Vertex Pharmaceuticals and CRISPR Therapeutics were granted conditional marketing authorization for CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) from the Medicines and Healthcare products Regulatory Agency (MHRA).
 
CASGEVY is authorized for the treatment of eligible patients 12 years and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. There are an estimated 2,000 patients eligible for CASGEVY in the U.K.
 
CASGEVY is a genetically modified autologous CD34+ cell enriched population that contains human hematopoietic stem and progenitor cells edited ex vivo by CRISPR/Cas9 at the erythroid-specific enhancer region of the BCL11A gene.
 
Exa-cel is also under review by the European Medicines Agency and the U.S. FDA. The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively.