UK – NICE turns down bluebird bio’s gene therapy Zynteglo

The UK’s National Institute for Health and Care Excellence (NICE) has rejected bluebird bio’s gene therapy Zynteglo for patients with transfusion-dependent beta-thalassaemia (TDT) in draft guidance.

The draft guidance, published today, said that clinical trials evaluating Zynteglo (betibeglogene autotemcel; beti-cel) were ‘small’, with participants not having been followed up for ‘very long’.

NICE did add, however, that these trials suggest that eventually certain TDT patients treated with Zynteglo may no longer require blood transfusion or may require them less often.

bluebird’s main clinical evidence for Zynteglo came from phase 1/2 studies of the gene therapy in TDT patients with non-beta0/beta0 genotypes.

The primary endpoint of these studies was transfusion independence (TI), with the secondary endpoint being transfusion reduction…