UK HTA looks disfavorably on new sickle cell gene therapies

15 March 2024

Britain’s health technology assessor (HTA) is moving to recommend against funding Casgevy (exagamglogene autotemcel), the second such blow for people with sickle cell disease (SCD) in the country.

The agency has already recommended not to fund Pfizer’s (NYSE: PFE) Oxbryta (voxelotor), although another review round could see that decision reversed.

Like Oxbryta, Casgevy is one of a new generation of gene therapies offering hope for the first time that there could be a transformational impact in SCD.

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