UK’s NICE Approves SCD Gene Therapy for NHS England
February 04, 2025
The United Kingdom’s National Institute for Health and Care Excellence
approved the first gene therapy to treat sickle cell disease (SCD) for use on England’s National Health Service on Jan. 31.
Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals) is a non-viral, ex vivo CRISPR/Cas9 gene-edited therapy. It works by editing a patient’s hematopoietic stem cells to increase fetal hemoglobin production in red blood cells, significantly reducing or eliminating the need for transfusions.
While the list price of exa-cel, a one-time treatment, is 1.65 million British pounds, NHS England secured a reduced price through a
reimbursement agreement with Vertex under the Innovative Medicines Fund. Exa-cel is now available under a managed access scheme for patients 12 years and older with certain types of severe SCD.
