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A U.K. watchdog balked at the cost-effectiveness of Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Thursday, recommending against funding the treatment unless uncertainties can be cleared up satisfactorily.

The U.K. became the first country to authorize Vertex’s Casgevy (exagamglogene autotemcel) when the Medicines and Healthcare products Regulatory Agency approved the treatment in November 2023. However, regulatory approval is just the first of two key steps to reaching patients in the U.K. Vertex must also show the therapy is cost-effective before it can be used routinely by the National Health Service (NHS).

The U.K. National Institute for Health and Care Excellence (NICE) recommended against the use of Casgevy in sickle cell disease in draft guidance. The therapy has a list price of $2.2 million in the U.S. but NICE’s guidance says the U.K. price is “commercial in confidence.” Vertex’s justification for the cost rests on the potential for a one-time treatment with Casgevy to provide a functional cure for sickle cell disease. 

Currently, NICE’s cost-effectiveness estimate for Casgevy exceeds the threshold it normally views as a good use of the...