CASGEVY (exagamglogene autotemcel [exa-cel]

July 1, 2026: Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced the FDA approved an expanded label for CASGEVY (exagamglogene autotemcel), allowing use in patients ages 2 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). The therapy was previously approved for patients 12 years and older; Vertex estimates ~5,500 additional children in the U.S. are now eligible. Vertex said it has more than 75 activated authorized treatment centers in the U.S. for CASGEVY.

The company noted regulatory review is underway in the Kingdom of Saudi Arabia and the United Kingdom for pediatric label expansion (Vertex says submissions were recently completed to expand use to children as young as five).

Vertex also described its CLIMB clinical program: completed Phase 1/2/3 open-label studies CLIMB-111 (TDT) and CLIMB-121 (SCD) in ages 12–35 with ~2 years follow-up; ongoing Phase 3 open-label studies CLIMB-141 (TDT) and CLIMB-151 (SCD) in ages 2–11, with enrollment and dosing complete for the 5–11 cohort; and long-term follow-up study CLIMB-131 (up to 15 years).