Galenus Prize 2025: Casgevy wins the joint prize in the Orphan Drugs category

Transfusion-dependent β-thalassemia and severe sickle cell disease Galenus Prize 2025: Casgevy wins the joint prize in the Orphan Drugs category

Exagamglogene autotemcel (Exa-cel; Casgevy) from Vertex Pharmaceuticals is currently the only approved gene-editing therapy for the single-dose treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. It is one of two compounds to receive the Galenus of Pergamon Award in the Orphan Drug category.