STAT August 7, 2024
LONDON — Patients in England with a rare blood disorder will now be able to receive the world’s first CRISPR-based medicine, after drugmaker Vertex Pharmaceuticals and U.K. health officials struck a reimbursement deal for the pricey treatment.
The National Institute for Health and Care Excellence, the U.K.’s cost-effectiveness watchdog, said early Thursday that it was recommending the treatment, called Casgevy, for certain patients with beta thalassemia, a condition that otherwise requires regular blood transfusions. With NICE’s positive review, and an agreement between Vertex and the National Health Service on the price, Casgevy...
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