Exagamglogene autotemcel (Casgevy)

Mechanism of action

Gene therapy

Diseases for which it is established

History of changes in FDA indication

  • 2023-12-08: Approved for the treatment of sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). (Based on CLIMB SCD-121)
  • 2024-01-16: Approved for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. (Based on CLIMB THAL-111)

Also known as

  • Generic name: exa-cel
  • Brand name: Casgevy