LONDON — People in England with sickle cell disease will now be able to access the world’s first CRISPR-based medicine under a pricing and reimbursement agreement reached between U.K. health officials and Vertex Pharmaceuticals.
The deal is notable because the U.K.’s cost-effectiveness watchdog initially raised concerns about the durability of the treatment, called Casgevy, in a preliminary ruling last year, saying it wasn’t yet recommending the therapy.
But in a statement Friday morning, the agency, the National Institute for Health and Care Excellence, called the one-time treatment a potential cure. It said Casgevy would be available through the National Health Service to patients 12 years and older under a managed access scheme, which will allow officials to track how long the benefits of the therapy last and whether patients who receive it have any ongoing sickle cell complications that require additional care.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in