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Jun 11, 2026 12:30 PM

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).

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Among the eight patients with sufficient follow-up, 100% achieved the primary endpoint of remaining VOC-free for at least 12 ...