Vertex Pharmaceuticals received FDA approval to expand the use of its gene therapy, Casgevy, to children aged 2 to 11. This one-time treatment targets patients with sickle cell disease (SCD) experiencing recurrent pain crises or transfusion-dependent beta-thalassemia (TDT).
Casgevy is now the first and only genetic therapy approved for this younger demographic for both blood disorders. The supplemental approval follows Phase 3 trial data demonstrating safety and efficacy consistent with results in older patients.
The expansion makes approximately 5,500 additional children in the U.S. eligible for the treatment. The FDA previously limited Casgevy use to patients aged 12 and older.