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

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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