Vertex says pediatric CASGEVY data show consistent benefits in rare blood disorders

2 min read Updated on 11 Jun 2026, 09:55 PM

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Jubin VScanX News Team

AI Summary

Vertex Pharmaceuticals Incorporated announced data demonstrating the clinical benefits of CASGEVY (exagamglogene autotemcel) in children ages 5–11 with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress and simultaneously published in the New England Journal of Medicine, show that efficacy and safety outcomes in this age group are consistent with the transformative profile established in adult and adolescent patients. These findings highlight the potential benefits of addressing vaso-occlusive crises (VOCs) and transfusion burden earlier in life to prevent long-term complications such as organ damage.

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Vertex Pharmaceuticals Incorporated 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. These findings highlight the potential benefits of addressing vaso-occlusive crises (VOCs) and transfusion burden earlier in life to prevent long-term complications such as organ damage.

"The data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits CASGEVY can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

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 19.0 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.

Study	Condition	Patients Dosed	Patients Achieving Primary Endpoint	Primary Endpoint	Mean Duration (months)
CLIMB-151	Severe SCD	11	8 / 8 (100%)	Free from VOCs for ≥12 months	19.0
CLIMB-141	TDT	15	8 / 8 (100%)	Transfusion independence for ≥12 months	23.4

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.