FDA Expands Casgevy Approval to Children as Young as 2 Years With Sickle Cell Disease or Transfusion-Dependent β-Thalassemia

The FDA has granted supplemental approval to exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals) for patients 2 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT), marking the first gene therapy approved for children as young as 2 years with SCD.¹

Previously, casgevy was approved for patients 12 years and older with SCD with recurrent VOCs or TDT.¹ This expansion broadens access to a one-time, autologous CRISPR/Cas9 genome-edited cell therapy for younger pediatric patients with serious inherited blood disorders