UK's NICE says it's not ready to recommend Vertex's Casgevy for sickle cell

14 Mar 2024
endpts.com
Drug ApprovalGene Therapy
The UK’s National Institute for Health and Care Excellence isn’t quite ready to recommend Vertex and CRISPR Therapeutics’ Casgevy for sickle cell disease, citing cost-effectiveness issues. NICE said in a draft guidance that it would need more data on the treatment’s effectiveness, as well as a commercial arrangement. Casgevy, a one-time treatment also known as exa-cel, was approved in the UK in November. The approval was the first ever for a therapy created with CRISPR gene editing, for both sickle cell disease and transfusion-dependent beta thalassemia. In the US, the FDA approved it for sickle cell in December and for beta thalassemia in January, with a price of $2.2 million.
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