BOSTON, July 1, 2026
Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved the expanded use of CASGEVY® (exagamglogene autotemcel) for the treatment of children aged 2 years and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT). The expanded indication makes CASGEVY the first and only approved genetic therapy for both diseases in children as young as two years old. The approval expands eligibility to approximately 5,500 additional pediatric patients in the United States beyond the previous approval for individuals aged 12 years and older. Vertex stated that more than 75 authorized treatment centers are now activated across the U.S. to provide access to the one-time therapy through established reimbursement pathways, while regulatory reviews for similar pediatric label expansions continue in the United Kingdom and Saudi Arabia.
FDA Approval Expands Access to One-Time Gene-Editing Therapy
The expanded approval significantly broadens access to CASGEVY for young children diagnosed with severe inherited blood disorders that can lead to lifelong complications and premature mortality. CASGEVY is a one-time, non-viral CRISPR/Cas9 gene-edited cell therapy that modifies a patient’s own hematopoietic stem cells to increase fetal hemoglobin production, helping prevent vaso-occlusive crises in sickle cell disease and reducing or eliminating the need for chronic blood transfusions in beta thalassemia. Vertex said earlier treatment may allow patients to receive the therapy before irreversible organ damage develops, offering the potential for durable long-term clinical benefits.
Clinical Studies Demonstrated Consistent Safety and Efficacy
The FDA’s decision builds on results from the completed Phase 1/2/3 CLIMB-111 and CLIMB-121 studies involving patients aged 12 to 35 years, along with ongoing Phase 3 CLIMB-141 and CLIMB-151 trials evaluating children between 2 and 11 years of age. Enrollment and dosing have been completed for the 5-to-11-year-old cohorts in both pediatric studies. Patients continue to be monitored through the long-term CLIMB-131 follow-up study, which is designed to assess the safety and durability of CASGEVY for up to 15 years following treatment. According to Vertex, clinical findings consistently demonstrated sustained reductions in vaso-occlusive crises for sickle cell disease and transfusion independence for many beta thalassemia patients across different age groups.
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Vertex Advances Global Expansion of Gene-Editing Therapy
Vertex continues to expand the global availability of CASGEVY following regulatory approvals in multiple regions. The company recently submitted applications to regulatory authorities in the United Kingdom and Saudi Arabia seeking approval to extend treatment eligibility to younger pediatric patients. CASGEVY represents one of the first commercially approved CRISPR/Cas9 gene-editing therapies worldwide and reflects Vertex’s broader strategy of developing transformative genetic medicines for severe diseases with significant unmet medical needs. The expanded pediatric indication further strengthens the company’s leadership in gene-editing therapeutics while offering earlier intervention opportunities for children living with sickle cell disease and transfusion-dependent beta thalassemia.
Source: Vertex Pharmaceuticals press release
