Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) reported new clinical data showing that CASGEVY (exagamglogene autotemcel) delivered efficacy and safety outcomes in children ages 5 to 11 with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT) The data were consistent with results previously observed in adolescent and adult patients.
Phase 3 Studies Show High Rates Of Clinical Response An interim analysis from the Phase 3 CLIMB-151 study in children with severe SCD showed all 11 treated patients remained free from vaso-occlusive crises (VOCs).
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The average VOC-free duration reached 19 months.
In the Phase 3 CLIMB-141 study evaluating children with TDT, 15 patients received CASGEVY.
