EXAGAMGLOGÈNE AUTOTEMCEL

USDailyMed:Exagamglogene autotemcel B06AX05(WHO) CA:℞-onlyUK:POM(Prescription only)US:℞-onlyEU:Rx-only DB15572 S53L777GM8 D12749 Exagamglogene autotemcel, sold under the brand nameCasgevy, is agene therapyused for the treatment ofsickle cell diseaseand transfusion-dependentbeta thalassemia.It was developed byVertex PharmaceuticalsandCRISPR Therapeutics. The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023.It was approved by the Kingdom of Bahrain'sNational Health Regulatory Authority(NHRA) to treatsickle-cell anemiaandbeta thalassemiaon 2 December 2023.It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizingCRISPR/Cas9 gene editingtechnology to be approved by the USFood and Drug Administration(FDA).The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache, and itching.