US FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease
United States, July 6 -- The U.S. Food and Drug Administration 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 ? 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. SCD affects red blood cells (RBCs), which contain hemoglobin, a protein that transports oxygen throughout the body. SCD can cause various symptoms and health issues, including episodes of severe pain called sickle cell crises or VOCs. Thal...
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