The approval expands the use of Casgevy to patients as young as 2 years old for the treatment of sickle cell disease and| beta thalassemia
The USFDA issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT). According to the agency, this represents the first gene therapy approved for patients as young as 2 years old with SCD.
Casgevy was previously approved for the treatment of patients aged 12 years and older with SCD or TDT. The new decision allows younger pediatric patients to access an additional treatment option for these life-threatening diseases, said Karim Mikhail, B Pharm, MS, acting director of the Center for Biologics Evaluation and Research (CBER), in a news release.