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).
Among the eight patients with sufficient follow-up, 100% achieved the primary endpoint of remaining VOC-free for at least 12 consecutive months. The average VOC-free duration reached 19 months.
In the Phase 3 CLIMB-141 study evaluating children with TDT, 15 patients received CASGEVY.
All eight patients with sufficient follow-up achieved transfusion independence for at least 12 consecutive months while maintaining a weighted average hemoglobin level of at least 9 g/dL. The average duration of transfusion independence was 23.4 months.
Safety Profile Remains Consistent
Vertex said the safety profile observed in younger patients was consistent with findings from studies in older individuals undergoing myeloablative conditioning and autologous transplant procedures.
The company reiterated that one previously disclosed death in a child with TDT was linked to severe veno-occlusive disease from busulfan conditioning and was not related to CASGEVY.
Vertex Pursues Regulatory Expansion
CASGEVY is currently approved in multiple countries for eligible patients aged 12 years and older with SCD accompanied by recurrent VOCs or TDT.
Vertex said the U.S. Food and Drug Administration is reviewing an application to expand the therapy’s use to younger children. The company has also submitted applications in the United Kingdom and Saudi Arabia seeking similar label expansions.
CASGEVY remains investigational for children ages 5 to 11.
VRTX Price Action: Vertex Pharmaceuticals shares were up 0.89% at $439.59 at the time of publication on Thursday, according to Benzinga Pro data.
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