UK Healthcare Regulator Approves Vertex Pharmaceuticals' Casgevy for Children Aged Five and Older

The UK’s MHRA approved Vertex Pharmaceuticals’ Casgevy gene-editing therapy for children aged five and older with severe sickle-cell disease or transfusion-dependent beta-thalassemia, lowering the previous minimum age of 12. The decision followed an assessment of the treatment’s safety, quality, and effectiveness; Casgevy modifies bone marrow stem cells to support production of functioning hemoglobin. Vertex remains the therapy’s exclusive license holder, manufacturer, and distributor, and the regulator will continue monitoring its safety.