15 December 2023 - If approved by the European Commission, patients 12 years of age and older with severe sickle cell disease or transfusion-dependent beta thalassaemia, for whom haematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available, would be eligible for treatment.

Vertex Pharmaceuticals announced today that the EMA’s CHMP has adopted a positive opinion for the conditional approval of Casgevy (exagamglogene autotemcel, a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease and transfusion-dependent beta thalassaemia.

Read Vertex Pharmaceuticals press release