On Thursday, the U.S. Food and Drug Administration (FDA) approved an expanded label for Vertex Pharmaceuticals Incorporated‘s (NASDAQ:VRTX) Casgevy (exagamglogene autotemcel).
The approval allows Casgevy to be used in patients aged 2 years and older with sickle cell disease (SCD), accompanied by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
The decision makes Casgevy the first approved genetic therapy available for children as young as 2 with either condition.
FDA Decision Broadens Patient Eligibility For Vertex Gene Therapy
The expanded indication significantly increases the number of eligible patients in the U.S. Vertex said approximately 5,500 additional children can now receive the one-time genetic therapy, building on the drug’s previous approval for patients aged 12 years and older.
