US drug developer Vertex Pharmaceuticals (Nasdaq: VRTX) revealed a reimbursement agreement with NHS England for eligible transfusion-dependent beta thalassemia (TDT) patients to access the CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), from today.
The reimbursement agreement comes as the UK’s health technology assessor the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending Casgevy’s use in the National Health Service (NHS).
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted Casgevy the first authorization in the world for a CRISPR-based gene-editing therapy on November 15, 2023.
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