Vertex Says Pediatric CASGEVY Data Show Consistent Benefits In Rare Blood Disorders

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). data-variant="card" data-news-mode="manual" > Read Also: Vertex's New Kidney Drug Delivers Big Trial Win, Stock Jumps Among the eight patients with sufficient follow-up, 100% achieved the primary endpoint of remaining VOC-free for at least 12 ... Full story available on Benzinga.com