FDA Expands Gene Therapy Approval to Include Beta-Thalassemia

The Food and Drug Administration on Tuesday expanded the approval for exagamglogene autotemcel (Casgevy, Vertex), a CRISPR/Cas9 gene-edited cell therapy, to include the treatment of transfusion-dependent beta-thalassemia (TDT) in patients ages 12 and older. FDA previously approved the gene therapy to treat patients with sickle cell disease (SCD) Dec. 8.

Exagamglogene autotemcel is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid-specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, which has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD and transfusion requirements in patients with TDT.

Outside of the United States, the European Medicines Agency recommended approval of exagamglogene autotemcel last month to treat SCD or TDT patients ages 12 and older for whom hematopoietic stem cell transplantation is appropriate and a suitable donor is not available. Vertex also announced last week that it received regulatory approval for exagamglogene autotemcel in Saudi Arabia.