.

Previously, the drug was FDA approved for the treatment of SCD in patients ages 12 and older. Voxelotor inhibits Hb polymerization, reduces sickling, and improves red blood cell survival.

GBT’s pediatric sNDA and NDA are based on data from the phase IIa HOPE-KIDS 1 Study (GBT440-007), which demonstrated improved Hb and reduced hemolysis in patients who received weight-based treatment with voxelotor dispersible tablets.

The agency has granted priority review status to both applications, with a Prescription Drug User Fee Act target action date of December 25, 2021.

Source: Global Blood Therapeutics press release, September 7, 2021.