The Food and Drug Administration (FDA) has expanded the approval of Casgevy® (exagamglogene autotemcel) to include patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
Exagamglogene autotemcel is an autologous genome edited hematopoietic stem cell-based gene therapy. It was previously approved for patients aged 12 years and older with SCD and TDT.
The approval in patients aged 5 years to less than 12 years with SCD was based on data from an open-label phase 3 trial (ClinicalTrials.gov Identifier: NCT05329649). The study included 11 pediatric patients who had a documented history of at least 2 protocol-defined severe VOC events annually during the 2 years prior to screening. Of the 11 patients who received exagamglogene autotemcel treatment, 8 were evaluated for efficacy.