STAT+: Vertex, U.K. reach deal to provide CRISPR-based medicine to thalassemia patients in England

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 can now be offered through the NHS in England.

Casgevy is a one-time treatment that uses CRISPR to edit a patient’s own blood stem cells, overcoming the mutations at the root of various inherited blood disorders. Clinical trial data indicated it can act effectively as a cure for many patients, though there are questions about just how long the benefits of the treatment will last. Its U.S. list price is $2.2 million, though the deal between England and Vertex means it will be offered through the NHS at an undisclosed discount.

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