FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

The U.S. Food and Drug Administration today 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 (VOCs) or transfusion-dependent β thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD. Casgevy has been previously approved for the treatment of patients aged 12 years and older with SCD with recurrent VOCs or TDT. “With today’s decision, pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,” said Karim Mikhail, B. Phar...